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Trial record 2 of 2 for:    START, deferiprone | thalassemia

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03591575
Recruitment Status : Active, not recruiting
First Posted : July 19, 2018
Last Update Posted : March 9, 2020
Sponsor:
Information provided by (Responsible Party):
Chiesi Canada Corp

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
Beta Thalassemia Major Anemia Iron Overload 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.

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Study Type : Interventional  (Clinical Trial)
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 : November 9, 2018
Estimated Primary Completion Date : November 1, 2020
Estimated Study Completion Date : November 1, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron Thalassemia
Drug Information available for: Deferiprone

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 Name: 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 Name: Placebo for deferiprone oral solution




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 [ Time Frame: 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 [ Time Frame: 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) [ Time Frame: 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% [ Time Frame: 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



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

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03591575


Locations
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Egypt
Ain Shams University
Cairo, Egypt
Cairo University
Cairo, Egypt
Pediatric Hospital of Cairo University
Cairo, Egypt
Indonesia
Cipto Mangunkusumo National Hospital
Jakarta, Indonesia
Sponsors and Collaborators
Chiesi Canada Corp
Investigators
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Study Director: Fernando Tricta, MD ApoPharma Inc.
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Responsible Party: Chiesi Canada Corp
ClinicalTrials.gov Identifier: NCT03591575    
Other Study ID Numbers: LA55-0417
First Posted: July 19, 2018    Key Record Dates
Last Update Posted: March 9, 2020
Last Verified: October 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Chiesi Canada Corp:
thalassemia
iron overload
chelation
deferiprone
Ferriprox
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Deferiprone
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Pharmaceutical Solutions
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action