A Phase 4 interventional study of Deferiprone oral solution and Placebo in Beta Thalassemia Major Anemia and Iron Overload, sponsored by Chiesi Canada Corp. Completed at 4 sites in 2 countries. Open to participants aged 6 Months to 9 Years. Per ClinicalTrials.gov, last updated 2024-03-15.
Sponsored by Chiesi Canada Corp (part of Chiesi Farmaceutici S.p.A.) · Phase 4, Interventional, and Treatment
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on iron chelation therapy. Half the patients in the study will receive deferiprone, and the other half will receive placebo, for up to 12 months.
This study will give deferiprone to infants and young children with thalassemia who have started receiving regular blood transfusions but whose iron load is not yet at the level where chelation treatment would normally begin. The purpose is to see if doing this will postpone the build-up of iron without causing serious side effects. Half the children in the study will be given deferiprone at a dose that is lower than what is normally prescribed, and the other half will be given placebo. All patients will receive the assigned product three times a day for up to 12 months. Tests for signs of iron overload will be done monthly, and a patient whose iron load reaches the level where chelation therapy would normally begin will be immediately taken out of the study and started on standard chelation therapy.
156 studies on the registry are indexed under Iron Overload; 6 are open to participants now.
This study's enrollment of 64 is close to the median of 60 across 97 interventional studies indexed under Iron Overload.
Browse Iron Overload studies →Chiesi Canada Corp is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
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.
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.
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.
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.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
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.
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.
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.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Subjects in this group will receive deferiprone oral solution at a dosage up to 75 milligrams per kilogram of body weight (mg/kg) per day, divided into 3 equal doses
Drug: Deferiprone oral solution
Subjects in this group will receive placebo solution at a volume equal to what they would receive if they were in the active arm, divided into 3 equal doses
Drug: Placebo
Liquid formulation of deferiprone, with a concentration of 80 mg/mL
Also known as: Ferriprox
Liquid solution that matches deferiprone oral solution in appearance and taste
Also known as: Placebo for deferiprone oral solution
The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12
Current treatment guidelines are that standard iron chelation therapy should begin after a serum ferritin level of 1000 μg/L has been reached; thus, patients who were at this level at two consecutive visits were removed from the study so that they could start on this therapy.
Time frame: 12 months
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points
The outcome measure was the percentage of patients in each group still below the serum ferritin threshold at Months 4, 8, and 12. Without adequate chelation therapy, most patients receiving red blood cell transfusions are likely to exceed this level within a few months. However, a safety feature of the study design was that patients who reached the serum ferritin threshold were withdrawn (i.e., so that they could begin standard chelation therapy).
Time frame: 4, 8, and 12 months
| Milestone | Deferiprone | Placebo |
|---|---|---|
| Started | 32 | 32 |
| Completed | 26 | 31 |
| Not completed | 6 | 1 |
| Withdrew: Adverse event | 2 | 0 |
| Withdrew: Withdrawal by subject | 4 | 1 |
Current treatment guidelines are that standard iron chelation therapy should begin after a serum ferritin level of 1000 μg/L has been reached; thus, patients who were at this level at two consecutive visits were removed from the study so that they could start on this therapy.
| Participants | Deferiprone | Placebo |
|---|---|---|
| The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12 | 21 | 12 |
The outcome measure was the percentage of patients in each group still below the serum ferritin threshold at Months 4, 8, and 12. Without adequate chelation therapy, most patients receiving red blood cell transfusions are likely to exceed this level within a few months. However, a safety feature of the study design was that patients who reached the serum ferritin threshold were withdrawn (i.e., so that they could begin standard chelation therapy).
| Participants | Deferiprone | Placebo |
|---|---|---|
| Number of patients still below threshold at Month 4 | 29 | 28 |
| Number of patients still below threshold at Month 8 | 22 | 20 |
| Number of patients still below threshold at Month 12 | 21 | 12 |
Collected over 12 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Deferiprone | 0/32 (0%) | 5/32 (15.6%) | 29/32 (90.6%) |
| Placebo | 0/32 (0%) | 2/32 (6.3%) | 29/32 (90.6%) |
| Event | Deferiprone | Placebo |
|---|---|---|
| NeutropeniaBlood and lymphatic system disorders | 4/32 | 1/32 |
| AgranulocytosisBlood and lymphatic system disorders | 1/32 | 0/32 |
| Autoimmune haemolytic anaemiaBlood and lymphatic system disorders | 0/32 | 1/32 |
| Dengue feverInfections and infestations | 1/32 | 0/32 |
| Bronchitis chronicRespiratory, thoracic and mediastinal disorders | 0/32 | 1/32 |
| Event | Deferiprone | Placebo |
|---|---|---|
| PyrexiaGeneral disorders | 20/32 | 18/32 |
| Neutrophil count decreasedInvestigations | 9/32 | 9/32 |
| NasopharyngitisInfections and infestations | 8/32 | 8/32 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 2/32 | 7/32 |
| BronchitisInfections and infestations | 3/32 | 6/32 |
| CoughRespiratory, thoracic and mediastinal disorders | 4/32 | 6/32 |
| DiarrhoeaGastrointestinal disorders | 4/32 | 5/32 |
| GastroenteritisInfections and infestations | 5/32 | 4/32 |
| Upper respiratory tract infectionInfections and infestations | 4/32 | 5/32 |
| PharyngitisInfections and infestations | 4/32 | 1/32 |
| Age, Continuous(Years) | Deferiprone | Placebo | Total |
|---|---|---|---|
| Mean | 3.03 ± 2.42 | 2.63 ± 1.70 | 2.83 ± 2.08 |
| Sex: Female, Male(Participants) | Deferiprone | Placebo | Total |
|---|---|---|---|
| Female | 12 | 11 | 23 |
| Male | 20 | 21 | 41 |
| Ethnicity (NIH/OMB)(Participants) | Deferiprone | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 1 | 1 |
| Not Hispanic or Latino | 32 | 31 | 63 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Deferiprone | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 6 | 3 | 9 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 26 | 29 | 55 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(Participants) | Deferiprone | Placebo | Total |
|---|---|---|---|
| Egypt | 26 | 29 | 55 |
| Indonesia | 6 | 3 | 9 |
| Serum ferritin (µg/L)(micrograms per liter) | Deferiprone | Placebo | Total |
|---|---|---|---|
| Mean | 513.84 ± 236.06 | 451.69 ± 196.43 | 482.76 ± 217.68 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is completed, as verified in Mar 2024. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Deferiprone