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

NCT03591575 | Completed Phase 4 Results DMC FDA Drug

• 1 other identifier: LA55-0417
• Study Type: interventional
• Target: 64
• Locations: 2 countries
• Sites: 4

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.

Conditions

Beta Thalassemia Major Anemia; Iron Overload

Keywords

thalassemia; iron overload; chelation; deferiprone; Ferriprox

Outcome Measures

Primary Outcomes (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

  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 Outcomes (1)

• Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time Points

  4, 8, and 12 months

Study Arms (2)

Deferiprone

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

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

Interventions

Deferiprone oral solution DRUG

Liquid formulation of deferiprone, with a concentration of 80 mg/mL

Also known as: Ferriprox

Deferiprone

Placebo DRUG

Liquid solution that matches deferiprone oral solution in appearance and taste

Also known as: Placebo for deferiprone oral solution

Placebo

Eligibility Criteria

• Age: 6 Months - 9 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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, with a minimum of 2 transfusions already completed
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Chiesi Canada Corp: lead

Study Sites (4)

Ain Shams University Children's Hospital

Cairo, Egypt

Location

Pediatric Hospital of Cairo University

Cairo, Egypt

Location

The Clinical Research Center, Faculty of Medicine, Cairo University

Cairo, Egypt

Location

Cipto Mangunkusumo National Hospital

Jakarta, Indonesia

Location

MeSH Terms

Conditions

Iron Overload; Thalassemia

Interventions

Deferiprone

Condition Hierarchy (Ancestors)

Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Caroline Fradette, PhD
• Organization: Chiesi Canada Corp.

c.fradette@chiesi.com

1-(800) 854-3534

Study Officials

• Mohsen El Alfy, MD

  Ain Shams University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 9, 2018
• First Posted: July 19, 2018
• Study Start: November 9, 2018
• Primary Completion: September 29, 2020
• Study Completion: September 29, 2020
• Last Updated: March 15, 2024
• Results First Posted: March 15, 2024

Record last verified: 2024-03

Locations

EG (3); ID (1)