An Open-Label Extension Study of PGN-EDODM1 in People Wit... | NCT07220603

Studies
An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)

NCT07220603

Sponsor

PepGen Inc

Status

Recruiting

Last Update Posted

Mar 30, 2026Actual

Enrollment

48Estimated

Phase

Phase 2

Conditions

Myotonic Dystrophy 1

Interventions

PGN-EDODM1

Countries

Canada

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

Mar 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 23, 2025Actual

Primary Completion Date

Jan 2029Estimated

Completion Date

Jan 2029Estimated

First Submitted Date

Oct 22, 2025

First Submission Date that Met QC Criteria

Oct 22, 2025

First Posted Date

Oct 24, 2025Actual

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

Mar 26, 2026

Last Update Posted Date

Mar 30, 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 long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.

Detailed Description

Not provided

Conditions Module

Conditions

Myotonic Dystrophy 1

Keywords

DM1
Myotonic Dystrophy 1
Myotonic Dystrophy
PepGen
PGN-EDODM1
Myotonic Muscular Dystrophy
Steinhert's Disease
Myotonic Dystrophies
Genetic Diseases, Inborn
Neuromuscular Diseases
Nervous System Diseases
Musculoskeletal Diseases
Myotonic Disorders
Muscular Disorders, Atrophic
Heredodegenerative Disorders, Nervous System
Muscular Diseases

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

48Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
PGN-EDODM1	Experimental	Participants will receive doses of PGN-EDODM1 once every 4 weeks (Q4W)	Drug: PGN-EDODM1

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 Week 108

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Participant has completed a prior study with PGN-EDODM1

Exclusion Criteria:

Abnormal laboratory tests at screening considered clinically significant by the Investigator
Use of an investigational drug (other than PGN-EDODM1), device, or product, within 30 days or 5 half-lives of the study drug (whichever is longer) prior to study entry

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
PepGen Patient Advocacy	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
D030342	Genetic Diseases, Inborn
D009468	Neuromuscular Diseases
D009422	Nervous System Diseases
D009140	Musculoskeletal Diseases
D020967	Myotonic Disorders
D020966	Muscular Disorders, Atrophic
D020271	Heredodegenerative Disorders, Nervous System
D009135	Muscular Diseases

Ancestor Terms

ID	Term
D009136	Muscular Dystrophies
D019636	Neurodegenerative Diseases
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided