An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
ConditionMyotonic Dystrophy 1
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorPepGen Inc
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Participant has completed a prior study with PGN-EDODM1
Disqualifiers
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
Trial design
Design model
Single group
Treatments tested in this trial
PGN-EDODM1
DrugAdministered by intravenous (IV) infusion
Treatment groups
48 Participants
are divided into 1 treatment groupGroup A: PGN-EDODM1Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Safety and tolerability as assessed by number of participants with Adverse Events (AEs)
Time frame
Baseline through Week 108 Source ClinicalTrials.gov