Status:
COMPLETED
A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia
Lead Sponsor:
ApoPharma
Conditions:
Friedreich's Ataxia
Eligibility:
All Genders
7-35 years
Phase:
PHASE2
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-te...
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 receiv...
Eligibility Criteria
Inclusion
- Subjects who completed the ApoPharma study LA29-0207
- Female subjects of childbearing potential must have a negative pregnancy test.
- 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.
- 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
- Serum Ferritin and Hemoglobin (Hb) levels are below the reference range for age and sex-matched controls.
- 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).
- Doubling of score on 9HPT or T25FW compared to their study baseline results in LA29-0207.
- 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.
- 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.
- Unable to comply with requirements of the protocol.
- Pregnant, breastfeeding or planning to become pregnant during the study period.
- QTc interval \>450ms.
- Have been on antioxidants prior to start of study treatment.
Key Trial Info
Start Date :
June 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2011
Estimated Enrollment :
36 Patients enrolled
Trial Details
Trial ID
NCT00897221
Start Date
June 1 2009
End Date
March 1 2011
Last Update
June 27 2011
Active Locations (4)
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1
Hospital Erasme
Brussels, Belgium
2
Hospital Necker-Enfants Malades
Paris, France
3
Fondazione IRCCS Istituto Neurologico "C. Besta"
Milan, Italy
4
La Fundacion Para la Investigacion Biomedica
Madrid, Spain