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

December 18, 2023 updated by: ApoPharma

Long-term Safety and Efficacy Study of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients With Sickle Cell Disease or Other Anemias

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

134

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada
        • Hospital for Sick Kids
  • Egypt
      • Alexandria, Egypt
        • Zagazig University
      • Cairo, Egypt
        • Cairo University
      • Cairo, Egypt
        • Ain Shams University
      • Cairo, Egypt
        • Pediatric Hospital of Cairo University
  • Saudi Arabia
      • Abha, Saudi Arabia
        • Asser Central Hospital
  • United Kingdom
      • London, United Kingdom
        • Evelina Children's Hospital
      • London, United Kingdom
        • Barts and The London
  • United States
    • California
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital Oakland
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan Comprehensive Cancer Center
      • Detroit, Michigan, United States, 48201
        • Children's Hospital of Michigan
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104-4399
        • The Children's Hospital of Philadephia
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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).
Drug: Deferiprone
Other Names:
  • Ferriprox tablets
  • Ferriprox oral solution
Experimental: 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).
Drug: Deferiprone
Other Names:
  • Ferriprox tablets
  • Ferriprox oral solution

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Patients With Adverse Events
Time Frame: From the first day of the study until the last study visit (Week 104 or early termination)
Number of patients with at least one adverse event (AE) of any type; number of patients with at least one serious adverse event, and number of patients who withdrew from the study due to an AE
From the first day of the study until the last study visit (Week 104 or early termination)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in Liver Iron Concentration (LIC)
Time Frame: One year, two years, and three years after the start of deferiprone therapy
LIC was measured by MRI, in units of mg of iron per gram of liver (dry weight). The change from baseline in LIC was determined for three different periods of exposure to deferiprone: one year, two years, and three years.
One year, two years, and three years after the start of deferiprone therapy
Change From Baseline in Cardiac MRI T2*
Time Frame: One year, two years, and three years after the start of deferiprone therapy
The change from baseline in cardiac MRI T2* was determined for three different periods of exposure to deferiprone: one year, two years, and three years
One year, two years, and three years after the start of deferiprone therapy
Change From Baseline in Serum Ferritin
Time Frame: One year, two years, and three years after the start of deferiprone therapy
The change from baseline in serum ferritin (SF) was determined for three different periods of exposure to deferiprone: one year, two years, and three years.
One year, two years, and three years after the start of deferiprone therapy

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

ApoPharma

Investigators

  • Principal Investigator: Janet Kwiatkowski, MD, Children's Hospital of Philadelphia

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 21, 2015

Primary Completion (Actual)

April 30, 2019

Study Completion (Actual)

August 21, 2019

Study Registration Dates

First Submitted

May 5, 2015

First Submitted That Met QC Criteria

May 11, 2015

First Posted (Estimated)

May 14, 2015

Study Record Updates

Last Update Posted (Actual)

January 10, 2024

Last Update Submitted That Met QC Criteria

December 18, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LA38-EXT

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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