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ClinicalTrials.gov public records Last synced May 22, 2026, 12:14 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 40 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Steinert's Disease Clear all
Recruiting No phase listed Observational
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1), DM1
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 88 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced May 22, 2026, 12:14 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
3 Years to 17 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced May 22, 2026, 12:14 AM EDT
Recruiting Phase 2Phase 3 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 27, 2025 · Synced May 22, 2026, 12:14 AM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced May 22, 2026, 12:14 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 24, 2025 · Synced May 22, 2026, 12:14 AM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
VX-670, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 64 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
8
States / cities
San Carlos, California • Gainesville, Florida • Fairway, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced May 22, 2026, 12:14 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced May 22, 2026, 12:14 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 17, 2023 · Synced May 22, 2026, 12:14 AM EDT
Completed Phase 1 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 22, 2021 · Synced May 22, 2026, 12:14 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), Early Repolarization Syndrome (ERS), Arrhythmogenic Cardiomyopathy (AC, ARVD/C), Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Normal Control Subjects
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 85 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2031
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced May 22, 2026, 12:14 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, Muscular Dystrophy, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 2, Congenital Myotonic Dystrophy, PROMM (Proximal Myotonic Myopathy), Steinert's Disease, Myotonic Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2028
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced May 22, 2026, 12:14 AM EDT
Recruiting No phase listed Observational
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 20, 2024 · Synced May 22, 2026, 12:14 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
Mexiletine, Placebo
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 80 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 18, 2018 · Synced May 22, 2026, 12:14 AM EDT
Enrolling by invitation Phase 3 Interventional
View directory record Official record
Conditions
Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert Disease
Interventions
Del-desiran (AOC 1001)
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years and older
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
17
States / cities
Los Angeles, California • Stanford, California • Denver, Colorado + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced May 22, 2026, 12:14 AM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced May 22, 2026, 12:14 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years and older
Enrollment
465 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2028
U.S. locations
5
States / cities
Boston, Massachusetts • Winston-Salem, North Carolina • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 23, 2025 · Synced May 22, 2026, 12:14 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 23, 2025 · Synced May 22, 2026, 12:14 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 10, 2025 · Synced May 22, 2026, 12:14 AM EDT
Completed No phase listed Observational
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 13, 2018 · Synced May 22, 2026, 12:14 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 17, 2024 · Synced May 22, 2026, 12:14 AM EDT
Completed Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
Congenital Myotonic Dystrophy
Interventions
Tideglusib, Placebo
Drug
Lead sponsor
AMO Pharma Limited
Industry
Eligibility
6 Years to 16 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
9
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2025 · Synced May 22, 2026, 12:14 AM EDT
Completed Phase 1 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 4, 2019 · Synced May 22, 2026, 12:14 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 5, 2022 · Synced May 22, 2026, 12:14 AM EDT
Completed Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 7, 2025 · Synced May 22, 2026, 12:14 AM EDT