A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
Recruiting
Phase 1
Phase 2
Interventional Study
Duchenne Muscular Dystrophy (DMD)
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 4 – 20
- Sex
- Male
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 24 (estimated)
- Sponsor
- Entrada Therapeutics, Inc. · Industry
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About This Trial
This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-45 is, learn about any side effects, and look at the potential positive effects of ENTR-601-45, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredien…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counselor.
2. Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigato…
Contacts
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