Trial · NCT01597440

Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia

NCT01597440, Phase 2, Terminated, Mendel Tuchman; results first posted 2017-04-26

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia. Trials, record NCT01597440. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01597440

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Trial

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

Title: Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia
NCT number: NCT01597440
Status: Terminated
Phases: Phase 2
Enrollment: 1 participants, actual
Lead sponsor: Mendel Tuchman
Conditions: Propionic Acidemia, Methylmalonic Acidemia
Interventions: N-carbamylglutamate, Standard of Care
First posted: 2012-05-14
Last updated: 2017-04-26
Results first posted: 2017-04-26

Source ClinicalTrials.gov

Registered outcomes2

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

List	Outcome	Time frame	Description
Primary	Neurodevelopment	30 months	Neurodevelopmental outcome as measured by Cognitive Composite (Bayley III), Motor Composite (Bayley III) and Functional Status Scale and safety of NCG treatment as measured by adverse events and laboratory blood tests
Secondary	Number of Participants With Adverse Events	Start of episode through 7 days or discharge (if earlier)	Safety is measured by tracking and detailing the number and type of adverse events and their severity based on the CTCAE.

Source ClinicalTrials.gov

Outcomes2

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Neurodevelopment	30 months	The study was closed prematurely. There is no analysis population.
SECONDARY	Number of Participants With Adverse Events	Start of episode through 7 days or discharge (if earlier)		Participants	NUMBER

Source ClinicalTrials.gov

Results by group1

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

Outcome	Group	Category	Value	Spread	Units
Number of Participants With Adverse Events	Carbaglu		1		Participants

Source ClinicalTrials.gov

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Carbaglu	1
Overall Study	STARTED	Placebo	0
Overall Study	COMPLETED	Carbaglu	1
Overall Study	COMPLETED	Placebo	0
Overall Study	NOT COMPLETED	Carbaglu	0
Overall Study	NOT COMPLETED	Placebo	0

Source ClinicalTrials.gov

Adverse events14

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	Events
other	Lethargy	Nervous system disorders	Carbaglu	1	1	1
other	Lethargy	Nervous system disorders	Placebo	0	0	0
other	Hyperventilation	Respiratory, thoracic and mediastinal disorders	Carbaglu	1	1	1
other	Hyperventilation	Respiratory, thoracic and mediastinal disorders	Placebo	0	0	0
other	Vomiting	Gastrointestinal disorders	Carbaglu	1	1	1
other	Vomiting	Gastrointestinal disorders	Placebo	0	0	0
other	Ketosis	Metabolism and nutrition disorders	Carbaglu	1	1	1
other	Ketosis	Metabolism and nutrition disorders	Placebo	0	0	0
other	Acidosis	Blood and lymphatic system disorders	Carbaglu	1	1	1
other	Acidosis	Blood and lymphatic system disorders	Placebo	0	0	0

1 to 10 of 14Source ClinicalTrials.gov

See all 14 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Carbaglu
Total, all-cause mortality	Placebo
Total, other adverse events	Carbaglu	1	1
Total, other adverse events	Placebo	0	0
Total, serious adverse events	Carbaglu	0	1
Total, serious adverse events	Placebo	0	0

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

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/NCT01597440

GET https://conductscience.org/api/trials/v1/NCT01597440/registered

API reference For AI assistants