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

Description

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).

Conditions

Myotonic Dystrophy 1

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Study Overview

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Study Details

Study overview

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).

A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (FREEDOM-DM1)

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

Condition
Myotonic Dystrophy 1
Intervention / Treatment

-

Contacts and Locations

Irvine

UCI Center for Clinical Research, Irvine, California, United States, 92697

Palo Alto

Stanford University, Palo Alto, California, United States, 94304

Atlanta

Rare Disease Research, LLC, Atlanta, Georgia, United States, 30329

Fairway

University of Kansas Medical Center, Fairway, Kansas, United States, 66205

Boston

Massachusetts General Hospital, Boston, Massachusetts, United States, 02114

Rochester

University of Rochester Medical Center, Rochester, New York, United States, 14642

Richmond

Virginia Commonwealth University, Richmond, Virginia, United States, 23298

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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%

Ages Eligible for Study

18 Years to 50 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

PepGen Inc,

Study Record Dates

2025-04