U.S., April 7 -- ClinicalTrials.gov registry received information related to the study (NCT07511556) titled 'First-in-human Study of UX016 in GNEM' on March 30.
Brief Summary: The main goal of this study is to evaluate the safety of UX016 and to evaluate the impact of UX016 on muscle strength in adults with GNE Myopathy (GNEM).
Study Start Date: Oct., 2026
Study Type: INTERVENTIONAL
Condition:
GNE Myopathy
Intervention:
DRUG: UX016
Tablets for oral use
OTHER: Placebo
Tablets for oral use. Tablets will match the UX016 tablets, but contain no active ingredients
Recruitment Status: NOT_YET_RECRUITING
Sponsor: Ultragenyx Pharmaceutical Inc
Published by HT Digital Content Services with permission from Health Daily Digest....