Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia
Status:
Completed
Trial end date:
2006-04-01
Target enrollment:
Participant gender:
Summary
This study will determine the highest amount of idebenone that can be taken without harmful
side effects in children, teenagers, and adults with Friedreich's ataxia, a progressive
degenerative disease that affects several body systems. Studies in France and Canada showed
that patients with Friedreich's ataxia who took idebenone had a decrease in the size of their
left ventricle (main pumping chamber of the heart), which is often enlarged in this disease.
It is possible that idebenone may also prevent the progression of nervous system degeneration
in Friedreich's ataxia.
Patients 5 years of age and older with Friedreich's ataxia may be eligible for this study.
Candidates are screened with a blood test and review of their medical records, including
genetic studies.
Participants undergo the following procedures during a 6-day hospital admission to the NIH
Clinical Center:
- Placement of an intravenous catheter (plastic tube inserted into a vein) for collecting
blood samples after drug administration
- Blood and urine tests
- Heart examination, including electrocardiogram (EKG), to assess heart function and size.
- Idebenone therapy: Patients take three tablets a day (at 7 AM, 1 PM and 7 PM) on days 2,
3 and 4 of hospitalization. Blood samples are collected through the IV tube at 0.5, 1,
2, 4, and 6 hours after the first dose on day 2, then at 1 hour after the first and
third doses every day, and then at 1, 2, 4, 8, 12, 24, 36, and 48 hours after the last
dose on day 4 to determine how the body uses and eliminates the drug.
- Monitoring for drug side effects: Patients have frequent checks of vital signs (blood
pressure, pulse, temperature, breathing rate) and a brief physical examination to check
for drug side effects from the start of drug therapy on day 2 until at least 43 hours
after the last dose on day 4.
Patients who experience no difficulties are discharged from the hospital after the sixth day
with a 1-month supply of medication to take 3 times a day at home. They are contacted by
phone every 2 weeks while taking the medication to check side effects. Blood tests are also
done every 2 weeks to check for any abnormalities.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)