Phase 3
N=70
Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
Friedreich's Ataxia
Bottom Line
View on ClinicalTrials.gov: NCT00537680 ↗Enrolled (actual)
70
Serious AEs
2.9%
Results posted
Dec 2011
Primary outcome: Primary: Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 — -2.5; -2.4; -1.3 ICARS points
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Idebenone (Drug); Placebo (Drug)
- Age
- Pediatric · 8+ yrs
- Sex
- All
- Sponsor
- Santhera Pharmaceuticals
- Primary completion
- Apr 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24 |
-2.5; -2.4; -1.3 | — |
| SECONDARY Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24 |
-1.6; -1.2; 0.6 | — |
| SECONDARY Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24 |
0.2; 0.6; 1.0 | — |
| SECONDARY Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12 |
-1.5; -1.6; -3.0 | — |
| SECONDARY Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle |
-0.032; 0.007; 0.010 | — |
| SECONDARY Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle |
-3.32; 1.53; 2.65 | — |
| SECONDARY Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI) |
-3.06; -2.56; -1.26 | — |
| SECONDARY Percent Change From Baseline to Week 24 in Left Ventricular Mass Index |
-4.5; -4.9; -1.7 | — |
| SECONDARY Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle |
-0.018; -0.004; -0.038 | — |
| SECONDARY Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle |
-0.075; 0.12; -3.04 | — |
| SECONDARY Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS) |
-0.014; 0.021; 0.063 | — |
| SECONDARY Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS) |
1.3; 2.6; 7.8 | — |
Summary
This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.
Eligibility Criteria
Inclusion criteria
- Documented diagnosis of Friedreich's Ataxia (FRDA) with confirmed FRDA mutations
- Patients 8 - 17 years of age at baseline
- Patients with a body weight greater than 25 kg/55 lbs at baseline
- Patients able to walk at least 10 meters without accompanying person (ICARS Walking Capacities score ≤6) at screening and baseline
- Patients who in the opinion of the investigator are able to comply with the requirements of the study, including swallowing the study medication
- Negative urine pregnancy test at screening and baseline (female patients of childbearing potential)
Exclusion criteria
- Treatment with idebenone, Coenzyme Q10 or vitamin E (if taken at a dose 5 times above the daily requirement) within the past month
- Patients with International Cooperative Ataxia Rating Scale (ICARS) score of greater than 54 or less than 10 at screening
- Pregnancy and/or breast-feeding
- Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of Aspartate Aminotransferase (AST; also known as GOT), Alanine Aminotransferase (ALT; also known as GPT) or creatinine
- History of abuse of drugs or alcohol
- Participation in the previous (Phase II) study of idebenone at the National Institutes of Health (NIH)
- Participation in a trial of another investigational drug within the last 3 months
Data sourced from ClinicalTrials.gov (NCT00537680). 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.