Clinical trial NCT02443545: Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
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 type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Iron Overload
Sickle Cell Disease
Other Anemias
Keywords: Iron overload
Sickle cell disease
Deferiprone
Ferriprox
Iron chelation
Lead sponsor: Chiesi Canada Corp
Sponsor class: INDUSTRY
First posted: 2015-05-14
Enrollment: 134
Sex: ALL
Minimum age (years): 3
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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 (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.
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

Primary outcomes:
measure: Number of Patients With Adverse Events
timeFrame: From the first day of the study until the last study visit (Week 104 or early termination)
Secondary outcomes:
measure: Change From Baseline in Liver Iron Concentration (LIC)
timeFrame: One year, two years, and three years after the start of deferiprone therapy
measure: Change From Baseline in Cardiac MRI T2*
timeFrame: One year, two years, and three years after the start of deferiprone therapy
measure: Change From Baseline in Serum Ferritin
timeFrame: One year, two years, and three years after the start of deferiprone therapy
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Ann Arbor
country: United States
facility: University of Michigan Comprehensive Cancer Center
state: Michigan
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadephia
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Toronto
country: Canada
facility: Hospital for Sick Kids
state: Ontario
city: Alexandria
country: Egypt
facility: Zagazig University
city: Cairo
country: Egypt
facility: Ain Shams University
city: Cairo
country: Egypt
facility: Cairo University
city: Cairo
country: Egypt
facility: Pediatric Hospital of Cairo University
city: Abha
country: Saudi Arabia
facility: Asser Central Hospital
city: London
country: United Kingdom
facility: Barts and The London
city: London
country: United Kingdom
facility: Evelina Children's Hospital
Study officials:
affiliation: Children's Hospital of Philadelphia
name: Janet Kwiatkowski, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Elalfy MS, Hamdy M, El-Beshlawy A, Ebeid FSE, Badr M, Kanter J, Inusa B, Adly AAM, Williams S, Kilinc Y, Lee D, Fradette C, Rozova A, Temin NT, Tricta F, Kwiatkowski JL. Deferiprone for transfusional iron overload in sickle cell disease and other anemias: open-label study of up to 3 years. Blood Adv. 2023 Feb 28;7(4):611-619. doi: 10.1182/bloodadvances.2021006778.