Myotonic Dystrophy Type 1 (DM1)Phase 3NCT07486934
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
A Phase 3 study testing zeleciment basivarsen (DYNE-101) in people with Myotonic Dystrophy Type 1 (DM1) and DM1, run by Dyne Therapeutics at 44 sites in 12 countries.
Trial summary
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1). Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Myotonic Dystrophy Type 1 (DM1), DM1, Myotonic Dystrophy
- What is being tested?
- zeleciment basivarsen (DYNE-101)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 44 sitesin 12 countries
- Study dates
- 14 May 2026to Jan 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (>) 100. Historical results from clinical testing are acceptable.
- Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the ankle are allowed).
- Body mass index (BMI) less than (<) 35 kilograms per meter square (kg/m^2).
Who cannot join
- A known diagnosis of congenital DM1.
- History of major surgical procedure (based on Investigator judgment) within 12 weeks prior to the start of screening, with the exception of implanted pacemaker or defibrillator.
- Use of glucagon-like peptide 1 (GLP-1) agonist/incretin medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments.
- Note: Other inclusion and exclusion criteria may apply.
Trial locations and contacts
44 sites in 12 countries. Contact the study team or a site near you to ask about taking part.
44 sites in 12 countries
United States14 sites, 14 recruiting
- Recruiting
Rare Disease Research, LLC
Atlanta, Georgia, 30329
Julia Zhu, 678-883-6897, julia.zhu@rarediseaseresearch.com
- Recruiting
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Demaya Starkes, 443-923-3832, starkesd@kennedykrieger.org
- Recruiting
Roy Blunt NextGen Precision Health Institute
Columbia, Missouri, 65211
Heather McHatton, 573-882-7619, heathermchatton@health.missouri.edu
- Recruiting
UF Health: University of Florida Clinical Research Center
Gainesville, Florida, 32610
Debbye Landers, 352-273-6181, debralanders@ufl.edu
- Recruiting
Rare Disease Research, LLC
Hillsborough, North Carolina, 27278
Hannah Nation, 984-314-2252, hannah.nation@rarediseaseresearch.com
- Recruiting
Houston Methodist Neurological Institute
Houston, Texas, 77030
Estefania Porras, 346-238-2398, eporras@houstonmethodist.org
- Recruiting
IU Health Neuroscience Center
Indianapolis, Indiana, 46202
Jennifer Terrell, 317-278-6266, jkramey@iu.edu
- Recruiting
UCSD - Altman Clinical and Translational Research Institute
La Jolla, California, 92037
Christian Farfan, cfarfan@health.ucsd.edu
- Recruiting
UCLA Medical Center (Department of Neurology)
Los Angeles, California, 90095
Dennis Fernando, 310-825-3264, defernando@mednet.ucla.edu
- Recruiting
Stanford Neuroscience Health Center
Palo Alto, California, 94304
Anita Chacko, 650-272-8986, neuromuscularresearch@stanford.edu
- Recruiting
Virginia Commonwealth University - Biotech One
Richmond, Virginia, 23298
Levi Headrick, levi.headrick@vcuhealth.org
- Recruiting
University of Rochester Medical Center
Rochester, New York, 14642
Emily Verdaasdonk, 716-274-3385, Emily_verdaasdonk@urmc.rochester.edu
- Recruiting
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, 84132
Michael Papadakis, 8015814474, m.papadakis@utah.edu
- Recruiting
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229
Tonya D Randolph, 210-450-8460, Randolpht@uthscsa.edu
France5 sites, 5 recruiting
- Recruiting
AP-HP, Hôpital Raymond-Poincaré
Garches, 92380
Pascal Laforet, +33 1 42 16 03 85, pascal.laforet@aphp.fr
- Recruiting
CHU Marseille - Hôpital de la Timone (APHM)
Marseille, 13385
Shahram Attarian, Professor, +33 4 91 38 65 79, shahram.attarian@ap-hm.fr
- Recruiting
CHU de Nantes - Hôtel-Dieu
Nantes, 44000
Yann Pereon, +33 2 40 08 36 17, yann.pereon@univ-nantes.fr
- Recruiting
Pitié-Salpêtrière Hospital
Paris, 75013
Nassima Ait Tahar, + 33 1 42 16 37 63, n.ait-tahar@institut-myologie.org
- Recruiting
CHU de Toulouse - Hôpital Pierre-Paul Riquet
Toulouse, 31300
Antoine Faurie-Grépon, +33 5 61 77 91 26, antoine.faurie-grepon@inserm.fr
