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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00897221
Recruitment Status : Completed
First Posted : May 12, 2009
Last Update Posted : June 27, 2011
Information provided by:

Brief Summary:

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

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 36 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (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
Study Start Date : June 2009
Actual Primary Completion Date : March 2011
Actual Study Completion Date : March 2011

Arm Intervention/treatment
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

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

Secondary Outcome Measures :
  1. The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS. [ Time Frame: 52 weeks ]

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   7 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00897221

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Hospital Erasme
Brussels, Belgium
Hospital Necker-Enfants Malades
Paris, France
Fondazione IRCCS Istituto Neurologico "C. Besta"
Milan, Italy
La Fundacion Para la Investigacion Biomedica
Madrid, Spain
Sponsors and Collaborators
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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
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Responsible Party: Dian Shaw, ApoPharma Inc. Identifier: NCT00897221    
Other Study ID Numbers: LA29-EXT
First Posted: May 12, 2009    Key Record Dates
Last Update Posted: June 27, 2011
Last Verified: June 2011
Keywords provided by ApoPharma:
Friedreich's ataxia
Additional relevant MeSH terms:
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Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Pharmaceutical Solutions
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action