A Phase 2 interventional study of Deferasirox granule formulation and Deferasirox DT formulation in Transfusion-dependent Anemia, sponsored by Novartis Pharmaceuticals. Completed at 43 sites in 17 countries. Open to participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2024-09-19.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 2, Interventional, and Treatment
This was a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin of a deferasirox granule formulation and a deferasirox dispersible tablet (DT) formulation in children and adolescents aged ≥ 2 and \< 18 years at enrolment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, to demonstrate the effect of improved compliance on iron burden.
Randomization was stratified by age groups (2 to \<10 years, 10 to \<18 years) and prior iron chelation therapy (Yes/ No). There were two study phases which include a 1 year core phase where participants were randomized to a 48 week treatment period to either Deferasirox DT or granules, and an optional extension phase where all participants received the granules up to 5 years. Participants who demonstrated benefit to granules or DT in the core phase, and/or expressed the wish to continue in the optional extension phase on granules, were offered this possibility until there was local access to the new formulation (granules or film-coated tablet (FCT)) or up to 5 years, whichever occurred first.
156 studies on the registry are indexed under Iron Overload; 6 are open to participants now.
This study's enrollment of 224 is above 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.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
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.
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.
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.
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.
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.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
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.
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.
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.
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.
Participants will be administered deferasirox dispersible tablets orally once daily based on body weight for 48 weeks.
Drug: Deferasirox DT formulation
Participants will be administered deferasirox granules orally once daily in the form of stick packs based on body weight for 48 weeks.
Drug: Deferasirox granule formulation
Deferasirox granules will be provided as stick packs containing 90 mg, 180 mg and 360 mg granules for oral use and will be administered based on body weight.
Also known as: ICL670
Deferasirox DT will be provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use and will be administered based on body weight.
Also known as: ICL670
Percentage of Overall Compliance Using Stick Pack or Tablet Counts in Iron Chelation Therapy (ICT)-naïve Participants During the Core Phase
Compliance was calculated as the ratio of total count consumed to total count prescribed of deferasirox granule stick packs or dispersible tablets, where total count consumed was derived from cumulative dispensed, returned and lost/wasted counts over 24 weeks of treatment and total count prescribed was derived from cumulative prescribed count over 24 weeks of treatment.
Time frame: 24 weeks
Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase
The analysis included the comparison of means between the two treatment arms of change from baseline after 24 weeks of treatment in serum ferritin in pediatric ICT naïve participants with iron overload. The endpoint was assessed at Week 25 visit.
Time frame: From Baseline to Week 25
Percentage of Overall Compliance Using Stick Pack or Tablet Counts in ICT-naïve Participants During the Core Phase
Compliance was calculated as the ratio of total count consumed to total count prescribed of deferasirox granule stick packs or dispersible tablets over 48 weeks of treatment.
Time frame: 48 weeks
Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase
The analysis included the comparison of means between the two treatment arms of change from baseline after 48 weeks of treatment in serum ferritin in pediatric ICT naïve participants with iron overload.
Time frame: From Baseline to 48 weeks
Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in Pre-treated Participants During the Core Phase
The analysis included the comparison of means between the two treatment arms of change from baseline after 25 weeks and after 48 weeks of treatment in serum ferritin in pre-treated participants. The analyses were performed at Week 25 and Week 48.
Time frame: From Baseline to Week 25 and Week 48
Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Patient Reported Outcomes (PRO) Questionnaires
Participants aged between 10 years and less than 18 years at enrollment completed PRO questionnaires by themselves. The mSICT questionnaire for PRO consisted of 3 domains: adherence, satisfaction/preference, and concerns. The adherence domain had a minimum score of 6 and maximum score of 30; a lower score for adherence indicates better adherence. Satisfaction/preference domain had a minimum score of 2 and maximum score of 10; a lower score for satisfaction/preference indicates better satisfaction/preference. Concerns domain had a minimum score of 3 and maximum score of 15; a higher score for concerns indicate fewer concerns.
Time frame: At Week 2, Week 3, Week 25 and Week 48
Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Observer Reported Outcomes (ObsRO) Questionnaire (Caregiver's Perspective)
The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses. The mSICT questionnaire consisted of 2 domains: adherence and concerns per caregiver's perspective. The adherence domain had a minimum score of 5 and a maximum score of 25; a lower score for adherence indicates better adherence. The concerns domain had a minimum score of 1 and a maximum score of 5; a higher score for concerns indicates fewer concerns.