Japan5 sites, 5 recruiting
- Recruiting
Niigata National Hospital, National Hospital Organization
Kashiwazaki, Niigata, 945-8585
Takashi Nakajima, nakajima-md@mqb.biglobe.ne.jp
- Recruiting
National Center Hospital, National Center of Neurology and Psychiatry (NCNP)
Kodaira, Tokyo, 187-8551
Hirofumi Komaki, komakihiro@ncnp.go.jp
- Recruiting
The University of Osaka Hospital
Suita-shi, Osaka, 565-0871
Masanori Takahashi, mtakahas@sahs.med.osaka-u.ac.jp
- Recruiting
National Hospital Organization Osaka Toneyama Medical Center
Toyonaka-Shi, Osaka, 560-8552
Tsuyoshi Matsumura, MD, PhD, 81668532001, matsumura.tsuyoshi.kq@mail.hosp.go.jp
- Recruiting
Yamaguchi University Hospital
Ube-Shi, Yamaguchi, 755-8505
Masayuki Nakamori, MD, PhD, Professor, mnakamor@yamaguchi-u.ac.jp
Germany4 sites, 4 recruiting
- Recruiting
Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit
Berlin, State of Berlin, 13125
Elisabetta Gazzerro, Dr med, 0049 172 23 53 192, Elisabetta.gazzerro@charite.de
- Recruiting
Universitätsklinikum Bonn AöR Zentrum für Neurologie Klinik für Neuroimmunologie und Neuromuskuläre Erkrankungen
Bonn, North Rhine-Westphalia, 53127
Jens Reimann, MD, +49228 287 31383, NME.Studien@ukbonn.de
- Recruiting
Universitätsmedizin Göttingen
Göttingen, Lower Saxony, 37075
Jana Zschuentzsch, 495513963099, j.zschuentzsch@med.uni-goettingen.de
- Recruiting
LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus Innenstadt
Munich, Bavaria, 80336
Riccardo Morbio, + 49 89 4400 72523, Riccardo.Morbio@med.uni-muenchen.de
Italy4 sites, 4 recruiting
- Recruiting
Centro Clinico Nemo Brescia, Fondazione Serena ETS
Gussago, Brescia, 25064
Massimiliano Filosto, +39 0306729137, studiclinicinemobrescia@centrocliniconemo.it
- Recruiting
Centro Clinico Nemo Milano, Fondazione Serena ETS
Milan, Milano, 20162
Erica Di Natale, +39 02 91433764, erica.dinatale@centrocliniconemo.it
- Recruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS - Department of Women's, Child and Public Health Sciences - UOC Neuropsichiatria Infantile
Roma, Roma, 00168
Lorenzo Stivala, +390630156330, lorenzo.stivala@policlinicogemelli.it
- Recruiting
Azienda Ospedaliero-Universitaria Sant'Andrea - UOC Neurologia
Roma, Roma, 00189
Matteo Garibaldi, +390633775525, matteo.garibaldi@uniroma1.it
Belgium2 sites, 2 recruiting
- Recruiting
UZA
Edegem, 2650
Lauren Meers, +32 3 821 30 00, Lauren.Meers@uza.be
- Recruiting
UZ Leuven
Leuven, 3000
Britt Van Lancker, 32 16 34 88 59, britt.vanlancker@uzleuven.be
Denmark2 sites, 2 recruiting
- Recruiting
Aarhus University Hospital, Department of Neurology
Aarhus, 8200
- Recruiting
Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077)
Copenhagen, 2100
Nicolai Rasmus Preisler, 45 35457257, nicolai.rasmus.preisler@regionh.dk
Netherlands2 sites, 2 recruiting
- Recruiting
University of Maastricht
Maastricht, 6229 HX
Catharina Faber, c.faber@mumc.nl
- Recruiting
Radboud University Medical Center
Nijmegen, 6525 GA
Silvano Gefferie, spierziektenresearchteam@radboudumc.nl
Spain2 sites, 2 recruiting
- Recruiting
Hospital Universitari Vall d'Hebron
Barcelona, Barcelona, 08035
Raul Juntas Morales, raul.juntas@vallhebron.cat
- Recruiting
Hospital Universitario Infanta Sofía
San Sebastián de los Reyes, Madrid, 28702
Gerardo Gutierrez Gutierrez, +34 91 191 40 00, g3.neuro@gmail.com
United Kingdom2 sites, 2 recruiting
- Recruiting
University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery
London, England, WC1N 3BG
Vino Vivekanandam, PhD, v.vivekanandam@ucl.ac.uk
- Recruiting
Northern Care Alliance NHS Foundation Trust
Salford, Lancashire, M6 8HD
Bethan Blackledge, +49 161 789 7373, bethan.blackledge@nca.nhs.uk
Australia1 site, 1 recruiting
- Recruiting
St Vincent's Hospital, Melbourne
Fitzroy, Victoria, 3065
Gayatri Jain, gayatri.jain@svha.org.au
Alan Lai, Alan.LAI@svha.org.au
New Zealand1 site, 1 recruiting
- Recruiting
University of Auckland
Auckland, Auckland, 1023
Neerja Singh, 6499232119, neerja.singh@auckland.ac.nz
About this study
Official title: A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).
