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NCT04231266 · ClinicalTrials.gov registry record · Phase 2
Multi-Center Study of ManNAc for GNE Myopathy
A Phase 2 study of GNE Myopathy, sponsored by Leadiant Biosciences.
- Active
- Registry status
- Phase 2
- Development phase
- 54
- Enrollment target
- 10
- Study locations
NCT04231266 is a Phase 2 study of GNE Myopathy that is active but no longer recruiting, run by Leadiant Biosciences. The registered enrollment target is 54 participants. The trial reports 10 study locations across 9 states.
The verdict
NCT04231266, a Phase 2 study of GNE Myopathy, is active but no longer recruiting, sponsored by Leadiant Biosciences.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 54 participants
- Enrollment target
- 10
- Study locations
Study Summary
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.
Conditions Studied
Interventions
- OTHER Placebo
- DRUG ManNAc
Study Locations (10)
New York
- Columbia University Medical Center - New York
- University of Rochester - Rochester
California
- UCLA - Los Angeles
Iowa
- University of Iowa - Iowa City
Kansas
- University of Kansas, Medical Center - Kansas City
Maryland
- NIH Clinical Center - Bethesda
Massachusetts
- Brigham and Women's Hospital - Boston
Missouri
- Washington University - St Louis
Ohio
- Ohio State University, Wexner Medical Center - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 54 participants |
| Start Date | 2022-04-05 |
| Est. Completion | 2025-10-30 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04231266
The ClinicalTrials.gov registry entry for NCT04231266 describes a study currently listed as active not recruiting, categorized as Phase 2. The registered enrollment target is 54 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Leadiant Biosciences, which has 4 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with GNE Myopathy appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04231266 reports 10 study locations spanning 9 distinct geographic areas - top geographies include New York, California, Iowa.
Frequently Asked Questions
What is clinical trial NCT04231266 about?
NCT04231266 is a clinical study titled "Multi-Center Study of ManNAc for GNE Myopathy". 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 the...
What is the current status of trial NCT04231266?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 54 participants. The study started on 2022-04-05. Estimated completion is 2025-10-30.
What conditions does trial NCT04231266 study?
This clinical trial studies the following conditions: GNE Myopathy.
What interventions are being tested in trial NCT04231266?
The interventions under investigation include: Placebo (OTHER), ManNAc (DRUG).
Who is sponsoring clinical trial NCT04231266?
This trial is sponsored by Leadiant Biosciences, which has 4 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04231266 being conducted?
This trial has 10 study locations across California, Iowa, Kansas, Maryland, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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