A six-month double-blind, randomized, placebo-controlled study investigating the safety and tolerability of deferiprone in participants with Friedreich's ataxia - Not applicable

Phase 1

Conditions: Friedreich's Ataxia
MedDRA version: 9.1Level: LLTClassification code 10017374Term: Friedreich's ataxia

Registration Number: EUCTR2007-003331-23-BE

Lead Sponsor: ApoPharma Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 80

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. In addition, a female subject must confirm that during the study and within 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 (IUDs) (in place for at least 3 months prior to first dose), 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.
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. Effective methods of contraception for males include condoms or sterilization or abstinence from sexual intercourse.
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.
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

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 enrol 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/agranulocytosis defined by an absolute neutrophil count (ANC) < 1.5 x 10E9/L or thrombocytopenia defined by a platelet count <150 x 10E9/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 enrolment 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 Greater than 450 ms at Baseline.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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