Health Trials

Clinical trials

2

Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

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.

Participants needed: 24
Trial details
Phase: Phase 2Age: 16-65Biological sex: AllType: InterventionalSponsor: PepGen IncUpdated: Apr 28, 2026Locations: 8
Eligibility criteria

Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK g... [+3]

Congenital DM1 [+5]

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Status: Recruiting

An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)

The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.

Participants needed: 48
Trial details
Phase: Phase 2Biological sex: AllType: InterventionalSponsor: PepGen IncUpdated: Mar 30, 2026Locations: 3
Eligibility criteria

Participant has completed a prior study with PGN-EDODM1

Abnormal laboratory tests at screening considered clinically significant by the... [+1]

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