Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic
Dystrophy Type 1
Research Question:
Is the drug, PGN EDODM1, safe and tolerable in people with myotonic dystrophy type
1 (also known as Steinert disease)?
Basic Study Information
Purpose:
The purpose of this study is to learn about the effects of an “investigational” drug,
PGN-EDODM1, and to see how safe and tolerable PGN-EDODM1 is for people with myotonic
dystrophy type 1 compared to placebo. The study consists of 2 periods: A Screening
Period (up to 30 days) and a Treatment and
Observation Period (16 weeks). You will come to the site for 6 visits over the course
of the study. Compensation is provided for participating. You are eligible if you
have a diagnosis of myotonic dystrophy type 1 and myotonia (muscles that do not relax
after contraction).
Location: University of Rochester Medical Center
Study Reference #: STUDY00008300
Lead Researcher (Principal Investigator)
Lead Researcher:
Johanna Hamel
Study Contact Information
Study Coordinator: Elizabeth Luebbe
Phone: (585) 275-7867
Email: elizabeth_luebbe@urmc.rochester.edu
Additional Study Details
Number of Visits:
6 to 10
Transportation Coverage:
Yes
Reimbursement:
Yes
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