A Phase 4 interventional study of Deferasirox in Iron Overload, sponsored by Novartis Pharmaceuticals. Completed at 10 sites in 7 countries. Open to participants aged 2 Years to 6 Years. Per ClinicalTrials.gov, last updated 2020-08-25.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 4, Interventional, and Treatment
This study employed a prospective, single-arm, global multi-center interventional open-label, non-randomized design to identify and assess safety profile of the crushed deferasirox FCT when administered up to 24 weeks in pediatric patients aged ≥2 to \<6 years with transfusional hemosiderosis. The study was designed to enroll a minimum of 40 patients. Forty-four patients were treated and analyzed.
The study included a screening period (from Day 0-14) with two visits at least 7 days apart to assess eligibility of patients that were chelation naïve or on a prior iron chelator treatment other than DFX. For Patients on DFX treatment prior to study entry only one screening visit (screening visit 1) were to occur to determine eligibility. Any current chelation therapy except deferasirox were to be discontinued to undergo a 5-day washout period prior to commencing a 24 week treatment period with crushed deferasirox FCT.
All patients were to have weekly visits for the first month to monitor renal function. Hepatic function were to be assessed biweekly during the first month. Thereafter, monthly safety assessments were to be performed, including the monitoring of serum ferritin values and trends in order to adapt patient treatment.
Eligibility, application of dosing standards and adjustments, as well as safety and serum ferritin assessments as specified in the protocol.
The planned duration of treatment was 24 weeks followed by a 30-day safety follow up.
156 studies on the registry are indexed under Iron Overload; 6 are open to participants now.
This study's enrollment of 44 is below the median of 60 across 97 interventional studies indexed under Iron Overload.
Browse Iron Overload studies →Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Child-Pugh class
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Drug: Deferasirox
Deferosirox was provided in tablet forms of 90, 180 and 360mg. Tablets were crushed in the home environment and administered by sprinkling the full dose on to soft food to be consumed immediately.
Also known as: ICL670
Number of Participants With Selected Gastrointestinal Disorders up to 24 Weeks
To assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal (GI) disorders (esophagitis, stomatitis, mouth ulceration, gastric ulcers, haemorrhage, abdominal pain, diarrhea, nausea, and vomiting). Only descriptive analysis performed.
Time frame: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
Adverse Events Profile
Analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event TEAEs and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters. Only descriptive analysis performed.
Time frame: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
Number of Participants With Notable Changes in ECG Values From Baseline
Safety measured by the notable post-baseline changes in ECG values (PR, QRS, QT, QTcF and HR intervals) compared to baseline. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
Time frame: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
Absolute Change From Baseline in Serum Ferritin (SF)
Absolute change from baseline over time in SF values up to 24 weeks of treatment were to be provided. Only descriptive analysis performed.
Time frame: Baseline (BL), Week 4, Week 8, Week 12, Week 16, EOT (Week 24)
Number of Participants With Worst Post-baseline Values in Selected Chemistry Parameters
Safety measured by the worst post-baseline severity grade observed in a patient calculated using the normal/low/high classifications based on local laboratory normal ranges, regardless of the baseline status. Baseline was defined as the last non-missing value on or prior to the first dose. The selected chemistry parameters were: Alanine aminotransferase (ALT), Alkaline phosphatase (ALP), Aspartate aminotransferase (AST), total bilirubin, direct bilirubin, serum creatinine and Urine protein creatinine ratio (UPCR) (Protein/Creatinine represented UPCR). Only descriptive analysis performed.
Time frame: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
Number of Participants With Clinically Significant Auditory Assessments Changes From Baseline
Safety measured by notable post-baseline changes in Auditory assessments (comprehensive audiometry threshold examination and speech recognition). Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
Time frame: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
Number of Participants With Clinically Significant Ocular Assessments Changes From Baseline
Safety measured by notable post-baseline changes in Ocular assessments (Distance visual acuity test, Applanation tonometry, lens photography, wide angle fundus photography of the retina and optic nerve). Ocular assessment were required at screening and end of Treatment; during treatment, they were to be performed at the discretion of the investigator based on patient reporting symptoms. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
Time frame: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.
Absolute Change From Baseline in Systolic and Diastolic Blood Pressures (mmHg)
Absolute change from baseline over time in systolic and diastolic blood pressures measurements were to be provided. Only descriptive analysis performed.
Time frame: Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
Absolute Change From Baseline in Pulse Rate (Bpm)
Absolute change from baseline over time in supine pulse rate was to be provided. Only descriptive analysis performed.
Time frame: Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
Absolute Change From Baseline in Body Temperature (°C)
Absolute change from baseline over time in body temperature measurements was to be provided. Only descriptive analysis performed.
Time frame: Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
Absolute Change From Baseline in Body Weight (kg)
Absolute change from baseline over time in body weight measurements was to be provided. Only descriptive analysis performed.
Time frame: Baseline (BL), Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Adherence
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed.
Time frame: Week 4, Week 12, EOT (Week 24)
Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Type of Medicine Child Like Scoring
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts.
Time frame: Baseline (BL), Week 4, Week 12, EOT (Week 24)
Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts.
Time frame: Baseline (BL), Week 4, Week 12, EOT (Week 24)
Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Rank Based on Child's Preference Scoring
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency count.
Time frame: Baseline (BL), Week 4, Week 12, EOT (Week 24)
Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Concerns
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child's response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver's responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed.
Time frame: Week 4, Week 12, EOT (Week 24)
Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Adherence
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed.
Time frame: Week 4, Week 12, EOT (Week 24)
Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Type of Medicine Child Like Scoring
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts.
Time frame: Week 4, Week 12, EOT (Week 24)
Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts.
Time frame: Week 4, Week 12, EOT (Week 24)
Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Rank Based on Child's Preference Scoring
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency counts.
Time frame: Week 4, Week 12, EOT (Week 24)
Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Concerns
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child's response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver's responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed.
Time frame: Week 4, Week 12, EOT (Week 24)
Palatability Score in Chelation Naive Participants
The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed.
Time frame: Week 4, Week 12, EOT (Week 24)
Number of Chelation Naive Participants With Palatability After Taste Item Scoring
The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts.
Time frame: Week 4, Week 12, EOT (Week 24)
Palatability Score in Participants Pre-treated With Deferasirox
The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed.