Time frame: At Week 2, Week 3, Week 25 and Week 48
Change Over-time in Domain Score of Modified Satisfaction With Iron Chelation Therapy (mSICT) Using Observer Reported Outcomes (ObsRO) Questionnaire (Child's Perspective)
The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses. The mSICT questionnaire is presented for 2 domains: adherence and concerns per child's perspective. The adherence domain had a minimum score of 6 and a maximum score of 30; a lower score for adherence indicates better adherence. The concerns domain had a minimum score of 2 and a maximum score of 10; a higher score for concerns indicates fewer concerns.
Time frame: At Week 2, Week 3, Week 25 and Week 48
Change Over-time in Domain Score of Palatability Using Patient Reported Outcomes (PRO) Questionnaires
The palatability questionnaire was used to measure: taste, aftertaste, whether medication was taken and how the participant perceived the amount of medication taken. This questionnaire had a minimum score of 0 and maximum score of 11; a higher score means better palatability. Participants aged between 10 years and less than 18 years at enrollment completed the PRO questionnaire by themselves.
Time frame: At Week 2, Week 3, Week 25 and Week 48
Change Over-time in Domain Score of Palatability Using Observer Reported Outcomes (ObsRO) Questionnaire
The palatability questionnaire was used to measure: taste, aftertaste, whether medication was taken and how the participant perceived the amount of medication taken. This questionnaire had a minimum score of 0 and maximum score of 11; a higher score means better palatability. The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses.
Time frame: At Week 2, Week 3, Week 25 and Week 48
Change Over Time in Weekly Dose Violation Rate Using Compliance Patient Reported Outcomes (PRO) Questionnaire
The compliance questionnaire consisted of 2 items: 1. To assess if the medication was taken (yes/no) and 2. To record of the time when the medication was taken (with a not applicable option for participants who did not take their medication). Daily diary records were used to calculate the rate of dose violation in each study arm (doses missed completely or not taken before 12 PM). The dose violation rate was calculated as: \[Number of dose violations / Drug exposure (days)\] \*100. Higher values represent more dose violations.
Time frame: At Week 1, Week 13, Week 25, Week 37 and Week 48
Change Over Time in Weekly Dose Violation Rate Using Compliance Observer Reported Outcomes (ObsRO) Questionnaire
The compliance questionnaire consisted of 2 items: 1. To assess if the medication was taken (yes/no) and 2. To record the time when the medication was taken (with a not applicable option for participants who did not take their medication). Daily diary records were used to calculate the rate of dose violation in each treatment arm (doses missed completely or not taken before 12 PM). The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses. The dose violation rate was calculated as: \[Number of dose violations / Drug exposure (days)\] \*100. Higher values represent more dose violations.
Time frame: At Week 1, Week 13, Week 25, Week 37 and Week 48
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) During the Core Phase
An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.
Time frame: From Baseline to 48 weeks
Pre-dose Concentrations of Deferasirox to Support the Assessment of Compliance
Pre-dose pharmacokinetic (PK) data from participants in the Pharmacokinetic Analysis Set 1 (PAS-1) were analyzed to assess variability of individual participant's compliance. A linear mixed effect power model to pre-dose samples which fulfill compliance criteria in terms of steady state (4 consecutive same doses prior to the PK sample drawn), time-windows (PK sample drawn 20 to 28 hours after previous dose) and without any vomiting episodes within the 4 hours prior to the PK sample were fitted. The model considered dose, treatment group, stratification factors and potential other factors, such as body weight as covariates.
Time frame: At Weeks 1, 3, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, and 45
Concentrations of Deferasirox Between 2 and 4 Hours Post-dose at Weeks 5 and 9
Post-dose pharmacokinetic (PK) data from participants in the Pharmacokinetic Analysis Set 1 (PAS-1) were analyzed along with Pre-dose PK data.
Time frame: At Week 5 and Week 9
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) During the Entire Granule Period
An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation after participant providing written informed consent for participation in the study. In the DFX Granules arm, AEs are reported since the initial randomization to the arm in the core phase and continuing in the extension phase. In the DFX cross-over arm, AEs are reported for participants since the participant crossed-over from dispersible tablet to granules in the extension phase only.
