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
Limb Girdle Muscular Dystrophy (LGMD) Natural History
5th February 2013
Condition: Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)
Intervention:
Sponsors: Atrium Health; Muscular Dystrophy Association
Completed
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
Withdrawn
Limb Girdle Muscular Dystrophy Type 2E Recruitment Study
10th April 2018
Condition: Limb-Girdle Muscular Dystrophy, Type 2E
Intervention:
Sponsors: Nationwide Children's Hospital; Myonexus Therapeutics
Recruiting
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
21st December 2018
Condition: Limb-Girdle Muscular Dystrophy
Interventions: Drug: Deflazacort; Drug: Placebo
Sponsor: PTC Therapeutics
Recruiting
Stem Cell Therapy in Limb Girdle Muscular Dystrophy
31st January 2014
Condition: Limb Girdle Muscular Dystrophy
Intervention: Biological: Autologous bone marrow mononuclear cell transplantation
Sponsor: Neurogen Brain and Spine Institute
Withdrawn
Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E
5th April 2018
Conditions: Limb-Girdle Muscular Dystrophy Type 2A; Limb-Girdle Muscular Dystrophy, Type 2E
Intervention:
Sponsor: Nationwide Children's Hospital
Recruiting
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
Completed
Limb-Girdle Muscular Dystrophy Type 2I in Norway
29th April 2019
Conditions: Limb Girdle Muscular Dystrophy, Type 2I; Limb Girdle Muscular Dystrophy; Muscular Dystrophies
Intervention:
Sponsors: University Hospital of North Norway; University of Tromso; Norwegian Muscle Disease Association (FFM); Norwegian National Advisory Unit on Rare Disorders (NKSD); Norwegian Competence Center for Sleep Disorders
Recruiting
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)
10th June 2019
Conditions: Limb Girdle Muscular Dystrophy; Muscular Dystrophies
Intervention:
Sponsors: Virginia Commonwealth University; Newcastle University; University of California, Irvine; University of Kansas; University of Colorado, Denver; Nationwide Children's Hospital; Washington University School of Medicine; University of Iowa; Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; University of Florida; Brigham and Women's Hospital
Recruiting
MRI on Persons With Mutations in POMT2 Gene (LGMD2N)
3rd May 2016
Condition: Limb-girdle Muscular Dystrophy
Intervention:
Sponsor: Rigshospitalet, Denmark
Completed
Gene Transfer Clinical Trial for LGMD2E (Beta-sarcoglycan Deficiency) Using scAAVrh74.MHCK7.hSGCB
29th August 2018
Condition: Limb-Girdle Muscular Dystrophy, Type 2E
Intervention: Drug: scAAVrh74.MHCK7.hSGCB
Sponsor: Sarepta Therapeutics, Inc.
Recruiting
Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I
15th February 2019
Condition: LGMD2I
Intervention:
Sponsor: Genethon
Not yet recruiting
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
Completed
Weekly Steroids in Muscular Dystrophy
13th August 2019
Conditions: Limb-girdle Muscular Dystrophy; Becker Muscular Dystrophy
Intervention: Drug: Prednisone
Sponsor: Northwestern University
Enrolling by invitation
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
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
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
Completed
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
Molecular Analysis of Patients With Neuromuscular Disease
19th October 2006
Conditions: Limb-girdle Muscular Dystrophy; Duchenne Muscular Dystrophy; Becker Muscular Dystrophy; Neuromuscular; Disorder, Hereditary
Intervention:
Sponsors: Boston Children’s Hospital; National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting
Clinical Outcome Study for Dysferlinopathy
30th August 2012
Conditions: Dysferlinopathy; LGMD2B; Miyoshi Myopathy
Intervention:
Sponsors: Newcastle-upon-Tyne Hospitals NHS Trust; Jain Foundation
Unknown status
The Global FKRP Patient Registry
28th June 2019
Conditions: LGMD2I; LGMDR9; Limb Girdle Muscular Dystrophy; Congenital Muscular Dystrophy; Muscle-Eye-Brain Disease; Walker-Warburg Syndrome; FKRP Gene Mutation
Intervention: Other: Patient Registry
Sponsor: Newcastle University
Recruiting
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
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 Association; University Hospital Southampton NHS Foundation Trust
Completed