Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

NCT ID: NCT02443545

Last Updated: 2024-01-10

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 134 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-05-21
• Study Completion Date: 2019-08-21

Brief Summary

This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.

Detailed Description

Deferiprone (brand name Ferriprox®) is an iron chelator that is approved in the United States and over 60 other countries for the treatment of iron overload in patients with thalassemia, when other treatments are inadequate. This study has been designed to evaluate the long-term efficacy, safety, and tolerability of deferiprone to treat iron overload in patients who have sickle cell disease or other anemias.

Only patients who have completed an earlier study, LA38-0411, are eligible to enroll in this one.

Conditions

Iron Overload; Sickle Cell Disease; Other Anemias

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Group 1: Deferiprone 3 years

Patients in this group are those who were randomized to the deferiprone arm in study LA38-0411, and hence will receive deferiprone for a total of 3 years (1 year in the initial study plus 2 years in the extension study).

Group Type: EXPERIMENTAL

Deferiprone

Intervention Type: DRUG

Group 2: Deferiprone 2 years

Patients in this group are those who were randomized to the deferoxamine arm in study LA38-0411, and hence will receive deferiprone for 2 years (both of them in the extension study).

Group Type: EXPERIMENTAL

Deferiprone

Intervention Type: DRUG

Interventions

Deferiprone

Intervention Type: DRUG

Other Intervention Names

Ferriprox tablets; Ferriprox oral solution

Eligibility Criteria

Inclusion Criteria

1. Completed study LA38-0411

2. Females of childbearing potential must have a negative pregnancy test result at Visit 1. In addition, if applicable, they must:

• Use an effective method of contraception according to local requirements, during the study and within 30 days following their last dose of study medication, OR
• Have had a tubal ligation (supporting evidence required), OR
• Have had a hysterectomy (supporting evidence required), OR
• Participate in a non-heterosexual lifestyle, OR
• Have a male sexual partner who has been sterilized (supporting evidence required)

3. Fertile heterosexual males and/or their partners must agree to use an effective method of contraception during the study and for 30 days following the last dose of study medication

4. All patients and/or their authorized legal representatives must provide signed and dated written informed consent prior to the first study intervention, and assent will be obtained from patients who are considered to be minors. Patients must be able to adhere to study restrictions, appointments, and evaluation schedules.

Exclusion Criteria

1. Plan to participate in another clinical trial at any time from the day of enrollment until 30 days post-treatment in the current study

2. For only those patients who were treated with deferoxamine in study LA38-0411 (Group 2): Presence of any medical condition (including clinically significant laboratory abnormalities, such as ALT (alanine aminotransferase) ≥ 5 x ULN or creatinine ≥ 2 x ULN), psychological condition, or psychiatric condition which in the opinion of the investigator would cause participation in the study to be unwise.

3. Pregnant, breastfeeding, or planning to become pregnant during the study period.

4. Treatment failure after 1 year on deferiprone which in the investigator's judgment indicates the need for the patient to be started on a different iron chelator

• Minimum Eligible Age: 3 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ApoPharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Janet Kwiatkowski, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

Locations

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, United States

Children's Hospital of Michigan

Detroit, Michigan, United States

The Children's Hospital of Philadephia

Philadelphia, Pennsylvania, United States

Medical University of South Carolina

Charleston, South Carolina, United States

Hospital for Sick Kids

Toronto, Ontario, Canada

Zagazig University

Alexandria, , Egypt

Ain Shams University

Cairo, , Egypt

Cairo University

Cairo, , Egypt

Pediatric Hospital of Cairo University

Cairo, , Egypt

Asser Central Hospital

Abhā, , Saudi Arabia

Barts and The London

London, , United Kingdom

Evelina Children's Hospital

London, , United Kingdom

Countries

United States; Canada; Egypt; Saudi Arabia; United Kingdom

References

Elalfy MS, Hamdy M, El-Beshlawy A, Ebeid FSE, Badr M, Kanter J, Inusa B, Adly AAM, Williams S, Kilinc Y, Lee D, Fradette C, Rozova A, Temin NT, Tricta F, Kwiatkowski JL. Deferiprone for transfusional iron overload in sickle cell disease and other anemias: open-label study of up to 3 years. Blood Adv. 2023 Feb 28;7(4):611-619. doi: 10.1182/bloodadvances.2021006778.

Reference Type: DERIVED

PMID: 36018224 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

LA38-EXT

Identifier Type: -

Identifier Source: org_study_id