Time frame: From Baseline to 305 weeks
Number of Participants With Adverse Events of Special Interest (AESI) During the Entire Granule Period
An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation after participant providing written informed consent for participation in the study. In the DFX Granules arm, AEs are reported since the initial randomization to the arm in the core phase and continuing in the extension phase. In the DFX cross-over arm, AEs are reported for participants since the participant crossed-over from dispersible tablet to granules in the extension phase only. AESI included active monitoring for renal toxicity; including renal failure, hepatic toxicity; including hepatic failure, and gastrointestinal hemorrhage
Time frame: From Baseline to 305 weeks
Exposure-Response Relationship in Relation to Pre- and Post-Dose Deferasirox Concentrations (PK/PD Relationship)
This outcome measure explores exposure-response relationships for measures of safety and effectiveness through serum creatinine change from baseline, notable serum creatinine values, serum creatinine clearance change form baseline and notable serum creatinine clearance categories, serum ferritin change from baseline, in relationship to derived PK parameters for pre- and post-dose deferasirox concentrations.
Time frame: From Baseline to 48 weeks
| Milestone | DFX DT | DFX Granule |
|---|---|---|
| Started | 112 | 112 |
| Completed | 87 | 99 |
| Not completed | 25 | 13 |
| Withdrew: Adverse event | 8 | 5 |
| Withdrew: Withdrawal by subject | 3 | 0 |
| Withdrew: Withdrawal by parent/guardian | 9 | 4 |
| Withdrew: Protocol violation | 1 | 2 |
| Withdrew: Physician decision | 3 | 0 |
| Withdrew: Lost to follow-up | 0 | 1 |
| Withdrew: Lack of efficacy | 0 | 1 |
| Withdrew: Recovery | 1 | 0 |
| Milestone | DFX DT | DFX Granule |
|---|---|---|
| Started | 69 | 77 |
| Completed | 42 | 46 |
| Not completed | 27 | 31 |
| Withdrew: Adverse event | 3 | 9 |
| Withdrew: Physician decision | 6 | 8 |
| Withdrew: Withdrawal by parent/guardian | 10 | 7 |
| Withdrew: Lack of efficacy | 2 | 3 |
| Withdrew: Withdrawal by subject | 2 | 2 |
| Withdrew: Protocol violation | 0 | 1 |
| Withdrew: Recovery | 1 | 1 |
| Withdrew: Death | 1 | 0 |
| Withdrew: Technical problems | 2 | 0 |
Compliance was calculated as the ratio of total count consumed to total count prescribed of deferasirox granule stick packs or dispersible tablets, where total count consumed was derived from cumulative dispensed, returned and lost/wasted counts over 24 weeks of treatment and total count prescribed was derived from cumulative prescribed count over 24 weeks of treatment.
| percentage of compliance | DFX DT | DFX Granule |
|---|---|---|
| Percentage of Overall Compliance Using Stick Pack or Tablet Counts in Iron Chelation Therapy (ICT)-naïve Participants During the Core Phase | 89.45 (85.29 to 93.61) | 91.78 (87.81 to 95.75) |
The analysis included the comparison of means between the two treatment arms of change from baseline after 24 weeks of treatment in serum ferritin in pediatric ICT naïve participants with iron overload. The endpoint was assessed at Week 25 visit.
| μg/L | DFX DT | DFX Granule |
|---|---|---|
| Baseline | 2063.7 (1807.17 to 2320.15) | 1955.5 (1712.57 to 2198.49) |
| Week 25 | 2216.3 (1929.59 to 2502.93) | 2228.4 (2011.69 to 2445.18) |
| Change from Baseline to Week 25 | 250.5 (-84.63 to 585.58) | 340.0 (115.48 to 564.59) |
Compliance was calculated as the ratio of total count consumed to total count prescribed of deferasirox granule stick packs or dispersible tablets over 48 weeks of treatment.
| percentage of compliance | DFX DT | DFX Granule |
|---|---|---|
| Percentage of Overall Compliance Using Stick Pack or Tablet Counts in ICT-naïve Participants During the Core Phase | 91.57 (87.65 to 95.49) | 94.80 (91.48 to 98.13) |
The analysis included the comparison of means between the two treatment arms of change from baseline after 48 weeks of treatment in serum ferritin in pediatric ICT naïve participants with iron overload.
