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14 Jun 2012

Trials and projects concerning neuromuscular diseases

Illustration for article: Trials and projects concerning neuromuscular diseases
The French Muscular Dystrophy Association (AFM) supports clinical trials designed to evaluate the efficacy of innovative treatments for neuromuscular diseases. This is a list of the ongoing trials in 2019.

Charcot-Marie-Tooth disease

Preclinical development permitted the lauchment of a phase II trial published in 2014 december, followed by phase 3 trial assessing long term safety and tolerability (PLEO-CMT-FU).

Type of therapy : Pharmacology
Product : PXT3003
Phase : Phase III
 

IFB088 or Sephin1 : randomized, double blind, placebo controlled study of single ascending doses and multiple ascending doses

Type of therapy : Pharmacology
Product : IFB088
Phase : Phase I
 

Duchenne and Becker muscular dystrophies

Randomized, double-blind trial versus placebo to assess the efficacy of the nebivolol to delay the occurrence and progression of cardiac insufficiency

Type of therapy : Pharmacology
Product : Nebivolol
Phase : Phase III
 

Rimeporide : safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses 

Type of therapy : Pharmacology
Product : Rimeporide
Phase : Phase Ib
 

Preclinical development of exon 53 skipping by morpholino antisense oligonucleotide permitted the lauchment of a phase I/II trial (SRP-053)

Type of therapy : Gene therapy
Product : Antisense oligonucleotide
Phase : Phase I/II
 

Microdystrophin -based gene therapy

Type of therapy : Gene therapy
Product : AAV-microdystrophin
Phase : Preclinical development
 

Inclusion Body Myositis

Rapamycin (RAPAMI)

Type of therapy : Pharmacology
Product : Rapamycin
Phase : Phase IIb
 

Limb girdle muscular dystrophies

Transfer of the CAPN3 gene in LGMD2A (LGMD R9 linked to FKRP)

Type of therapy : Gene therapy
Product : AAV-CAPN3
Phase : Preclinical development
 

Transfer of the SGCG gene in LGMD2C (LGMD R9 linked to FKRP)

Type of therapy : Gene therapy
Product : AAV-SGCG
Phase : Preclinical development
 

Transfer of the FKRP gene in LGMD2I (LGMD R9 linked to FKRP)

Type of therapy : Gene therapy
Product : AAV-FKRP
Phase : Preclinical development

 

Myotonic dystrophy type 1

Randomized, double-blind trial versus placebo to assess the efficacy of Metformine. Published in 2018 August.

Type of therapy : Pharmacology
Product : Metformine
Phase : Phase II
 

Non-dystrophic myotonias

Randomized, cross-over, double-blind phase III trial versus placebo permitted the european marketing authorization of Mexiletin

Type of therapy : Pharmacology
Product : Mexiletin
Phase : European marketing authorization

 

Oculopharyngeal Muscular Dystrophy (OPMD)

Treatment of dysphagia in oculopharyngeal muscular dystrophy by autologous transplantation of myoblasts 

Type of therapy : Cell therapy
Product : Autologous myoblasts
Phase : Phase II

 

Spinal muscular atrophy

Preclinical development of gene therapy : proof of concept in the mice with AAV-SMN

Type of therapy : Gene therapy
Product : AAV-SMN
Phase : Preclinical development
 

X-linked myotubular myopathy

Transfer of the MTM gene 
Type of therapy : Gene therapy
Product : AAV-MTM
Phase : Phase I/II