Read the detailed description
The study consists of three periods: a Screening period (up to 8 weeks), Placebo-Controlled Period (48 weeks) and a Long-Term Extension Period (24 weeks).
An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety and tolerability data of this study at regular intervals.
- Being tested
- zeleciment basivarsen (DYNE-101), Placebo
- Keywords
- DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Steinert, Myotonic Muscular Dystrophy, HARMONIA, Dyne Therapeutics, Dyne, DYNE-101, zeleciment basivarsen, z-basivarsen
- Medical subject headings
- Myotonic Dystrophy, Myotonia, Myotonic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
zeleciment basivarsen (DYNE-101)Drug
Administered by IV infusion
PlaceboDrug
Administered by IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Placebo-Controlled Period: DYNE-101 | Experimental | Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks. Receives: Drug: zeleciment basivarsen (DYNE-101) |
| Placebo-Controlled Period: Placebo | Placebo comparator | Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks. Receives: Drug: Placebo |
| Long-Term Extension Period: DYNE-101 | Experimental | Participants who receive DYNE-101 in Placebo-Controlled Period will continue to received DYNE-101, Q8W for up to 24 weeks.
Participants who received placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks. Receives: Drug: zeleciment basivarsen (DYNE-101); Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
5 Times Sit-To-Stand (5×STS) Time
Measured: Baseline, Week 49
Secondary outcomes (21)
Video Hand Opening Time (vHOT) [Middle Finger]
Measured: Baseline, Week 49
Quantitative Muscle Testing (QMT) Total
Measured: Baseline, Week 49
Clinician Global Impression of Change (CGI-C)
Measured: Week 49
10-Meter Walk/Run Test (10-MWRT) Velocity (m/s)
Measured: Baseline, Week 49
Patient Global Impression of Change (PGI-C)
Measured: Week 49
DM1-ACTIV^C Total Score
Measured: Baseline, Week 49
Myotonic Dystrophy Health Index (MDHI) Total
Measured: Baseline, Week 49
Patient Global Impression of Severity (PGI-S)
Measured: Baseline, Week 49
Clinician Global Impression of Severity (CGI-S)
Measured: Baseline, Week 49
9-Hole Peg Test (9-HPT) Time
Measured: Baseline, Week 49
Myotonic Dystrophy Health Index (MDHI) Subscale Scores
Measured: Baseline, Week 49
Maximum Observed Plasma Drug Concentration (Cmax) of DYNE-101
Measured: Pre-dose, and at multiple time points up to Week 73
Time to Maximum Concentration (tmax) of DYNE-101
Measured: Pre-dose, and at multiple time points up to Week 73
Area Under the Concentration-Time Curve (AUC) from Hour 0 to the Last Measurable Concentration (AUC0-tlast) of DYNE-101
Measured: Pre-dose, and at multiple time points up to Week 73
Area Under the Concentration-Time Curve Extrapolated to Infinity (AUC0-∞) of DYNE-101
Measured: Pre-dose, and at multiple time points up to Week 73
Apparent Terminal Elimination Rate Constant (λZ) of DYNE-101
Measured: Pre-dose, and at multiple time points up to Week 73
Apparent Terminal Elimination Half-Life (t½) of DYNE-101
Measured: Pre-dose, and at multiple time points up to Week 73
Plasma clearance (CL) of DYNE-101
Measured: Pre-dose, and at multiple time points up to Week 73
Volume of Distribution at the Terminal Phase (Vz), if Appropriate of DYNE-101
Measured: Pre-dose, and at multiple time points up to Week 73
Volume of Distribution at Steady State (Vss), if Appropriate of DYNE-101
Measured: Pre-dose, and at multiple time points up to Week 73
Number of Participants With Antidrug Antibodies (ADAs)
Measured: Up to Week 73
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2026
- Study start14 May 2026
- Primary completionJul 2028 (estimated)
- Study completionJan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic Dystrophy Type 1 (DM1)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dyne Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07486934
- Sponsor's protocol ID
- DYNE101-DM1-301
- Other IDs
- 2025-522957-20-00, 2025
- Status last verified
- Apr 2026
- First submitted
- 17 Mar 2026
- Last updated
- 6 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1
- ClinicalTrials.gov ID
- NCT07486934
- Acronym / sponsor ID
- DYNE101-DM1-301
- Keywords
- DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Dyne TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 14 May 2026 (actual)
- Primary completion
- Jul 2028 (estimated)
- Study completion
- Jan 2029 (estimated)
- First posted
- 23 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).