| μg/L | DFX DT | DFX Granule |
|---|---|---|
| Change From Baseline in Serum Ferritin (SF) for Both Study Drug Formulations in ICT naïve Participants During the Core Phase | 305.8 (4.28 to 607.24) | 317.0 (69.10 to 564.88) |
The analysis included the comparison of means between the two treatment arms of change from baseline after 25 weeks and after 48 weeks of treatment in serum ferritin in pre-treated participants. The analyses were performed at Week 25 and Week 48.
| μg/L | DFX DT | DFX Granule |
|---|---|---|
| Change from Baseline to Week 25 | 59.0 (-210.88 to 328.79) | 150.3 (-59.43 to 360.01) |
| Change from Baseline to Week 48 | 207.7 (-94.29 to 509.68) | 215.7 (-50.47 to 481.95) |
Participants aged between 10 years and less than 18 years at enrollment completed PRO questionnaires by themselves. The mSICT questionnaire for PRO consisted of 3 domains: adherence, satisfaction/preference, and concerns. The adherence domain had a minimum score of 6 and maximum score of 30; a lower score for adherence indicates better adherence. Satisfaction/preference domain had a minimum score of 2 and maximum score of 10; a lower score for satisfaction/preference indicates better satisfaction/preference. Concerns domain had a minimum score of 3 and maximum score of 15; a higher score for concerns indicate fewer concerns.
| score on a scale | DFX DT | DFX Granule |
|---|---|---|
| Adherence (Week 2) | 9.5 ± 2.30 | 7.6 ± 1.99 |
| Adherence (Week 3) | 10.9 ± 4.95 | 6.6 ± 0.79 |
| Adherence (Week 25) | 11.9 ± 3.93 | 9.2 ± 3.31 |
| Adherence (Week 48) | 12.9 ± 4.17 | 8.4 ± 2.29 |
| Satisfaction/preference (Week 2) | 5.2 ± 2.09 | 2.9 ± 1.36 |
| Satisfaction/preference (Week 3) | 4.0 ± 1.32 | 3.1 ± 1.22 |
| Satisfaction/preference (Week 25) | 5.5 ± 2.37 | 3.0 ± 1.10 |
| Satisfaction/preference (Week 48) | 4.8 ± 2.24 | 3.1 ± 0.92 |
| Concerns (Week 2) | 13.1 ± 2.18 | 14.5 ± 1.06 |
| Concerns (Week 3) | 13.4 ± 2.10 | 14.4 ± 0.80 |
| Concerns (Week 25) | 11.5 ± 3.13 | 14.5 ± 1.21 |
| Concerns (Week 48) | 12.8 ± 2.20 | 13.5 ± 2.75 |
The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses. The mSICT questionnaire consisted of 2 domains: adherence and concerns per caregiver's perspective. The adherence domain had a minimum score of 5 and a maximum score of 25; a lower score for adherence indicates better adherence. The concerns domain had a minimum score of 1 and a maximum score of 5; a higher score for concerns indicates fewer concerns.
| score on a scale | DFX DT | DFX Granule |
|---|---|---|
| Adherence (Week 2) | 7.7 ± 2.84 | 5.8 ± 1.32 |
| Adherence (Week 3) | 7.8 ± 2.64 | 5.8 ± 1.43 |
| Adherence (Week 25) | 7.1 ± 2.35 | 6.5 ± 1.69 |
| Adherence (Week 48) | 7.5 ± 2.53 | 6.8 ± 2.57 |
| Concerns (Week 2) | 3.9 ± 1.32 | 4.5 ± 0.94 |
| Concerns (Week 3) | 4.1 ± 1.31 | 4.7 ± 0.71 |
| Concerns (Week 25) | 4.3 ± 1.00 | 4.5 ± 0.89 |
| Concerns (Week 48) | 4.0 ± 1.24 | 4.6 ± 0.77 |
The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses. The mSICT questionnaire is presented for 2 domains: adherence and concerns per child's perspective. The adherence domain had a minimum score of 6 and a maximum score of 30; a lower score for adherence indicates better adherence. The concerns domain had a minimum score of 2 and a maximum score of 10; a higher score for concerns indicates fewer concerns.
