Phase 3
Completed N=232
A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients
Source: ClinicalTrials.gov NCT00905268 ↗Enrolled (actual)
232
Serious AEs
11.2%
Results posted
Jun 2016
Primary outcomePrimary: Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 — 1.6; 1.7; 1.2; 1.1 units on a scale
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
The purpose of this trial is to study the efficacy, safety and tolerability of idebenone in 12 months of treatment in children and adults with Friedreich's Ataxia. This is a randomised placebo-controlled double-blind trial conducted in Europe. Efficacy outcomes include measures of neurological impairment and function, and measures of the heart.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 |
1.6; 1.7; 1.2; 1.1 | — |
| SECONDARY Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52 |
0.9; 1.2; 1.4; 0.9 | — |
| SECONDARY Proportion of Patients Improving (Responding) on ICARS by a Clinically Relevant Margin |
18.2; 23.6; 23.7; 31 | — |
| SECONDARY Proportion of Patients Improving on Left Ventricular Peak Systolic Strain Rate or Showing a Reduction in Left Ventricular Mass Index (LVMI) With no Worsening in Strain Rate |
50; 51.4; 30.3; 44.1 | — |
| SECONDARY Change in Peak Systolic Strain Rate From Baseline to Week 52 |
0.007; -0.004; 0.085; 0.024 | — |
| SECONDARY Change in Peak Workload From Baseline to Week 52 |
1.15; -7.41; -6.91; -1.54 | — |
Eligibility Criteria
Inclusion Criteria
- Documented diagnosis of FRDA with confirmed FRDA mutations
- Patients 8 years of age or older at baseline
- Patients with body weight ≥ 25kg
- Patients who in the opinion of the investigator are able to comply with the requirements of the study, including swallowing the medication
- Negative urine pregnancy test at screening and at baseline (women of childbearing potential)
Exclusion Criteria
- Treatment with idebenone or Coenzyme Q10 within the past 1 month
- Pregnancy and/or breast-feeding
- Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of SGOT, SGPT, or creatinine
- Past or present history of abuse of drugs or alcohol
Data sourced from ClinicalTrials.gov (NCT00905268). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.