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

PepGen

Status and phase: Enrolling; Phase 2

Conditions: Myotonic Dystrophy 1

Treatments: Drug: PGN-EDODM1

Study type: Interventional

Funder types: Industry

Identifiers

NCT07220603

PGN-EDODM1-103

Details and patient eligibility

About

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.

Enrollment: 48 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participant has completed a prior study with PGN-EDODM1

Exclusion criteria

• Abnormal laboratory tests at screening considered clinically significant by the Investigator
• Use of an investigational drug (other than PGN-EDODM1), device, or product, within 30 days or 5 half-lives of the study drug (whichever is longer) prior to study entry

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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48 participants in 1 patient group

PGN-EDODM1

Experimental group

Description:

Participants will receive doses of PGN-EDODM1 once every 4 weeks (Q4W)

Treatment:

Drug: PGN-EDODM1