A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia

This will be a multi-centre, double-blind, randomized, placebo-controlled clinical trial. A total of 80 patients with Friedreich's ataxia will be enrolled. Eligible patients will receive deferiprone oral solution or placebo at a total daily dose of 20 mg/kg/day, 40 mg/kg/day or 60 mg/kg/day, divided into two-daily doses for 6 months.

Inclusion criteria

• Diagnosis of FRDA, with confirmed mutation (excludes point mutation) in the frataxin (FXN) gene and GAA repeats ≥ 400 on the shorter allele.
• Males or females aged 7 to 35 years.
• 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.
• Neurological testing: A FARS score >20 and <85 at Screening and Baseline.
• Female subjects of childbearing potential must have a negative pregnancy test at Baseline.
• 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.
• 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.

Exclusion criteria

• Iron deficiency defined as ferritin levels below the reference range for age- and sex-matched controls
• 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 enroll if the score has not doubled compared to screening).
• Abnormal ALT, greater than 2.0 times the upper limit of normal on two consecutive assessments.
• Serum creatinine outside the normal reference range.
• History or evidence of neutropenia defined by an absolute neutrophil count (ANC) < 1.5 x 109/L or thrombocytopenia defined by a platelet count <150 x 109/L.
• Refusal to participate in screening procedures or unable to participate in screening procedures or unable to comply with the requirements of the protocol.
• Receiving any investigational drug products or having received any investigational product within 30 days prior to enrollment into this study.
• Subjects who have previously taken deferiprone.
• 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.
• Pregnant, breastfeeding or planning to become pregnant during the study period.
• History of malignancy.
• History of alcohol or drug abuse.
• 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.
• Hypersensitivity to the active substance (deferiprone) or any of the excipients in the oral solution.
• QT interval > 450 msec at Baseline.