NCT07220603

Brief Summary

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
48

participants targeted

Target at P25-P50 for phase_2

Timeline
32mo left

Started Dec 2025

Typical duration for phase_2

Geographic Reach
1 country

3 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress12%
Dec 2025Jan 2029

First Submitted

Initial submission to the registry

October 22, 2025

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 24, 2025

Completed
2 months until next milestone

Study Start

First participant enrolled

December 23, 2025

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2029

Last Updated

March 30, 2026

Status Verified

March 1, 2026

Enrollment Period

3 years

First QC Date

October 22, 2025

Last Update Submit

March 26, 2026

Conditions

Myotonic Dystrophy 1

Keywords

DM1Myotonic Dystrophy 1Myotonic DystrophyPepGenPGN-EDODM1Myotonic Muscular DystrophySteinhert's DiseaseMyotonic DystrophiesGenetic Diseases, InbornNeuromuscular DiseasesNervous System DiseasesMusculoskeletal DiseasesMyotonic DisordersMuscular Disorders, AtrophicHeredodegenerative Disorders, Nervous SystemMuscular Diseases

Outcome Measures

Primary Outcomes (1)

  • Safety and tolerability as assessed by number of participants with Adverse Events (AEs)

    Baseline through Week 108

Study Arms (1)

PGN-EDODM1

EXPERIMENTAL

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

Drug: PGN-EDODM1

Interventions

PGN-EDODM1DRUG

Administered by intravenous (IV) infusion

PGN-EDODM1

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Participant has completed a prior study with PGN-EDODM1

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

University of Calgary

Calgary, Alberta, T2N 4Z6, Canada

RECRUITING

Ottawa Hospital Research Institute

Ottawa, Ontario, Canada

RECRUITING

CIUSSS du Saguenay-Lac-Saint-Jean

Saguenay, Canada

RECRUITING

MeSH Terms

Conditions

Myotonic DystrophyGenetic Diseases, InbornNeuromuscular DiseasesNervous System DiseasesMusculoskeletal DiseasesMyotonic DisordersMuscular Disorders, AtrophicHeredodegenerative Disorders, Nervous SystemMuscular Diseases

Condition Hierarchy (Ancestors)

Muscular DystrophiesNeurodegenerative DiseasesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

PepGen Patient Advocacy

CONTACT

781-797-0979clinicaltrials@pepgen.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 22, 2025

First Posted

October 24, 2025

Study Start

December 23, 2025

Primary Completion (Estimated)

January 1, 2029

Study Completion (Estimated)

January 1, 2029

Last Updated

March 30, 2026

Record last verified: 2026-03

Locations

CA(3)