Trial · NCT00992459
Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders
NCT00992459, Phase 3, Completed, Amgen; results first posted 2013-08-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders. Trials, record NCT00992459. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00992459
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders
- NCT number
- NCT00992459
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 46 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Urea Cycle Disorders
- Interventions
- HPN-100, Buphenyl (NaPBA)
- First posted
- 2009-10-09
- Last updated
- 2024-07-09
- Results first posted
- 2013-08-12
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | The Primary Endpoint Was the 24-hour Area Under the Curve for Blood Ammonia (NH324-hour AUC) on Days 14 and 28. | pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 | Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. Arm A day 14 and Arm B day 28 data were combined as a NaPBA treatment Arm. Arm B day 14 and Arm A day 28 data were combined as a HPN-100 treatment Arm. |
| Secondary | Correlation Between Urinary Phenylacetylglutamine (PAGN) Excretion Over 24 Hours (U-PAGN24-hour Excr) and Venous Ammonia - Area Under the Concentration-time Curve From Time 0 (Predose) to 24 Hours (AUC0-24) | 28 Days | The correlation between 24-hour urinary phenylacetylglutamine (PAGN) excretion (U-PAGN24-hour Excr) and venous ammonia AUC0-24 was summarized and the correlation was tested using the Spearman rank-order correlation. |
| Secondary | Maximum Ammonia Values Observed on NaPBA Versus HPN-100 | pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 | Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. |
| Secondary | Rate (Percentage) of Ammonia Values Above Upper Limit of Normal (ULN) on NaPBA Versus HPN-100 | on Day 14 and Day 28 | NaPBA treated arm: total 345 blood samples were collected. HPN-100 treated arm: 343 blood samples were collected. |
| Secondary | Number and Severity of Symptomatic Hyperammonemic Crises | 29 Days | Severity of symptomatic hyperammonemic crises was measured by peak ammonia level (µmol/L) when it is \>= 100 µmol/L. |
| Secondary | Rate of Adverse Events in Each Treatment Group | 29 Days | |
| Secondary | Cmax for PAA of NaPBA and HPN-100 in Plasma | pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 | Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. |
| Secondary | Cmax for PBA of NaPBA and HPN-100 in Plasma | pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 | Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. |
| Secondary | Cmax PAGN of NaPBA and HPN-100 in Plasma | pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 | Blood samples were collected at pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28. |
| Secondary | U-PAGN24-hour Excr of NaPBA and HPN-100 | 24 hours on Day 14 of each treatments |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | The Primary Endpoint Was the 24-hour Area Under the Curve for Blood Ammonia (NH324-hour AUC) on Days 14 and 28. | pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 | Intent-to-Treat (ITT) (N = 45): Patients receiving any amount of NaPBA or HPN-100 comprise the ITT population. ITT population was to be used for the primary analysis of this endpoint. One subject who withdrew from study after receiving one dose of NaPBA yielding N=44 | μmol∙h/L | MEAN |
| SECONDARY | Correlation Between Urinary Phenylacetylglutamine (PAGN) Excretion Over 24 Hours (U-PAGN24-hour Excr) and Venous Ammonia - Area Under the Concentration-time Curve From Time 0 (Predose) to 24 Hours (AUC0-24) | 28 Days | Intent-to-Treat (ITT) (N = 45): Patients receiving any amount of NaPBA or HPN-100 comprise the ITT population. ITT population was to be used for the primary analysis of this secondary endpoint. One subject who withdrew from study after receiving one dose of NaPBA yielding N=44 | correlation coefficient | NUMBER |
| SECONDARY | Maximum Ammonia Values Observed on NaPBA Versus HPN-100 | pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 | Intent-to-Treat (ITT) (N = 45): Patients receiving any amount of NaPBA or HPN-100 comprise the ITT population. ITT population was to be used for the primary analysis of this endpoint. One subject who withdrew from study after receiving one dose of NaPBA yielding N=44 | µmol/L | MEAN |
| SECONDARY | Rate (Percentage) of Ammonia Values Above Upper Limit of Normal (ULN) on NaPBA Versus HPN-100 | on Day 14 and Day 28 | Intent-to-Treat (ITT) (N = 45): Patients receiving any amount of NaPBA or HPN-100 comprise the ITT population. ITT population was to be used for the primary analysis of this secondary endpoint. One subject who withdrew from study after receiving one dose of NaPBA yielding N=44 | samples | NUMBER |
| SECONDARY | Number and Severity of Symptomatic Hyperammonemic Crises | 29 Days | Safety population: (N=45 for NaPBA; N = 44 for HPN): Patients receiving any amount of NaPBA or HPN-100 comprise the Safety population. Safety population was to be used for safety analysis performed. | events | NUMBER |
