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ClinicalTrials.gov public records Last synced Sep 17, 2026, 4:48 PM EDT

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Showing 1–24 of 46 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Limb-Girdle Muscular Dystrophy Clear all
Recruiting No phase listed Observational

Natural History Study for Charcot Marie Tooth Disease

NCT05902351
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease Type 2, Charcot-Marie-Tooth Disease, Type 2C, Charcot-Marie-Tooth Disease Type 2A2B, Charcot-Marie-Tooth Disease Type 2B2, Charcot-Marie-Tooth Disease Type 2A1, Charcot-Marie-Tooth Disease Type 4B1, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease Type 2B1, Charcot-Marie-Tooth Disease Type 2U (Diagnosis), Charcot-Marie-Tooth Disease Type 4A, Charcot-Marie-Tooth Disease, Type 4A, Axonal Form, Charcot-Marie-Tooth Disease Type 2A2A, Charcot-Marie-Tooth Disease Type 2S (Disorder), Charcot-Marie-Tooth Disease and Deafness, Charcot-Marie-Tooth Disease Type 4B2, Charcot-Marie-Tooth Disease Type 4H, Charcot-Marie-Tooth Disease Type 1F, Charcot-Marie-Tooth Disease Type 4C, Charcot-Marie-Tooth Disease Type 4E, Charcot-Marie-Tooth Disease Type 1D, Charcot-Marie-Tooth Disease Type 2Q (Diagnosis), Charcot-Marie-Tooth Disease Type 2A2, Charcot-Marie-Tooth Disease Type 2N (Diagnosis), Charcot-Marie-Tooth Disease Type 2B5, Charcot-Marie-Tooth Disease Type 2D, Charcot-Marie-Tooth Disease Type 4D, Charcot-Marie-Tooth Disease Type 2K, Charcot-Marie-Tooth Disease Type 2L (Diagnosis), Charcot-Marie-Tooth Disease Type 2T, Charcot-Marie-Tooth Disease Type 2I, Charcot-Marie-Tooth Disease Type 2J, Charcot-Marie-Tooth Disease Type 2E, Charcot-Marie-Tooth Disease Type 2G, Charcot-Marie-Tooth Disease Type 1C, Charcot-Marie-Tooth Disease Type 2R, Charcot-Marie-Tooth Disease Type 2O (Diagnosis), Charcot-Marie-Tooth Disease Type 2M, Charcot-Marie-Tooth Disease Type 2P, Charcot-Marie-Tooth Disease Type 2Y, Charcot-Marie-Tooth Disease Type 4F (Diagnosis), Charcot-Marie-Tooth Disease Type 4B3, Charcot-Marie-Tooth Disease Type 2H, HNPP, X-Linked Charcot-Marie-Tooth Disease, Charcot Marie Tooth Disease (CMT)
Interventions
Not listed
Lead sponsor
Hereditary Neuropathy Foundation
Government
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Sep 17, 2026, 4:48 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

NCT05230459
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
Interventions
AB-1003 dose level 1, AB-1003 dose level 2, Placebo
Genetic · Drug
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2032
U.S. locations
6
States / cities
Irvine, California • Iowa City, Iowa • Kansas City, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 17, 2026, 4:48 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluation of Limb-Girdle Muscular Dystrophy

NCT00893334
View directory record Official record
Conditions
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency), Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency), Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 6, 2014 · Synced Sep 17, 2026, 4:48 PM EDT
Completed No phase listed Observational

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)

NCT03981289
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy, Muscular Dystrophies
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
4 Years to 65 Years
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
9
States / cities
Irvine, California • Aurora, Colorado • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2026 · Synced Sep 17, 2026, 4:48 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

A Study To Evaluate The Pharmacokinetics, Excretion, Mass Balance and Metabolism of PF-07265803

NCT05286281
View directory record Official record
Conditions
Healthy
Interventions
PF-07265803
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older · Male only
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Mar 28, 2024 · Synced Sep 17, 2026, 4:48 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

What Makes Exercise Feel More Difficult to Women With and Without Type 2 Diabetes

NCT00785005
View directory record Official record
Conditions
Type 2 Diabetes
Interventions
Not listed
Lead sponsor
University of Colorado, Denver
Other
Eligibility
50 Years to 70 Years · Female only
Enrollment
99 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2011
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Oct 13, 2022 · Synced Sep 17, 2026, 4:48 PM EDT
Completed No phase listed Observational

Biomarker Development in LGMD2i

NCT04202627
View directory record Official record
Conditions
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Interventions
Not listed
Lead sponsor
ML Bio Solutions, Inc.
Industry
Eligibility
10 Years to 65 Years
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
10
States / cities
Irvine, California • Aurora, Colorado • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2023 · Synced Sep 17, 2026, 4:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Trial of PF-06252616 in Ambulatory Participants With LGMD2I

NCT02841267
View directory record Official record
Conditions
LGMD2I
Interventions
PF 06252616
Drug
Lead sponsor
Kathryn Wagner
Other
Eligibility
18 Years to 99 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 18, 2020 · Synced Sep 17, 2026, 4:48 PM EDT
Completed Phase 1 Interventional

rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies

NCT02710500
View directory record Official record
Conditions
Dysferlinopathy
Interventions
rAAVrh74.MHCK7.DYSF.DV
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated May 12, 2021 · Synced Sep 17, 2026, 4:48 PM EDT
Active, not recruiting Phase 3 Interventional

