A Clinical Study of PGN-EDODM1 in People With Myotonic Dy... | NCT06667453

Studies
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

NCT06667453

Sponsor

PepGen Inc

Status

Recruiting

Last Update Posted

Apr 28, 2026Actual

Enrollment

24Estimated

Phase

Phase 2

Conditions

Myotonic Dystrophy 1

Interventions

PGN-EDODM1
Placebo

Countries

Canada
New Zealand
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2026

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Dec 10, 2024Actual

Primary Completion Date

Mar 2027Estimated

Completion Date

Mar 2027Estimated

First Submitted Date

Oct 22, 2024

First Submission Date that Met QC Criteria

Oct 29, 2024

First Posted Date

Oct 31, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 23, 2026

Last Update Posted Date

Apr 28, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

24Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
PGN-EDODM1	Drug	Administered by intravenous (IV) infusion	PGN-EDODM1

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Safety and tolerability as assessed by number of participants with Adverse Events (AEs)		Baseline through Day 112

Secondary Outcomes

Measure	Description	Time Frame
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

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

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 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

Note: Other inclusion and exclusion criteria may apply.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

16 Years

Maximum Age

65 Years

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
PepGen	Contact	781-797-0979		clinicaltrials@pepgen.com

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of Calgary	Recruiting	Calgary	Alberta	T2N 4Z6	Canada

Condition Browse Module

MeSH Terms

ID	Term
D009223	Myotonic Dystrophy

Ancestor Terms

ID	Term
D009136	Muscular Dystrophies
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases

Browse Leaves

Not provided

Browse Branches

Not provided