Trial · NCT02346461

An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy

NCT02346461, Phase 2, Completed, National Human Genome Research Institute (NHGRI); results first posted 2019-04-16

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy. Trials, record NCT02346461. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02346461

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy
NCT number: NCT02346461
Status: Completed
Phases: Phase 2
Enrollment: 12 participants, actual
Lead sponsor: National Human Genome Research Institute (NHGRI)
Conditions: GNE Myopathy
Interventions: ManNAc, ManNAc
First posted: 2015-01-27
Last updated: 2019-04-16
Results first posted: 2019-04-16

Source ClinicalTrials.gov

Registered outcomes7

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Mean Area Under the Curve (AUClast) of Plasma ManNAc (Baseline-adjusted)	Day 7	The mean area under the plasma ManNAc concentration-versus time curve from time 0 (dosing) to the time of last quantifiable concentration.
Primary	Maximum Observed Plasma Concentration (Cmax) of ManNAc (Baseline-adjusted)	Day 7	The maximum (or peak) plasma ManNAc concentration that the drug achieves in the body after the drug has been administrated.
Primary	The Time to Cmax (Tmax) for ManNAc	Day 7	The time taken to achieve the maximum observed plasma concentration for ManNAc .
Primary	Half-life (t ½) for ManNAc	Day 7	The amount of time it takes for plasma ManNAc concentration to decline by half.
Primary	Mean Area Under the Curve (AUClast) of Plasma Neu5Ac (Baseline-adjusted)	Day 7	The mean area under the plasma Neu5Ac concentration-versus time curve from time 0 (dosing) to the time of last quantifiable concentration.
Primary	Maximum Observed Plasma Concentration (Cmax) of Neu5Ac (Baseline-adjusted)	Day 7	The maximum (or peak) plasma Neu5Ac concentration that the drug achieves in the body after the drug has been administrated.
Primary	The Time to Cmax (Tmax) for Neu5Ac	Day 7	The time taken to achieve the maximum observed plasma concentration for Neu5Ac.

Source ClinicalTrials.gov

Outcomes7

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Mean Area Under the Curve (AUClast) of Plasma ManNAc (Baseline-adjusted)	Day 7	All subjects received ManNAc	hr*ng/mL	MEAN
PRIMARY	Maximum Observed Plasma Concentration (Cmax) of ManNAc (Baseline-adjusted)	Day 7	All subjects received ManNAc	ng/mL	GEOMETRIC_MEAN
PRIMARY	The Time to Cmax (Tmax) for ManNAc	Day 7	All subjects received ManNAc	hours	MEDIAN
PRIMARY	Half-life (t ½) for ManNAc	Day 7	All subjects received ManNAc	hours	MEDIAN
PRIMARY	Mean Area Under the Curve (AUClast) of Plasma Neu5Ac (Baseline-adjusted)	Day 7	All subjects received ManNAc	hr*ng/mL	MEAN
PRIMARY	Maximum Observed Plasma Concentration (Cmax) of Neu5Ac (Baseline-adjusted)	Day 7	All subjects received ManNAc	ng/mL	GEOMETRIC_MEAN
PRIMARY	The Time to Cmax (Tmax) for Neu5Ac	Day 7	All subjects received ManNAc	hours	MEDIAN

Source ClinicalTrials.gov

Results by group14

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Mean Area Under the Curve (AUClast) of Plasma ManNAc (Baseline-adjusted)	ManNAc: Cohort A	7461	Standard Deviation 1776	hr*ng/mL
Mean Area Under the Curve (AUClast) of Plasma ManNAc (Baseline-adjusted)	ManNAc: Cohort B	9432	Standard Deviation 2710	hr*ng/mL
Maximum Observed Plasma Concentration (Cmax) of ManNAc (Baseline-adjusted)	ManNAc: Cohort A	1588	Standard Deviation 28.9	ng/mL
Maximum Observed Plasma Concentration (Cmax) of ManNAc (Baseline-adjusted)	ManNAc: Cohort B	1774	Standard Deviation 21.2	ng/mL
The Time to Cmax (Tmax) for ManNAc	ManNAc: Cohort A	2.0	Standard Deviation 0.4	hours
The Time to Cmax (Tmax) for ManNAc	ManNAc: Cohort B	2.5	Standard Deviation 0.8	hours
Half-life (t ½) for ManNAc	ManNAc: Cohort A	2.0	Standard Deviation 0.3	hours
Half-life (t ½) for ManNAc	ManNAc: Cohort B	2.1	Standard Deviation 1.1	hours
Mean Area Under the Curve (AUClast) of Plasma Neu5Ac (Baseline-adjusted)	ManNAc: Cohort A	4206	Standard Deviation 1352	hr*ng/mL
Mean Area Under the Curve (AUClast) of Plasma Neu5Ac (Baseline-adjusted)	ManNAc: Cohort B	5175	Standard Deviation 1421	hr*ng/mL

1 to 10 of 14Source ClinicalTrials.gov

See all 14 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	ManNAc: Cohort A	6
Overall Study	STARTED	ManNAc: Cohort B	6
Overall Study	COMPLETED	ManNAc: Cohort A	6
Overall Study	COMPLETED	ManNAc: Cohort B	6
Overall Study	NOT COMPLETED	ManNAc: Cohort A	0
Overall Study	NOT COMPLETED	ManNAc: Cohort B	0

Source ClinicalTrials.gov

Adverse events132

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Abdominal distension	Gastrointestinal disorders	ManNAc 3 g - 7 Days	2	6
other	Abdominal distension	Gastrointestinal disorders	ManNAc 6 g - 7 Days	1	6
other	Abdominal distension	Gastrointestinal disorders	ManNAc 6 g - Day 8 to 30 Months	2	12
other	Abdominal pain	Gastrointestinal disorders	ManNAc 3 g - 7 Days	1	6
other	Abdominal pain	Gastrointestinal disorders	ManNAc 6 g - 7 Days	0	6
other	Abdominal pain	Gastrointestinal disorders	ManNAc 6 g - Day 8 to 30 Months	0	12
other	Activated partial thromboplastin time prolonged	Investigations	ManNAc 3 g - 7 Days	0	6
other	Activated partial thromboplastin time prolonged	Investigations	ManNAc 6 g - 7 Days	2	6
other	Activated partial thromboplastin time prolonged	Investigations	ManNAc 6 g - Day 8 to 30 Months	7	12
other	Alanine aminotransferase increased	Investigations	ManNAc 3 g - 7 Days	2	6

1 to 10 of 132Source ClinicalTrials.gov

See all 132 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	ManNAc 3 g - 7 Days	0	6
Total, all-cause mortality	ManNAc 6 g - 7 Days	0	6
Total, all-cause mortality	ManNAc 6 g - Day 8 to 30 Months	0	12
Total, other adverse events	ManNAc 3 g - 7 Days	5	6
Total, other adverse events	ManNAc 6 g - 7 Days	6	6
Total, other adverse events	ManNAc 6 g - Day 8 to 30 Months	12	12
Total, serious adverse events	ManNAc 3 g - 7 Days	0	6
Total, serious adverse events	ManNAc 6 g - 7 Days	0	6
Total, serious adverse events	ManNAc 6 g - Day 8 to 30 Months	0	12

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
33893973	DERIVED	PubMed
34257421	DERIVED	PubMed

Grants0

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.

None in this release.

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT02346461

GET https://conductscience.org/api/trials/v1/NCT02346461/events?page=2

API reference For AI assistants