Trial · NCT00551200
Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders
NCT00551200, Phase 2, Completed, Amgen; results first posted 2015-05-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders. Trials, record NCT00551200. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00551200
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders
- NCT number
- NCT00551200
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 14 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Urea Cycle Disorders
- Interventions
- HPN-100, BUPHENYL®
- First posted
- 2007-10-30
- Last updated
- 2024-07-10
- Results first posted
- 2015-05-12
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve) | At steady state (1 week) on each medication (Buphenyl® alone, HPN-100 alone), and at steady state (1 week) after each dose escalation | Data were collected at pre-first dose and at 30 minutes and 1, 2, 4, 5, 6, 8, 10, 12, and 24 hours post first dose. |
| Primary | Number of Subjects Experienced Adverse Events | during the period on 100% Buphenyl (up to 4 weeks) or HPN-100 (up to 10 weeks) | |
| Primary | Number of Subjects Experienced Serious Adverse Events | during the period subjects on 100% Buphenyl (up to 4 weeks) or HPN-100 (up to 10 weeks) | |
| Secondary | Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites) | At steady state (1 week) on each medication (Buphenyl® alone, HPN-100 alone) | measured AUC0-24 (Area under the curve from time 0 (pre-dose) to 24 hours) for each metabolite in plasma. Data were collected at 30 minutes and 1, 2, 4, 5, 6, 8, 10, 12, and 24 hours post-first dose. |
| Secondary | Drug Preference for HPN-100 or Buphenyl® (as Assessed by Global Preference Question) | End of Study |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| SECONDARY | Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites) | At steady state (1 week) on each medication (Buphenyl® alone, HPN-100 alone) | μg*h/mL | MEAN | |
| PRIMARY | Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve) | At steady state (1 week) on each medication (Buphenyl® alone, HPN-100 alone), and at steady state (1 week) after each dose escalation | μmol/L | MEAN | |
| SECONDARY | Drug Preference for HPN-100 or Buphenyl® (as Assessed by Global Preference Question) | End of Study | participants | NUMBER | |
| PRIMARY | Number of Subjects Experienced Adverse Events | during the period on 100% Buphenyl (up to 4 weeks) or HPN-100 (up to 10 weeks) | participants | NUMBER | |
| PRIMARY | Number of Subjects Experienced Serious Adverse Events | during the period subjects on 100% Buphenyl (up to 4 weeks) or HPN-100 (up to 10 weeks) | participants | NUMBER |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites) | NaPBA Steady State | AUC0-24 PBA (phenylbutyrate) in plasma | 740 | Standard Deviation 363 | μg*h/mL |
| Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites) | HPN-100 Steady State | AUC0-24 PBA (phenylbutyrate) in plasma | 540 | Standard Deviation 325 | μg*h/mL |
| Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites) | NaPBA Steady State | AUC0-24 PAA (phenylacetate) in plasma | 596 | Standard Deviation 738 | μg*h/mL |
| Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites) | HPN-100 Steady State | AUC0-24 PAA (phenylacetate) in plasma | 575 | Standard Deviation 970 | μg*h/mL |
| Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites) | NaPBA Steady State | AUC0-24 PAGN (phenylacetylglutamine) in plasma | 1133 | Standard Deviation 352 | μg*h/mL |
| Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites) | HPN-100 Steady State | AUC0-24 PAGN (phenylacetylglutamine) in plasma | 1098 | Standard Deviation 485 | μg*h/mL |
| Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve) | NaPBA Steady State | in peak | 79.1 | Standard Deviation 40.1 | μmol/L |
| Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve) | HPN-100 Steady State | in peak | 56.3 | Standard Deviation 27.9 | μmol/L |
| Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve) | NaPBA Steady State | in TNAUC (time-normalized area under the curve) | 38.4 | Standard Deviation 19.6 | μmol/L |
| Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve) | HPN-100 Steady State | in TNAUC (time-normalized area under the curve) | 26.5 | Standard Deviation 10.7 | μmol/L |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Buphenyl to HPN-100 | 14 | |
| Overall Study | COMPLETED | Buphenyl to HPN-100 | 10 | |
| Overall Study | NOT COMPLETED | Buphenyl to HPN-100 | 4 |
Source ClinicalTrials.gov
Adverse events18
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 | Nausea | Gastrointestinal disorders | Buphenyl | 2 | 14 | |
| other | Nausea | Gastrointestinal disorders | HPN-100 | 0 | 10 | |
| other | Dyspepsia | Gastrointestinal disorders | Buphenyl | 1 | 14 | |
| other | Dyspepsia | Gastrointestinal disorders | HPN-100 | 0 | 10 | |
| other | Abdominal Pain | Gastrointestinal disorders | Buphenyl | 2 | 14 | |
| other | Abdominal Pain | Gastrointestinal disorders | HPN-100 | 0 | 10 | |
| other | Gastrooesophageal Reflux Disease | Gastrointestinal disorders | Buphenyl | 1 | 14 | |
| other | Gastrooesophageal Reflux Disease | Gastrointestinal disorders | HPN-100 | 0 | 10 | |
| other | Increased Appetite | Metabolism and nutrition disorders | Buphenyl | 1 | 14 | |
| other | Increased Appetite | Metabolism and nutrition disorders | HPN-100 | 3 | 10 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Buphenyl | ||
| Total, all-cause mortality | HPN-100 | ||
| Total, other adverse events | Buphenyl | 7 | 14 |
| Total, other adverse events | HPN-100 | 5 | 10 |
| Total, serious adverse events | Buphenyl | 1 | 14 |
| Total, serious adverse events | HPN-100 | 0 | 10 |
Source ClinicalTrials.gov
Papers4
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Buphenyl vs HPN-100: Number of Subjects Experienced Adverse Events | Trial comparisons |
| Buphenyl vs HPN-100: Number of Subjects Experienced Serious Adverse Events | Trial comparisons |
| Buphenyl 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/NCT00551200
GET https://conductscience.org/api/trials/v1/NCT00551200/registered