Back to resultsSafety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children (START)
Primary Purpose
Beta Thalassemia Major Anemia, Iron Overload
Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Deferiprone oral solution
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Beta Thalassemia Major Anemia focused on measuring thalassemia, iron overload, chelation, deferiprone, Ferriprox
Eligibility Criteria
Inclusion Criteria:
- Male or female aged ≥ 6 months to < 10 years
- Confirmed diagnosis of beta-thalassemia, as determined by high performance liquid chromatography (HPLC) or DNA testing
- Started on a red blood cell (RBC) transfusion regimen
- Screening level of serum ferritin 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:
- 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.
Sites / Locations
- Ain Shams University
- Cairo University
- Pediatric Hospital of Cairo University
- Cipto Mangunkusumo National Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Deferiprone
Placebo
Arm Description
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
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
Outcomes
Primary Outcome Measures
The percentage of patients in each treatment group who still have a serum ferritin level < 1000 micrograms per liter (μg/L) at Month 12
A serum ferritin level of 1000 μg/L is the threshold for when standard chelation therapy begins
Secondary Outcome Measures
Time to reach a serum ferritin level ≥ 1000 μg/L
Without adequate chelation therapy, most patients receiving regular red blood cell transfusions are likely to exceed this level within 12 months
Time to reach a labile plasma iron (LPI) value ≥ 0.6 micromoles (µM)
Without adequate chelation therapy, most patients receiving regular red blood cell transfusions are likely to exceed this level within 12 months
Time to reach a transferrin saturation (TSAT) value ≥ 60%
Without adequate chelation therapy, most patients receiving regular red blood cell transfusions are likely to exceed this level within 12 months
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03591575
Brief Title
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
Acronym
START
Official Title
Safety and Efficacy of Early-start Deferiprone Treatment in Infants and Young Children Newly Diagnosed With Transfusion-dependent Beta Thalassemia
Study Type
Interventional
2. Study Status
Record Verification Date
February 2021
Overall Recruitment Status
Completed
Study Start Date
November 9, 2018 (Actual)
Primary Completion Date
September 29, 2020 (Actual)
Study Completion Date
September 29, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Chiesi Canada Corp
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Beta Thalassemia Major Anemia, Iron Overload
Keywords
thalassemia, iron overload, chelation, deferiprone, Ferriprox
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
The placebo solution will have the same appearance and flavor as deferiprone oral solution, and will be administered at a volume matching that required for the dose of active product.
Allocation
Randomized
Enrollment
64 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Deferiprone
Arm Type
Experimental
Arm Description
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
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
Deferiprone oral solution
Other Intervention Name(s)
Ferriprox
Intervention Description
Liquid formulation of deferiprone, with a concentration of 80 mg/mL
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Placebo for deferiprone oral solution
Intervention Description
Liquid solution that matches deferiprone oral solution in appearance and taste
Primary Outcome Measure Information:
Title
The percentage of patients in each treatment group who still have a serum ferritin level < 1000 micrograms per liter (μg/L) at Month 12
Description
A serum ferritin level of 1000 μg/L is the threshold for when standard chelation therapy begins
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Time to reach a serum ferritin level ≥ 1000 μg/L
Description
Without adequate chelation therapy, most patients receiving regular red blood cell transfusions are likely to exceed this level within 12 months
Time Frame
Up to 12 months
Title
Time to reach a labile plasma iron (LPI) value ≥ 0.6 micromoles (µM)
Description
Without adequate chelation therapy, most patients receiving regular red blood cell transfusions are likely to exceed this level within 12 months
Time Frame
Up to 12 months
Title
Time to reach a transferrin saturation (TSAT) value ≥ 60%
Description
Without adequate chelation therapy, most patients receiving regular red blood cell transfusions are likely to exceed this level within 12 months
Time Frame
Up to 12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
9 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female aged ≥ 6 months to < 10 years
Confirmed diagnosis of beta-thalassemia, as determined by high performance liquid chromatography (HPLC) or DNA testing
Started on a red blood cell (RBC) transfusion regimen
Screening level of serum ferritin 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:
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Fernando Tricta, MD
Organizational Affiliation
ApoPharma Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Ain Shams University
City
Cairo
Country
Egypt
Facility Name
Cairo University
City
Cairo
Country
Egypt
Facility Name
Pediatric Hospital of Cairo University
City
Cairo
Country
Egypt
Facility Name
Cipto Mangunkusumo National Hospital
City
Jakarta
Country
Indonesia
12. IPD Sharing Statement
Learn more about this trial
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
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