Time frame: Week 4, Week 12, EOT (Week 24)
Number of Participants Pre-treated With Deferasirox With Palatability After Taste Item Scoring
The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts.
Time frame: Baseline, Week 4, Week 12, EOT (Week 24)
GI Symptom Score in Chelation Naive Participants
The GI symptom score was calculated from responses to 5 questions, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT.
Time frame: Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
Number of Participants With GI Bowel Movements Item Scoring in Chelation Naive Participants
The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 ("None"), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = "5 - 10" and 6 = "11 or more". The GI bowel movements item score was presented descriptively using frequency counts.
Time frame: Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
GI Symptom Score in Participants Pre-treated With Deferasirox
The GI symptom score was calculated from responses to 5 questions, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT.
Time frame: Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
Number of Participants With GI Bowel Movements Item Scoring in Participants Pre-treated With Deferasirox
The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 ("None"), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = "5 - 10" and 6 = "11 or more". The GI bowel movements item score was presented descriptively using frequency counts.
Time frame: Baseline, Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)
The study was conducted in 10 sites in 7 countries (one in Egypt, three in Italy, one in Lebanon, one in Oman, two in Thailand, one in the United Arab Emirates, and one in the United Kingdom).
| Milestone | Deferasirox |
|---|---|
| Started | 44 |
| Completed | 39 |
| Not completed | 5 |
| Withdrew: Adverse event | 3 |
| Withdrew: Physician decision | 1 |
| Withdrew: Withdrawal by subject | 1 |
To assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal (GI) disorders (esophagitis, stomatitis, mouth ulceration, gastric ulcers, haemorrhage, abdominal pain, diarrhea, nausea, and vomiting). Only descriptive analysis performed.
| Participants | Deferasirox |
|---|---|
| Oesophagitis | 0 |
| Barrett's esophagitis | 0 |
| Stomatitis | 0 |
| Mouth ulceration | 0 |
| Gastric ulcer | 0 |
| Gastrointestinal haemorrhage | 0 |
| Abdominal pain | 2 |
| Diarrhoea | 4 |
| Nausea | 0 |
| Vomiting | 2 |
Analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event TEAEs and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters. Only descriptive analysis performed.
| Participants | Deferasirox |
|---|---|
| On treatment-related AE | 22 |
| On treatment related SAE | 0 |
| On treatment Deaths | 0 |
Safety measured by the notable post-baseline changes in ECG values (PR, QRS, QT, QTcF and HR intervals) compared to baseline. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
| Participants | Deferasirox |
|---|---|
| Number of Participants With Notable Changes in ECG Values From Baseline | 0 |
Absolute change from baseline over time in SF values up to 24 weeks of treatment were to be provided. Only descriptive analysis performed.
| ug/L | Deferasirox |
|---|---|
| Baseline (BL) | 2152.7 ± 1039.06 |
| Change from BL @ Week 4 | -118.4 ± 590.20 |
| Change from BL @ Week 8 | -17.5 ± 665.68 |
| Change from BL @ Week 12 | -52.8 ± 790.26 |
| Change from BL @ Week 16 | 71.9 ± 988.30 |
| Change from BL @ Week 20 | -64.3 ± 791.92 |
| Change from BL @ EOT (Week 24) | -140.7 ± 824.01 |
Safety measured by the worst post-baseline severity grade observed in a patient calculated using the normal/low/high classifications based on local laboratory normal ranges, regardless of the baseline status. Baseline was defined as the last non-missing value on or prior to the first dose. The selected chemistry parameters were: Alanine aminotransferase (ALT), Alkaline phosphatase (ALP), Aspartate aminotransferase (AST), total bilirubin, direct bilirubin, serum creatinine and Urine protein creatinine ratio (UPCR) (Protein/Creatinine represented UPCR). Only descriptive analysis performed.
| Participants | Deferasirox |
|---|---|
| Alanine Aminotransferase — Normal | 16 |
| Alanine Aminotransferase — Low | 0 |
| Alanine Aminotransferase — High | 28 |
| Alanine Aminotransferase — High & Low | 0 |
| Alanine Aminotransferase — Missing | 0 |
| Alkaline Phosphatase — Normal | 25 |
| Alkaline Phosphatase — Low | 2 |
| Alkaline Phosphatase — High | 17 |
| Alkaline Phosphatase — High & Low | 0 |
| Alkaline Phosphatase — Missing | 0 |
| Aspartate Aminotransferase — Normal | 14 |
| Aspartate Aminotransferase — Low | 0 |
| Aspartate Aminotransferase — High | 30 |
| Aspartate Aminotransferase — High & Low | 0 |
| Aspartate Aminotransferase — Missing | 0 |
| Bilirubin — Normal | 16 |
| Bilirubin — Low | 0 |
| Bilirubin — High | 28 |
| Bilirubin — High & Low | 0 |
| Bilirubin — Missing | 0 |
| Creatinine — Normal | 6 |
| Creatinine — Low | 29 |
| Creatinine — High | 6 |
| Creatinine — High & Low | 3 |
| Creatinine — Missing | 0 |
| Direct Bilirubin — Normal | 16 |
| Direct Bilirubin — Low | 0 |
| Direct Bilirubin — High | 28 |
| Direct Bilirubin — High & Low | 0 |
| Direct Bilirubin — Missing | 0 |
| Protein/Creatinine — Normal | 20 |
| Protein/Creatinine — Low | 1 |
| Protein/Creatinine — High | 17 |
| Protein/Creatinine — High & Low | 6 |
| Protein/Creatinine — Missing | 0 |
Safety measured by notable post-baseline changes in Auditory assessments (comprehensive audiometry threshold examination and speech recognition). Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
| Participants | Deferasirox |
|---|---|
| Number of Participants With Clinically Significant Auditory Assessments Changes From Baseline | 0 |
Safety measured by notable post-baseline changes in Ocular assessments (Distance visual acuity test, Applanation tonometry, lens photography, wide angle fundus photography of the retina and optic nerve). Ocular assessment were required at screening and end of Treatment; during treatment, they were to be performed at the discretion of the investigator based on patient reporting symptoms. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
| Participants | Deferasirox |
|---|---|
| Number of Participants With Clinically Significant Ocular Assessments Changes From Baseline | 0 |
Absolute change from baseline over time in systolic and diastolic blood pressures measurements were to be provided. Only descriptive analysis performed.