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

NCT06246513
View directory record Official record
Conditions
Limb-girdle Muscular Dystrophy
Interventions
SRP-9003, Glucocorticoid
Biological · Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
4
States / cities
La Jolla, California • Columbus, Ohio • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Sep 17, 2026, 4:48 PM EDT
Completed No phase listed Observational

Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy

NCT00457912
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Not listed
Enrollment
277 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2018
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 24, 2018 · Synced Sep 17, 2026, 4:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT02579239
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy
Interventions
ATYR1940, Placebo
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
3
States / cities
Irvine, California • Baltimore, Maryland • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 18, 2023 · Synced Sep 17, 2026, 4:48 PM EDT
Completed No phase listed Observational

Cardiac Magnetic Resonance in Children With Muscular Dystrophy

NCT01081080
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
8 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 10, 2013 · Synced Sep 17, 2026, 4:48 PM EDT
Terminated Phase 1Phase 2 Interventional

Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

NCT03652259
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophy, Type 2E
Interventions
SRP-9003
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 15 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 19, 2026 · Synced Sep 17, 2026, 4:48 PM EDT
Recruiting No phase listed Observational

Molecular Analysis of Patients With Neuromuscular Disease

NCT00390104
View directory record Official record
Conditions
Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy, Limb-girdle Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Week to 100 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 23, 2023 · Synced Sep 17, 2026, 4:48 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety Study of Lenalidomide Plus R-CHOP Chemotherapy Versus Placebo Plus R-CHOP Chemotherapy in Untreated ABC Type Diffuse Large B-cell Lymphoma

NCT02285062
View directory record Official record
Conditions
Lymphoma, Large B-Cell, Diffuse
Interventions
lenalidomide, Placebo, Rituximab, Cyclophosphamide, Doxorubicin, prednisone, vincristine
Drug
Lead sponsor
Celgene
Industry
Eligibility
18 Years to 80 Years
Enrollment
570 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
22
States / cities
Fayetteville, Arkansas • Orange, California • Sacramento, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jun 21, 2023 · Synced Sep 17, 2026, 4:48 PM EDT
Terminated Phase 1 Interventional

Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States

NCT06747273
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3
Interventions
SRP-9004
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
2
States / cities
Columbus, Ohio • Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced Sep 17, 2026, 4:48 PM EDT
Active, not recruiting Phase 3 Interventional

Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)

NCT05775848
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
Interventions
BBP-418 (ribitol), Placebo
Drug · Other
Lead sponsor
ML Bio Solutions, Inc.
Industry
Eligibility
12 Years to 60 Years
Enrollment
112 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
13
States / cities
Little Rock, Arkansas • Irvine, California • Aurora, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Sep 17, 2026, 4:48 PM EDT
Active, not recruiting Phase 2 Interventional

Study of BBP-418 in Patients With LGMD2I

NCT04800874
View directory record Official record
Conditions
LGMD2I
Interventions
BBP-418
Drug
Lead sponsor
ML Bio Solutions, Inc.
Industry
Eligibility
12 Years to 55 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Sep 17, 2026, 4:48 PM EDT
Terminated Phase 3 Interventional Results available

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

NCT03783923
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophy
Interventions
Deflazacort
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
8
States / cities
Atlanta, Georgia • Iowa City, Iowa • Kansas City, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 26, 2022 · Synced Sep 17, 2026, 4:48 PM EDT
Withdrawn Phase 3 Interventional

A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants

NCT06952686
View directory record Official record
Conditions
Muscular Dystrophies, Limb-Girdle
Interventions
SRP-9005, Corticosteroid
Biological · Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 5, 2025 · Synced Sep 17, 2026, 4:48 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Stem Cell Quantification In Peripheral Blood After Hyperbaric Oxygen Therapy Treatments

NCT01740908
View directory record Official record
Conditions
Changes in Peripheral Blood Population Numbers and Type.
Interventions
Hyperbaric Oxygen Treatment
Other
Lead sponsor
Restorix Research Institute, LLLP
Other
Eligibility
18 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Anacortes, Washington
Source: ClinicalTrials.gov public record
Updated Dec 3, 2012 · Synced Sep 17, 2026, 4:48 PM EDT
Completed Phase 2 Interventional Results available

Weekly Steroids in Muscular Dystrophy

NCT04054375
View directory record Official record
Conditions
Limb-girdle Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Prednisone
Drug
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 20, 2023 · Synced Sep 17, 2026, 4:48 PM EDT
Terminated Phase 3 Interventional Results available

A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation

NCT03439514
View directory record Official record
Conditions
Dilated Cardiomyopathy, Lamin A/C Gene Mutation
Interventions
ARRY-371797 (PF-07265803), Placebo
Drug · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
56
States / cities
Birmingham, Alabama • Mobile, Alabama • Chandler, Arizona + 26 more
Source: ClinicalTrials.gov public record
Updated Jan 8, 2024 · Synced Sep 17, 2026, 4:48 PM EDT