NCT06204809

Brief Summary

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).

Trial Health

90
On Track

Trial Health Score

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

Enrollment
24

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Dec 2023

Geographic Reach
3 countries

12 active sites

Status
completed

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

First Submitted

Initial submission to the registry

December 5, 2023

Completed
7 days until next milestone

Study Start

First participant enrolled

December 12, 2023

Completed
1 month until next milestone

First Posted

Study publicly available on registry

January 12, 2024

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 28, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 28, 2025

Completed
Last Updated

February 13, 2026

Status Verified

February 1, 2026

Enrollment Period

1.9 years

First QC Date

December 5, 2023

Last Update Submit

February 12, 2026

Conditions

Myotonic Dystrophy 1

Keywords

Myotonic DystrophyMuscular DystrophiesGenetic Diseases, InbornNeuromuscular DiseasesNervous System DiseasesMusculoskeletal DiseasesMyotonic DisordersMuscular Disorders, AtrophicHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesMuscular DiseasesSteinert Disease

Outcome Measures

Primary Outcomes (1)

  • Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs

    Baseline to Week 16

Secondary Outcomes (4)

  • Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1

    Baseline up to Day 3

  • Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1

    Baseline up to Day 3

  • Apparent Terminal Half-Life (t½) of PGN-EDODM1

    Baseline up to Day 3

  • Area Under the Concentration-time Curve of PGN-EDODM1

    Baseline up to Day 3

Study Arms (2)

PGN-EDODM1

EXPERIMENTAL

PGN-EDODM1 for infusion

Drug: PGN-EDODM1 for infusion

Placebo

PLACEBO COMPARATOR

0.9% NaCl

Other: Placebo

Interventions

PGN-EDODM1 for infusionDRUG

Single dose of PGN-EDODM1 by intravenous (IV) infusion

PGN-EDODM1
PlaceboOTHER

Administered by IV infusion

Placebo

Eligibility Criteria

Age18 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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%

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

UCI Center for Clinical Research

Irvine, California, 92697, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

Rare Disease Research

Atlanta, Georgia, 30329, United States

Location

University of Kansas Medical Center

Fairway, Kansas, 66205, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

University of Calgary

Calgary, Alberta, T3M 1M4, Canada

Location

Ottawa Hospital Research Institute (OHRI)

Ottawa, Ontario, Canada

Location

CIUSSS du Saguenay-Lac-Saint-Jean

Chicoutimi, Quebec, Canada

Location

University College London Hospital

London, UK, NW1 2PG, United Kingdom

Location

Salford Royal Hospital

Salford, United Kingdom

Location

MeSH Terms

Conditions

Myotonic DystrophyMuscular DystrophiesGenetic Diseases, InbornNeuromuscular DiseasesNervous System DiseasesMusculoskeletal DiseasesMyotonic DisordersMuscular Disorders, AtrophicHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesMuscular Diseases

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 5, 2023

First Posted

January 12, 2024

Study Start

December 12, 2023

Primary Completion

October 28, 2025

Study Completion

October 28, 2025

Last Updated

February 13, 2026

Record last verified: 2026-02

Locations

US(7)CA(3)GB(2)