| millimetre of mercury (mmHg) | Deferasirox |
|---|---|
| Systolic BP-Baseline (BL) | 100.0 ± 10.10 |
| Systolic BP-Change from BL @ Week 2 | -0.1 ± 11.27 |
| Systolic BP-Change from BL @ Week 3 | -2.3 ± 7.93 |
| Systolic BP-Change from BL @ Week 4 | -0.3 ± 12.56 |
| Systolic BP-Change from BL @ Week 8 | 0.3 ± 8.53 |
| Systolic BP-Change from BL @ Week 12 | -1.4 ± 12.16 |
| Systolic BP-Change from BL @ Week 16 | -1.9 ± 11.76 |
| Systolic BP-Change from BL @ Week 20 | -0.6 ± 11.67 |
| Systolic BP-Change from BL @ EOT (Week 24) | -2.6 ± 11.54 |
| Diastolic BP-Baseline (BL) | 60.0 ± 7.85 |
| Diastolic BP-Change from BL @ Week 2 | 0.7 ± 6.13 |
| Diastolic BP-Change from BL @ Week 3 | -2.1 ± 9.60 |
| Diastolic BP-Change from BL @ Week 4 | 1.0 ± 10.24 |
| Diastolic BP-Change from BL @ Week 8 | -0.6 ± 13.21 |
| Diastolic BP-Change from BL @ Week 12 | 1.7 ± 11.42 |
| Diastolic BP-Change from BL @ Week 16 | -0.5 ± 10.28 |
| Diastolic BP-Change from BL @ Week 20 | -0.1 ± 11.70 |
| Diastolic BP-Change from BL @ EOT (Week 24) | -0.4 ± 7.89 |
Absolute change from baseline over time in supine pulse rate was to be provided. Only descriptive analysis performed.
| beats per minute (bpm) | Deferasirox |
|---|---|
| Baseline (BL) | 102.4 ± 10.73 |
| Change from BL @ Week 2 | -1.1 ± 10.51 |
| Change from BL @ Week 3 | -2.7 ± 16.18 |
| Change from BL @ Week 4 | -3.1 ± 12.85 |
| Change from BL @ Week 8 | -1.0 ± 15.47 |
| Change from BL @ Week 12 | -0.3 ± 12.69 |
| Change from BL @ Week 16 | -2.2 ± 8.99 |
| Change from BL @ Week 20 | 0.8 ± 12.55 |
| Change from BL @ EOT (Week 24) | 1.1 ± 12.10 |
Absolute change from baseline over time in body temperature measurements was to be provided. Only descriptive analysis performed.
| Celsius degree (°C) | Deferasirox |
|---|---|
| Baseline (BL) | 36.43 ± 0.416 |
| Change from BL @ Week 2 | -0.03 ± 0.285 |
| Change from BL @ Week 3 | -0.10 ± 0.353 |
| Change from BL @ Week 4 | 0.00 ± 0.360 |
| Change from BL @ Week 8 | 0.02 ± 0.336 |
| Change from BL @ Week 12 | -0.03 ± 0.413 |
| Change from BL @ Week 16 | 0.10 ± 0.400 |
| Change from BL @ Week 20 | 0.03 ± 0.372 |
| Change from BL @ EOT (Week 24) | 0.08 ± 0.453 |
Absolute change from baseline over time in body weight measurements was to be provided. Only descriptive analysis performed.
| kilogram (kg) | Deferasirox |
|---|---|
| Baseline (BL) | 15.26 ± 3.109 |
| Change from BL @ Week 4 | 0.08 ± 0.290 |
| Change from BL @ Week 8 | 0.19 ± 0.460 |
| Change from BL @ Week 12 | 0.31 ± 0.482 |
| Change from BL @ Week 16 | 0.25 ± 0.433 |
| Change from BL @ Week 20 | 0.43 ± 0.532 |
| Change from BL @ EOT (Week 24) | 0.44 ± 0.600 |
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed.
| Score | Deferasirox |
|---|---|
| Child's perspective overall score-Week 4 | -2.4 (-3.8 to -1.0) |
| Child's perspective overall score-Week 12 | -2.6 (-4.5 to -0.7) |
| Child's perspective overall score-EOT | -1.9 (-3.5 to -0.2) |
| Caregiver's perspective overall score-Week 4 | -1.4 (-2.9 to 0.1) |
| Caregiver's perspective overall score-Week 12 | -1.4 (-3.3 to 0.4) |
| Caregiver's perspective overall score-EOT | -1.0 (-2.6 to 0.6) |
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts.
| Participants | Deferasirox |
|---|---|
| BL — Tablet to dissolve in liquid | 0 |
| BL — Tablet (taken once a day) | 4 |
| BL — Tablet (taken 3 times a day) | 0 |
| BL — Tablet crushed | 11 |
| BL — Sprinkle powder on food | 14 |
| BL — Injection | 0 |
| BL — I don't know | 3 |
| Wk 4 — Tablet to dissolve in liquid | 0 |
| Wk 4 — Tablet (taken once a day) | 1 |
| Wk 4 — Tablet (taken 3 times a day) | 0 |
| Wk 4 — Tablet crushed | 14 |
| Wk 4 — Sprinkle powder on food | 14 |
| Wk 4 — Injection | 0 |
| Wk 4 — I don't know | 1 |
| Wk 12 — Tablet to dissolve in liquid | 1 |
| Wk 12 — Tablet (taken once a day) | 1 |
| Wk 12 — Tablet (taken 3 times a day) | 0 |
| Wk 12 — Tablet crushed | 13 |
| Wk 12 — Sprinkle powder on food | 12 |
| Wk 12 — Injection | 0 |
| Wk 12 — I don't know | 0 |
| EOT — Tablet to dissolve in liquid | 0 |
| EOT — Tablet (taken once a day) | 4 |
| EOT — Tablet (taken 3 times a day) | 0 |
| EOT — Tablet crushed | 9 |
| EOT — Sprinkle powder on food | 15 |
| EOT — Injection | 0 |
| EOT — I don't know | 0 |
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts.
