NCT06667453

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 dystrophy type 1 (DM1) compared to placebo.

Trial Health

80
On Track

Trial Health Score

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

Enrollment
24

participants targeted

Target at below P25 for phase_2

Timeline
10mo left

Started Dec 2024

Geographic Reach
3 countries

8 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 Progress63%
Dec 2024Mar 2027

First Submitted

Initial submission to the registry

October 22, 2024

Completed
9 days until next milestone

First Posted

Study publicly available on registry

October 31, 2024

Completed
1 month until next milestone

Study Start

First participant enrolled

December 10, 2024

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2027

Last Updated

April 28, 2026

Status Verified

April 1, 2026

Enrollment Period

2.2 years

First QC Date

October 22, 2024

Last Update Submit

April 23, 2026

Conditions

Myotonic Dystrophy 1

Keywords

DM1Myotonic Dystrophy 1Myotonic DystrophyPepGenPGN-EDODM1Myotonic Muscular DystrophySteinert's Disease

Outcome Measures

Primary Outcomes (1)

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

    Baseline through Day 112

Secondary Outcomes (8)

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

    Day 1 through Day 84

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

    Day 1 through Day 84

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

    Day 1 through Day 84

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

    Day 1 through Day 84

  • Change in splicing index in skeletal muscle tissue

    Baseline through Day 91

  • +3 more secondary outcomes

Study Arms (2)

PGN-EDODM1

EXPERIMENTAL

Participants will be randomized to receive ascending doses of PGN-EDODM1, once every 4 weeks (Q4W) for 12 weeks

Drug: PGN-EDODM1

Placebo

PLACEBO COMPARATOR

Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks

Other: Placebo

Interventions

PGN-EDODM1DRUG

Administered by intravenous (IV) infusion

PGN-EDODM1
PlaceboOTHER

Administered by intravenous (IV) infusion

Placebo

Eligibility Criteria

Age16 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

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
  • Presence of myotonia
  • Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed
  • Body Mass Index (BMI) of \< 35.0 kg/m\^2

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 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

University of Calgary

Calgary, Alberta, T2N 4Z6, Canada

RECRUITING

Ottawa Hospital Research Institute (OHRI)

Ottawa, Ontario, Canada

RECRUITING

CIUSSS du Saguenay-Lac-Saint-Jean

Chicoutimi, Quebec, Canada

RECRUITING

Montreal Neurological Institute

Montreal, Quebec, H3A 2B4, Canada

RECRUITING

Pacific Clinical Research Network Auckland

Takapuna, Auckland, 0622, New Zealand

ACTIVE NOT RECRUITING

Salford Royal Hospital

Salford, England, United Kingdom

RECRUITING

University College London Hospitals NHS Foundation Trust

London, UK, United Kingdom

RECRUITING

Newcastle Upon Tyne Hospitals

Newcastle upon Tyne, NE7 7DN, United Kingdom

RECRUITING

MeSH Terms

Conditions

Myotonic Dystrophy

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesMyotonic DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

PepGen

CONTACT

781-797-0979clinicaltrials@pepgen.com

Study Design

Study Type
interventional
Phase
phase 2
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

October 22, 2024

First Posted

October 31, 2024

Study Start

December 10, 2024

Primary Completion (Estimated)

March 1, 2027

Study Completion (Estimated)

March 1, 2027

Last Updated

April 28, 2026

Record last verified: 2026-04

Locations

GB(3)CA(4)NZ(1)