- Conditions
- Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)
- Interventions
- Not listed
- Lead sponsor
- Wake Forest University Health Sciences
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2017
- U.S. locations
- 1
- States / cities
- Charlotte, North Carolina
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- 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
- 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
- Conditions
- Muscular Dystrophies
- Interventions
- rAAV1.tMCK.human-alpha-sarcoglycan- First cohort, Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort
- Genetic
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- LGMD2C
- Interventions
- ATA-200
- Biological
- Lead sponsor
- Atamyo Therapeutics
- Industry
- Eligibility
- 6 Years to 13 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2031
- U.S. locations
- 1
- States / cities
- Gainsville, Florida
- 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
- Conditions
- Dysferlinopathy, LGMD2B Now Classified as LGMDR2, Miyoshi Myopathy
- Interventions
- Not listed
- Lead sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
- Other
- Eligibility
- 10 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2024
- U.S. locations
- 6
- States / cities
- Orange, California • Palo Alto, California • St Louis, Missouri + 3 more
- 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
- Conditions
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan and Epilepsy), Alpha-Dystroglycanopathy (Dystroglycanopathy, Congenital With or Without Mental Retardation (Formerly MDC1C)), Alpha-Dystroglycanopathy (Fukuyama CMD), Alpha-Dystroglycanopathy (LGMDR09 FKRP Related (Formerly LGMD2I)), Alpha-Dystroglycanopathy (LGMDR11 POMT1 Related (Formerly LGMD2K)), Alpha-Dystroglycanopathy (LGMDR13 FKTN Related (Formerly LGMD2M)), Alpha-Dystroglycanopathy (LGMDR14 POMT2 Related (Formerly LGMD2N)), Alpha-Dystroglycanopathy (LGMDR15 POMGnT1 Related (Formerly LGMD2O)), Alpha-Dystroglycanopathy (LGMDR19 GMPPB Related (Formerly LGMD2T)), Alpha-Dystroglycanopathy (LGMDR20 ISPD Related (Formerly LGMD2U)), Alpha-Dystroglycanopathy (LGMDR24 POMGnT2 Related), Alpha-Dystroglycanopathy (Muscle Eye Brain Disease (MEB)), Alpha-Dystroglycanopathy (Walker Warburg Syndrome (WWS)), Choline Kinase B Receptor - CHKB, Collagen VI Related Disorders, Collagen XII Related Disorders, Congenital Muscular Dystrophy Not Otherwise Specified (Including Merosin Positive), Congenital Muscular Dystrophy With Cataracts and Intellectual Disability (MDCCAID), Congenital Muscular Dystrophy With Joint Hyperlaxity, Congenital Muscular Dystrophy With Rigid Spine Related to ACTA1, Emery-Dreifuss Muscular Dystrophy, GOLGA2-related Congenital Muscle Dystrophy With Brain Involvement, LMNA Related Disorders, Merosin Deficient CMD (Full or Partial), Nesprin Related MD (SYNE1), SELENON Related Disorders (Previously Known as SEPN1), SELENON Related Myopathy (Aka SEPN1), Telethonin CMD, Congenital Myasthenic Syndrome, Limb-Girdle Muscular Dystrophy, LGMDD01 - DNAJB6 (Formerly LGMD1D), LGMDD05 - Collagen VI Related Bethlem Myopathy (Dominant), LGMDR07 - Telethonin (TCAP) Related (Formerly LGMD2G), LGMDR08 - TRIM Related (Formerly LGMD2H), LGMDR09 - FKRP Related (Formerly LGMD2I), LGMDR10 - Titin (TTN) Related (Formerly LGMD2J), LGMDR11 - POMT1 Related (Formerly LGMD2K), LGMDR13 - Fukutin (FKTN) Related (Formerly LGMD2M), LGMDR14 - POMT2 Related (Formerly LGMD2N), LGMDR15 - POMGnT1 Related (Formerly LGMD2O), LGMDR16 - DAG1 Related Dystroglycanopathy (Formerly LGMD2P), LGMDR17 - Plectin (PLEC) Related (Formerly LGMD2Q), LGMDR18 - TRAPPC11 Related (Formerly LGMD2S), LGMDR19 - GMPPB Related (Formerly LGMD2T), LGMDR20 - ISPD Related (Formerly LGMD2U), LGMDR22 - Collagen VI Related Bethlem Myopathy (Recessive), LGMDR23 - LAMA2 Related, LGMDR24 - POMGnT2 Related
- Interventions
- Not listed
- Lead sponsor
- Cure CMD
- Other
- Eligibility
- Not listed
- Enrollment
- 4,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2029
- U.S. locations
- 1
- States / cities
- Lakewood, California
- Conditions
- Type 2 Diabetes
- Interventions
- Exercise program, Ascorbic Acid (Vitamin C)
- Behavioral · Drug
- Lead sponsor
- University of Colorado, Denver
- Other
- Eligibility
- 30 Years to 55 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
- 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
- Conditions
- LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y
- Interventions
- Not listed
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 6 Years to 50 Years
- Enrollment
- 1,000 participants
- Timeline
- 2023 – 2029
- U.S. locations
- 1
- States / cities
- Richmond, Virginia
- 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
- 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 · Other
- Lead sponsor
- AskBio Inc
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 10 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
- Conditions
- Limb Girdle Muscular Dystrophy
- Interventions
- SRP-6004
- Genetic
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Muscular Dystrophies, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy
- Interventions
- Retrograde high pressure transvenous perfusion with normal saline
- Other
- Lead sponsor
- University of North Carolina, Chapel Hill
- Other
- Eligibility
- 21 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2014
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
- 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
- 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
- Conditions
- Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Katherine Mathews
- Other
- Eligibility
- Not listed
- Enrollment
- 190 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2030
- U.S. locations
- 1
- States / cities
- Iowa City, Iowa
- 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
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy, FSHD, Cerebral Palsy, Becker Muscular Dystrophy
- Interventions
- Abilitech Assist
- Device
- Lead sponsor
- AbiliTech Medical Inc.
- Industry
- Eligibility
- 10 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Saint Paul, Minnesota