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

Myotonic Dystrophy 1 Clinical Trial

— FREEDOM-DM1  

Official title:

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06204809
• Other study ID #: PGN-EDODM1-101
• Status: Recruiting
• Phase: Phase 1
• Start date: December 12, 2023
• Est. completion date: April 2025

Study information

• Verified date: June 2024
• Source: PepGen Inc
• Contact: PepGen
• Phone: 781-797-0979
• Email: clinicaltrials[@]pepgen.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 24
• Est. completion date: April 2025
• Est. primary completion date: April 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 50 Years

Eligibility

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

• 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 exclusion criteria may apply.

Related Conditions & MeSH terms

• Myotonic Dystrophy
• Myotonic Dystrophy 1

Intervention

Drug:
• PGN-EDODM1
Administered by IV infusion

Other:
• Placebo
Administered by IV infusion

Locations

Country	Name	City	State
Canada	CIUSSS du Saguenay-Lac-Saint-Jean	Chicoutimi	Quebec
Canada	Ottawa Hospital Research Institute (OHRI)	Ottawa	Ontario
United States	Rare Disease Research, LLC	Atlanta	Georgia
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of Rochester Medical Center	Rochester	New York

Sponsors (1)

Lead Sponsor	Collaborator
PepGen Inc

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1		Baseline up to Day 3
Secondary	Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1		Baseline up to Day 3
Secondary	Apparent Terminal Half-Life (t½) of PGN-EDODM1		Baseline up to Day 3
Secondary	Area Under the Concentration-time Curve of PGN-EDODM1		Baseline up to Day 3