| score on a scale | DFX DT | DFX Granule |
|---|---|---|
| Adherence (Week 2) | 12.1 ± 4.57 | 8.2 ± 2.28 |
| Adherence (Week 3) | 11.7 ± 3.78 | 8.2 ± 2.64 |
| Adherence (Week 25) | 11.1 ± 3.82 | 9.1 ± 2.64 |
| Adherence (Week 48) | 11.3 ± 3.99 | 9.1 ± 3.00 |
| Concerns (Week 2) | 8.5 ± 2.29 | 9.2 ± 1.66 |
| Concerns (Week 3) | 8.7 ± 2.00 | 8.8 ± 2.04 |
| Concerns (Week 25) | 8.6 ± 1.94 | 8.7 ± 1.85 |
| Concerns (Week 48) | 8.8 ± 1.75 | 9.0 ± 1.82 |
The palatability questionnaire was used to measure: taste, aftertaste, whether medication was taken and how the participant perceived the amount of medication taken. This questionnaire had a minimum score of 0 and maximum score of 11; a higher score means better palatability. Participants aged between 10 years and less than 18 years at enrollment completed the PRO questionnaire by themselves.
| score on a scale | DFX DT | DFX Granule |
|---|---|---|
| Week 2 | 8.8 ± 3.32 | 10.3 ± 1.91 |
| Week 3 | 9.6 ± 2.83 | 10.9 ± 0.24 |
| Week 25 | 9.2 ± 2.70 | 10.4 ± 1.80 |
| Week 48 | 9.4 ± 3.07 | 11.0 ± 0.00 |
The palatability questionnaire was used to measure: taste, aftertaste, whether medication was taken and how the participant perceived the amount of medication taken. This questionnaire had a minimum score of 0 and maximum score of 11; a higher score means better palatability. The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses.
| score on a scale | DFX DT | DFX Granule |
|---|---|---|
| Week 2 | 8.9 ± 3.13 | 10.9 ± 0.90 |
| Week 3 | 9.4 ± 2.88 | 10.8 ± 0.79 |
| Week 25 | 9.3 ± 2.83 | 10.6 ± 1.73 |
| Week 48 | 9.0 ± 3.11 | 10.9 ± 0.96 |
The compliance questionnaire consisted of 2 items: 1. To assess if the medication was taken (yes/no) and 2. To record of the time when the medication was taken (with a not applicable option for participants who did not take their medication). Daily diary records were used to calculate the rate of dose violation in each study arm (doses missed completely or not taken before 12 PM). The dose violation rate was calculated as: \[Number of dose violations / Drug exposure (days)\] \*100. Higher values represent more dose violations.
| percentage of days with dose violations | DFX DT | DFX Granule |
|---|---|---|
| Week 1 | 26.86 ± 37.966 | 26.79 ± 38.992 |
| Week 13 | 12.78 ± 31.000 | 23.41 ± 35.906 |
| Week 25 | 13.19 ± 29.614 | 25.93 ± 42.583 |
| Week 37 | 18.89 ± 32.745 | 30.16 ± 41.921 |
| Week 48 | 2.86 ± 7.559 | 52.38 ± 52.424 |
The compliance questionnaire consisted of 2 items: 1. To assess if the medication was taken (yes/no) and 2. To record the time when the medication was taken (with a not applicable option for participants who did not take their medication). Daily diary records were used to calculate the rate of dose violation in each treatment arm (doses missed completely or not taken before 12 PM). The ObsRO questionnaires for participants aged between 2 years and less than 10 years were designed as observations made by caregivers such as the parent or legal guardian. The caregivers continued completing the ObsRO questionnaires even after the participant turned 10 years for consistency in responses. The dose violation rate was calculated as: \[Number of dose violations / Drug exposure (days)\] \*100. Higher values represent more dose violations.
| percentage of days with dose violations | DFX DT | DFX Granule |
|---|---|---|
| Week 1 | 18.80 ± 29.912 | 27.79 ± 37.577 |
| Week 13 | 10.38 ± 27.374 | 18.65 ± 35.513 |
| Week 25 | 7.79 ± 23.025 | 13.72 ± 31.813 |
| Week 37 | 13.86 ± 31.769 | 20.01 ± 37.632 |
| Week 48 | 13.56 ± 32.211 | 14.50 ± 31.123 |
An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.
