Status:

RECRUITING

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

Lead Sponsor:

PepGen Inc

Conditions:

Myotonic Dystrophy 1

Eligibility:

All Genders

16-60 years

Phase:

PHASE2

Brief Summary

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 dy...

Eligibility Criteria

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

Note: Other inclusion and exclusion criteria may apply.

Key Trial Info

Start Date :

December 10 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

August 1 2026

Estimated Enrollment :

24 Patients enrolled

Trial Details

Trial ID

NCT06667453

Start Date

December 10 2024

End Date

August 1 2026

Last Update

March 2 2026

Active Locations (8)

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Page 1 of 2 (8 locations)

1

University of Calgary

Calgary, Alberta, Canada, T2N 4Z6

2

Ottawa Hospital Research Institute (OHRI)

Ottawa, Ontario, Canada

3

CIUSSS du Saguenay-Lac-Saint-Jean

Chicoutimi, Quebec, Canada

4

Montreal Neurological Institute

Montreal, Quebec, Canada, H3A 2B4

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