A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
ConditionMyotonic Dystrophy 1
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age16-65
SponsorPepGen Inc
About this trial
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.
Eligibility criteria
Qualifiers
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/m²
Disqualifiers
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
Trial design
Treatments tested in this trial
- PGN-EDODM1
- Placebo
Treatment groups
24 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov