Multi-Center Study of ManNAc for GNE Myopathy
Public ClinicalTrials.gov record NCT04231266. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy of ManNAc in Subjects With GNE Myopathy
Study identification
- NCT ID
- NCT04231266
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- Leadiant Biosciences, Inc.
- Industry
- Enrollment
- 54 participants
Conditions and interventions
Conditions
Interventions
- ManNAc Drug
- Placebo Other
Drug · Other
Eligibility (public fields only)
- Age range
- 18 Years to 70 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 4, 2022
- Primary completion
- Jun 26, 2025
- Completion
- Oct 29, 2025
- Last update posted
- Jun 12, 2025
2022 – 2025
United States locations
- U.S. sites
- 10
- U.S. states
- 9
- U.S. cities
- 10
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCLA | Los Angeles | California | 90095 | — |
| University of Iowa | Iowa City | Iowa | 52242 | — |
| University of Kansas, Medical Center | Kansas City | Kansas | 66160 | — |
| NIH Clinical Center | Bethesda | Maryland | 20892 | — |
| Brigham and Women's Hospital | Boston | Massachusetts | 02115 | — |
| Washington University | St Louis | Missouri | 63110 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| University of Rochester | Rochester | New York | 14642 | — |
| Ohio State University, Wexner Medical Center | Columbus | Ohio | 43210 | — |
| University of Utah | Salt Lake City | Utah | 84112 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04231266, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 12, 2025 · Synced Jul 21, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04231266 live on ClinicalTrials.gov.