Multi-Center Study of ManNAc for GNE Myopathy

PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

54

Participants

Timeline

Start Date

April 5, 2022

Primary Completion Date

June 27, 2025

Study Completion Date

October 30, 2025

Conditions
GNE Myopathy
Interventions
DRUG

ManNAc

Oral N-acetyl-D-mannosamine monohydrate (ManNAc)

OTHER

Placebo

Placebo

Trial Locations (10)

10032

Columbia University Medical Center, New York

14642

University of Rochester, Rochester

20892

NIH Clinical Center, Bethesda

43210

Ohio State University, Wexner Medical Center, Columbus

52242

University of Iowa, Iowa City

63110

Washington University, St Louis

66160

University of Kansas, Medical Center, Kansas City

84112

University of Utah, Salt Lake City

90095

UCLA, Los Angeles

02115

Brigham and Women's Hospital, Boston

Sponsors
All Listed Sponsors
collaborator

Brigham and Women's Hospital

OTHER

collaborator

National Human Genome Research Institute (NHGRI)

NIH

collaborator

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

collaborator

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

NIH

collaborator

NIH (NIAMS and NIND) as part of NeuroNext

UNKNOWN

lead

Leadiant Biosciences, Inc.

INDUSTRY

NCT04231266 - Multi-Center Study of ManNAc for GNE Myopathy | Biotech Hunter | Biotech Hunter