A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia

This study has been completed.
Sponsor:
Information provided by:
ApoPharma
ClinicalTrials.gov Identifier:
NCT00897221
First received: May 8, 2009
Last updated: June 24, 2011
Last verified: June 2011
  Purpose

The primary objective of this study is to evaluate the long-term safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).

The secondary objective is to evaluate the long-term efficacy of deferiprone for the treatment of FRDA.

The tertiary objectives are to evaluate the effect of deferiprone on:

  1. cardiac function,
  2. quality of life, and
  3. functional status.

Condition Intervention Phase
Friedreich's Ataxia
Drug: Deferiprone oral solution 100mg/mL
Drug: Deferiprone oral solution 100 mg/mL
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label, Single Treatment, Safety and Efficacy, Long-term Study of Deferiprone in Subjects With Friedreich's Ataxia

Resource links provided by NLM:


Further study details as provided by ApoPharma:

Primary Outcome Measures:
  • The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events. [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS. [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Enrollment: 36
Study Start Date: June 2009
Study Completion Date: March 2011
Primary Completion Date: March 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Dose 1
Deferiprone oral solution 20 mg/kg/day
Drug: Deferiprone oral solution 100mg/mL
Deferiprone oral solution (20 mg/kg/day)
Other Name: Ferriprox
Experimental: Dose 2
Deferiprone oral solution 40 mg/kg/day
Drug: Deferiprone oral solution 100 mg/mL
Deferiprone oral solution(40mg/kg/day)
Other Name: Ferriprox

Detailed Description:

This is a multi-centre, open-label, non-randomized, single treatment, safety and efficacy study. All subjects who completed the LA29-0207 study are eligible for participation. Participants will receive deferiprone oral solution at the same dose (20 or 40 mg/kg/day) that they were assigned for LA29-0207. The duration of treatment will be 52 weeks.

  Eligibility

Ages Eligible for Study:   7 Years to 35 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Subjects who completed the ApoPharma study LA29-0207
  2. Female subjects of childbearing potential must have a negative pregnancy test.
  3. Male subjects must confirm that he and/or his female partner will use an effective method of contraception for the length of the trial and for 30 days following completion of the study or early termination.
  4. Signed and witnessed written informed consent/assent, obtained prior to the first study intervention, as well as the ability to adhere to study restrictions, appointments and evaluation schedules.

Exclusion Criteria:

  1. Serum Ferritin and Hemoglobin (Hb) levels are below the reference range for age and sex-matched controls.
  2. Unable to complete T25FW AND with a score > 5 minutes in the 9HPT. Subjects who can complete T25FW or with a score ≤ 5 minutes in the 9HPT will be allowed to enrol).
  3. Doubling of score on 9HPT or T25FW compared to their study baseline results in LA29-0207.
  4. History or evidence of neutropenia/agranulocytosis defined by a confirmed absolute neutrophil count (ANC) < 1.5 x 109/L or thrombocytopenia defined by a platelet count <150 x 109/L.
  5. Occurrence of SAEs or any other AEs during the LA29-0207 study, which in the opinion of the investigator cause the patient's participation in the extension study to be inappropriate.
  6. Unable to comply with requirements of the protocol.
  7. Pregnant, breastfeeding or planning to become pregnant during the study period.
  8. QTc interval >450ms.
  9. Have been on antioxidants prior to start of study treatment.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00897221

Locations
Belgium
Hospital Erasme
Brussels, Belgium
France
Hospital Necker-Enfants Malades
Paris, France
Italy
Fondazione IRCCS Istituto Neurologico "C. Besta"
Milan, Italy
Spain
La Fundacion Para la Investigacion Biomedica
Madrid, Spain
Sponsors and Collaborators
ApoPharma
Investigators
Principal Investigator: Massimo Pandolfo, M.D. Hospital Erasme, Brussels, Belgium
Principal Investigator: Arnold Munnich, M.D. Hospital Necker-Enfants Malades, Paris, France
Principal Investigator: Franco Taroni, M.D. Fondazione IRCCS Istituto Neurologico "C. Besta", Milan, Italy
Principal Investigator: Javier Arpa, M.D. La Fundaction Para la Investigacion Biomedica, Madrid, Spain
  More Information

No publications provided

Responsible Party: Dian Shaw, ApoPharma Inc.
ClinicalTrials.gov Identifier: NCT00897221     History of Changes
Other Study ID Numbers: LA29-EXT
Study First Received: May 8, 2009
Last Updated: June 24, 2011
Health Authority: Belgium: Federal Agency for Medicinal Products and Health Products
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Italy: Ethics Committee
Spain: Spanish Agency of Medicines

Keywords provided by ApoPharma:
Friedreich's ataxia

Additional relevant MeSH terms:
Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Degenerations
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Pharmaceutical Solutions
Deferiprone
Therapeutic Uses
Pharmacologic Actions
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on September 16, 2014