Overview

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

Status:
Completed
Trial end date:
2011-03-01
Target enrollment:
0
Participant gender:
All
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.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
ApoPharma
Treatments:
Deferiprone
Pharmaceutical Solutions
Criteria
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.