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

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

30th June 2016

Conditions:   Facioscapulohumeral Muscular Dystrophy;   Limb Girdle Muscular Dystrophy
Intervention:   Drug: ATYR1940
Sponsor:   aTyr Pharma, Inc.
Enrolling by invitation - verified December 2016

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Acceptance and Commitment Therapy for Muscle Disease

20th 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
Recruiting - verified June 2016

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MRI on Persons With Mutations in POMT2 Gene (LGMD2N)

27th April 2016

Condition:   Limb-girdle Muscular Dystrophy
Intervention:  
Sponsor:   Rigshospitalet, Denmark
Active, not recruiting - verified October 2016

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rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies

11th February 2016

Condition:   Dysferlinopathy
Intervention:   Drug: rAAVrh74.MHCK7.DYSF.DV
Sponsor:   Nationwide Children's Hospital
Recruiting - verified November 2016

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MRI and Muscle Involvement in Patients With Mutations in GMPPB

1st December 2015

Condition:   Limb-girdle Muscular Dystrophy
Intervention:  
Sponsor:   Rigshospitalet, Denmark
Completed - verified April 2016

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

28th September 2015

Conditions:   Limb-Girdle Muscular Dystrophies;   Facioscapulohumeral Muscular Dystrophy
Interventions:   Biological: ATYR1940;   Biological: Placebo
Sponsor:   aTyr Pharma, Inc.
Completed - verified January 2017

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Cell Therapy in Limb Girdle Muscular Dystrophy

12th September 2014

Condition:   Limb Girdle Muscular Dystrophy
Intervention:   Biological: Stem Cell
Sponsor:   Neurogen Brain and Spine Institute
Completed - verified September 2016

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

11th June 2014

Conditions:   Becker Muscular Dystrophy;   Limb-Girdle Muscular Dystrophy Type 2I
Intervention:  
Sponsor:   Rigshospitalet, Denmark
Completed - verified November 2016

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

24th July 2013

Condition:   Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
Intervention:   Drug: scAAVrh74.tMCK.hSGCA
Sponsor:   Jerry R. Mendell
Enrolling by invitation - verified March 2016

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Limb Girdle Muscular Dystrophy (LGMD) Natural History

18th January 2013

Condition:   Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)
Intervention:  
Sponsors:   Carolinas Healthcare System;   Muscular Dystrophy Association
Recruiting - verified February 2015

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Clinical Outcome Study for Dysferlinopathy

28th August 2012

Conditions:   Dysferlinopathy;   LGMD2B;   Miyoshi Myopathy
Intervention:  
Sponsors:   Newcastle-upon-Tyne Hospitals NHS Trust;   Jain Foundation
Active, not recruiting - verified October 2015

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Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

18th 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 - verified June 2016

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Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy

1st April 2009

Conditions:   Muscular Dystrophies;   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy;   Limb-Girdle Muscular Dystrophy
Intervention:   Other: Retrograde high pressure transvenous perfusion with normal saline
Sponsors:   University of North Carolina, Chapel Hill;   National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);   University of North Carolina
Completed - verified January 2015

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Deflazacort in Dysferlinopathies

6th September 2007

Conditions:   LGMD2B;   Miyoshi Myopathy;   Dysferlinopathy
Interventions:   Drug: deflazacort;   Drug: placebo
Sponsor:   Ludwig-Maximilians - University of Munich
Completed - verified August 2015

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Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy

6th 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 - verified July 2016

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16 Mar 2011