| Participants | Deferasirox |
|---|---|
| BL — Taste | 6 |
| BL — Aftertaste | 5 |
| BL — Convenience | 5 |
| BL — Number of pills | 0 |
| BL — No/Less side effects | 0 |
| BL — Can correctly prepare the medicine | 0 |
| BL — Easier to remember to take the medicine | 1 |
| BL — Number of times he/she has to take the medicine | 0 |
| BL — No/Less pain on the injection site | 0 |
| BL — Gain personal time with their family and friends | 2 |
| BL — Other | 1 |
| Wk 4 — Taste | 0 |
| Wk 4 — Aftertaste | 0 |
| Wk 4 — Convenience | 4 |
| Wk 4 — Number of pills | 0 |
| Wk 4 — No/Less side effects | 0 |
| Wk 4 — Can correctly prepare the medicine | 0 |
| Wk 4 — Easier to remember to take the medicine | 0 |
| Wk 4 — Number of times he/she has to take the medicine | 0 |
| Wk 4 — No/Less pain on the injection site | 0 |
| Wk 4 — Gain personal time with their family and friends | 2 |
| Wk 4 — Other | 8 |
| Wk 12 — Taste | 0 |
| Wk 12 — Aftertaste | 0 |
| Wk 12 — Convenience | 2 |
| Wk 12 — Number of pills | 0 |
| Wk 12 — No/Less side effects | 0 |
| Wk 12 — Can correctly prepare the medicine | 0 |
| Wk 12 — Easier to remember to take the medicine | 0 |
| Wk 12 — Number of times he/she has to take the medicine | 0 |
| Wk 12 — No/Less pain on the injection site | 0 |
| Wk 12 — Gain personal time with their family and friends | 3 |
| Wk 12 — Other | 8 |
| EOT — Taste | 0 |
| EOT — Aftertaste | 0 |
| EOT — Convenience | 0 |
| EOT — Number of pills | 0 |
| EOT — No/Less side effects | 0 |
| EOT — Can correctly prepare the medicine | 0 |
| EOT — Easier to remember to take the medicine | 0 |
| EOT — Number of times he/she has to take the medicine | 0 |
| EOT — No/Less pain on the injection site | 0 |
| EOT — Gain personal time with their family and friends | 7 |
| EOT — Other | 3 |
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency count.
| Participants | Deferasirox |
|---|---|
| BL -Tablet to dissolve in liquid — 1 | 0 |
| BL -Tablet to dissolve in liquid — 2 | 1 |
| BL -Tablet to dissolve in liquid — 3 | 12 |
| BL -Tablet to dissolve in liquid — 4 | 7 |
| BL -Tablet to dissolve in liquid — 5 | 11 |
| BL -Tablet to dissolve in liquid — 6 | 1 |
| Week 4 -Tablet to dissolve in liquid — 1 | 0 |
| Week 4 -Tablet to dissolve in liquid — 2 | 11 |
| Week 4 -Tablet to dissolve in liquid — 3 | 8 |
| Week 4 -Tablet to dissolve in liquid — 4 | 3 |
| Week 4 -Tablet to dissolve in liquid — 5 | 5 |
| Week 4 -Tablet to dissolve in liquid — 6 | 3 |
| Week 12-Tablet to dissolve in liquid — 1 | 1 |
| Week 12-Tablet to dissolve in liquid — 2 | 7 |
| Week 12-Tablet to dissolve in liquid — 3 | 7 |
| Week 12-Tablet to dissolve in liquid — 4 | 4 |
| Week 12-Tablet to dissolve in liquid — 5 | 7 |
| Week 12-Tablet to dissolve in liquid — 6 | 0 |
| EOT-Tablet to dissolve in liquid — 1 | 0 |
| EOT-Tablet to dissolve in liquid — 2 | 8 |
| EOT-Tablet to dissolve in liquid — 3 | 8 |
| EOT-Tablet to dissolve in liquid — 4 | 7 |
| EOT-Tablet to dissolve in liquid — 5 | 4 |
| EOT-Tablet to dissolve in liquid — 6 | 1 |
| BL-Tablet (taken once a day) — 1 | 6 |
| BL-Tablet (taken once a day) — 2 | 3 |
| BL-Tablet (taken once a day) — 3 | 15 |
| BL-Tablet (taken once a day) — 4 | 6 |
| BL-Tablet (taken once a day) — 5 | 2 |
| BL-Tablet (taken once a day) — 6 | 0 |
| Week 4-Tablet (taken once a day) — 1 | 3 |
| Week 4-Tablet (taken once a day) — 2 | 0 |
| Week 4-Tablet (taken once a day) — 3 | 9 |
| Week 4-Tablet (taken once a day) — 4 | 18 |
| Week 4-Tablet (taken once a day) — 5 | 0 |
| Week 4-Tablet (taken once a day) — 6 | 0 |
| Week 12-Tablet (taken once a day) — 1 | 1 |
| Week 12-Tablet (taken once a day) — 2 | 0 |
| Week 12-Tablet (taken once a day) — 3 | 11 |
| Week 12-Tablet (taken once a day) — 4 | 13 |
| Week 12-Tablet (taken once a day) — 5 | 1 |
| Week 12-Tablet (taken once a day) — 6 | 0 |
| EOT-Tablet (taken once a day) — 1 | 4 |
| EOT-Tablet (taken once a day) — 2 | 0 |
| EOT-Tablet (taken once a day) — 3 | 11 |
| EOT-Tablet (taken once a day) — 4 | 13 |
| EOT-Tablet (taken once a day) — 5 | 0 |
| EOT-Tablet (taken once a day) — 6 | 0 |
| BL-Tablet (taken 3 times a day) — 1 | 0 |
| BL-Tablet (taken 3 times a day) — 2 | 0 |
| BL-Tablet (taken 3 times a day) — 3 | 0 |
| BL-Tablet (taken 3 times a day) — 4 | 14 |
| BL-Tablet (taken 3 times a day) — 5 | 17 |
| BL-Tablet (taken 3 times a day) — 6 | 1 |
| Week 4-Tablet (taken 3 times a day) — 1 | 0 |
