Clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy - Scientific guideline

This document provides guidance on the development of any medicinal product for the treatment of Duchenne or Becker muscular dystrophy. It addresses the identification of the target population, study design and choice of efficacy endpoints and safety parameters.

Keyword: Duchenne and Becker muscular dystrophy, paediatric population, genetic neuromuscular disorder, molecular diagnosis, motor function, muscle strength

Current effective version

Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy

Adopted Reference Number: EMA/CHMP/236981/2011 Corr.1 Summary:

Recent advances in basic and clinical research have opened new perspectives for future therapeutic options in Duchenne and Becker muscular dystrophy (DBMD). The increasing number of clinical trials that recruit a rather small number of patients and the high degree of variability of disease progression for these disorders have raised a number of issues relating to the study design, including the choice of appropriate clinical efficacy endpoints, the definition of reliable surrogate outcome measures the need for subgroup analyses within the heterogeneous patient population and the possibility of data extrapolation.

Document history - First version (current)

Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy

Adopted Reference Number: EMA/CHMP/236981/2011 Corr.1 Summary:

Recent advances in basic and clinical research have opened new perspectives for future therapeutic options in Duchenne and Becker muscular dystrophy (DBMD). The increasing number of clinical trials that recruit a rather small number of patients and the high degree of variability of disease progression for these disorders have raised a number of issues relating to the study design, including the choice of appropriate clinical efficacy endpoints, the definition of reliable surrogate outcome measures the need for subgroup analyses within the heterogeneous patient population and the possibility of data extrapolation.

Overview of comments received on 'Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy'

Reference Number: EMA/30262/2016 Summary:

Overview of comments received on 'Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy' (EMA/CHMP/236981/2011)

Draft guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy

Draft: consultation closed Consultation dates: 01/03/2013 to 31/08/2013 Reference Number: EMA/CHMP/236981/2011 Summary:

Recent advances in basic and clinical research have opened new perspectives for future therapeutic options in Duchenne and Becker muscular dystrophy. This guideline is intended to provide guidance for the evaluation of medicinal products in the treatment of these diseases, including study design, the choice of appropriate efficacy endpoints and the definition of reliable surrogate outcome measures.

Concept paper on the need for a guideline on the treatment of Duchenne and Becker muscular dystrophy

Draft: consultation closed Consultation dates: 23/06/2011 to 30/09/2011 Reference Number: EMA/CHMP/CNSWP/236981/2011 Summary:

Neuromuscular diseases encompass a broad spectrum of diverse muscular disorders (e.g. inherited myopathies, metabolic and inflammatory myopathies), diseases of neuromuscular transmission (e.g. myasthenia gravis, Lambert-Eaton myasthenic syndrome, hereditary neuromuscular disorders) as well as disorders of the upper and lower motoneurons (e.g. amyotrophic lateral sclerosis, spinal muscular atrophy). Neuromuscular diseases can involve both, muscles that are moved voluntarily and those that function automatically, e.g. for breathing.