Myotonic Dystrophy Type 1(DM1) Myotonic dystrophy, myotonic dystrophy type 1, DM1, muscular dystrophy, muscle disease, muscle weakness, myotonia, muscle stiffness, difficulty relaxing muscles, hand grip problems, facial weakness, difficulty walking, genetic disorder, inherited disease, neuromuscular disease, nervous system disorder, progressive disease, Steinert disease, muscle atrophy, fatigue, daily activity limitations, swallowing difficulty, respiratory muscle weakness, cardiac involvement,
Conditions
Interventions
Administered by IV infusion
Drug: DYNE-101
Drug: Placebo
DYNE-101, zeleciment basivarsen, DM1 treatment, myotonic dystrophy therapy, antisense oligonucleotide, RNA-targeted therapy, investigational drug, intravenous infusion, IV administration, dosing every
Sponsors
Sato Toshiyuki
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size >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). Note: Other inclusion criteria may apply.
Exclusion criteria
Exclusion criteria: -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 exclusion criteria may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 5xSTS time, change from baseline at Week 49 | — |
Secondary
| Measure | Time frame |
|---|---|
| - Video hand opening time (vHOT; middle finger), change from baseline at Week 49 - Quantitative Muscle Testing (QMT) total, change from baseline at Week 49 - Clinician Global Impression of Change (CGI-C) at Week 49 - 10-meter walk/run test (10-MWRT) velocity (m/s), change from baseline at Week 49 - Patient Global Impression of Change (PGI-C) at Week 49 - DM1-ACTIVC total score, change from baseline at Week 49 - Myotonic Dystrophy Health Index (MDHI) total, change from baseline at Week 49 | — |
Countries
Australia, Belgium, Canada, Denmark, France, Germany, Italy, Japan, New Zealand, Spain, The Netherlands, The Republic of Korea, The United Kingdom, The United States
Contacts
Public ContactToshiyuki Sato
PPD-SNBL K.K.
Outcome results
None listed