| Week 4-Tablet (taken 3 times a day) — 2 | 1 |
| Week 4-Tablet (taken 3 times a day) — 3 | 1 |
| Week 4-Tablet (taken 3 times a day) — 4 | 6 |
| Week 4-Tablet (taken 3 times a day) — 5 | 21 |
| Week 4-Tablet (taken 3 times a day) — 6 | 1 |
| Week 12-Tablet (taken 3 times a day) — 1 | 0 |
| Week 12-Tablet (taken 3 times a day) — 2 | 2 |
| Week 12-Tablet (taken 3 times a day) — 3 | 1 |
| Week 12-Tablet (taken 3 times a day) — 4 | 6 |
| Week 12-Tablet (taken 3 times a day) — 5 | 17 |
| Week 12-Tablet (taken 3 times a day) — 6 | 0 |
| EOT-Tablet (taken 3 times a day) — 1 | 0 |
| EOT-Tablet (taken 3 times a day) — 2 | 1 |
| EOT-Tablet (taken 3 times a day) — 3 | 1 |
| EOT-Tablet (taken 3 times a day) — 4 | 3 |
| EOT-Tablet (taken 3 times a day) — 5 | 23 |
| EOT-Tablet (taken 3 times a day) — 6 | 0 |
| BL-Tablet crushed — 1 | 12 |
| BL-Tablet crushed — 2 | 14 |
| BL-Tablet crushed — 3 | 3 |
| BL-Tablet crushed — 4 | 3 |
| BL-Tablet crushed — 5 | 0 |
| BL-Tablet crushed — 6 | 0 |
| Week 4-Tablet crushed — 1 | 10 |
| Week 4-Tablet crushed — 2 | 9 |
| Week 4-Tablet crushed — 3 | 11 |
| Week 4-Tablet crushed — 4 | 0 |
| Week 4-Tablet crushed — 5 | 0 |
| Week 4-Tablet crushed — 6 | 0 |
| Week 12-Tablet crushed — 1 | 8 |
| Week 12-Tablet crushed — 2 | 9 |
| Week 12-Tablet crushed — 3 | 6 |
| Week 12-Tablet crushed — 4 | 2 |
| Week 12-Tablet crushed — 5 | 0 |
| Week 12-Tablet crushed — 6 | 1 |
| EOT-Tablet crushed — 1 | 8 |
| EOT-Tablet crushed — 2 | 9 |
| EOT-Tablet crushed — 3 | 8 |
| EOT-Tablet crushed — 4 | 2 |
| EOT-Tablet crushed — 5 | 1 |
| EOT-Tablet crushed — 6 | 0 |
| BL-Sprinkle powder on food — 1 | 16 |
| BL-Sprinkle powder on food — 2 | 13 |
| BL-Sprinkle powder on food — 3 | 1 |
| BL-Sprinkle powder on food — 4 | 1 |
| BL-Sprinkle powder on food — 5 | 1 |
| BL-Sprinkle powder on food — 6 | 0 |
| Week 4-Sprinkle powder on food — 1 | 19 |
| Week 4-Sprinkle powder on food — 2 | 8 |
| Week 4-Sprinkle powder on food — 3 | 0 |
| Week 4-Sprinkle powder on food — 4 | 1 |
| Week 4-Sprinkle powder on food — 5 | 2 |
| Week 4-Sprinkle powder on food — 6 | 0 |
| Week 12-Sprinkle powder on food — 1 | 15 |
| Week 12-Sprinkle powder on food — 2 | 8 |
| Week 12-Sprinkle powder on food — 3 | 1 |
| Week 12-Sprinkle powder on food — 4 | 1 |
| Week 12-Sprinkle powder on food — 5 | 1 |
| Week 12-Sprinkle powder on food — 6 | 0 |
| EOT-Sprinkle powder on food — 1 | 16 |
| EOT-Sprinkle powder on food — 2 | 10 |
| EOT-Sprinkle powder on food — 3 | 0 |
| EOT-Sprinkle powder on food — 4 | 2 |
| EOT-Sprinkle powder on food — 5 | 0 |
| EOT-Sprinkle powder on food — 6 | 0 |
| BL-Injection — 1 | 0 |
| BL-Injection — 2 | 0 |
| BL-Injection — 3 | 0 |
| BL-Injection — 4 | 0 |
| BL-Injection — 5 | 0 |
| BL-Injection — 6 | 32 |
| Week 4-Injection — 1 | 0 |
| Week 4-Injection — 2 | 0 |
| Week 4-Injection — 3 | 0 |
| Week 4-Injection — 4 | 1 |
| Week 4-Injection — 5 | 1 |
| Week 4-Injection — 6 | 28 |
| Week 12-Injection — 1 | 1 |
| Week 12-Injection — 2 | 0 |
| Week 12-Injection — 3 | 0 |
| Week 12-Injection — 4 | 0 |
| Week 12-Injection — 5 | 0 |
| Week 12-Injection — 6 | 25 |
| EOT-Injection — 1 | 0 |
| EOT-Injection — 2 | 0 |
| EOT-Injection — 3 | 0 |
| EOT-Injection — 4 | 1 |
| EOT-Injection — 5 | 0 |
| EOT-Injection — 6 | 27 |
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child's response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver's responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed.
| Score | Deferasirox |
|---|---|
| Child's perspective overall score-Week 4 | 1.1 (0.6 to 1.7) |
| Child's perspective overall score-Week 12 | 1.2 (0.5 to 1.9) |
| Child's perspective overall score-EOT | 0.8 (0.0 to 1.5) |
| Caregiver's perspective overall score-Week 4 | 0.4 (-0.1 to 0.8) |
| Caregiver's perspective overall score-Week 12 | 0.4 (-0.2 to 0.9) |
| Caregiver's perspective overall score-EOT | 0.2 (-0.3 to 0.7) |
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed.
| Score | Deferasirox |
|---|---|
| Child's perspective overall score-Week 4 | 10.4 ± 2.98 |
| Child's perspective overall score-Week 12 | 9.8 ± 2.99 |
| Child's perspective overall score-EOT | 10.3 ± 3.43 |
| Caregiver's perspective overall score-Week 4 | 9.0 ± 3.97 |
| Caregiver's perspective overall score-Week 12 | 9.9 ± 5.30 |
| Caregiver's perspective overall score-EOT | 10.6 ± 5.03 |
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts.
| Participants | Deferasirox |
|---|---|
| Wk 4 — Tablet to dissolve in liquid | 0 |
| Wk 4 — Tablet (taken once a day) | 1 |
| Wk 4 — Tablet (taken 3 times a day) | 0 |
| Wk 4 — Tablet crushed | 5 |
| Wk 4 — Sprinkle powder on food | 3 |
| Wk 4 — Injection | 0 |
| Wk 4 — I don't know | 2 |
| Wk 12 — Tablet to dissolve in liquid | 1 |
| Wk 12 — Tablet (taken once a day) | 0 |
| Wk 12 — Tablet (taken 3 times a day) | 0 |
| Wk 12 — Tablet crushed | 5 |
| Wk 12 — Sprinkle powder on food | 3 |
| Wk 12 — Injection | 0 |
| Wk 12 — I don't know | 0 |
| EOT — Tablet to dissolve in liquid | 4 |
| EOT — Tablet (taken once a day) | 0 |
| EOT — Tablet (taken 3 times a day) | 0 |
| EOT — Tablet crushed | 3 |
| EOT — Sprinkle powder on food | 2 |
| EOT — Injection | 0 |
| EOT — I don't know | 0 |
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts.
| Participants | Deferasirox |
|---|---|
| Wk 4 — Taste | 0 |
| Wk 4 — Aftertaste | 1 |
| Wk 4 — Convenience | 2 |
| Wk 4 — Number of pills | 1 |
| Wk 4 — No/Less side effects | 0 |
| Wk 4 — Can correctly prepare the medicine | 1 |
| Wk 4 — Easier to remember to take the medicine | 1 |
| Wk 4 — Number of times he/she has to take the medicine | 1 |
| Wk 4 — No/Less pain on the injection site | 0 |
| Wk 4 — Gain personal time with their family and friends | 1 |
| Wk 4 — Other | 1 |
| Wk 12 — Taste | 0 |
| Wk 12 — Aftertaste | 0 |
| Wk 12 — Convenience | 2 |
| Wk 12 — Number of pills | 0 |
| Wk 12 — No/Less side effects | 0 |
| Wk 12 — Can correctly prepare the medicine | 0 |
| Wk 12 — Easier to remember to take the medicine | 0 |
| Wk 12 — Number of times he/she has to take the medicine | 0 |
| Wk 12 — No/Less pain on the injection site | 1 |
| Wk 12 — Gain personal time with their family and friends | 2 |
| Wk 12 — Other | 1 |
| EOT — Taste | 0 |
| EOT — Aftertaste | 0 |
| EOT — Convenience | 1 |
| EOT — Number of pills | 0 |
| EOT — No/Less side effects | 1 |
| EOT — Can correctly prepare the medicine | 1 |
| EOT — Easier to remember to take the medicine | 2 |
| EOT — Number of times he/she has to take the medicine | 0 |
| EOT — No/Less pain on the injection site | 1 |
| EOT — Gain personal time with their family and friends | 2 |
| EOT — Other | 0 |
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency counts.
| Participants | Deferasirox |
|---|---|
| Wk 4 -Tablet to dissolve in liquid — 1 | 2 |
| Wk 4 -Tablet to dissolve in liquid — 2 | 1 |
| Wk 4 -Tablet to dissolve in liquid — 3 | 2 |
| Wk 4 -Tablet to dissolve in liquid — 4 | 4 |
| Wk 4 -Tablet to dissolve in liquid — 5 | 1 |
| Wk 4 -Tablet to dissolve in liquid — 6 | 0 |
| Wk 12-Tablet to dissolve in liquid — 1 | 2 |
| Wk 12-Tablet to dissolve in liquid — 2 | 3 |
| Wk 12-Tablet to dissolve in liquid — 3 | 2 |
| Wk 12-Tablet to dissolve in liquid — 4 | 0 |
| Wk 12-Tablet to dissolve in liquid — 5 | 2 |
| Wk 12-Tablet to dissolve in liquid — 6 | 0 |
| EOT-Tablet to dissolve in liquid — 1 | 4 |
| EOT-Tablet to dissolve in liquid — 2 | 2 |
| EOT-Tablet to dissolve in liquid — 3 | 1 |
| EOT-Tablet to dissolve in liquid — 4 | 2 |
| EOT-Tablet to dissolve in liquid — 5 | 0 |
| EOT-Tablet to dissolve in liquid — 6 | 0 |
| Wk 4-Tablet (taken once a day) — 1 | 1 |
| Wk 4-Tablet (taken once a day) — 2 | 3 |
| Wk 4-Tablet (taken once a day) — 3 | 5 |
| Wk 4-Tablet (taken once a day) — 4 | 1 |
| Wk 4-Tablet (taken once a day) — 5 | 0 |
| Wk 4-Tablet (taken once a day) — 6 | 0 |
| Wk 12-Tablet (taken once a day) — 1 | 0 |
| Wk 12-Tablet (taken once a day) — 2 | 0 |
| Wk 12-Tablet (taken once a day) — 3 | 2 |
| Wk 12-Tablet (taken once a day) — 4 | 6 |
| Wk 12-Tablet (taken once a day) — 5 | 1 |
| Wk 12-Tablet (taken once a day) — 6 | 0 |
| EOT-Tablet (taken once a day) — 1 | 0 |
| EOT-Tablet (taken once a day) — 2 | 1 |
| EOT-Tablet (taken once a day) — 3 | 3 |
| EOT-Tablet (taken once a day) — 4 | 5 |
| EOT-Tablet (taken once a day) — 5 | 0 |
| EOT-Tablet (taken once a day) — 6 | 0 |
| Wk 4-Tablet (taken 3 times a day) — 1 | 1 |
| Wk 4-Tablet (taken 3 times a day) — 2 | 0 |
| Wk 4-Tablet (taken 3 times a day) — 3 | 1 |
| Wk 4-Tablet (taken 3 times a day) — 4 | 3 |
| Wk 4-Tablet (taken 3 times a day) — 5 | 4 |
| Wk 4-Tablet (taken 3 times a day) — 6 | 1 |
| Wk 12-Tablet (taken 3 times a day) — 1 | 0 |
| Wk 12-Tablet (taken 3 times a day) — 2 | 1 |
| Wk 12-Tablet (taken 3 times a day) — 3 | 2 |
