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Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants with Myotonic Dystrophy Type 1

Myotonic Dystrophy 1

The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).

The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).

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Participation Requirements

  • Sex:

    ALL

  • Eligible Ages:

    18 to 50

Participation 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

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
* Medications specific for the treatment of myotonia within 2 weeks prior to screening
* Percent predicted forced vital capacity (FVC) \<40%

Note: Other inclusion and exclusion criteria may apply.

Study Location

CIUSSS du Saguenay-Lac-Saint-Jean
CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Quebec
Canada

Contact Study Team

Primary Contact

Patient Advocacy

[email protected]
Ottawa Hospital Research Institute (OHRI)
Ottawa Hospital Research Institute (OHRI)
Ottawa, Ontario
Canada

Contact Study Team

Primary Contact

Patient Advocacy

[email protected]
Study Sponsored By
PepGen Inc
Participants Required
More Information
Study ID: NCT06204809