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ClinicalTrials.gov public records Last synced Sep 17, 2026, 9:36 PM EDT

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Showing 25–44 of 20 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Steinert Clear all
Recruiting No phase listed Observational

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension

NCT07700225
View directory record Official record
Conditions
DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type-1, Myotonic Dystrophy, Type 1 (DM1), Myotonic Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years to 70 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 17, 2026, 9:36 PM EDT
Recruiting Phase 2Phase 3 Interventional

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

NCT05004129
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 Sep 17, 2026, 9:36 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy

NCT06089018
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 Sep 17, 2026, 9:36 PM EDT
Enrolling by invitation Phase 2 Interventional

A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I

NCT06926621
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
VX-670
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
2
States / cities
St Louis, Missouri • Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 9, 2026 · Synced Sep 17, 2026, 9:36 PM EDT
Completed Phase 1Phase 2 Interventional

Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

NCT05027269
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 Sep 17, 2026, 9:36 PM EDT
Terminated No phase listed Observational

Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)

NCT03867435
View directory record Official record
Conditions
Myotonic Dystrophy Type-1, Myotonic Dystrophy Type-2
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
11 Years to 70 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 3, 2021 · Synced Sep 17, 2026, 9:36 PM EDT
Recruiting No phase listed Observational

Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy

NCT06075693
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
88 participants
Timeline
2022 – 2028
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 23, 2025 · Synced Sep 17, 2026, 9:36 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy

NCT03059264
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 Sep 17, 2026, 9:36 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

NCT03692312
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 Sep 17, 2026, 9:36 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

NCT06844214
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
SAR446268
Biological
Lead sponsor
Sanofi
Industry
Eligibility
10 Years to 55 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
4
States / cities
Gainesville, Florida • Tampa, Florida • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Sep 17, 2026, 9:36 PM EDT
Recruiting No phase listed Observational

Long-Term Development of Muscular Dystrophy Outcome Assessments

NCT05989620
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 17, 2025 · Synced Sep 17, 2026, 9:36 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2

NCT05854433
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Sep 17, 2026, 9:36 PM EDT
Completed Phase 1 Interventional

Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

NCT06204809
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 12, 2026 · Synced Sep 17, 2026, 9:36 PM EDT
Recruiting No phase listed Observational

Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

NCT03981575
View directory record Official record
Conditions
Myotonic Dystrophy 1, DM1
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years to 70 Years
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
10
States / cities
La Jolla, California • Los Angeles, California • Denver, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Sep 17, 2026, 9:36 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

NCT06185764
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
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
8
States / cities
San Carlos, California • Gainesville, Florida • Fairway, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Sep 17, 2026, 9:36 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

NCT00577577
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 Sep 17, 2026, 9:36 PM EDT
Recruiting Phase 1Phase 2 Interventional

Study of ATX-01 in Participants With DM1

NCT06300307
View directory record Official record
Conditions
Myotonic Dystrophy 1
Interventions
ATX-01, Placebo
Drug
Lead sponsor
ARTHEx Biotech S.L.
Industry
Eligibility
18 Years to 64 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 9, 2026 · Synced Sep 17, 2026, 9:36 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

NCT05481879
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
DYNE-101, Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
8
States / cities
Stanford, California • Gainesville, Florida • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Sep 17, 2026, 9:36 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

NCT01931644
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 Sep 17, 2026, 9:36 PM EDT
Completed Phase 1Phase 2 Interventional

A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

NCT02312011
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 5, 2022 · Synced Sep 17, 2026, 9:36 PM EDT