At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- DYNE-101 (drug), Placebo (drug)
- How long the study runs
- Study runs about 82 months (dates as stated)
- About the drug or intervention
- DYNE-101 — drug: Administered by IV infusion · Placebo — drug: Administered by IV infusion
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 18 Years to 65 Years
- Who
- All
- Number of participants
- 127
- Started
- 2022-09-05
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for myotonic dystrophy type 1 (dm1)
- • Phase1/Phase2 - 127 participants
- • The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1)
Who can take part?
- • Ages 18 Years to 65 Years
- • Diagnosed with myotonic dystrophy type 1 (dm1)
Where?
- • London - University College London Hospitals
- • Newcastle upon Tyne - John Walton Muscular Dystrophy Research Centre
- • Salford - Salford Royal Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (208 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years - 65 Years
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Diagnosis of DM1 with trinucleotide repeat size \>100.
- ✓Age of onset of DM1 muscle symptoms ≥12 years.
- ✓Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator.
- ✓Hand grip strength and ankle dorsiflexion strength.
- ✓Able to complete 10-MWRT, stair ascend/descend (MAD cohorts only), and 5×STS at screening without the use of...
Who cannot take part
- ✗History of anaphylaxis.
- ✗Medical condition other than DM1 that would significantly impact ambulation or participation in functional assessments.
- ✗Treatment with medications that can improve myotonia within a period of 5 half-lives of the medication prior to...
- ✗Percent predicted forced vital capacity (FVC) \<50%.
- ✗History of tibialis anterior biopsy within 3 months of Day 1 or planning to undergo tibialis anterior biopsies...
See the full criteria
Where Is This Study? (3 UK sites)
University College London Hospitals
London NW1 2BU, United Kingdom
Rajinder Sidhu - Associate Director, Research Governance and Operations
uclh.jro-communications@nhs.net020 3447 9825John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, United Kingdom
Salford Royal Hospital
Salford M6 8HD, United Kingdom
