Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias

NCT02041299 | Terminated Phase 4 Results DMC

• 1 other identifier: LA38-0411
• Study Type: interventional
• Target: 230
• Locations: 7 countries
• Sites: 30

Brief Summary

This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body. We will be comparing deferiprone with deferoxamine, another drug that removes iron from the body.

Conditions

Iron Overload; Sickle Cell Disease; Other Anemias

Keywords

sickle cell disease; Iron overload; Deferiprone; Ferriprox; deferoxamine; Chelation

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Liver Iron Concentration (LIC)

  LIC was measured by MRI. A score \>7 mg/g dw is indicative of iron overload.

  Change from baseline to Week 52

Secondary Outcomes (3)

• Change From Baseline in Cardiac Iron

  Change from baseline to Week 52

• Change From Baseline in Serum Ferritin

  Change from baseline to Week 52

• Change in Patient-reported Quality of Life, as Measured by the Short Form Health Survey (SF-36) or the Child Health Questionnaire (CHQ-PF50).

  Change from baseline to Week 52

Study Arms (2)

Deferiprone

EXPERIMENTAL

Patients randomized to the deferiprone arm will be prescribed either tablets or liquid medication. Deferiprone is taken orally, at a dosage that is calculated in terms of milligrams per kilogram of body weight (mg/kg) and is divided into 3 equal doses taken approximately 8 hours apart. The daily dosage is 75 mg/kg (25 mg/kg per dose) for patients with less severe iron load, and 99 mg/kg (33 mg/kg per dose) for those with more severe iron load.

Drug: Deferiprone

Deferoxamine

ACTIVE COMPARATOR

Patients randomized to the deferoxamine arm will be prescribed the drug as per the approved US prescribing information. Deferoxamine is administered as a subcutaneous infusion over 8-12 hours, 5 to 7 days a week. The dosage is 20 mg/kg (children) or 40 mg/kg (adults) in patients with less severe iron load, and up to 40 mg/kg (children) or 50 mg/kg (adults) in those with more severe iron load.

Drug: Deferoxamine

Interventions

Deferiprone DRUG

Also known as: Ferriprox tablets, Deferiprone oral solution

Deferiprone

Deferoxamine DRUG

Deferoxamine

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female ≥ 2 years of age;

You may not qualify if:

• Baseline LIC \>7 mg/g dw (measured by MRI);
• Patients who have received no less than 20 transfusions of RBCs;
• Patients who have received at least 1 transfusion per year in the last 2 years and who are expected to have a continuing requirement (based on Investigator's judgement) during the duration of the trial
• Thalassemia syndromes;
• Myelodysplastic syndrome (MDS) or myelofibrosis;
• Diamond Blackfan anemia;
• Primary bone marrow failure;
• Baseline LIC \>30 mg/g dw (measured by MRI);
• Unable or unwilling to undergo a 7 day washout period if currently being treated with deferiprone or deferoxamine or deferasirox;
• Previous discontinuation of treatment with deferiprone or deferoxamine due to adverse events;
• History or presence of hypersensitivity or idiosyncratic reaction to deferiprone or deferoxamine;
• Treated with hydroxyurea within 30 days;
• History of malignancy;
• Evidence of abnormal liver function (serum ALT level(s) \> 5 times upper limit of normal at screening or creatinine levels \>2 times upper limit of normal at screening);
• A serious, unstable illness, as judged by the Investigator, during the past 3 months before screening/baseline visit including but not limited to: hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease;

Sponsors & Collaborators

• ApoPharma: lead

Study Sites (33)

Children's Hospital Oakland

Oakland, California, 94609, United States

Location

University of Illinois at Chicago

Chicago, Illinois, 60612, United States

Location

Children's Hospital

New Orleans, Louisiana, 70118, 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

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Centro Infantil Boldrini

Campinas, Brazil

Location

Hospital de Clínicas de Porto Alegre-HCPA,

Rio Branco, 90035-903, Brazil

Location

Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO

Rio de Janeiro, 20211-030, Brazil

Location

Casa de Saúde Santa Marcelina

São Paulo, Brazil

Location

Universidade Federal de São Paulo

São Paulo, Brazil

Location

Hospital for Sick Kids

Toronto, Ontario, Canada

Location

Mansoura University Children's Hospital

Al Mansurah, Egypt

Location

Alexandria University

Alexandria, Egypt

Location

Zagazig University

Alexandria, Egypt

Location

Ains Shams University

Cairo, Egypt

Location

Cairo University

Cairo, Egypt

Location

Pediatric Hospital of Cairo University

Cairo, Egypt

Location

King Abdulaziz University Hospital

Jeddah, Western Region, 80215, Saudi Arabia

Location

Asser Central Hospital

Abhā, Saudi Arabia

Location

King Khalid University Hospital

Riyadh, Saudi Arabia

Location

National Center for Bone Marrow Transplantation

Tunis, Bad Saadoun, Tunisia

Location

Farhat Hached Hospital, Hematology Department

Sousse, Tunisia

Location

Principal Military Hospital of Instruction of Tunis

Tunis, Tunisia

Location

Cukurova University

Adana, Turkey (Türkiye)

Location

Hacettepe University

Ankara, Turkey (Türkiye)

Location

Istanbul University

Istanbul, Turkey (Türkiye)

Location

Hammersmith Hospital

London, W12 0HS, United Kingdom

Location

Barts and The London

London, United Kingdom

Location

Evelina Children's Hospital

London, United Kingdom

Location

Imperial College Healthcare NHS Trust

London, United Kingdom

Location

Related Publications (1)

• Kwiatkowski JL, Hamdy M, El-Beshlawy A, Ebeid FSE, Badr M, Alshehri A, Kanter J, Inusa B, Adly AAM, Williams S, Kilinc Y, Lee D, Tricta F, Elalfy MS. Deferiprone vs deferoxamine for transfusional iron overload in SCD and other anemias: a randomized, open-label noninferiority study. Blood Adv. 2022 Feb 22;6(4):1243-1254. doi: 10.1182/bloodadvances.2021004938.

  PMID: 34847228 DERIVED

MeSH Terms

Conditions

Iron Overload; Anemia, Sickle Cell

Interventions

Deferiprone; Deferoxamine

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; Hydroxamic Acids; Hydroxylamines; Amines; Organic Chemicals; Hydroxy Acids; Carboxylic Acids

Limitations and Caveats

The trial was terminated early as the pool of potential participants was exhausted and it was determined that the number of patients already randomized would be sufficient to assess the primary endpoint.

Results Point of Contact

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

ftricta@chiesi.com

1-416-558-6342

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: January 15, 2014
• First Posted: January 22, 2014
• Study Start: April 17, 2014
• Primary Completion: April 20, 2019
• Study Completion: June 18, 2019
• Last Updated: August 10, 2021
• Results First Posted: August 10, 2021

Record last verified: 2021-07

Locations

GB (4); TU (3); CA (1); US (8); EG (6); BR (5); SA (3)