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 : Unknown
Verified January 2019 by ApoPharma.
Recruitment status was: Enrolling by invitation
First Posted : May 14, 2015
Last Update Posted : January 14, 2019
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|Condition or disease||Intervention/treatment||Phase|
|Iron Overload Sickle Cell Disease Other Anemias||Drug: Deferiprone||Phase 4|
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|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|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 :||October 2021|
|Estimated Study Completion Date :||April 2022|
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)..
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).
- 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
- 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*
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||3 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Completed study LA38-0411
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)
- 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
- 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.
- Plan to participate in another clinical trial at any time from the day of enrollment until 30 days post-treatment in the current study
- 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.
- Pregnant, breastfeeding, or planning to become pregnant during the study period.
- 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
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
|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|
|Hospital for Sick Kids|
|Toronto, Ontario, Canada|
|Ain Shams University|
|Pediatric Hospital of Cairo University|
|Asser Central Hospital|
|Abha, Saudi Arabia|
|Barts and The London|
|London, United Kingdom|
|Evelina Children's Hospital|
|London, United Kingdom|
|Principal Investigator:||Janet Kwiatkowski, MD||Children's Hospital of Philadelphia|
|Other Study ID Numbers:||
|First Posted:||May 14, 2015 Key Record Dates|
|Last Update Posted:||January 14, 2019|
|Last Verified:||January 2019|
Sickle cell disease
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Iron Metabolism Disorders
Iron Chelating Agents
Molecular Mechanisms of Pharmacological Action