| Participants | DFX DT - Core Phase | DFX Granule - Core Phase |
|---|---|---|
| All AEs | 108 | 100 |
| All SAEs | 23 | 27 |
Pre-dose pharmacokinetic (PK) data from participants in the Pharmacokinetic Analysis Set 1 (PAS-1) were analyzed to assess variability of individual participant's compliance. A linear mixed effect power model to pre-dose samples which fulfill compliance criteria in terms of steady state (4 consecutive same doses prior to the PK sample drawn), time-windows (PK sample drawn 20 to 28 hours after previous dose) and without any vomiting episodes within the 4 hours prior to the PK sample were fitted. The model considered dose, treatment group, stratification factors and potential other factors, such as body weight as covariates.
| μmol/L | DFX DT | DFX Granule |
|---|---|---|
| Week 1 | 2.93 ± 369.5 | 1.40 ± 235.4 |
| Week 3 | 14.2 ± 137.0 | 11.8 ± 117.1 |
| Week 5 | 14.4 ± 115.7 | 11.7 ± 127.5 |
| Week 9 | 20.1 ± 115.8 | 12.1 ± 97.1 |
| Week 13 | 19.7 ± 124.2 | 13.5 ± 107.5 |
| Week 17 | 18.4 ± 109.4 | 13.1 ± 135.3 |
| Week 21 | 19.6 ± 120.4 | 13.4 ± 163.6 |
| Week 25 | 17.1 ± 147.2 | 15.9 ± 108.6 |
| Week 29 | 23.8 ± 111.3 | 13.2 ± 156.6 |
| Week 33 | 21.4 ± 184.0 | 14.1 ± 175.6 |
| Week 37 | 20.8 ± 137.1 | 14.6 ± 135.3 |
| Week 41 | 21.8 ± 131.6 | 15.5 ± 134.7 |
| Week 45 | 28.3 ± 141.2 | 19.0 ± 117.0 |
Post-dose pharmacokinetic (PK) data from participants in the Pharmacokinetic Analysis Set 1 (PAS-1) were analyzed along with Pre-dose PK data.
| μmol/L | DFX DT | DFX Granule |
|---|---|---|
| Week 5 (3 hour post-dose) | 65.2 ± 80.5 | 53.2 ± 86.2 |
| Week 9 (3 hour post-dose) | 70.4 ± 77.0 | 59.8 ± 61.7 |
An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation after participant providing written informed consent for participation in the study. In the DFX Granules arm, AEs are reported since the initial randomization to the arm in the core phase and continuing in the extension phase. In the DFX cross-over arm, AEs are reported for participants since the participant crossed-over from dispersible tablet to granules in the extension phase only.
| Participants | DFX DT Cross-over Granule - Optional Extension Phase | DFX Granules - Core and Optional Extension Phase |
|---|---|---|
| AEs | 64 | 106 |
| Suspected AEs | 48 | 73 |
| SAEs | 22 | 38 |
| Suspected SAEs | 4 | 7 |
An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation after participant providing written informed consent for participation in the study. In the DFX Granules arm, AEs are reported since the initial randomization to the arm in the core phase and continuing in the extension phase. In the DFX cross-over arm, AEs are reported for participants since the participant crossed-over from dispersible tablet to granules in the extension phase only. AESI included active monitoring for renal toxicity; including renal failure, hepatic toxicity; including hepatic failure, and gastrointestinal hemorrhage
| Participants | DFX DT Cross-over Granule - Optional Extension Phase | DFX Granules - Core and Optional Extension Phase |
|---|---|---|
| Any AESI | 52 | 79 |
| Gastrointestinal hemorrhages | 3 | 4 |
| Hearing loss | 5 | 5 |
| Lens opacities, Retinal changes and Optic neuritis | 0 | 2 |
| Liver disorders - Hepatic failure | 1 | 0 |
| Liver disorders - Increased liver transaminases | 16 | 46 |
| Peripheral blood cytopenias | 5 | 7 |
| Renal disorders - Acute renal failure | 1 | 0 |
| Renal disorders - Increased serum creatinine | 4 | 8 |
| Renal disorders -Proteinuria | 40 | 49 |
| Renal disorders - Renal tubular disorders | 4 | 0 |
| Severe Cutaneous Adverse Reactions (SCARs) | 0 | 1 |
This outcome measure explores exposure-response relationships for measures of safety and effectiveness through serum creatinine change from baseline, notable serum creatinine values, serum creatinine clearance change form baseline and notable serum creatinine clearance categories, serum ferritin change from baseline, in relationship to derived PK parameters for pre- and post-dose deferasirox concentrations.
