Iron Overload Clinical Trial
— STARTOfficial title:
Safety and Efficacy of Early-start Deferiprone Treatment in Infants and Young Children Newly Diagnosed With Transfusion-dependent Beta Thalassemia
Verified date | March 2024 |
Source | Chiesi Canada Corp |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 64 |
Est. completion date | September 29, 2020 |
Est. primary completion date | September 29, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 6 Months to 9 Years |
Eligibility | Inclusion Criteria: 1. Male or female aged = 6 months to < 10 years 2. Confirmed diagnosis of beta-thalassemia, as determined by high performance liquid chromatography (HPLC) or DNA testing 3. Started on a red blood cell (RBC) transfusion regimen, with a minimum of 2 transfusions already completed 4. Screening level of serum ferritin (SF) greater than >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: 1. Prior use of iron chelation 2. Diagnosis of hepatitis B or C, or HIV infection 3. 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 4. Disorders associated with neutropenia (absolute neutrophil count < 1.5 x 10^9/L) prior to the initiation of study medication 5. 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. 6. Presence of any medical condition which in the opinion of the investigator would cause participation in the study to be unwise. |
Country | Name | City | State |
---|---|---|---|
Egypt | Ain Shams University Children's Hospital | Cairo | |
Egypt | Pediatric Hospital of Cairo University | Cairo | |
Egypt | The Clinical Research Center, Faculty of Medicine, Cairo University | Cairo | |
Indonesia | Cipto Mangunkusumo National Hospital | Jakarta |
Lead Sponsor | Collaborator |
---|---|
Chiesi Canada Corp |
Egypt, Indonesia,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
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 | 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. | 12 months | |
Secondary | 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). | 4, 8, and 12 months |
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