NCT01027884

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10-18 Year Old Patients With Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 65 (actual)
• Sponsor: Santhera Pharmaceuticals · Industry
• Sex: Male
• Age: 10 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD. Furthermore, the safety and tolerability of idebenone was assessed.

Detailed description

This study was a Phase III, multicenter, randomized, double-blind, placebo-controlled efficacy and safety study. DMD patients (ambulatory and non-ambulatory) at age 10-18 years were enrolled at sites in Europe and North America. Study subjects were randomized in a 1:1 ratio to receive either idebenone (900 mg/day) or placebo 3 times a day with meals for 52 weeks. The primary endpoint was the difference between Catena®/Raxone® and placebo in the change from Baseline to week 52 in Peak Expiratory Flow (PEF as percent predicted, PEF%p, a measure of respiratory muscle strength) as measured by hospital-based spirometry. PEF was also measured by the patient at home using the hand-held ASMA-1 device (secondary endpoint). Other respiratory endpoints included Forced Expiratory Volume in 1 second (as percent predicted, FEV1%p, an additional measure of respiratory muscle strength) and Forced Vital Capacity (as percent predicted, FVC%p, a measure of restrictive lung disease predictive of morbidity and mortality in DMD).

Conditions

• Muscular Dystrophy, Duchenne
• Ambulatory Care

Interventions

Type	Name	Description
DRUG	Placebo	Placebo (900 mg/day) 2 tabl (150 mg each) x 3 times orally with meals
DRUG	Idebenone	Idebenone (900 mg/day) 2 tabl (150 mg each) x 3 times orally with meals

Timeline

• Start date: 2009-07-01
• Primary completion: 2014-01-01
• Completion: 2014-04-01
• First posted: 2009-12-09
• Last updated: 2015-10-19
• Results posted: 2015-10-19

Locations

23 sites across 10 countries: United States, Austria, Belgium, France, Germany, Italy, Netherlands, Spain, Sweden, Switzerland