Results for this outcome have not been posted.
Collected over From initial randomization up to 48 weeks (Core phase) and Up to 5 years from entering the extension phase (Optional Extension phase). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| DFX DT- Core Phase | 0/111 (0%) | 23/111 (20.7%) | 104/111 (93.7%) |
| DFX DT Cross-over Granule - Optional Extension Phase | 1/69 (1.4%) | 22/69 (31.9%) | 63/69 (91.3%) |
| DFX Granules - Core Phase | 0/110 (0%) | 27/110 (24.5%) | 97/110 (88.2%) |
| DFX Granules - Optional Extension Phase | 0/77 (0%) | 20/77 (26%) | 63/77 (81.8%) |
| Event | DFX DT- Core Phase | DFX DT Cross-over Granule - Optional Extension Phase | DFX Granules - Core Phase | DFX Granules - Optional Extension Phase |
|---|---|---|---|---|
| GastroenteritisInfections and infestations | 0/111 | 4/69 | 2/110 | 4/77 |
| Sickle cell anaemia with crisisBlood and lymphatic system disorders | 5/111 | 0/69 | 3/110 | 2/77 |
| Fanconi syndrome acquiredRenal and urinary disorders | 1/111 | 3/69 | 0/110 | 0/77 |
| CholelithiasisHepatobiliary disorders | 1/111 | 0/69 | 0/110 | 3/77 |
| PyrexiaGeneral disorders | 4/111 | 1/69 | 4/110 | 2/77 |
| PharyngitisInfections and infestations | 0/111 | 2/69 | 0/110 | 1/77 |
| SinusitisInfections and infestations | 0/111 | 2/69 | 0/110 | 0/77 |
| TonsillitisInfections and infestations | 2/111 | 2/69 | 0/110 | 1/77 |
| Upper respiratory tract infectionInfections and infestations | 0/111 | 2/69 | 0/110 | 2/77 |
| Haemoglobin decreasedInvestigations | 1/111 | 2/69 | 0/110 | 0/77 |
| Event | DFX DT- Core Phase | DFX DT Cross-over Granule - Optional Extension Phase | DFX Granules - Core Phase | DFX Granules - Optional Extension Phase |
|---|---|---|---|---|
| Urine protein/creatinine ratio increasedInvestigations | 39/111 | 36/69 | 27/110 | 28/77 |
| Upper respiratory tract infectionInfections and infestations | 34/111 | 23/69 | 31/110 | 24/77 |
| PyrexiaGeneral disorders | 24/111 | 15/69 | 26/110 | 16/77 |
| NasopharyngitisInfections and infestations | 12/111 | 14/69 | 11/110 | 11/77 |
| CoughRespiratory, thoracic and mediastinal disorders | 12/111 | 14/69 | 15/110 | 9/77 |
| Alanine aminotransferase increasedInvestigations | 15/111 | 11/69 | 20/110 | 11/77 |
| PharyngitisInfections and infestations | 3/111 | 12/69 | 11/110 | 7/77 |
| Bilirubin conjugated increasedInvestigations | 16/111 | 2/69 | 12/110 | 3/77 |
| VomitingGastrointestinal disorders | 15/111 | 9/69 | 9/110 | 4/77 |
| DiarrhoeaGastrointestinal disorders | 14/111 | 7/69 | 9/110 | 6/77 |
Full Analysis Set (FAS) was defined according to the Intent to Treat (ITT) principle, and included all randomized participants.
| Age, Continuous(years) | DFX DT | DFX Granule | Total |
|---|---|---|---|
| Mean | 5.8 ± 3.89 | 5.9 ± 3.94 | 5.9 ± 3.90 |
| Sex/Gender, Customized(Participants) | DFX DT | DFX Granule | Total |
|---|---|---|---|
| Female | 54 | 56 | 110 |
| Male | 58 | 56 | 114 |
| Ethnicity (NIH/OMB)(Participants) | DFX DT | DFX Granule | Total |
|---|---|---|---|
| Hispanic or Latino | 7 | 7 | 14 |
| Not Hispanic or Latino | 104 | 105 | 209 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Race (NIH/OMB)(Participants) | DFX DT | DFX Granule | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 38 | 44 | 82 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 11 | 8 | 19 |
| White | 57 | 52 | 109 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 6 | 8 | 14 |
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
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Studies of the drug tested here: Deferasirox