Ignite Creation Date:
2026-03-26 @ 3:19 PM
Ignite Modification Date:
2026-03-31 @ 1:10 PM
Study NCT ID:
NCT07486934
Status:
NOT_YET_RECRUITING
Last Update Posted:
2026-03-23
First Post:
2026-03-17
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Efficacy, Safety, and Tolerability of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009223', 'term': 'Myotonic Dystrophy'}, {'id': 'D009222', 'term': 'Myotonia'}, {'id': 'D020967', 'term': 'Myotonic Disorders'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020879', 'term': 'Neuromuscular Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 150}}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2026-04', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2026-03', 'completionDateStruct': {'date': '2029-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-03-17', 'studyFirstSubmitDate': '2026-03-17', 'studyFirstSubmitQcDate': '2026-03-17', 'lastUpdatePostDateStruct': {'date': '2026-03-23', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-03-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Fatigue and Daytime Sleepiness Scale (FDSS)', 'timeFrame': 'Up to Week 73'}, {'measure': 'Cognitive function as measured by individual and composite Cogstate cognition scores', 'timeFrame': 'Up to Week 73'}, {'measure': 'Digital functional assessments as measured by the ActiGraph LEAP® wearable device', 'timeFrame': 'Up to Week 73'}], 'primaryOutcomes': [{'measure': '5 Times Sit-To-Stand (5×STS) Time', 'timeFrame': 'Baseline, Week 49'}], 'secondaryOutcomes': [{'measure': 'Video Hand Opening Time (vHOT) [Middle Finger]', 'timeFrame': 'Baseline, Week 49'}, {'measure': 'Quantitative Muscle Testing (QMT) Total', 'timeFrame': 'Baseline, Week 49'}, {'measure': 'Clinician Global Impression of Change (CGI-C)', 'timeFrame': 'Week 49'}, {'measure': '10-Meter Walk/Run Test (10-MWRT) Velocity (m/s)', 'timeFrame': 'Baseline, Week 49'}, {'measure': 'Patient Global Impression of Change (PGI-C)', 'timeFrame': 'Week 49'}, {'measure': 'DM1-ACTIV^C Total Score', 'timeFrame': 'Baseline, Week 49'}, {'measure': 'Myotonic Dystrophy Health Index (MDHI) Total', 'timeFrame': 'Baseline, Week 49'}, {'measure': 'Patient Global Impression of Severity (PGI-S)', 'timeFrame': 'Baseline, Week 49'}, {'measure': 'Clinician Global Impression of Severity (CGI-S)', 'timeFrame': 'Baseline, Week 49'}, {'measure': '9-Hole Peg Test (9-HPT) Time', 'timeFrame': 'Baseline, Week 49'}, {'measure': 'Myotonic Dystrophy Health Index (MDHI) Subscale Scores', 'timeFrame': 'Baseline, Week 49'}, {'measure': 'Maximum Observed Plasma Drug Concentration (Cmax) of DYNE-101', 'timeFrame': 'Pre-dose, and at multiple time points up to Week 73'}, {'measure': 'Time to Maximum Concentration (tmax) of DYNE-101', 'timeFrame': 'Pre-dose, and at multiple time points up to Week 73'}, {'measure': 'Area Under the Concentration-Time Curve (AUC) from Hour 0 to the Last Measurable Concentration (AUC0-tlast) of DYNE-101', 'timeFrame': 'Pre-dose, and at multiple time points up to Week 73'}, {'measure': 'Area Under the Concentration-Time Curve Extrapolated to Infinity (AUC0-∞) of DYNE-101', 'timeFrame': 'Pre-dose, and at multiple time points up to Week 73'}, {'measure': 'Apparent Terminal Elimination Rate Constant (λZ) of DYNE-101', 'timeFrame': 'Pre-dose, and at multiple time points up to Week 73'}, {'measure': 'Apparent Terminal Elimination Half-Life (t½) of DYNE-101', 'timeFrame': 'Pre-dose, and at multiple time points up to Week 73'}, {'measure': 'Plasma clearance (CL) of DYNE-101', 'timeFrame': 'Pre-dose, and at multiple time points up to Week 73'}, {'measure': 'Volume of Distribution at the Terminal Phase (Vz), if Appropriate of DYNE-101', 'timeFrame': 'Pre-dose, and at multiple time points up to Week 73'}, {'measure': 'Volume of Distribution at Steady State (Vss), if Appropriate of DYNE-101', 'timeFrame': 'Pre-dose, and at multiple time points up to Week 73'}, {'measure': 'Number of Participants With Antidrug Antibodies (ADAs)', 'timeFrame': 'Up to Week 73'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'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'], 'conditions': ['Myotonic Dystrophy Type 1 (DM1)', 'DM1', 'Myotonic Dystrophy', 'Steinert Disease', 'Steinert']}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to assess the efficacy, safety, and tolerability of DYNE-101 for the treatment of myotonic dystrophy 1 (DM1).', 'detailedDescription': '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).\n\nAn 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (\\>) 100. Historical results from clinical testing are acceptable.\n* 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).\n* Body mass index (BMI) less than (\\<) 35 kilograms per meter square (kg/m\\^2).\n\nExclusion Criteria:\n\n* A known diagnosis of congenital DM1.\n* 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.\n* 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.\n\nNote: Other inclusion and exclusion criteria may apply."}, 'identificationModule': {'nctId': 'NCT07486934', 'briefTitle': 'Efficacy, Safety, and Tolerability of DYNE-101 in Participants With Myotonic Dystrophy Type 1', 'organization': {'class': 'INDUSTRY', 'fullName': 'Dyne Therapeutics'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'DYNE101-DM1-301'}, 'secondaryIdInfos': [{'id': '2025-522957-20-00', 'type': 'CTIS'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Placebo-Controlled Period: DYNE-101', 'description': 'Participants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks.', 'interventionNames': ['Drug: DYNE-101']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo-Controlled Period: Placebo', 'description': 'Participants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks.', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Long-Term Extension Period: DYNE-101', 'description': 'Participants who receive DYNE-101 in Placebo-Controlled Period will continue to received DYNE-101, Q8W for up to 24 weeks.\n\nParticipants who received placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.', 'interventionNames': ['Drug: DYNE-101', 'Drug: Placebo']}], 'interventions': [{'name': 'DYNE-101', 'type': 'DRUG', 'description': 'Administered by IV infusion', 'armGroupLabels': ['Long-Term Extension Period: DYNE-101', 'Placebo-Controlled Period: DYNE-101']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Administered by IV infusion', 'armGroupLabels': ['Long-Term Extension Period: DYNE-101', 'Placebo-Controlled Period: Placebo']}]}, 'contactsLocationsModule': {'centralContacts': [{'name': 'Dyne Clinical Trials', 'role': 'CONTACT', 'email': 'clinicaltrials@dyne-tx.com', 'phone': '+1-781-317-1919'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Dyne Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}