Friedreich's ataxia MedDRA version: 9.1 Level: LLT Classification code 10017374 Term: Friedreich's ataxia
Conditions
Sponsors
ApoPharma Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subjects who completed the ApoPharma study LA29-0207. 2. Female subjects of childbearing potential must have a negative pregnancy test. In addition, a female subject must confirm that during the study and for 30 days following the completion of the study or early termination she: • will use an effective method of contraception, OR • has had a tubal ligation (supporting evidence required), OR • has had a hysterectomy (supporting evidence required), OR • participates in a non-heterosexual lifestyle, OR • indicates her only male sexual partner has been sterilized (supporting evidence required). Effective methods of contraception include oral contraceptives, intrauterine devices (RIDs), diaphragm or condom, providing they are used with contraceptive foam or cream, or abstinence from sexual intercourse. Supporting evidence for sterilization consists of a surgical report or letter from the family physician. 3. Heterosexual, sexually active 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. Effective methods of contraception for males include condoms or sterilization or abstinence from sexual intercourse or participation in a non-heterosexual lifestyle. 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. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 less than or equal to 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) 450ms. 9. Have been on antioxidants prior to start of study treatment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate the long-term safety and tolerability of deferiprone in subjects with Friedreich’s ataxia (FRDA).;Secondary Objective: 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.;Primary end point(s): Evaluation of long-term safety and tolerability of deferiprone in subjects with Friedreich's ataxia. 9-Hole Peg Test The 9HPT is a quantitative test of the functionality of the upper extremities. The test will be administered by a trained examiner (not necessarily the Investigator) in accordance with the Schedule of Events. The subject is instructed to place pegs into set holes as quickly as possible and then remove all the pegs. The test is performed two consecutive times with the dominant hand, followed by two consecutive times with the non-dominant hand. The maximum time allotted for each test (dominant or non-dominant hand) will be 5 minutes. The total time in seconds it takes the subject to complete the task is recorded. The average time for the four tests will be evaluated as a measure of treatment efficacy. Timed 25-Foot Walk The T25FW is a quantitative mobility and leg function performance test. The test will be administered by a trained examiner (not necessarily the Investigator) in accordance with the Schedule of Events. The subject is instructed to walk 25 feet along a pre-defined straight course as quickly as possible. The task will be immediately repeated by having the subject walk back to the starting point. Subjects will be allowed to use assistive devices when completing this task. The total time in seconds it takes the subject to walk 25 feet will be recorded for each test. The average time for the two tests will be evaluated as a measure of treatment efficacy. Low Contrast Letter Acuity LCLA is a quantit | — |
Countries
Belgium, France, Italy, Spain
Outcome results
None listed