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

NCT ID: NCT06667453

Last Updated: 2025-12-22

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

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-10

Study Completion Date

2026-08-31

Brief Summary

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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.

Detailed Description

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Conditions

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Myotonic Dystrophy 1

Keywords

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DM1 Myotonic Dystrophy 1 Myotonic Dystrophy PepGen PGN-EDODM1 Myotonic Muscular Dystrophy Steinert's Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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PGN-EDODM1

Participants will be randomized to receive ascending doses of PGN-EDODM1, once every 4 weeks (Q4W) for 12 weeks

Group Type EXPERIMENTAL

PGN-EDODM1

Intervention Type DRUG

Administered by intravenous (IV) infusion

Placebo

Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Administered by intravenous (IV) infusion

Interventions

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PGN-EDODM1

Administered by intravenous (IV) infusion

Intervention Type DRUG

Placebo

Administered by intravenous (IV) infusion

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
* Medical Research Council (MRC) score of ≥ Grade 4- in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
* Presence of myotonia
* Body Mass Index (BMI) of \< 32.0 kg/m\^2

Exclusion Criteria

* Congenital DM1
* Known history or presence of any clinically significant conditions that may interfere with study safety assessments
* Abnormal laboratory tests at screening considered clinically significant by the Investigator
* Medications specific for the treatment of myotonia within 2 weeks prior to screening
* Percent predicted forced vital capacity (FVC) \<40%
* Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening
Minimum Eligible Age

16 Years

Maximum Eligible Age

60 Years

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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University of Calgary

Calgary, Alberta, Canada

Site Status RECRUITING

Ottawa Hospital Research Institute (OHRI)

Ottawa, Ontario, Canada

Site Status RECRUITING

CIUSSS du Saguenay-Lac-Saint-Jean

Chicoutimi, Quebec, Canada

Site Status RECRUITING

Montreal Neurological Institute

Montreal, Quebec, Canada

Site Status RECRUITING

Salford Royal Hospital

Salford, England, United Kingdom

Site Status RECRUITING

University College London Hospitals NHS Foundation Trust

London, UK, United Kingdom

Site Status RECRUITING

Newcastle Upon Tyne Hospitals

Newcastle upon Tyne, , United Kingdom

Site Status RECRUITING

Countries

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Canada United Kingdom

Central Contacts

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PepGen

Role: CONTACT

Phone: 781-797-0979

Email: [email protected]

Facility Contacts

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Patient Advocacy

Role: primary

Jessica MacGregor

Role: primary

Jessica Tremblay

Role: primary

Patient Advocacy

Role: primary

Patient Advocacy

Role: primary

Patient Advocacy

Role: primary

Patient Advocacy

Role: primary

Other Identifiers

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PGN-EDODM1-102

Identifier Type: -

Identifier Source: org_study_id