Detailed description
The study consists of three periods: a Screening period (up to 8 weeks), Placebo-Controlled Period (48 weeks) and a Long-Term Extension Period (24 weeks).
An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety and tolerability data of this study at regular intervals.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo-Controlled Period: DYNE-101 | Experimental | Drug: zeleciment basivarsen (DYNE-101)Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks. |
| Placebo-Controlled Period: Placebo | Placebo comparator | Drug: PlaceboParticipants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks. |
| Long-Term Extension Period: DYNE-101 | Experimental | Drug: zeleciment basivarsen (DYNE-101), Drug: PlaceboParticipants who receive DYNE-101 in Placebo-Controlled Period will continue to received DYNE-101, Q8W for up to 24 weeks. Participants who received placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (>) 100. Historical results from clinical testing are acceptable.
- Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the ankle are allowed).
- Body mass index (BMI) less than (<) 35 kilograms per meter square (kg/m^2).
Key exclusion criteria (4 listed)
- A known diagnosis of congenital DM1.
- History of major surgical procedure (based on Investigator judgment) within 12 weeks prior to the start of screening, with the exception of implanted pacemaker or defibrillator.
- Use of glucagon-like peptide 1 (GLP-1) agonist/incretin medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments.
- Note: Other inclusion and exclusion criteria may apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| zeleciment basivarsen (DYNE-101)Administered by IV infusion | Drug | Long-Term Extension Period: DYNE-101, Placebo-Controlled Period: DYNE-101 |
| PlaceboAdministered by IV infusion | Drug | Long-Term Extension Period: DYNE-101, Placebo-Controlled Period: Placebo |
Primary outcome
5 Times Sit-To-Stand (5×STS) Time
Time frame: Baseline, Week 49
21 secondary outcomes
Video Hand Opening Time (vHOT) [Middle Finger]
Time frame: Baseline, Week 49
Quantitative Muscle Testing (QMT) Total
Time frame: Baseline, Week 49
Clinician Global Impression of Change (CGI-C)
Time frame: Week 49
10-Meter Walk/Run Test (10-MWRT) Velocity (m/s)
Time frame: Baseline, Week 49
Patient Global Impression of Change (PGI-C)
Time frame: Week 49
DM1-ACTIV^C Total Score
Time frame: Baseline, Week 49
Myotonic Dystrophy Health Index (MDHI) Total
Time frame: Baseline, Week 49
Patient Global Impression of Severity (PGI-S)
Time frame: Baseline, Week 49
Clinician Global Impression of Severity (CGI-S)
Time frame: Baseline, Week 49
9-Hole Peg Test (9-HPT) Time
Time frame: Baseline, Week 49
Myotonic Dystrophy Health Index (MDHI) Subscale Scores
Time frame: Baseline, Week 49
Maximum Observed Plasma Drug Concentration (Cmax) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Time to Maximum Concentration (tmax) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Area Under the Concentration-Time Curve (AUC) from Hour 0 to the Last Measurable Concentration (AUC0-tlast) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Area Under the Concentration-Time Curve Extrapolated to Infinity (AUC0-∞) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Apparent Terminal Elimination Rate Constant (λZ) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Apparent Terminal Elimination Half-Life (t½) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Plasma clearance (CL) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Volume of Distribution at the Terminal Phase (Vz), if Appropriate of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Volume of Distribution at Steady State (Vss), if Appropriate of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Number of Participants With Antidrug Antibodies (ADAs)
Time frame: Up to Week 73
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
44 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing zeleciment basivarsen (DYNE-101) in people with Myotonic Dystrophy Type 1 (DM1) and DM1, run by Dyne Therapeutics at 44 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 44 sites in United States, France, Japan, Germany, Italy and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dyne Clinical Trials (+1-781-317-1919, clinicaltrials@dyne-tx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
5 Times Sit-To-Stand (5×STS) Time (time frame: Baseline, Week 49). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2026.