Overview
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.
Principal investigator
Eligibility criteria
Inclusion Criteria:
* Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
* Medical Research Council (MRC) score of ≥ Grade 4 in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
* Presence of myotonia
* Body Mass Index (BMI) of \< 32.0 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.
* Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
* Medical Research Council (MRC) score of ≥ Grade 4 in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
* Presence of myotonia
* Body Mass Index (BMI) of \< 32.0 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.
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