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Showing 1–24 of 140 matching trials from the live ClinicalTrials.gov search.
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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
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
14
States / cities
Little Rock, Arkansas • Palo Alto, California • Sacramento, California + 11 more
Active, not recruiting Phase 2 Interventional

Study of BBP-418 in Patients With LGMD2I

NCT04800874
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
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
Completed Not applicable Interventional Accepts healthy volunteers

Orthotic Dose Response Study

NCT02629731
Conditions
Ankle Osteoarthritis, Flat Foot or Pes Planus
Interventions
custom orthoses with varying degrees of hindfoot posting
Device
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
18 Years to 80 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2023
U.S. locations
2
States / cities
Seattle, Washington
Conditions
Lower Extremity Surgery, Bunion, Metatarsophalangeal Fushion, Midfoot Fusion, Hindfoot Fushion, Total Ankle Arthroplasty
Interventions
Exparel, Bupivacaine Hydrochloride
Drug
Lead sponsor
Pacira Pharmaceuticals, Inc
Industry
Eligibility
18 Years and older
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
5
States / cities
Phoenix, Arizona • Pasadena, California • Boston, Massachusetts + 2 more
Conditions
Fraser Syndrome, Fryns Syndrome, Chromosomal Abnormalities, Abnormalities, Multiple
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Terminated No phase listed Observational Accepts healthy volunteers

Genetic Disease Gene Identification

NCT00916903
Conditions
Congenital Vertical Talus, Familial Encephalopathy With Neuroserpin Inclusion Bodies, Idiopathic Generalised Epilepsy, Familial Dementia, X-linked Mental Retardation
Interventions
Not listed
Lead sponsor
State University of New York - Upstate Medical University
Other
Eligibility
6 Months and older
Enrollment
176 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2015
U.S. locations
1
States / cities
Syracuse, New York
Conditions
Diabetic Foot Ulcers, Equinus Deformity
Interventions
Not listed
Lead sponsor
Georgetown University
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
End Stage Renal Failure, Hemodialysis Access Failure, Arterio-venous Fistula
Interventions
One stage approach
Procedure
Lead sponsor
Tze-Woei Tan
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Tucson, Arizona
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
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
Urothelial Cancer, Receptors, Fibroblast Growth Factor
Interventions
Archival Tumor Tissue Sample
Other
Lead sponsor
Janssen Research & Development, LLC
Industry
Eligibility
18 Years and older
Enrollment
3,679 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
23
States / cities
Tucson, Arizona • Denver, Colorado • Newark, Delaware + 20 more
Conditions
Clubfeet, Adolescent Idiopathic Scoliosis
Interventions
Not listed
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
1 Day to 99 Years
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Lexington, Kentucky
Conditions
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases
Interventions
Deramiocel (CAP-1002), Placebo
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
10 Years and older · Male only
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
20
States / cities
Phoenix, Arizona • Little Rock, Arkansas • La Jolla, California + 17 more
Conditions
Lysosomal Storage Disease, Cholesterol Metabolism
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Day to 99 Years
Enrollment
344 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Clubfoot
Interventions
Not listed
Lead sponsor
University of British Columbia
Other
Eligibility
Not listed
Enrollment
370 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2013
U.S. locations
1
States / cities
Houston, Texas
Conditions
Gastric Adenocarcinoma
Interventions
Derazantinib, Derazantinib-paclitaxel-ramucirumab combination
Drug
Lead sponsor
Basilea Pharmaceutica
Industry
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
2
States / cities
Orlando, Florida • New York, New York
Conditions
Fractures, Bone, Fractures, Closed, Fractures, Open, Fracture of Femur, Fracture Nonunion, Fracture of Tibia, Limb Deformity, Limb Fracture, Limb Defect, Limb Asymmetry
Interventions
Fitbone Transport and Lengthening System
Device
Lead sponsor
Orthofix Inc.
Industry
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
9
States / cities
Los Angeles, California • Orange, California • Atlanta, Georgia + 6 more
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
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
Active, not recruiting No phase listed Observational

LGMD R1 Natural History Study

NCT05618080
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
Hypochondroplasia
Interventions
Infigratinib
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 18 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
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