| Wk 12-Tablet (taken 3 times a day) — 4 | 1 |
| Wk 12-Tablet (taken 3 times a day) — 5 | 5 |
| Wk 12-Tablet (taken 3 times a day) — 6 | 0 |
| EOT-Tablet (taken 3 times a day) — 1 | 0 |
| EOT-Tablet (taken 3 times a day) — 2 | 0 |
| EOT-Tablet (taken 3 times a day) — 3 | 0 |
| EOT-Tablet (taken 3 times a day) — 4 | 0 |
| EOT-Tablet (taken 3 times a day) — 5 | 9 |
| EOT-Tablet (taken 3 times a day) — 6 | 0 |
| Wk 4-Tablet crushed — 1 | 1 |
| Wk 4-Tablet crushed — 2 | 6 |
| Wk 4-Tablet crushed — 3 | 1 |
| Wk 4-Tablet crushed — 4 | 1 |
| Wk 4-Tablet crushed — 5 | 1 |
| Wk 4-Tablet crushed — 6 | 0 |
| Wk 12-Tablet crushed — 1 | 1 |
| Wk 12-Tablet crushed — 2 | 5 |
| Wk 12-Tablet crushed — 3 | 2 |
| Wk 12-Tablet crushed — 4 | 0 |
| Wk 12-Tablet crushed — 5 | 0 |
| Wk 12-Tablet crushed — 6 | 1 |
| EOT-Tablet crushed — 1 | 1 |
| EOT-Tablet crushed — 2 | 4 |
| EOT-Tablet crushed — 3 | 4 |
| EOT-Tablet crushed — 4 | 0 |
| EOT-Tablet crushed — 5 | 0 |
| EOT-Tablet crushed — 6 | 0 |
| Wk 4-Sprinkle powder on food — 1 | 5 |
| Wk 4-Sprinkle powder on food — 2 | 0 |
| Wk 4-Sprinkle powder on food — 3 | 0 |
| Wk 4-Sprinkle powder on food — 4 | 1 |
| Wk 4-Sprinkle powder on food — 5 | 3 |
| Wk 4-Sprinkle powder on food — 6 | 1 |
| Wk 12-Sprinkle powder on food — 1 | 5 |
| Wk 12-Sprinkle powder on food — 2 | 0 |
| Wk 12-Sprinkle powder on food — 3 | 1 |
| Wk 12-Sprinkle powder on food — 4 | 2 |
| Wk 12-Sprinkle powder on food — 5 | 1 |
| Wk 12-Sprinkle powder on food — 6 | 0 |
| EOT-Sprinkle powder on food — 1 | 4 |
| EOT-Sprinkle powder on food — 2 | 2 |
| EOT-Sprinkle powder on food — 3 | 1 |
| EOT-Sprinkle powder on food — 4 | 2 |
| EOT-Sprinkle powder on food — 5 | 0 |
| EOT-Sprinkle powder on food — 6 | 0 |
| Wk 4-Injection — 1 | 0 |
| Wk 4-Injection — 2 | 0 |
| Wk 4-Injection — 3 | 1 |
| Wk 4-Injection — 4 | 0 |
| Wk 4-Injection — 5 | 1 |
| Wk 4-Injection — 6 | 8 |
| Wk 12-Injection — 1 | 1 |
| Wk 12-Injection — 2 | 0 |
| Wk 12-Injection — 3 | 0 |
| Wk 12-Injection — 4 | 0 |
| Wk 12-Injection — 5 | 0 |
| Wk 12-Injection — 6 | 8 |
| EOT-Injection — 1 | 0 |
| EOT-Injection — 2 | 0 |
| EOT-Injection — 3 | 0 |
| EOT-Injection — 4 | 0 |
| EOT-Injection — 5 | 0 |
| EOT-Injection — 6 | 9 |
The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child's response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver's responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed.
| Score | Deferasirox |
|---|---|
| Child's perspective overall score-Week 4 | 9.7 ± 0.65 |
| Child's perspective overall score-Week 12 | 9.4 ± 0.88 |
| Child's perspective overall score-EOT | 8.9 ± 1.36 |
| Caregiver's perspective overall score-Week 4 | 3.7 ± 1.42 |
| Caregiver's perspective overall score-Week 12 | 3.9 ± 1.76 |
| Caregiver's perspective overall score-EOT | 3.4 ± 1.59 |
The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed.
| Score | Deferasirox |
|---|---|
| Week 4 | 10.4 ± 0.97 |
| Week 12 | 10.9 ± 0.33 |
| EOT (Week 24) | 10.8 ± 0.67 |
The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts.
| Participants | Deferasirox |
|---|---|
| Wk 4 — Very Good | 3 |
| Wk 4 — Good | 4 |
| Wk 4 — Neither good nor bad | 3 |
| Wk 4 — Bad | 1 |
| Wk 4 — Very bad | 0 |
| Wk 12 — Very Good | 2 |
| Wk 12 — Good | 5 |
| Wk 12 — Neither good nor bad | 2 |
| Wk 12 — Bad | 0 |
| Wk 12 — Very bad | 0 |
| EOT — Very Good | 1 |
| EOT — Good | 4 |
| EOT — Neither good nor bad | 3 |
| EOT — Bad | 1 |
| EOT — Very bad | 0 |
The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed.
| Score | Deferasirox |
|---|---|
| Week 4 | 10.7 ± 1.29 |
| Week 12 | 10.3 ± 2.13 |
| EOT (Week 24) | 10.6 ± 1.57 |
The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts.
| Participants | Deferasirox |
|---|---|
| BL — Very Good | 5 |
| BL — Good | 5 |
| BL — Neither good nor bad | 15 |
| BL — Bad | 7 |
| BL — Very bad | 0 |
| Wk 4 — Very Good | 1 |
| Wk 4 — Good | 13 |
| Wk 4 — Neither good nor bad | 16 |
| Wk 4 — Bad | 0 |
| Wk 4 — Very bad | 0 |
| Wk 12 — Very Good | 4 |
| Wk 12 — Good | 9 |
| Wk 12 — Neither good nor bad | 12 |
| Wk 12 — Bad | 2 |
| Wk 12 — Very bad | 0 |
| EOT — Very Good | 0 |
| EOT — Good | 11 |
| EOT — Neither good nor bad | 14 |
| EOT — Bad | 3 |
| EOT — Very bad | 0 |
The GI symptom score was calculated from responses to 5 questions, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT.
