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A PHASE 1 PLACEBO-CONTROLLED STUDY TO ASSESS PGN-EDODM1 IN ADULT PARTICIPANTS WITH MYOTONIC DYSTROP

UCI Specialty Area: Other
Principal Investigator: Namita Goyal
Official Title: A PHASE 1 PLACEBO-CONTROLLED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SINGLE ASCENDING DOSES OF PGN-EDODM1 IN ADULT PARTICIPANTS WITH MYOTONIC DYSTROPHY TYPE 1 (FREEDOM-DM1)
A Study On:
Other

Study Description

Eligibility

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

Congenital DM1

Known history or presence of any clinically significant conditions that may interfere with study safety assessments

Abnormal laboratory tests at screening

Medications specific for the treatment of myotonia within 2 weeks prior to screening

Percent predicted forced vital capacity (FVC) <40%

Note: Other inclusion and exclusion criteria may apply.

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University of California, Irvine - Study Information Sheet

UCI Center for Clinical Research Potential Participant Database

Lead Researcher
Maheswari Senthil, MD
UCI Center for Clinical Research

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