Phase 4
N=64
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
Beta Thalassemia Major Anemia · Iron Overload
Bottom Line
View on ClinicalTrials.gov: NCT03591575 ↗Enrolled (actual)
64
Serious AEs
10.9%
Results posted
Mar 2024
Primary outcome: Primary: 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 Participants — p=0.0446
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Deferiprone oral solution (Drug); Placebo (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Chiesi Canada Corp
- Primary completion
- Sep 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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 | 0.0446 sig |
| SECONDARY Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points |
29; 28; 22; 20; 21; 12 | 0.0446 sig |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Male or female aged ≥ 6 months to 200 μg/L but not more than 600 μg/L. Since SF level may be impacted by the presence of infection, it must additionally be verified that the child has had no signs of infection in the previous 7 days, including the day of screening, and that the level of C-reactive protein (CRP) is no greater than 20% higher than the normal range for the patient's age. If there are signs of infection and/or the CRP level is above this threshold, the SF level must be checked again a minimum of one week later.
Exclusion Criteria
- Prior use of iron chelation
- Diagnosis of hepatitis B or C, or HIV infection
- Evidence of abnormal liver or kidney function at screening: serum alanine transaminase (ALT) level > 5 times upper limit of normal or creatinine levels >2 times upper limit of normal
- Disorders associated with neutropenia (absolute neutrophil count < 1.5 x 10^9/L) prior to the initiation of study medication
- A serious, unstable illness, as judged by the investigator, during the previous 3 months before screening/baseline visit including but not limited to hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease.
- Presence of any medical condition which in the opinion of the investigator would cause participation in the study to be unwise.
Data sourced from ClinicalTrials.gov (NCT03591575). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.