| SECONDARY | Rate of Adverse Events in Each Treatment Group | 29 Days | Safety population: (N=45 for NaPBA; N = 44 for HPN): Patients receiving any amount of NaPBA or HPN-100 comprise the Safety population. Safety population was to be used for safety analysis performed. | participants | NUMBER |
| SECONDARY | Cmax for PAA of NaPBA and HPN-100 in Plasma | pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 | Intent-to-Treat (ITT) (N = 45): Patients receiving any amount of NaPBA or HPN-100 comprise the ITT population. ITT population was to be used for the primary analysis of this secondary endpoint. One subject who withdrew from study after receiving one dose of NaPBA yielding N=44 | μg/mL | MEAN |
| SECONDARY | Cmax for PBA of NaPBA and HPN-100 in Plasma | pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 | Intent-to-Treat (ITT) (N = 45): Patients receiving any amount of NaPBA or HPN-100 comprise the ITT population. ITT population was to be used for the primary analysis of this secondary endpoint. One subject who withdrew from study after receiving one dose of NaPBA yielding N=44 | μg/mL | MEAN |
| SECONDARY | Cmax PAGN of NaPBA and HPN-100 in Plasma | pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 | Intent-to-Treat (ITT) (N = 45): Patients receiving any amount of NaPBA or HPN-100 comprise the ITT population. ITT population was to be used for the primary analysis of this secondary endpoint. One subject who withdrew from study after receiving one dose of NaPBA yielding N=44 | μg/mL | MEAN |
| SECONDARY | U-PAGN24-hour Excr of NaPBA and HPN-100 | 24 hours on Day 14 of each treatments | Intent-to-Treat (ITT) (N = 45): Patients receiving any amount of NaPBA or HPN-100 comprise the ITT population. ITT population was to be used for the primary analysis of this secondary endpoint. One subject who withdrew from study after receiving one dose of NaPBA yielding N=44 | μg | 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 |
|---|---|---|---|---|---|
| The Primary Endpoint Was the 24-hour Area Under the Curve for Blood Ammonia (NH324-hour AUC) on Days 14 and 28. | NaPBA | 976.6 | Standard Deviation 865.9 | μmol∙h/L | |
| The Primary Endpoint Was the 24-hour Area Under the Curve for Blood Ammonia (NH324-hour AUC) on Days 14 and 28. | HPN-100 | 865.35 | Standard Deviation 660.53 | μmol∙h/L | |
Correlation Between Urinary Phenylacetylglutamine (PAGN) Excretion Over 24 Hours (U-PAGN24-hour Excr) and Venous Ammonia - Area Under the Concentration-time Curve From Time 0 (Predose) to 24 Hours (AUC0-24) | NaPBA | 0.437 | correlation coefficient | ||
Correlation Between Urinary Phenylacetylglutamine (PAGN) Excretion Over 24 Hours (U-PAGN24-hour Excr) and Venous Ammonia - Area Under the Concentration-time Curve From Time 0 (Predose) to 24 Hours (AUC0-24) | HPN-100 | 0.219 | correlation coefficient | ||
| Maximum Ammonia Values Observed on NaPBA Versus HPN-100 | NaPBA | 70.83 | Standard Deviation 66.705 | µmol/L | |
| Maximum Ammonia Values Observed on NaPBA Versus HPN-100 | HPN-100 | 60.94 | Standard Deviation 46.213 | µmol/L | |
| Rate (Percentage) of Ammonia Values Above Upper Limit of Normal (ULN) on NaPBA Versus HPN-100 | NaPBA | 125 | samples | ||
| Rate (Percentage) of Ammonia Values Above Upper Limit of Normal (ULN) on NaPBA Versus HPN-100 | HPN-100 | 122 | samples | ||
| Number and Severity of Symptomatic Hyperammonemic Crises | NaPBA | 1 | events | ||
| Number and Severity of Symptomatic Hyperammonemic Crises | HPN-100 | 0 | events |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Arm A | 22 | |
| Overall Study | STARTED | Arm B | 24 | |
| Overall Study | COMPLETED | Arm A | 21 | |
| Overall Study | COMPLETED | Arm B | 23 | |
| Overall Study | NOT COMPLETED | Arm A | 1 | |
| Overall Study | NOT COMPLETED | Arm B | 1 |
Source ClinicalTrials.gov
Adverse events36
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 | Abdominal discomfort | Gastrointestinal disorders | NaPBA | 3 | 45 | |
| other | Abdominal discomfort | Gastrointestinal disorders | HPN-100 | 0 | 44 | |
| other | Oral discomfort | General disorders | NaPBA | 2 | 45 | |
| other | Oral discomfort | General disorders | HPN-100 | 0 | 44 | |
| other | abdominal pain | Gastrointestinal disorders | NaPBA | 2 | 45 | |
| other | abdominal pain | Gastrointestinal disorders | HPN-100 | 3 | 44 | |
| other | decreased appetite | Metabolism and nutrition disorders | NaPBA | 2 | 45 | |
| other | decreased appetite | Metabolism and nutrition disorders | HPN-100 | 3 | 44 | |
| other | diarrhea | Gastrointestinal disorders | NaPBA | 3 | 45 | |
| other | diarrhea | Gastrointestinal disorders | HPN-100 | 7 | 44 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | NaPBA | ||
| Total, all-cause mortality | HPN-100 | ||
| Total, other adverse events | NaPBA | 23 | 45 |
| Total, other adverse events | HPN-100 | 27 | 44 |
| Total, serious adverse events | NaPBA | 1 | 45 |
| Total, serious adverse events | HPN-100 | 1 | 44 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| NaPBA vs HPN-100: Rate of Adverse Events in Each Treatment Group | Trial comparisons |
| NaPBA vs HPN-100: serious adverse events | 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.
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/NCT00992459
GET https://conductscience.org/api/trials/v1/NCT00992459/events?page=2