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A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia

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ClinicalTrials.gov Identifier: NCT00530127
Recruitment Status : Completed
First Posted : September 17, 2007
Last Update Posted : June 2, 2010
Sponsor:
Information provided by:
ApoPharma

Tracking Information
First Submitted Date  ICMJE September 13, 2007
First Posted Date  ICMJE September 17, 2007
Last Update Posted Date June 2, 2010
Study Start Date  ICMJE April 2008
Actual Primary Completion Date July 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 14, 2007)
The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events [ Time Frame: 6 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT00530127 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 14, 2007)
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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
Official Title  ICMJE A Six-month Double-blind, Randomized, Placebo-controlled Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
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).
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Friedreich's Ataxia
Intervention  ICMJE
  • Drug: placebo
    Same dose and frequency as treatment
  • Drug: deferiprone
    100 mg/mL
  • Drug: placebo
    Same dosage and frequency as study drug
Study Arms  ICMJE
  • Placebo Comparator: A
    Placebo solution
    Intervention: Drug: placebo
  • Experimental: B
    Deferiprone oral solution 20 mg/kg/day
    Intervention: Drug: deferiprone
  • Experimental: C
    Deferiprone oral solution 40 mg/kg/day
    Intervention: Drug: deferiprone
  • Placebo Comparator: D
    Placebo solution
    Intervention: Drug: placebo
  • Experimental: E
    deferiprone oral solution 60 mg/kg/day
    Intervention: Drug: deferiprone
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Estimated Enrollment  ICMJE
 (submitted: September 14, 2007)
80
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE July 2009
Actual Primary Completion Date July 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 7 Years to 35 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Belgium,   Canada,   France,   Italy,   Spain
Removed Location Countries United Kingdom,   United States
 
Administrative Information
NCT Number  ICMJE NCT00530127
Other Study ID Numbers  ICMJE LA29-0207
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Dian Shaw, ApoPharma Inc.
Study Sponsor  ICMJE ApoPharma
Collaborators  ICMJE Not Provided
Investigators  ICMJE
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
PRS Account ApoPharma
Verification Date May 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP