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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02443545
Recruitment Status : Enrolling by invitation
First Posted : May 14, 2015
Last Update Posted : January 14, 2019
Sponsor:
Information provided by (Responsible Party):
ApoPharma

Tracking Information
First Submitted Date  ICMJE May 5, 2015
First Posted Date  ICMJE May 14, 2015
Last Update Posted Date January 14, 2019
Study Start Date  ICMJE May 2015
Estimated Primary Completion Date October 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 11, 2015)
  • 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
  • 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
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 11, 2015)
  • 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
  • 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.
  • 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). ]
  • 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*
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
Official Title  ICMJE Long-term Safety and Efficacy Study of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients With Sickle Cell Disease or Other Anemias
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Iron Overload
  • Sickle Cell Disease
  • Other Anemias
Intervention  ICMJE Drug: Deferiprone
Other Names:
  • Ferriprox tablets
  • Deferiprone oral solution
Study Arms  ICMJE
  • 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)..
    Intervention: Drug: Deferiprone
  • 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).
    Intervention: Drug: Deferiprone
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: May 11, 2015)
300
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 2022
Estimated Primary Completion Date October 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Egypt,   Saudi Arabia,   United Kingdom,   United States
Removed Location Countries Brazil
 
Administrative Information
NCT Number  ICMJE NCT02443545
Other Study ID Numbers  ICMJE LA38-EXT
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party ApoPharma
Study Sponsor  ICMJE ApoPharma
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Janet Kwiatkowski, MD Children's Hospital of Philadelphia
PRS Account ApoPharma
Verification Date January 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP