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

Phase 4

Terminated

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.

Recruitment & Eligibility

Inclusion Criteria

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.

Exclusion Criteria

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 (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.
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

Arm && Interventions

Group	Intervention	Description
Group 2: Deferiprone 2 years	Deferiprone	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).
Group 1: Deferiprone 3 years	Deferiprone	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).

Primary Outcome Measures

Name	Time	Method
Number of Patients With Adverse Events	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

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Liver Iron Concentration (LIC)	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.
Change From Baseline in Cardiac MRI T2*	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
Change From Baseline in Serum Ferritin	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.

Locations (13): Zagazig University
🇪🇬
Alexandria, Egypt
Children's Hospital of Michigan
🇺🇸
Detroit, Michigan, United States
Hospital for Sick Kids
🇨🇦
Toronto, Ontario, Canada
University of Michigan Comprehensive Cancer Center
🇺🇸
Ann Arbor, Michigan, United States
Barts and The London
🇬🇧
London, United Kingdom
Ain Shams University
🇪🇬
Cairo, Egypt
Pediatric Hospital of Cairo University
🇪🇬
Cairo, Egypt
Medical University of South Carolina
🇺🇸
Charleston, South Carolina, United States
The Children's Hospital of Philadephia
🇺🇸
Philadelphia, Pennsylvania, United States
Cairo University
🇪🇬
Cairo, Egypt
Evelina Children's Hospital
🇬🇧
London, United Kingdom
UCSF Benioff Children's Hospital Oakland
🇺🇸
Oakland, California, United States
Asser Central Hospital
🇸🇦
Abha, Saudi Arabia