Trial · NCT04231266
Multi-Center Study of ManNAc for GNE Myopathy
NCT04231266, Phase 2, Active not recruiting, Leadiant Biosciences, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Multi-Center Study of ManNAc for GNE Myopathy. Trials, record NCT04231266. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04231266
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Multi-Center Study of ManNAc for GNE Myopathy
- NCT number
- NCT04231266
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 54 participants, actual
- Lead sponsor
- Leadiant Biosciences, Inc.
- Conditions
- GNE Myopathy
- Interventions
- ManNAc, Placebo
- First posted
- 2020-01-18
- Last updated
- 2025-06-13
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Muscle strength of ankle dorsiflexion, knee flexion, knee extension, shoulder abduction, elbow flexion and grip measured by fixed-frame Quantitative Muscle Assessment (QMA) | Minimum 2 years, until 24 months from randomization of last subject | The primary endpoint is the change in muscle strength decline under treatment compared to placebo. The primary analysis is based on the disease progression ratio (γ) comparing the rate of progression from baseline until last visit, under placebo to that under treatment. |
| Secondary | Inclusion Body Myositis Functional Rating Scale (IBMFRS) | Minimum 2 years, until 24 months from randomization of last subject | Change in patient-reported function as measured by the Inclusion Body Myositis Functional Rating Scale (IBMFRS). |
| Other | Adverse Events | Minimum 2 years, until 24 months from randomization of last subject | Safety and tolerability will be evaluated by comparing the frequency of adverse events (AEs) across groups, collected using information from in-person assessments, clinical laboratory tests, vital signs, electronic diary reports, and physical examinations. |
| Other | Correlation muscle strength measured Exploratory GNEM Functional Questions (ExGNEM) | Minimum 2 years, until 24 months from randomization of last subject | Evaluate the effect of ManNAc on patient-reported physical functioning assessed by ExGNEM, a six-question rating scale that assesses lower body function. |
| Other | Adult Myopathy Assessment Tool | Minimum 2 years, until 24 months from randomization of last subject | Evaluate the effect of ManNAc on physical function as measured by the Adult Myopathy Assessment Tool (AMAT), a 13-item standardized test that assesses physical performance, to be performed at baseline and every 6 months thereafter until completion of the study. |
| Other | Six-Minute Walk Test | Minimum 2 years, until 24 months from randomization of last subject | Evaluate the effect of ManNAc on physical function as measured by the Six-Minute Walk Test (whenever possible) to be performed at baseline and every 6 months thereafter until completion of the study. |
| Other | Timed Up and Go Test | Minimum 2 years, until 24 months from randomization of last subject | Evaluate the effect of ManNAc on physical function as measured by the Timed Up and Go, performance test to evaluate functional mobility, to be performed at baseline and every 6 months thereafter until completion of the study. |
| Other | Functional Reach Test | Minimum 2 years, until 24 months from randomization of last subject | Evaluate the effect of ManNAc on physical function as measured by the Functional Reach, a measure of stability, to be performed at baseline and every 6 months thereafter until completion of the study. |
| Other | Jebsen Hand Function Test | Baseline and every 12 months thereafter | Evaluate the effect of ManNAc on physical function as measured by the Jebsen Hand Function Test, a performance measure which assesses unilateral hand function, to be performed at baseline and every 6 months thereafter until completion of the study. |
| Other | Inclusion Body Myositis Functional Rating Scale | Minimum 2 years, until 24 months from randomization of last subject | Evaluate effect of ManNAc on activities of daily living (ADLs) compared to placebo as measured by the Inclusion Body Myositis Functional Rating Scale (IBMFRS), a patient-reported outcome completed at baseline, and every 6 months thereafter until completion of the study. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| U01AR070498 | A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy of ManNAc in Subjects with GNE Myopathy | AMATO, ANTHONY ARNOLD | BRIGHAM AND WOMEN'S HOSPITAL | NATIONAL INSTITUTE OF ARTHRITIS AND MUSCULOSKELETAL AND SKIN DISEASES | 2017 to 2023 |
Source NIH RePORTER
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04231266
GET https://conductscience.org/api/trials/v1/NCT04231266/registered