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Tests treatment safety and results for Myotonic Dystrophy Type 1 (DM1)
Official title Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
ClinicalTrials.gov ID: NCT05481879
What this study is testing
What is DYNE-101?
DYNE-101 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for myotonic dystrophy type 1 (dm1).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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 (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- 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 assistive devices such as canes...
You likely can't join if
- History of major surgical procedure within 12 weeks prior to the start of investigative product administration or an expectation of a major surgical...
- 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 performing screening assessments.
- Electrocardiogram (ECG) with the corrected QT interval by Fridericia's Formula (QTcF) ≥450 milliseconds (ms) in men and QTcF ≥460 ms in women, PR...
- Percent predicted forced vital capacity (FVC) \<50%.
See the full eligibility criteria
- 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 assistive devices such as canes, walkers, or orthoses.
- History of major surgical procedure within 12 weeks prior to the start of investigative product administration or an expectation of a major surgical procedure (eg, implantation of cardiac defibrillator) during the study.
- 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 performing screening assessments.
- Electrocardiogram (ECG) with the corrected QT interval by Fridericia's Formula (QTcF) ≥450 milliseconds (ms) in men and QTcF ≥460 ms in women, PR ≥240 ms, left bundle-branch block, or a conduction defect, which is...
- 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 during study period for reasons unrelated to the study.
- Participant has a history of suicide attempt, suicidal behavior, or has any suicidal ideation within 6 months prior to Screening that meets criteria at a level of 4 or 5 of the Columbia Suicide Severity Rating Scale...
- Use of glucagon-like peptide 1 (GLP-1) agonist medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening...
- Significant weight loss during study participation may impact weight-based dosing, performance on muscle function assessments, and pharmacodynamic (PD) biomarkers. Note: Other inclusion and exclusion criteria may apply.
The study team makes the final eligibility decision.
Where it's taking place
- Stanford, California, United States
- Gainesville, Florida, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- St Louis, Missouri, United States
- Rochester, New York, United States
- Denton, Texas, United States
- Richmond, Virginia, United States
- Fitzroy, Victoria, Australia
- Nantes, France
- Paris, France
- Berlin, Germany
- Munich, Germany
- Milan, Italy
- Rome, Italy
- Nijmegen, Netherlands
- Auckland, New Zealand
- London, United Kingdom
- Newcastle upon Tyne, United Kingdom
- Salford, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Stanford, California, United States; Gainesville, Florida, United States; Indianapolis, Indiana, United States; Iowa City, Iowa, United States; St Louis, Missouri, United States; Rochester, New York, United States and 14 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.