Clinical Trials Logo
  1. Home
  2. Friedreich's Ataxia
  3. NCT00905268

A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients — MICONOS  

Friedreich's Ataxia Clinical Trial

Official title:
A Phase III Double-blind, Randomised, Placebo-controlled Study of the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Clinical Trial 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.

Clinical Trial Description

Idebenone, a short-chain analogue of Co-enzyme Q10 (CoQ10), has the potential to moderate underlying causes of Friedreich's Ataxia through its antioxidant activity and its role as an electron carrier in the respiratory chain promoting mitochondrial ATP production.

The current 12-month placebo-controlled treatment study in 232 patients aims to confirm the positive effect of idebenone on neurological function, as for instance observed in the recent 48-patient, 6-month NICOSIA study in children, using the International Cooperative Ataxia Rating Scale (ICARS) and the newly developed Friedreich's Ataxia Rating Scale (FARS).

In addition, the study aims to confirm the positive effect on cardiomyopathy associated with FRDA observed in several small studies. Cardiac anatomy and function will be assessed using echocardiography, tissue Doppler imaging and cardiac MRI methods in patients with FRDA cardiomyopathy. In addition exercise capacity, measured as peak workload, will be assessed in patients able to comply with a modified exercise protocol. ;

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT00905268
Study type Interventional
Source Santhera Pharmaceuticals
Contact
Status Completed
Phase Phase 3
Start date April 2006
Completion date January 2010
View Details
See also
  Status Clinical Trial Phase
Completed NCT02660112 - (+) Epicatechin to Treat Friedreich's Ataxia Phase 2
Recruiting NCT02497534 - Biomarkers in Friedreich's Ataxia
Completed NCT04102501 - A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia Phase 3
Completed NCT01962363 - EPI-743 in Friedreich's Ataxia Point Mutations Phase 2
Completed NCT02179333 - Preliminary Study of the Scale To Assess Ataxia and Neurologic Dysfunction (STAND)
Completed NCT01016366 - Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia Phase 2
Recruiting NCT02069509 - Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)
Terminated NCT00803868 - Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia Phase 2/Phase 3
Completed NCT02797080 - Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia Phase 3
Completed NCT02415127 - Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Phase 3
Completed NCT00897221 - A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia Phase 2
Completed NCT02840669 - A Study to Characterize the Cardiac Phenotype of Individuals With Friedreich's Ataxia (CARFA Study) N/A
Completed NCT00697073 - Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients Phase 3
Recruiting NCT02316314 - Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)
Completed NCT00631202 - Efficacy of Epoetin Alfa in Patients With Friedreich's Ataxia Phase 2
Completed NCT01728064 - Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia Phase 2
Completed NCT02593773 - Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study Phase 3
Completed NCT01035671 - Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia Phase 2
Completed NCT00811681 - Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept Phase 3
Completed NCT00537680 - Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Phase 3