- Conditions
- Myotonic Dystrophy 1, Excessive Daytime Sleepiness
- Interventions
- Pitolisant Oral Tablet, Placebo oral tablet
- Drug
- Lead sponsor
- Harmony Biosciences Management, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 11
- States / cities
- Irvine, California • Aurora, Colorado • Tampa, Florida + 8 more
Search Results
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Showing 1–24
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- Conditions
- Myotonic Dystrophy, Congenital Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Dystrophia Myotonica, Dystrophia Myotonica 1, Dystrophia Myotonica 2, Myotonia Dystrophica, Myotonic Dystrophy, Congenital, Myotonic Myopathy, Proximal, PROMM (Proximal Myotonic Myopathy), Proximal Myotonic Myopathy, Steinert Disease, Steinert Myotonic Dystrophy, Steinert's Disease, Myotonia Atrophica
- Interventions
- Patient-entered data
- Other
- Lead sponsor
- Myotonic Dystrophy Foundation
- Other
- Eligibility
- Not listed
- Enrollment
- 3,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2030
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Myotonic Dystrophy Type 1, Congenital DM1, Juvenile DM1, Childhood DM1
- Interventions
- Not listed
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 0 Years to 17 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2030
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
- Interventions
- Not listed
- Lead sponsor
- Sanguine Biosciences
- Industry
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 17,667 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2024
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Myotonic Dystrophy 1
- Interventions
- PGN-EDODM1 for infusion, Placebo
- Drug · Other
- Lead sponsor
- PepGen Inc
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 7
- States / cities
- Irvine, California • Palo Alto, California • Atlanta, Georgia + 4 more
- Conditions
- Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1
- Interventions
- Ranolazine
- Drug
- Lead sponsor
- Ohio State University
- Other
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Myotonic Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Wake Forest University Health Sciences
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 75 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2029
- U.S. locations
- 1
- States / cities
- Winston-Salem, North Carolina
- Conditions
- Myotonic Dystrophy 1
- Interventions
- Not listed
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 12 Years to 90 Years
- Enrollment
- 125 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- 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
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases
- Interventions
- Placebo, AOC 1001
- Drug
- Lead sponsor
- Avidity Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 66 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Palo Alto, California • Denver, Colorado + 5 more
- Conditions
- Congenital Myotonic Dystrophy
- Interventions
- Tideglusib
- Drug
- Lead sponsor
- AMO Pharma Limited
- Industry
- Eligibility
- 6 Years to 45 Years
- Enrollment
- 76 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 10
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 7 more
- Conditions
- DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Steinert, Myotonic Muscular Dystrophy
- Interventions
- AOC 1001 (del-desiran), Placebo
- Drug
- Lead sponsor
- Avidity Biosciences, Inc.
- Industry
- Eligibility
- 16 Years to 65 Years
- Enrollment
- 159 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 18
- States / cities
- Stanford, California • Denver, Colorado • Gainesville, Florida + 15 more
- Conditions
- Myotonic Dystrophy Type 1
- Interventions
- IONIS-DMPKRx, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 20 Years to 55 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 8
- States / cities
- Stanford, California • Gainesville, Florida • Kansas City, Kansas + 5 more
- Conditions
- Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Duchenne Type, Muscular Dystrophy, Duchenne and Becker Types, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Genetic Disease, Inborn, Genetic Disease, X-Linked, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neuromuscular Diseases (NMD)
- Interventions
- Zeleciment Rostudirsen (DYNE-251), Placebo
- Drug
- Lead sponsor
- Dyne Therapeutics
- Industry
- Eligibility
- 4 Years to 18 Years · Male only
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 1
- States / cities
- Hillsborough, North Carolina
- Conditions
- Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy
- Interventions
- ERX-963, Placebo
- Drug
- Lead sponsor
- Expansion Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 4
- States / cities
- Palo Alto, California • Miami, Florida • Iowa City, Iowa + 1 more
- Conditions
- Myotonic Dystrophy Type 1
- Interventions
- Not listed
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 6
- States / cities
- Stanford, California • Gainesville, Florida • Kansas City, Kansas + 3 more
- Conditions
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Myotonic Muscular Dystrophy
- Interventions
- AOC 1001, Placebo
- Drug
- Lead sponsor
- Avidity Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Palo Alto, California • Denver, Colorado + 5 more
- Conditions
- Congenital Myotonic Dystrophy, CDM
- Interventions
- Not listed
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- Up to 59 Months
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Fairway, Kansas • Rochester, New York + 1 more
- Conditions
- Myotonic Dystrophy Type 1
- Interventions
- rhIGF-I/rhIGFBP-3, placebo
- Drug
- Lead sponsor
- Insmed Incorporated
- Industry
- Eligibility
- 21 Years to 65 Years
- Enrollment
- 69 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 12
- States / cities
- Orange, California • Sacramento, California • Kansas City, Kansas + 9 more
- Conditions
- Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
- Interventions
- Non-interventional study
- Other
- Lead sponsor
- Wake Forest University Health Sciences
- Other
- Eligibility
- 30 Years to 65 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Winston-Salem, North Carolina
- Conditions
- Muscular Dystrophy, Arrhythmia, Sudden Cardiac Death
- Interventions
- Screening
- Other
- Lead sponsor
- Indiana University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 448 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1996 – 2015
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- Congenital Myotonic Dystrophy
- Interventions
- Natural history
- Other
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 0 Years to 15 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2025
- U.S. locations
- 1
- States / cities
- Richmond, Virginia
- Conditions
- Myotonic Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 215 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2027
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Dallas, Texas
- Conditions
- Myotonic Dystrophy Type 1
- Interventions
- Not listed
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 8 Years to 17 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Stanford, California