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.
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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…
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