Phase 3 N=68 Treatment

Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Friedreich's Ataxia

Bottom Line

View on ClinicalTrials.gov: NCT00697073 ↗

Enrolled (actual)

Serious AEs

5.9%

Results posted

Aug 2011

Primary outcome: Primary: Change in ICARS — 1.1 units on a scale

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Idebenone (Drug)
Age: Pediatric, Adult · 8+ yrs
Sex: All
Sponsor: Santhera Pharmaceuticals
Primary completion: May 2010

Outcome Measures

Outcome	Result	p-value
PRIMARY Change in ICARS	1.1	—
SECONDARY FARS (Friedreich's Ataxia Rating Scale)	2.3	—
SECONDARY Nature of Adverse Events	117; 97; 75	—
SECONDARY Frequency/Number of Mild, Moderate, and Severe Adverse Events	30; 32; 4	—

Summary

This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.

Eligibility Criteria

Inclusion criteria

Friedreich's ataxia patients completing core study SNT-III-002 (NCT00537680) and presenting at Week 24 (Visit 5) of that study
Body weight ≥ 25kg/55 lbs
Negative urine pregnancy test
Patients who in the opinion of the investigator are able to comply with the requirements of this study

Exclusion criteria

Adverse events during the course of SNT-III-002(NCT00537680)which in the opinion of the investigator are attributable to idebenone and preclude further treatment with idebenone
Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 2 times the upper limit of normal of AST, ALT or creatinine
Treatment with coenzyme Q10, vitamin E (if taken at a dose 5 times above the daily requirement) or other sources of idebenone within the past month
Parallel participation in another clinical drug trial
Past or present history of abuse of drugs or alcohol
Pregnancy or breast-feeding

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00697073). 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.