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

NCT ID: NCT07220603

Last Updated: 2026-01-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

48 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-12-23

Study Completion Date

2029-01-31

Brief Summary

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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.

Detailed Description

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Conditions

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Myotonic Dystrophy 1

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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PGN-EDODM1

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

Group Type EXPERIMENTAL

PGN-EDODM1

Intervention Type DRUG

Administered by intravenous (IV) infusion

Interventions

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PGN-EDODM1

Administered by intravenous (IV) infusion

Intervention Type DRUG

Eligibility Criteria

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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
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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PepGen Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Ottawa Hospital Research Institute

Ottawa, Ontario, Canada

Site Status RECRUITING

Countries

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Canada

Central Contacts

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PepGen Patient Advocacy

Role: CONTACT

781-797-0979

Facility Contacts

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Study Coordinator

Role: primary

613-798-5555 ext. 19627

Other Identifiers

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PGN-EDODM1-103

Identifier Type: -

Identifier Source: org_study_id

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