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Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia

NCT00078481 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2008-03-04

No results posted yet for this study

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.

Conditions

  • Friedreich Ataxia

Interventions

DRUG

Idebenone

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    lead NIH

Study Design

Purpose
TREATMENT

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-02-29
Completion
2006-04-30

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00078481 on ClinicalTrials.gov