Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia - Tabular View

This Tabular View shows the required WHO registration data elements as marked by ^†

Tracking Information

First Received Date ^†

September 7, 2007

Last Updated Date

April 15, 2009

Start Date ^†

December 2007

Current Primary Outcome Measures ^†

ICARS [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Original Primary Outcome Measures ^†

Same as current

Change History

Complete list of historical versions of study NCT00537680 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^†

FARS [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
ADL of FARS [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
FACT [ Time Frame: 6 Months ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ^†

Same as current

Descriptive Information

Brief Title ^†

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

Official Title ^†

A Phase III Double-Blind, Randomized, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Brief 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.

Detailed Description

The study involves 6 clinic visits and upon completion the possibility to join a 12 months extension study were all patients will receive high dose Idebenone.

Study Phase

Phase III

Study Type ^†

Interventional

Study Design ^†

Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study

Condition ^†

Friedreich's Ataxia

Intervention ^†

Drug: Idebenone
Drug: Placebo

Study Arms / Comparison Groups

Experimental: mid dose Idebenone
Experimental: high dose Idebenone

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^†

Active, not recruiting

Enrollment ^†

Estimated Completion Date

May 2009

Estimated Primary Completion Date

May 2009 (final data collection date for primary outcome measure)

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

Gender

Both

Ages

8 Years to 17 Years

Accepts Healthy Volunteers

Contacts ^††

Location Countries ^†

United States

Expanded Access Status

Administrative Information

NCT ID ^†

NCT00537680

Responsible Party

Thomas Meier, PhD / Chief Scientific Officer, Santhera Pharmaceuticals (Switzerland) Ltd.

Secondary IDs ^††

Study Sponsor ^†

Santhera Pharmaceuticals

Collaborators ^††

Investigators ^†

Principal Investigator:	Susan Perlman, MD	University of California, Los Angeles
Principal Investigator:	David Lynch, MD	Children's Hospital of Philadelphia

Information Provided By

Santhera Pharmaceuticals

Verification Date

April 2009

^† Required WHO trial registration data element.
^†† WHO trial registration data element that is required only if it exists.