Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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

May 8, 2009
June 24, 2011
June 2009
March 2011   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT00897221 on ClinicalTrials.gov Archive Site
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 ]
Same as current
Not Provided
Not Provided
 
A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia
An Open-label, Single Treatment, Safety and Efficacy, Long-term Study of Deferiprone in Subjects With Friedreich's Ataxia

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.

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.

Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Friedreich's Ataxia
  • Drug: Deferiprone oral solution 100mg/mL
    Deferiprone oral solution (20 mg/kg/day)
    Other Name: Ferriprox
  • Drug: Deferiprone oral solution 100 mg/mL
    Deferiprone oral solution(40mg/kg/day)
    Other Name: Ferriprox
  • Experimental: Dose 1
    Deferiprone oral solution 20 mg/kg/day
    Intervention: Drug: Deferiprone oral solution 100mg/mL
  • Experimental: Dose 2
    Deferiprone oral solution 40 mg/kg/day
    Intervention: Drug: Deferiprone oral solution 100 mg/mL
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
36
March 2011
March 2011   (final data collection date for primary outcome measure)

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.
Both
7 Years to 35 Years
No
Contact information is only displayed when the study is recruiting subjects
Belgium,   France,   Italy,   Spain
 
NCT00897221
LA29-EXT
Yes
Dian Shaw, ApoPharma Inc.
ApoPharma
Not Provided
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
ApoPharma
June 2011

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