Donate
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 2012.

 

Congenital Muscular Dystrophies (MDC1A and collagen VI disorders)

Evaluation of a anti-apoptotic drug
  • Type of therapy : Pharmacology
  • Product : Omigapil
  • Phase : Phase I

Duchenne and Becker muscular dystrophies

Randomised, 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

Open-label study for previously treated ataluren (PTC124) patients with nonsense mutation dystrophinopathy 

  • Type of therapy : Gene therapy
  • Product : Ataluren (PTC 124)
  • Phase : Phase III 
Exon 44 skipping by antisense oligonucleotide
  • Type of therapy : Gene therapy
  • Product : Antisense oligonucleotide
  • Phase : Phase I/II
Exon 45 skipping by antisense oligonucleotide
  • Type of therapy : Gene therapy
  • Product : Antisense oligonucleotide
  • Phase : Phase II/B
Exon 53 skipping by antisense oligonucleotide
  • Type of therapy : Gene therapy
  • Product : Morpholino oligomer
  • Phase : Phase IIb
  • Type of therapy : Gene therapy
  • Product : AAV-U7
  • Phase : Preclinical development
Tamoxifen
  • Type of therapy : Pharmacology
  • Product : Tamoxifen
  • Phase : Preclinical development

Facioscapulohumeral muscular dystrophy

Autologous transplantation of myoblasts
  • Type of therapy : Cell therapy
  • Product : Autologous myoblasts
  • Phase : Phase I/II

Limb girdle muscular dystrophies

Transfer of the alpha-sarcoglycan gene in alpha- sarcoglycanopathy

  • Type of therapy : Gene therapy
  • Product : AAV-SGCA
  • Phase : Preclinical development
Evaluation of a mannosidase-I inhibitor in alpha-sarcoglycanopathy
  • Type of therapy : Pharmacology
  • Product : Kifunensine
  • Phase : Preclinical development

Myotonic dystrophy type 1

Metformine
  • Type of therapy : Pharmacology
  • Product : Metformine
  • Phase : Preclinical development
Non-dystrophic myotonias
  • Randomised, cross-over, double-blind trial versus placebo to assess the efficacy and the tolerance of mexiletine
  • Type of therapy : Pharmacology
  • Product : Mexiletine
  • Phase : Phase III

Oculopharyngeal Muscular Dystrophy (OPMD)

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

Periodic paralysis

  • Type of therapy : Pharmacology
  • Product : Dichlorphenamide
  • Phase: Phase III

Spinal muscular atrophy

  • Type of therapy : Pharmacology
  • Product : Riluzole
  • Phase : Phase III
  • Type of therapy : Pharmacology
  • Product : Olesoxime (TRO 19 622)
  • Phase : Phase II

X-linked myotubular myopathy

  • Type of therapy : Gene therapy
  • Product : AAV-MTM
  • Phase : Preclinical development