Clinical trial NCT00530127 (phase 1/phase 2, status: completed)
Conditions: friedreich's ataxia
ICD codes: G11.11
Drugs: placebo, deferiprone, deferiprone, placebo, deferiprone
SMILES: CN1C(=O)C=C(N2CCC[C@@H](N)C2)N(CC2=C(C=CC=C2)C#N)C1=O, CN1C=CC(=O)C(O)=C1C, CN1C=CC(=O)C(O)=C1C, CN1C(=O)C=C(N2CCC[C@@H](N)C2)N(CC2=C(C=CC=C2)C#N)C1=O, CN1C=CC(=O)C(O)=C1C
Eligibility criteria:

        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.

          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.

          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.

        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 enroll 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 defined by an absolute neutrophil count (ANC) < 1.5
             x 109/L or thrombocytopenia defined by a platelet count <150 x 109/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 enrollment 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 > 450 msec at Baseline.