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

NCT02443545 | Terminated Phase 4 Results DMC

• 1 other identifier: LA38-EXT
• Study Type: interventional
• Target: 134
• Locations: 5 countries
• Sites: 13

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.

Conditions

Iron Overload; Sickle Cell Disease; Other Anemias

Keywords

Iron overload; Sickle cell disease; Deferiprone; Ferriprox; Iron chelation

Outcome Measures

Primary Outcomes (1)

• Number of Patients With Adverse Events

  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

  From the first day of the study until the last study visit (Week 104 or early termination)

Secondary Outcomes (3)

• Change From Baseline in Liver Iron Concentration (LIC)

  One year, two years, and three years after the start of deferiprone therapy

• Change From Baseline in Cardiac MRI T2*

  One year, two years, and three years after the start of deferiprone therapy

• Change From Baseline in Serum Ferritin

  One year, two years, and three years after the start of deferiprone therapy

Study Arms (2)

Group 1: Deferiprone 3 years

EXPERIMENTAL

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

Group 2: Deferiprone 2 years

EXPERIMENTAL

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

Interventions

Deferiprone DRUG

Also known as: Ferriprox tablets, Ferriprox oral solution

Group 1: Deferiprone 3 years; Group 2: Deferiprone 2 years

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• ApoPharma: lead

Study Sites (13)

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 48109, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

The Children's Hospital of Philadephia

Philadelphia, Pennsylvania, 19104-4399, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Hospital for Sick Kids

Toronto, Ontario, Canada

Location

Zagazig University

Alexandria, Egypt

Location

Ain Shams University

Cairo, Egypt

Location

Cairo University

Cairo, Egypt

Location

Pediatric Hospital of Cairo University

Cairo, Egypt

Location

Asser Central Hospital

Abhā, Saudi Arabia

Location

Barts and The London

London, United Kingdom

Location

Evelina Children's Hospital

London, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 36018224 DERIVED

MeSH Terms

Conditions

Iron Overload; Anemia, Sickle Cell

Interventions

Deferiprone

Condition Hierarchy (Ancestors)

Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Limitations and Caveats

The trial was terminated early upon recommendation of the Data and Safety Monitoring Board (DSMB), which felt that sufficient information had been obtained.

Results Point of Contact

• Title: Fernando Tricta, MD
• Organization: Chiesi Canada Corp.

f.tricta@chiesi.com

1-800-854-3534

Study Officials

• Janet Kwiatkowski, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 5, 2015
• First Posted: May 14, 2015
• Study Start: May 21, 2015
• Primary Completion: April 30, 2019
• Study Completion: August 21, 2019
• Last Updated: January 10, 2024
• Results First Posted: January 10, 2024

Record last verified: 2023-12

Locations

SA (1); GB (2); US (5); CA (1); EG (4)