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

Phase 3

Completed

• Conditions: Friedreich's Ataxia
• Interventions: Drug: Idebenone

• Registration Number: NCT00697073
• Lead Sponsor: Santhera Pharmaceuticals

Brief Summary

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

Detailed Description

The study involves 6 clinic visits.

Study Timeline

• Study First Submitted: 2008-06-11
• Study First Submitted QC: 2008-06-12
• Study First Posted: 2008-06-13
• Study Start: 2008-07
• Primary Completion: 2010-05
• Study Completion: 2010-05
• Results First Submitted: 2011-06-07
• Results First Submitted QC: 2011-07-11
• Results First Posted: 2011-08-04
• Status Verified: 2023-06
• Last Update Submitted: 2023-06-22
• Last Update Posted: 2023-07-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 68

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	Idebenone	high dose Idebenone

Primary Outcome Measures

Name	Time	Method
Change in ICARS	baseline and 12 months	International Cooperative Ataxia Rating Scale (ICARS): ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability.; A total score of 0 points represents the best possible score, and a value of 100 points is the worst possible score. Therefore, a negative change in ICARS score represents an improvement. A higher score also indicates more disability.

Secondary Outcome Measures

Name	Time	Method
Nature of Adverse Events	12 Months
Frequency/Number of Mild, Moderate, and Severe Adverse Events	12 months
FARS (Friedreich's Ataxia Rating Scale)	baseline and 12 Months	FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population. The FARS consists of three subscales, comprising a general score for ataxia, a score for activities of daily living (ADL) and a neurological examination. The scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability.

Trial Locations

Locations (2)

David Geffen School of Medicine, UCLA; 🇺🇸 Los Angeles, California, United States

The Children's Hopsital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States