Current trials in LGMD

TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trials that are currently ongoing.

Nevertheless, we cannot ourselves keep an up-to-date overview of all the trials that are currently happening across the world. The most comprehensive online listing of trials is at clinicaltrials.gov - companies are obliged to provide details of their trials there.

The table below lists all LGMD trials registered or updated on clinicaltrials.gov in the last 1000 days. Because it links directly to clinicaltrials.gov it is automatically updated whenever the information on clinicaltrials.gov changes.

Please note that TREAT-NMD can take no responsibility for the accuracy of the information below, which is pulled directly from clinicaltrials.gov.

Trials for LGMD listed on clinicaltrials.gov

User-centred Assistive System for Arm Functions in Neuromuscular Subjects

25th April 2017

Conditions:   Muscular Dystrophies;   Muscular Dystrophy, Duchenne;   Muscular Dystrophy, Becker;   Muscular Dystrophy, Limb-Girdle Type 2
Interventions:   Device: Assistive device (Armon Ayura) test;   Device: Assistive device (Jaeco Wrex) test
Sponsors:   Politecnico di Milano;   IRCCS Eugenio Medea;   Villa Beretta Rehabilitation Center
Recruiting

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Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy

19th July 2016

Conditions:   Facioscapulohumeral Muscular Dystrophy;   Limb Girdle Muscular Dystrophy
Intervention:   Drug: ATYR1940
Sponsor:   aTyr Pharma, Inc.
Completed

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MRI on Persons With Mutations in POMT2 Gene (LGMD2N)

3rd May 2016

Condition:   Limb-girdle Muscular Dystrophy
Intervention:  
Sponsor:   Rigshospitalet, Denmark
Completed

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Acceptance and Commitment Therapy for Muscle Disease

22nd June 2016

Condition:   Muscle Diseases
Intervention:   Behavioral: Acceptance and Commitment Therapy (ACT)
Sponsors:   King's College Hospital NHS Trust;   King's College London;   Barts & The London NHS Trust;   Muscular Dystrophy UK;   University Hospital Southampton NHS Foundation Trust
Recruiting

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rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies

16th March 2016

Condition:   Dysferlinopathy
Intervention:   Drug: rAAVrh74.MHCK7.DYSF.DV
Sponsor:   Nationwide Children's Hospital
Active, not recruiting

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MRI and Muscle Involvement in Patients With Mutations in GMPPB

18th December 2015

Condition:   Limb-girdle Muscular Dystrophy
Intervention:  
Sponsor:   Rigshospitalet, Denmark
Completed

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The Safety and Biological Activity of ATYR1940 in Patients With Limb Girdle or Facioscapulohumeral Muscular Dystrophies

19th October 2015

Conditions:   Limb-Girdle Muscular Dystrophies;   Facioscapulohumeral Muscular Dystrophy
Interventions:   Biological: ATYR1940;   Biological: Placebo
Sponsor:   aTyr Pharma, Inc.
Completed

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Cell Therapy in Limb Girdle Muscular Dystrophy

22nd September 2014

Condition:   Limb Girdle Muscular Dystrophy
Intervention:   Biological: Stem Cell
Sponsor:   Neurogen Brain and Spine Institute
Completed

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Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I

17th June 2014

Conditions:   Becker Muscular Dystrophy;   Limb-Girdle Muscular Dystrophy Type 2I
Intervention:  
Sponsor:   Rigshospitalet, Denmark
Completed

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Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA

5th November 2013

Condition:   Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
Intervention:   Drug: scAAVrh74.tMCK.hSGCA
Sponsor:   Jerry R. Mendell
Active, not recruiting

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Limb Girdle Muscular Dystrophy (LGMD) Natural History

5th February 2013

Condition:   Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)
Intervention:  
Sponsors:   Carolinas Healthcare System;   Muscular Dystrophy Association
Completed

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Clinical Outcome Study for Dysferlinopathy

30th August 2012

Conditions:   Dysferlinopathy;   LGMD2B;   Miyoshi Myopathy
Intervention:  
Sponsors:   Newcastle-upon-Tyne Hospitals NHS Trust;   Jain Foundation
Active, not recruiting

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Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

20th May 2010

Conditions:   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy;   Limb Girdle Muscular Dystrophy
Intervention:   Drug: Coenzyme Q10 and Lisinopril
Sponsors:   Cooperative International Neuromuscular Research Group;   United States Department of Defense
Enrolling by invitation

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Deflazacort in Dysferlinopathies

10th September 2007

Conditions:   LGMD2B;   Miyoshi Myopathy;   Dysferlinopathy
Interventions:   Drug: deflazacort;   Drug: placebo
Sponsor:   Ludwig-Maximilians - University of Munich
Completed

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Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy

9th April 2007

Condition:   Limb-Girdle Muscular Dystrophy
Intervention:  
Sponsors:   Nationwide Children's Hospital;   National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);   Muscular Dystrophy Association
Active, not recruiting

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Molecular Analysis of Patients With Neuromuscular Disease

19th October 2006

Conditions:   Limb-girdle Muscular Dystrophy;   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy;   Facioscapulohumeral Muscular Dystrophy
Intervention:  
Sponsors:   Boston Children’s Hospital;   National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting

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12 Apr 2017