Trial · NCT00345605

Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder

NCT00345605, Phase 2, Completed, Brendan Lee; results first posted 2014-11-05

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder. Trials, record NCT00345605. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00345605

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Trial

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

Title: Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
NCT number: NCT00345605
Status: Completed
Phases: Phase 2
Enrollment: 12 participants, actual
Lead sponsor: Brendan Lee
Conditions: Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
Interventions: Sodium Phenylbutyrate, Arginine
First posted: 2006-06-28
Last updated: 2018-01-31
Results first posted: 2014-11-05

Source ClinicalTrials.gov

Registered outcomes7

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

List	Outcome	Time frame	Description
Primary	Measures of Liver Function: AST and ALT	Measured after each 1-week treatment period	Plasma aspartate aminotransferase (AST) and alanine aminotransferase (ALT) levels were measured.
Primary	Measures of Liver Function: PT and PTT	Measured after each 1-week treatment period	Prothrombin time (PT) and partial thromboplastin time (PTT) were measured PT measures factors I (fibrinogen), II (prothrombin), V, VII, and X, while PTT is a performance indicator of the efficacy of the common coagulation pathways.
Primary	Measures of Liver Function: Coagulation Factors	Measured after each 1-week treatment period	Plasma levels of coagulation factors I and IX were used as measures of hepatic synthetic function since the treatment duration was short.
Primary	Measures of Liver Function: INR	Measured after each 1-week treatment period	The result (in seconds) for a prothrombin time performed on a normal individual will vary according to the type of analytical system employed. This is due to the variations between different batches of manufacturer's tissue factor used in the reagent to perform the test. The INR was devised to standardize the results. Each manufacturer assigns an ISI value (International Sensitivity Index) for any tissue factor they manufacture. The ISI value indicates how a particular batch of tissue factor compares to an international reference tissue factor. The ISI is usually between 1.0 and 2.0. The INR is the ratio of a patient's prothrombin time to a normal (control) sample, raised to the power of the ISI value for the analytical system being used.
Secondary	Argininosuccinic Acid Levels	Measured after each 1-week treatment period
Secondary	Arginine Levels	Measured after each 1-week treatment period
Secondary	Urea Production Rate	Measured after each 1-week treatment period

Source ClinicalTrials.gov

Outcomes7

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

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Measures of Liver Function: AST and ALT	Measured after each 1-week treatment period	IU/L	MEAN
SECONDARY	Argininosuccinic Acid Levels	Measured after each 1-week treatment period	micromole/l	MEDIAN
SECONDARY	Arginine Levels	Measured after each 1-week treatment period	micromoles/L	MEDIAN
SECONDARY	Urea Production Rate	Measured after each 1-week treatment period	micromoles/kg/hr	MEAN
PRIMARY	Measures of Liver Function: PT and PTT	Measured after each 1-week treatment period	seconds	MEAN
PRIMARY	Measures of Liver Function: Coagulation Factors	Measured after each 1-week treatment period	mg/dL	MEAN
PRIMARY	Measures of Liver Function: INR	Measured after each 1-week treatment period	seconds	MEAN

Source ClinicalTrials.gov

Results by group20

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

Outcome	Group	Category	Value	Spread	Units
Measures of Liver Function: AST and ALT	Low-dose Arginine Plus Buphenyl	Plasma AST	36.2	Standard Error 10.9	IU/L
Measures of Liver Function: AST and ALT	High Dose Arginine Alone	Plasma AST	52	Standard Error 15.7	IU/L
Measures of Liver Function: AST and ALT	Low-dose Arginine Plus Buphenyl	ALT	31.7	Standard Error 9.55	IU/L
Measures of Liver Function: AST and ALT	High Dose Arginine Alone	ALT	57.86	Standard Error 17.44	IU/L
Argininosuccinic Acid Levels	High-dose Arginine Alone		69	Inter-Quartile Range 44 to 165	micromole/l
Argininosuccinic Acid Levels	Low-dose Arginine Plus Buphenyl		53	Inter-Quartile Range 29 to 118	micromole/l
Arginine Levels	High-dose Arginine Alone		129	Inter-Quartile Range 83 to 197	micromoles/L
Arginine Levels	Low-dose Arginine Plus Buphenyl		53	Inter-Quartile Range 41 to 61	micromoles/L
Urea Production Rate	High-dose Arginine Alone		215	Standard Deviation 56	micromoles/kg/hr
Urea Production Rate	Low-dose Arginine Plus Buphenyl		97	Standard Deviation 35	micromoles/kg/hr

1 to 10 of 20Source ClinicalTrials.gov

See all 20 results by group

Participant flow30

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

Period	Milestone	Group	Participants
Initial Washout	STARTED	High Dose First	6
Initial Washout	STARTED	Low Dose First	6
Initial Washout	COMPLETED	High Dose First	6
Initial Washout	COMPLETED	Low Dose First	6
Initial Washout	NOT COMPLETED	High Dose First	0
Initial Washout	NOT COMPLETED	Low Dose First	0
Arm 1	STARTED	High Dose First	6
Arm 1	STARTED	Low Dose First	6
Arm 1	COMPLETED	High Dose First	6
Arm 1	COMPLETED	Low Dose First	5

1 to 10 of 30Source ClinicalTrials.gov

See all 30 participant flow

Adverse events4

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	Vomiting	Gastrointestinal disorders	High-dose Arginine Alone	1	12	1
other	Vomiting	Gastrointestinal disorders	Low-dose Arginine Plus Buphenyl	1	12	1
other	Hyperammonemia	Metabolism and nutrition disorders	High-dose Arginine Alone	1	12	1
other	Hyperammonemia	Metabolism and nutrition disorders	Low-dose Arginine Plus Buphenyl	1	12	1

Source ClinicalTrials.gov

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	High-dose Arginine Alone
Total, all-cause mortality	Low-dose Arginine Plus Buphenyl
Total, other adverse events	High-dose Arginine Alone	1	12
Total, other adverse events	Low-dose Arginine Plus Buphenyl	1	12
Total, serious adverse events	High-dose Arginine Alone	0	12
Total, serious adverse events	Low-dose Arginine Plus Buphenyl	0	12

Source ClinicalTrials.gov

Papers5

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
8794176	BACKGROUND	PubMed
10869432	BACKGROUND	PubMed
12970464	BACKGROUND	PubMed
14522733	BACKGROUND	PubMed
14606100	BACKGROUND	PubMed

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
U54HD061221	Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (RDCRN) (U54 Clinical Trial Optional)	GROPMAN, ANDREA LYNNE; AH MEW, NICHOLAS; NAGAMANI, SANDESH CHAKRAVARTHY SREENATH	CHILDREN'S RESEARCH INSTITUTE	EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT	2009 to 2023

Source NIH RePORTER

Trial comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
High-dose Arginine Alone vs Low-dose Arginine Plus Buphenyl: serious adverse events

Source Trial comparisons

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.

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API reference For AI assistants