Clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis - Scientific guideline

This document provides guidance on the clinical investigation of medicinal products for the treatment of myotrophic lateral sclerosis. It focuses on the design of studies for disease-modifying as well as symptomatic treatments, the choice of meaningful outcome parameters and the clinical relevance of functional tests of disability including motor and respiratory functions and their relationship to survival.

Keywords: Amyotrophic lateral sclerosis (ALS), motor neuron disease

Current effective version

Guideline on clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis

AdoptedLegal effective date: 01/06/2016Reference Number: EMA/531686/2015 Corr. 1Summary:

Guideline on clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis (ALS)

Document history - First version (current)

Guideline on clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis

AdoptedLegal effective date: 01/06/2016Reference Number: EMA/531686/2015 Corr. 1Summary:

Guideline on clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis (ALS)

Overview of comments received on ' Guideline on clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis (ALS)' (EMA/531686/2015)

Reference Number: EMA/CHMP/131550/2015

Draft guideline on clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis

Consultation dates: 01/08/2013 to 31/01/2014Draft: consultation closedReference Number: EMA/CHMP/40105/2013Summary:

This document replaces and updates the previous points to consider on amyotrophic lateral sclerosis and focuses on the design of studies for disease-modifying as well as symptomatic treatments in this therapeutic area, the choice of meaningful outcome parameters and the clinical relevance of functional tests of disability, including motor and respiratory functions and their relationship to survival.

Concept paper on the need for revision of the points to consider on clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis

Consultation dates: 01/04/2012 to 30/06/2012Draft: consultation closedReference Number: EMA/CHMP/59352/2012Summary:

Amyotrophic lateral sclerosis (ALS) is a rare progressive motor neuron disease (reported incidence 0.8-2.4 per 100 000 person per year) characterised by axonal degeneration and death of motor neurons leading to progressive denervation and atrophy of skeletal muscles, paralysis and, in the majority of cases, death.

Document history - Superseded document

Points to consider on clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis

AdoptedLegal effective date: 01/05/2001Reference Number: CPMP/EWP/565/98