Clinical trialNCT04231266Active, not recruitingPhase 2Interventional

Multi-Center Study of ManNAc for GNE Myopathy

SponsorLeadiant Biosciences, Inc.Enrollment54GNE Myopathy

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

GNE myopathy is a rare genetic muscle disease characterized by progressive muscle atrophy and weakness. The disease is caused by mutations in the gene that encodes the enzyme that initiates and regulates N-acetylneuraminic acid (Neu5Ac) biosynthesis and glycan sialylation. Currently, there is no therapy available for this disease. N-Acetylmannosamine (ManNAc), an orphan drug in development for GNE myopathy, is an uncharged monosaccharide and the first committed precursor in Neu5Ac biosynthesis. In this randomized, double-blind, placebo-controlled trial the efficacy and long-term safety of ManNAc will be evaluated in subjects with GNE myopathy.

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Anthony A. Amato, MD
Brigham and Women's Hospital
Principal Investigator
clinicaltrials.gov ↗
Francis Rossignol, MD
National Institutes of Health (NIH)
Principal Investigator
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

10 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

Ucla
Los Angeles, California, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas, Medical Center
Kansas City, Kansas, United States
Nih Clinical Center
Bethesda, Maryland, United States
Brigham and Women'S Hospital
Boston, Massachusetts, United States
Washington University
St Louis, Missouri, United States
Columbia University Medical Center
New York, New York, United States
University of Rochester
Rochester, New York, United States
Ohio State University, Wexner Medical Center
Columbus, Ohio, United States
University of Utah
Salt Lake City, Utah, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

N-acetylmannosamine

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 5 Apr 2022
Primary completion: 27 Jun 2025
Completion: 30 Oct 2025
Last update posted: 13 Jun 2025
Registered enrollment: 54
Registry id: NCT04231266

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.