- 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
Search Results
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Showing 1–24
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- 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
- 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
- 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
- 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
- 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
- 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
- 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
- Conditions
- Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Interventions
- ATYR1940
- Drug
- Lead sponsor
- aTyr Pharma, Inc.
- Industry
- Eligibility
- 16 Years to 25 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 3
- States / cities
- Irvine, California • Stanford, California • Salt Lake City, Utah
- 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
- 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
- Conditions
- Limb Girdle Muscular Dystrophy
- Interventions
- SRP-9003
- Genetic
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years to 50 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2028
- U.S. locations
- 2
- States / cities
- Columbus, Ohio • Memphis, Tennessee
- 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
- 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
- Conditions
- Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
- Interventions
- BBP-418 (ribitol)
- Drug
- Lead sponsor
- ML Bio Solutions, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 107 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 12
- States / cities
- Little Rock, Arkansas • Orange, California • Aurora, Colorado + 8 more
- 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
- 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
- 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
- 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
- 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
- Conditions
- Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2I
- Interventions
- Not listed
- Lead sponsor
- The Emmes Company, LLC
- Industry
- Eligibility
- 14 Years and older
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022
- U.S. locations
- 1
- States / cities
- Kingston, Massachusetts
- Conditions
- Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Limb-Girdle Muscular Dystrophy
- Interventions
- MYO-029
- Drug
- Lead sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 108 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 9
- States / cities
- Washington D.C., District of Columbia • Kansas City, Kansas • Baltimore, Maryland + 6 more
- Conditions
- Limb-girdle Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 205 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2030
- U.S. locations
- 11
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • La Jolla, California + 8 more
- Conditions
- Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A, Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type R1, LGMD2A
- Interventions
- Not listed
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 12 Years to 50 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 9
- States / cities
- Orange, California • Aurora, Colorado • Gainesville, Florida + 6 more
- 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