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

Last updated: December 18, 2023
Sponsor: ApoPharma
Overall Status: Terminated

Phase

4

Condition

Red Blood Cell Disorders

Sickle Cell Disease

Anemia

Treatment

Deferiprone

Clinical Study ID

NCT02443545
LA38-EXT
  • Ages > 3
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Completed study LA38-0411
  2. Females of childbearing potential must have a negative pregnancy test result at Visit
  3. In addition, if applicable, they must:
  • Use an effective method of contraception according to local requirements, duringthe 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)
  1. Fertile heterosexual males and/or their partners must agree to use an effective methodof contraception during the study and for 30 days following the last dose of studymedication
  2. All patients and/or their authorized legal representatives must provide signed anddated written informed consent prior to the first study intervention, and assent willbe obtained from patients who are considered to be minors. Patients must be able toadhere to study restrictions, appointments, and evaluation schedules.

Exclusion

Exclusion Criteria:

  1. Plan to participate in another clinical trial at any time from the day of enrollmentuntil 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 laboratoryabnormalities, such as ALT (alanine aminotransferase) ≥ 5 x ULN or creatinine ≥ 2 xULN), psychological condition, or psychiatric condition which in the opinion of theinvestigator 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 judgmentindicates the need for the patient to be started on a different iron chelator

Study Design

Total Participants: 134
Treatment Group(s): 1
Primary Treatment: Deferiprone
Phase: 4
Study Start date:
May 21, 2015
Estimated Completion Date:
August 21, 2019

Study 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.

Connect with a study center

  • Centro Infantil Boldrini

    Campinas,
    Brazil

    Site Not Available

  • Hospital de Clínicas de Porto Alegre

    Porto Alegre,
    Brazil

    Site Not Available

  • Instituto Estadual de Hematologia

    Rio de Janeiro,
    Brazil

    Site Not Available

  • Casa de Saúde Santa Marcelina

    São Paulo,
    Brazil

    Site Not Available

  • Hospital for Sick Kids

    Toronto, Ontario
    Canada

    Site Not Available

  • Zagazig University

    Alexandria,
    Egypt

    Site Not Available

  • Ain Shams University

    Cairo,
    Egypt

    Site Not Available

  • Cairo University

    Cairo,
    Egypt

    Site Not Available

  • Pediatric Hospital of Cairo University

    Cairo,
    Egypt

    Site Not Available

  • Mansoura University Children's Hospital

    Mansoura,
    Egypt

    Site Not Available

  • Asser Central Hospital

    Abha,
    Saudi Arabia

    Site Not Available

  • King Abdulaziz University Hospital

    Jeddah,
    Saudi Arabia

    Site Not Available

  • King Khalid University Hospital

    Riyadh,
    Saudi Arabia

    Site Not Available

  • Barts and The London

    London,
    United Kingdom

    Site Not Available

  • Evelina Children's Hospital

    London,
    United Kingdom

    Site Not Available

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California 94609
    United States

    Site Not Available

  • University of Illinois at Chicago

    Chicago, Illinois 60612
    United States

    Site Not Available

  • Children's Hospital

    New Orleans, Louisiana 70118
    United States

    Site Not Available

  • University of Michigan Comprehensive Cancer Center

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • Children's Hospital of Michigan

    Detroit, Michigan 48201
    United States

    Site Not Available

  • The Children's Hospital of Philadephia

    Philadelphia, Pennsylvania 19104-4399
    United States

    Site Not Available

  • Medical University of South Carolina

    Charleston, South Carolina 29425
    United States

    Site Not Available

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