Phase 3
N=232
A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients
Friedreich's Ataxia
Bottom Line
View on ClinicalTrials.gov: NCT00905268 ↗Enrolled (actual)
232
Serious AEs
11.2%
Results posted
Jun 2016
Primary outcome: 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 units on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- idebenone (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 8+ yrs
- Sex
- All
- Sponsor
- Santhera Pharmaceuticals
- Primary completion
- Jan 2010
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 | — |
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.
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.