A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (NCT06667453) | Clinical Trial Compass
RecruitingPhase 2
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Canada, New Zealand24 participantsStarted 2024-12-10
Plain-language 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.
Who can participate
Age range16 Years – 65 Years
SexALL
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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.
What they're measuring
1
Safety and tolerability as assessed by number of participants with Adverse Events (AEs)