Safety Study of Idebenone to Treat Friedreich's Ataxia
Status:
Completed
Trial end date:
2006-04-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)