Safety Study of Idebenone to Treat Friedreich's Ataxia

Completed · Phase 1

Conditions studied: Friedreich Ataxia

In brief

This study will determine the highest dose of idebonone that can safely be given to patients with Friedrich's ataxia, an inherited degenerative disease that causes loss of muscle coordination, speech problems, weakness and sensory loss. Enlargement of the left ventricle (the large pumping chamber of the heart) is also common in this disease. In studies in France and Canada, patients with Friedrich's ataxia who were given idebonone, an antioxidant similar to the dietary supplement coenzyme Q, had a decrease in the size of their left ventricle. Patients 5 years and older with Friedrich's ataxia may be eligible for this study. Pregnant and lactating women may not participate. Candidates will be screened with a medical history and physical examination and a review of genetic studies. Patients who have not had genetic studies will be offered genetic counseling and testing to confirm or rule out Friedrich's ataxia. Participants will be admitted to the NIH Clinical Center for 3 days. They will have blood and urine tests and a heart evaluation, including an echocardiogram-a procedure that uses sound waves to produce images of the heart, and an electrocardiogram-a study of the electrical activity of the heart. When these tests have been completed, patients will take an idebonone capsule. They will be monitored for side effects for 72 hours. Blood samples will be collected through an intravenous catheter (flexible plastic tube placed in a vein) 0.5, 1, 2, 3, 4, 6, 12, 24, 48 and 72 hours after the drug is taken to determine how long it takes for the drug to be eliminated from the body. Patients will return for a follow-up visit within 1 to 8 weeks. Those who experienced no serious side effects may receive another, higher dose of the drug, with at least 6 days between doses.

Key facts

Study ID: NCT00015808
Run by: National Institute of Neurological Disorders and Stroke (NINDS)
People needed: 100
Starts: 2001-05-01
Expected to finish: 2006-04-01
Last updated by the study team: 2006-07-12

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Diagnosis of FRDA with confirmed FRDA mutations.
Age greater than or equal to five years old.
No exposure to idebenone or coenzyme Q10 for a period of at least one week prior to onset of the medication phase of the study.
Written, informed consent (and assent, if applicable).

You may not qualify if…

History of a hypersensitivity reaction to idebenone or coenzyme
Q10.
Pregnant or lactating women. All women of child-bearing potential must have negative serum pregnancy prior to the medication phase of the study.
Age less than five years old.
Platelet count, lymphocyte count or hemoglobin below the lower limit of normal.
Alkaline phosphatase, SGOT, SGPT greater than 1.5 times the upper limit of normal. Bilirubin greater than 1.2 g/dl.
Creatinine greater than 1.5 times the upper limit of normal.
Clinically significant medical disease that, in the judgment of the investigators, would expose the patient to undue risk of harm or prevent the patient from completing the study.

Where it is running

National Institute of Neurological Disorders and Stroke (NINDS) — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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