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A Study Investigating the Safety and Tolerability 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: NCT00530127
Recruitment Status : Completed
First Posted : September 17, 2007
Last Update Posted : June 2, 2010
Information provided by:

Brief Summary:

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

The secondary objective is to evaluate the efficacy of deferiprone for the treatment of FRDA, as assessed by a 9-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich's Ataxia Rating Scale (FARS).

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

  1. cardiac function as measured by changes in Left Ventricular Shortening Fraction (LVSF), Left Ventricular Ejection Fraction (LVEF) and Left Ventricular (LV) mass using echocardiogram (ECHO),
  2. quality of life using quality-of-life surveys, and
  3. functional status using Activities of Daily Living (ADL).

Condition or disease Intervention/treatment Phase
Friedreich's Ataxia Drug: placebo Drug: deferiprone Phase 1 Phase 2

Detailed Description:
This will be a multi-centre, double-blind, randomized, placebo-controlled clinical trial. A total of 80 patients with Friedreich's ataxia will be enrolled. Eligible patients will receive deferiprone oral solution or placebo at a total daily dose of 20 mg/kg/day, 40 mg/kg/day or 60 mg/kg/day, divided into two-daily doses for 6 months.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Six-month Double-blind, Randomized, Placebo-controlled Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
Study Start Date : April 2008
Actual Primary Completion Date : July 2009
Actual Study Completion Date : July 2009

Arm Intervention/treatment
Placebo Comparator: A
Placebo solution
Drug: placebo
Same dose and frequency as treatment

Experimental: B
Deferiprone oral solution 20 mg/kg/day
Drug: deferiprone
100 mg/mL

Experimental: C
Deferiprone oral solution 40 mg/kg/day
Drug: deferiprone
100 mg/mL

Placebo Comparator: D
Placebo solution
Drug: placebo
Same dosage and frequency as study drug

Experimental: E
deferiprone oral solution 60 mg/kg/day
Drug: deferiprone
100 mg/mL

Primary Outcome Measures :
  1. The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. The efficacy endpoints will be change in the score for 9-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich's Ataxia Rating Scale (FARS). [ Time Frame: 6 months ]

Information from the National Library of Medicine

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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. Diagnosis of FRDA, with confirmed mutation (excludes point mutation) in the frataxin (FXN) gene and GAA repeats ≥ 400 on the shorter allele.
  2. Males or females aged 7 to 35 years.
  3. No exposure to idebenone, coenzyme Q10, vitamin C, vitamin E or other antioxidants as a supplement or as a drug therapy for a period of at least one month prior to start of treatment and during the study.
  4. Neurological testing: A FARS score >20 and <85 at Screening and Baseline.
  5. Female subjects of childbearing potential must have a negative pregnancy test at Baseline.
  6. If the subject is a heterosexual, sexually-active male, he confirms 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.
  7. 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 schedule.

Exclusion Criteria:

  1. Iron deficiency defined as ferritin levels below the reference range for age- and sex-matched controls
  2. Unable to complete T25FW AND with score > 5 minutes in the 9HPT. (Subjects who can complete T25FW or with a score ≤ 5 minutes in the 9HPT will be allowed to enroll if the score has not doubled compared to screening).
  3. Abnormal ALT, greater than 2.0 times the upper limit of normal on two consecutive assessments.
  4. Serum creatinine outside the normal reference range.
  5. History or evidence of neutropenia defined by an absolute neutrophil count (ANC) < 1.5 x 109/L or thrombocytopenia defined by a platelet count <150 x 109/L.
  6. Refusal to participate in screening procedures or unable to participate in screening procedures or unable to comply with the requirements of the protocol.
  7. Receiving any investigational drug products or having received any investigational product within 30 days prior to enrollment into this study.
  8. Subjects who have previously taken deferiprone.
  9. Subjects who, in the opinion of the Investigator, represent poor medical, psychological or psychiatric risks, and for whom participation in an investigational trial would be unwise.
  10. Pregnant, breastfeeding or planning to become pregnant during the study period.
  11. History of malignancy.
  12. History of alcohol or drug abuse.
  13. Investigators, site personnel directly affiliated with this study and their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biological or legally adopted.
  14. Hypersensitivity to the active substance (deferiprone) or any of the excipients in the oral solution.
  15. QT interval > 450 msec at Baseline.

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): NCT00530127

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Murdoch Children's Research Institute
Victoria, Australia, 3052
Hospital Erasme
Brussels, Belgium, 1070
Canada, Ontario
McMaster University
Hamilton, Ontario, Canada, L8N 3Z5
Hospital Necker-Enfants Malades
Paris, France, 75743
Fondazione IRCCS Istituto Neurologico "C. Besta"
Milan, Italy, 20133
La Fundacion Para la Investigacion Biomedica
Madrid, Spain, 261
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 Fondazione IRCCS Istituto Neurologico "C. Besta"
Principal Investigator: Martin Delatycki Murdoch Children's Research Institute, Vicotria, Australia
Principal Investigator: Javier Arpa La Fundaction Para la Investigacion Biomedica, Madrid, Spain
Principal Investigator: Mark Tarnopolsky, MD McMaster University
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Responsible Party: Dian Shaw, ApoPharma Inc. Identifier: NCT00530127    
Other Study ID Numbers: LA29-0207
First Posted: September 17, 2007    Key Record Dates
Last Update Posted: June 2, 2010
Last Verified: May 2010
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
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action