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

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)

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06204809

Acronym

FREEDOM-DM1

Enrollment

Registered

2024-01-12

Start date

2023-12-12

Completion date

2025-10-28

Last updated

2026-02-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Myotonic Dystrophy 1

Keywords

Myotonic Dystrophy, Muscular Dystrophies, Genetic Diseases, Inborn, Neuromuscular Diseases, Nervous System Diseases, Musculoskeletal Diseases, Myotonic Disorders, Muscular Disorders, Atrophic, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases, Muscular Diseases, Steinert Disease

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).

Interventions

DRUGPGN-EDODM1 for infusion

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

OTHERPlacebo

Administered by IV infusion

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to 60 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
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

Countries

Canada, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 14, 2026