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A Dose-Range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients with Myotonic Dystrophy Type 1

Principal Investigator:
Doris
Leung

A Phase 1/2a Blinded, Placebo-Controlled Study to Assess the Safety, Tolerability, and Dose-Range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients with Myotonic Dystrophy Type 1

This is a phase 1/2a blinded, placebo-controlled, randomized, multiple ascending dose study to examine the safety and tolerability of multiple doses of ISIS 598769 administered subcutaneously to adult patients with DM1. Approximately 36 subjects (4 dose cohorts; 8 subjects per cohort in first 3 cohorts, 12 subjects in the highest dose cohort) are planned to be enrolled into this multiple-dose study of ISIS 598769. The number of subjects may be higher if subjects must be replaced and/or if the size or number of the cohorts is expanded in order to obtain further experience with some dose levels. The maximum number of enrolled subjects could range between 36 and 48 subjects.

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