A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Recruiting
Phase 2
Interventional Study
Myotonic Dystrophy 1
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 16 – 65
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 24 (estimated)
- Sponsor
- PepGen Inc · Industry
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About This Trial
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
* Presence of myotonia
* Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed
* Body Mass Index (BM…
Contacts