| Score | Deferasirox |
|---|---|
| Week 2 | 6.8 ± 2.82 |
| Week 3 | 7.1 ± 2.85 |
| Week 4 | 7.0 ± 3.52 |
| Week 8 | 6.8 ± 2.64 |
| Week 12 | 6.2 ± 1.79 |
| Week 16 | 7.0 ± 2.18 |
| Week 20 | 5.9 ± 1.29 |
| EOT (Week 24) | 6.8 ± 1.92 |
The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 ("None"), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = "5 - 10" and 6 = "11 or more". The GI bowel movements item score was presented descriptively using frequency counts.
| Participants | Deferasirox |
|---|---|
| Week 2 — 3 | 1 |
| Week 2 — 4 | 1 |
| Week 2 — 5 to 10 | 8 |
| Week 2 — 11 or more | 2 |
| Week 3 — 3 | 1 |
| Week 3 — 4 | 0 |
| Week 3 — 5 to 10 | 7 |
| Week 3 — 11 or more | 2 |
| Week 4 — 3 | 0 |
| Week 4 — 4 | 1 |
| Week 4 — 5 to 10 | 8 |
| Week 4 — 11 or more | 2 |
| Week 8 — 3 | 0 |
| Week 8 — 4 | 3 |
| Week 8 — 5 to 10 | 6 |
| Week 8 — 11 or more | 2 |
| Week 12 — 3 | 0 |
| Week 12 — 4 | 2 |
| Week 12 — 5 to 10 | 6 |
| Week 12 — 11 or more | 1 |
| Week 16 — 3 | 0 |
| Week 16 — 4 | 2 |
| Week 16 — 5 to 10 | 5 |
| Week 16 — 11 or more | 2 |
| Week 20 — 3 | 0 |
| Week 20 — 4 | 1 |
| Week 20 — 5 to 10 | 7 |
| Week 20 — 11 or more | 2 |
| EOT (Week 24) — 3 | 2 |
| EOT (Week 24) — 4 | 0 |
| EOT (Week 24) — 5 to 10 | 6 |
| EOT (Week 24) — 11 or more | 1 |
The GI symptom score was calculated from responses to 5 questions, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT.
| Score | Deferasirox |
|---|---|
| Week 2 | 6.3 ± 1.91 |
| Week 3 | 6.0 ± 1.63 |
| Week 4 | 6.4 ± 1.81 |
| Week 8 | 6.4 ± 2.34 |
| Week 12 | 6.5 ± 1.83 |
| Week 16 | 6.7 ± 2.10 |
| Week 20 | 7.3 ± 2.37 |
| EOT (Week 24) | 7.6 ± 2.95 |
The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 ("None"), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = "5 - 10" and 6 = "11 or more". The GI bowel movements item score was presented descriptively using frequency counts.
| Participants | Deferasirox |
|---|---|
| Baseline (BL) — 3 | 0 |
| Baseline (BL) — 4 | 1 |
| Baseline (BL) — 5 to 10 | 30 |
| Baseline (BL) — 11 or more | 1 |
| Week 2 — 3 | 0 |
| Week 2 — 4 | 1 |
| Week 2 — 5 to 10 | 29 |
| Week 2 — 11 or more | 2 |
| Week 3 — 3 | 0 |
| Week 3 — 4 | 1 |
| Week 3 — 5 to 10 | 26 |
| Week 3 — 11 or more | 1 |
| Week 4 — 3 | 0 |
| Week 4 — 4 | 1 |
| Week 4 — 5 to 10 | 28 |
| Week 4 — 11 or more | 1 |
| Week 8 — 3 | 1 |
| Week 8 — 4 | 1 |
| Week 8 — 5 to 10 | 29 |
| Week 8 — 11 or more | 1 |
| Week 12 — 3 | 1 |
| Week 12 — 4 | 0 |
| Week 12 — 5 to 10 | 26 |
| Week 12 — 11 or more | 0 |
| Week 16 — 3 | 0 |
| Week 16 — 4 | 1 |
| Week 16 — 5 to 10 | 29 |
| Week 16 — 11 or more | 1 |
| Week 20 — 3 | 0 |
| Week 20 — 4 | 1 |
| Week 20 — 5 to 10 | 28 |
| Week 20 — 11 or more | 2 |
| EOT (Week 24) — 3 | 0 |
| EOT (Week 24) — 4 | 1 |
| EOT (Week 24) — 5 to 10 | 25 |
| EOT (Week 24) — 11 or more | 2 |
Collected over Treatment-emergent adverse events (TEAEs) were collected from first dose of study treatment until end of study treatment plus 30 days post-treatment, assessed for approximately 28 weeks.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Deferasirox | 0/44 (0%) | 2/44 (4.5%) | 29/44 (65.9%) |
| Event | Deferasirox |
|---|---|
| PyrexiaGeneral disorders | 1/44 |
| BronchitisInfections and infestations | 1/44 |
| Event | Deferasirox |
|---|---|
| Urine protein/creatinine ratio increasedInvestigations | 12/44 |
| Upper respiratory tract infectionInfections and infestations | 10/44 |
| Blood creatinine increasedInvestigations | 9/44 |
| Alanine aminotransferase increasedInvestigations | 7/44 |
| Bilirubin conjugated increasedInvestigations | 6/44 |
| CoughRespiratory, thoracic and mediastinal disorders | 6/44 |
| PyrexiaGeneral disorders | 5/44 |
| DiarrhoeaGastrointestinal disorders | 4/44 |
| GastroenteritisInfections and infestations | 4/44 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 3/44 |
| Age, Continuous(Years) | Deferasirox |
|---|---|
| Mean | 3.05 ± 1.056 |
| Sex: Female, Male(Participants) | Deferasirox |
|---|---|
| Female | 17 |
| Male | 27 |
| Race/Ethnicity, Customized(Participants) | Deferasirox |
|---|---|
| Caucasian | 36 |
| Asian | 8 |
| Baseline Body Mass Index(kilogram per square metre (kg/m^2)) | Deferasirox |
|---|---|
| Mean | 16.40 ± 2.009 |
| Baseline iron chelation naive participants(Participants) | Deferasirox |
|---|---|
| Chelation naive = Yes | 10 |
| Chelation naive = No | 34 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Ran from Jan 2018 to primary completion in Dec 2019, 22 months.
Results were first posted in Aug 2020, 7 months after primary completion.
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Studies of the drug tested here: Deferasirox