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

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ClinicalTrials.gov Identifier: NCT02443545
Recruitment Status : Enrolling by invitation
First Posted : May 14, 2015
Last Update Posted : September 27, 2018
Sponsor:
Information provided by (Responsible Party):
ApoPharma

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.

Condition or disease Intervention/treatment Phase
Iron Overload Sickle Cell Disease Other Anemias Drug: Deferiprone Phase 4

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  (Clinical Trial)
Estimated Enrollment : 300 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-term Safety and Efficacy Study of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients With Sickle Cell Disease or Other Anemias
Study Start Date : May 2015
Estimated Primary Completion Date : September 2021
Estimated Study Completion Date : March 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia Iron

Arm Intervention/treatment
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
  • Deferiprone oral solution

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
  • Deferiprone oral solution




Primary Outcome Measures :
  1. Number of subjects with adverse events (AEs) [ Time Frame: From the first day of the study until the last study visit (Week 104 or early termination) ]
    Number of participants in each group with AEs, by frequency, severity, time to onset, duration, and relatedness to study product

  2. Number of subjects with serious adverse events (SAEs) [ Time Frame: From the first day of the study until 30 days after the last dose ]
    Number of participants in each group with SAEs, by frequency, severity, time to onset, duration, and relatedness to study product

  3. Discontinuations due to adverse events [ Time Frame: From the first day of the study until Week 104 ]
    Number of participants discontinued from the study because of adverse events


Secondary Outcome Measures :
  1. Change from baseline to end of study in liver iron concentration (LIC) [ Time Frame: Group 1: From Week 0 of study LA38-0411 until the last visit of study LA38-EXT (Week 104 or early termination). Group 2: From Week 0 of study LA38-EXT until the last visit (Week 104 or early termination). ]
    LIC will be determined by MRI

  2. Change from baseline to end of study in cardiac MRI T2* [ Time Frame: Group 1: From Week 0 of study LA38-0411 until the last visit of study LA38-EXT (Week 104 or early termination). Group 2: From Week 0 of study LA38-EXT until the last visit (Week 104 or early termination). ]
    Myocardial MRI T2* will be used to monitor the myocardial iron load resulting from chronic transfusions. Low T2* levels reflect a high cardiac iron concentration and high T2* values reflect low cardiac iron.

  3. Change from baseline to end of study in serum ferritin [ Time Frame: Group 1: From Week 0 of study LA38-0411 until the last visit of study LA38-EXT (Week 104 or early termination). Group 2: From Week 0 of study LA38-EXT until the last visit (Week 104 or early termination). ]
  4. Responder analysis [ Time Frame: Group 1: From Week 0 of study LA38-0411 to Weeks 0, 52, and 104 of LA38-EXT (1, 2, and 3 years of deferiprone treatment, respectively). Group 2: From Week 0 of study LA38-EXT to Weeks 52 and 104 (1 and 2 years of deferiprone treatment, respectively). ]
    The percentage of patients who show a ≥20% decline from baseline in LIC or serum ferritin or a ≥20% increase from baseline in cardiac MRI T2*



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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 ≥ 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

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): NCT02443545


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

Responsible Party: ApoPharma
ClinicalTrials.gov Identifier: NCT02443545     History of Changes
Other Study ID Numbers: LA38-EXT
First Posted: May 14, 2015    Key Record Dates
Last Update Posted: September 27, 2018
Last Verified: September 2018

Keywords provided by ApoPharma:
Iron overload
Sickle cell disease
Deferiprone
Ferriprox
Iron chelation

Additional relevant MeSH terms:
Anemia
Anemia, Sickle Cell
Iron Overload
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Iron
Deferiprone
Trace Elements
Micronutrients
Growth Substances
Physiological Effects of Drugs
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action