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

Phase 1

Recruiting

• Conditions: Myotonic Dystrophy 1
• Interventions: Drug: PGN-EDODM1 for infusion; Other: Placebo

• Registration Number: NCT06204809
• Lead Sponsor: PepGen Inc

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-12-05
• Study Start: 2023-12-12
• Study First Submitted QC: 2024-01-03
• Study First Posted: 2024-01-12
• Status Verified: 2024-10
• Last Update Submitted: 2024-12-05
• Last Update Posted: 2024-12-10
• Primary Completion: 2025-04
• Study Completion: 2025-04

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 32

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

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
PGN-EDODM1	PGN-EDODM1 for infusion	PGN-EDODM1 for infusion
Placebo	Placebo	0.9% NaCl

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Area Under the Concentration-time Curve of PGN-EDODM1	Baseline up to Day 3
Apparent Terminal Half-Life (t½) of PGN-EDODM1	Baseline up to Day 3
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

Trial Locations

Locations (9)

UCI Center for Clinical Research; 🇺🇸 Irvine, California, United States

Stanford University; 🇺🇸 Palo Alto, California, United States

University of Kansas Medical Center; 🇺🇸 Fairway, Kansas, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

University of Rochester Medical Center; 🇺🇸 Rochester, New York, United States

Virginia Commonwealth University; 🇺🇸 Richmond, Virginia, United States

Ottawa Hospital Research Institute (OHRI); 🇨🇦 Ottawa, Ontario, Canada

CIUSSS du Saguenay-Lac-Saint-Jean; 🇨🇦 Chicoutimi, Quebec, Canada

Salford Royal Hospital; 🇬🇧 Salford, United Kingdom