Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.

Clear filters
ClinicalTrials.gov public records Last synced Sep 23, 2026, 12:52 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 35 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Dystrophia Myotonica 1 Clear all
Completed Phase 2 Interventional Results available

Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

NCT04886518
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 Sep 3, 2026 · Synced Sep 23, 2026, 12:52 AM 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 23, 2026, 12:52 AM 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
5
States / cities
Gainesville, Florida • Tampa, Florida • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 20, 2026 · Synced Sep 23, 2026, 12:52 AM EDT
Enrolling by invitation Phase 3 Interventional

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

NCT07008469
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
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
19
States / cities
Los Angeles, California • Stanford, California • Denver, Colorado + 16 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Sep 23, 2026, 12:52 AM 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 23, 2026, 12:52 AM EDT
Recruiting No phase listed Observational

Myotonic Dystrophy Family Registry

NCT02398786
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 Sep 23, 2026, 12:52 AM 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 23, 2026, 12:52 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy

NCT02269865
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 20, 2014 · Synced Sep 23, 2026, 12:52 AM 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 23, 2026, 12:52 AM 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 23, 2026, 12:52 AM 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 23, 2026, 12:52 AM 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 23, 2026, 12:52 AM EDT
Completed Phase 1 Interventional

A Trial of MLN2704 in Subjects With Metastatic Androgen Independent Prostate Cancer

NCT00052000
View directory record Official record
Conditions
Prostate Cancer
Interventions
MLN2704 (DM1 conjugated monoclonal antibody MLN591)
Drug
Lead sponsor
Millennium Pharmaceuticals, Inc.
Industry
Eligibility
Male only
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
2
States / cities
Baltimore, Maryland • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 28, 2007 · Synced Sep 23, 2026, 12:52 AM 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 23, 2026, 12:52 AM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)

NCT00233519
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
SomatoKine/IPLEX
Drug
Lead sponsor
University of Rochester
Other
Eligibility
21 Years to 60 Years
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2008
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2012 · Synced Sep 23, 2026, 12:52 AM EDT
Completed No phase listed Observational

Multicenter Observational Study of Myotonic Dystrophy Type 1

NCT02308657
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 25, 2017 · Synced Sep 23, 2026, 12:52 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

NCT00082108
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 Sep 23, 2026, 12:52 AM 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 23, 2026, 12:52 AM EDT
Completed Phase 2 Interventional Results available

Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

NCT01406873
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 Sep 23, 2026, 12:52 AM EDT
Recruiting No phase listed Observational

Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy

NCT07630389
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Sep 23, 2026, 12:52 AM EDT
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 23, 2026, 12:52 AM EDT
Completed Phase 2 Interventional

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

NCT05479981
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 Sep 10, 2026 · Synced Sep 23, 2026, 12:52 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy

NCT07362875
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 Sep 23, 2026, 12:52 AM 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 23, 2026, 12:52 AM EDT