START: Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children

Sponsor
Chiesi Canada Corp (Industry)
Overall Status
Completed
CT.gov ID
NCT03591575
Collaborator
(none)
64
4
2
22.7
16
0.7

Study Details

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Deferiprone oral solution
  • Drug: Placebo
Phase 4

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.

Study Design

Study Type:
Interventional
Actual Enrollment :
64 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes 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.
Primary Purpose:
Treatment
Official Title:
Safety and Efficacy of Early-start Deferiprone Treatment in Infants and Young Children Newly Diagnosed With Transfusion-dependent Beta Thalassemia
Actual Study Start Date :
Nov 9, 2018
Actual Primary Completion Date :
Sep 29, 2020
Actual Study Completion Date :
Sep 29, 2020

Arms and Interventions

Arm Intervention/Treatment
Experimental: Deferiprone

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
Liquid formulation of deferiprone, with a concentration of 80 mg/mL
Other Names:
  • Ferriprox
  • Placebo Comparator: Placebo

    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 solution that matches deferiprone oral solution in appearance and taste
    Other Names:
  • Placebo for deferiprone oral solution
  • Outcome Measures

    Primary Outcome Measures

    1. The percentage of patients in each treatment group who still have a serum ferritin level < 1000 micrograms per liter (μg/L) at Month 12 [12 months]

      A serum ferritin level of 1000 μg/L is the threshold for when standard chelation therapy begins

    Secondary Outcome Measures

    1. Time to reach a serum ferritin level ≥ 1000 μg/L [Up to 12 months]

      Without adequate chelation therapy, most patients receiving regular red blood cell transfusions are likely to exceed this level within 12 months

    2. Time to reach a labile plasma iron (LPI) value ≥ 0.6 micromoles (µM) [Up to 12 months]

      Without adequate chelation therapy, most patients receiving regular red blood cell transfusions are likely to exceed this level within 12 months

    3. Time to reach a transferrin saturation (TSAT) value ≥ 60% [Up to 12 months]

      Without adequate chelation therapy, most patients receiving regular red blood cell transfusions are likely to exceed this level within 12 months

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Months to 9 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    4. 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:
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Ain Shams University Cairo Egypt
    2 Cairo University Cairo Egypt
    3 Pediatric Hospital of Cairo University Cairo Egypt
    4 Cipto Mangunkusumo National Hospital Jakarta Indonesia

    Sponsors and Collaborators

    • Chiesi Canada Corp

    Investigators

    • Study Director: Fernando Tricta, MD, ApoPharma Inc.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Chiesi Canada Corp
    ClinicalTrials.gov Identifier:
    NCT03591575
    Other Study ID Numbers:
    • LA55-0417
    First Posted:
    Jul 19, 2018
    Last Update Posted:
    Feb 10, 2021
    Last Verified:
    Feb 1, 2021
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Chiesi Canada Corp
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Feb 10, 2021