Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Recruiting
Phase 1
Phase 2
Interventional Study
Myotonic Dystrophy Type 1 (DM1)
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 – 65
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 116 (estimated)
- Sponsor
- Dyne Therapeutics · Industry
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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 (168 weeks) in both multiple-as…
Trial Locations
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Eligibility Criteria
Inclusion 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…
Contacts