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Recruiting NCT07220603

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

Phase II Interventional Myotonic Dystrophy 1

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: PGN-EDODM1.
Who it may be relevant to
Registry conditions: Myotonic Dystrophy 1. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Canada
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

An Open-Label Extension Study Evaluating Safety and Pharmacokinetics in Participants With Myotonic Dystrophy Type 1 (FREEDOM-OLE)

Overview

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.

Interventions

  • Drug PGN-EDODM1
    Administered by intravenous (IV) infusion

Primary outcome measures

  • Safety and tolerability as assessed by number of participants with Adverse Events (AEs) [Time frame: Baseline through Week 108]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Canada · 3 centers
  • University of Calgary — Calgary
  • Ottawa Hospital Research Institute — Ottawa
  • CIUSSS du Saguenay-Lac-Saint-Jean — Saguenay

Identifiers

NCT: NCT07220603 · PGN-EDODM1-103

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