Trial · NCT00947544
Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
NCT00947544, Phase 2, Completed, Amgen; results first posted 2013-12-25
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders. Trials, record NCT00947544. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00947544
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
- NCT number
- NCT00947544
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 17 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Urea Cycle Disorders
- Interventions
- HPN-100, NaPBA
- First posted
- 2009-07-28
- Last updated
- 2024-07-11
- Results first posted
- 2013-12-25
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Rate of Adverse Events During the Switchover Part of the Study Rate of Adverse Events (Number of Participants Showing Adverse Events) | 1 week on each treatment for a total of 2 week. | To evaluate the safety and PK characteristics of HPN-100 compared with sodium phenylbutyrate (NaPBA) in pediatric patients with urea cycle disorders (UCDs) |
| Secondary | Number and Causes of Hyperammonemic Events (Safety Extension) | 1 year | Number of Subjects with at Least One Hyperammonemic Crisis. Hyperammonemic crisis is defined as follows: • Clinical symptoms associated with ammonia of ≥ 100 µmol/L |
| Secondary | Blood Ammonia Control | Day 7 (NaPBA) and Day 14 (HPN-100) | To evaluate control of blood ammonia by HPN-100 compared with NaPBA in pediatric patients with UCDs. |
| Secondary | NH3 Cmax on NaPBA vs. HPN-100 on the Last Day of Treatment With Each Drug | Day 7 (NaPBA) and Day 14 (HPN-100) | blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). |
| Secondary | Average Ammonia Values on NaPBA vs. HPN-100 on the Last Day of Treatment With Each Drug (Switch Over) | Day 7 (NaPBA) and Day 14 (HPN-100) | blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). |
| Secondary | Rate (Percentage) of Ammonia Values Above Upper Limit of Normal (ULN) on NaPBA vs. HPN-100 | Day 7 (NaPBA) and Day 14 (HPN-100) | blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). |
| Secondary | Urinary PAGN 24-hour Excretion Values on NaPBA vs. HPN-100 (Switch Over) | Day 7 (NaPBA) and Day 14 (HPN-100) | Urinary PAGN (phenylacetylglutamine) 24-hour excretion. Urine was collect during 0-12 hrs and 12-24 hrs. |
| Secondary | Plasma PAA (Phenylacetate) AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug | Day 7 (NaPBA) and Day 14 (HPN-100) | blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). |
| Secondary | Plasma PBA (Phenylbutyrate) AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug | Day 7 (NaPBA) and Day 14 (HPN-100) | blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). |
| Secondary | Plasma PAGN AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug | Day 7 (NaPBA) and Day 14 (HPN-100) | blood samples were collected at pre-dose, 4, 8, 12, 16, 20, and 24 hour post dose on both Day 7 (NaPBA) and Day 14 (HPN-100). |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Rate of Adverse Events During the Switchover Part of the Study Rate of Adverse Events (Number of Participants Showing Adverse Events) | 1 week on each treatment for a total of 2 week. | participants | NUMBER | |
| SECONDARY | Number and Causes of Hyperammonemic Events (Safety Extension) | 1 year | participants | NUMBER | |
| SECONDARY | Blood Ammonia Control | Day 7 (NaPBA) and Day 14 (HPN-100) | μmol∙h/L | MEAN | |
| SECONDARY | NH3 Cmax on NaPBA vs. HPN-100 on the Last Day of Treatment With Each Drug | Day 7 (NaPBA) and Day 14 (HPN-100) | μmol/L | MEAN | |
| SECONDARY | Average Ammonia Values on NaPBA vs. HPN-100 on the Last Day of Treatment With Each Drug (Switch Over) | Day 7 (NaPBA) and Day 14 (HPN-100) | µmol/L | MEAN | |
| SECONDARY | Rate (Percentage) of Ammonia Values Above Upper Limit of Normal (ULN) on NaPBA vs. HPN-100 | Day 7 (NaPBA) and Day 14 (HPN-100) | percentage of sample | NUMBER | |
| SECONDARY | Urinary PAGN 24-hour Excretion Values on NaPBA vs. HPN-100 (Switch Over) | Day 7 (NaPBA) and Day 14 (HPN-100) | μg | MEAN | |
| SECONDARY | Plasma PAA (Phenylacetate) AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug | Day 7 (NaPBA) and Day 14 (HPN-100) | μg•h/mL AUC 0-24 | MEAN | |
| SECONDARY | Plasma PBA (Phenylbutyrate) AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug | Day 7 (NaPBA) and Day 14 (HPN-100) | µg*h/ml AUC 0-24 | MEAN | |
| SECONDARY | Plasma PAGN AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug | Day 7 (NaPBA) and Day 14 (HPN-100) | μg*h/mL AUC 0-24 | MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group23
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Rate of Adverse Events During the Switchover Part of the Study Rate of Adverse Events (Number of Participants Showing Adverse Events) | HPN-100 | 4 | participants | ||
| Rate of Adverse Events During the Switchover Part of the Study Rate of Adverse Events (Number of Participants Showing Adverse Events) | NaPBA | 2 | participants | ||
| Number and Causes of Hyperammonemic Events (Safety Extension) | Pre-Enrollment (NaPBA) | Number of subjects with at least 1 HAC | 5 | participants | |
| Number and Causes of Hyperammonemic Events (Safety Extension) | Safety Extension (HPN-100) | Number of subjects with at least 1 HAC | 3 | participants | |
| Number and Causes of Hyperammonemic Events (Safety Extension) | Pre-Enrollment (NaPBA) | Number of Crises | 8 | participants | |
| Number and Causes of Hyperammonemic Events (Safety Extension) | Safety Extension (HPN-100) | Number of Crises | 3 | participants | |
| Blood Ammonia Control | HPN-100 | 603.83 | Standard Deviation 187.92 | μmol∙h/L | |
| Blood Ammonia Control | NaPBA | 814.62 | Standard Deviation 322.36 | μmol∙h/L | |
| NH3 Cmax on NaPBA vs. HPN-100 on the Last Day of Treatment With Each Drug | HPN-100 | 47.77 | Standard Deviation 12.8 | μmol/L | |
| NH3 Cmax on NaPBA vs. HPN-100 on the Last Day of Treatment With Each Drug | NaPBA | 55.66 | Standard Deviation 21.61 | μmol/L |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Switch-Over Period (2 Weeks) | STARTED | Swich Over and Safety Extension | 11 | |
| Switch-Over Period (2 Weeks) | STARTED | Safety Extension Only | 0 | |
| Switch-Over Period (2 Weeks) | COMPLETED | Swich Over and Safety Extension | 11 | |
| Switch-Over Period (2 Weeks) | COMPLETED | Safety Extension Only | 0 | |
| Switch-Over Period (2 Weeks) | NOT COMPLETED | Swich Over and Safety Extension | 0 | |
| Switch-Over Period (2 Weeks) | NOT COMPLETED | Safety Extension Only | 0 | |
| Safety-Extension Period (12 Months) | STARTED | Swich Over and Safety Extension | 11 | |
| Safety-Extension Period (12 Months) | STARTED | Safety Extension Only | 6 | |
| Safety-Extension Period (12 Months) | COMPLETED | Swich Over and Safety Extension | 10 | |
| Safety-Extension Period (12 Months) | COMPLETED | Safety Extension Only | 6 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events17
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 pain upper | Gastrointestinal disorders | HPN-100 | 3 | 17 | |
| other | Vomiting | Gastrointestinal disorders | HPN-100 | 4 | 17 | |
| other | Upper respiratory tract infection | Infections and infestations | HPN-100 | 4 | 17 | |
| other | Pyrexia | General disorders | HPN-100 | 2 | 17 | |
| other | Nasopharyngitis | Infections and infestations | HPN-100 | 2 | 17 | |
| other | Sinusitis | Infections and infestations | HPN-100 | 2 | 17 | |
| other | ALT increased | Investigations | HPN-100 | 2 | 17 | |
| other | Anion gap increased | Investigations | HPN-100 | 2 | 17 | |
| other | AST increased | Investigations | HPN-100 | 2 | 17 | |
| other | Decreased appetite | Metabolism and nutrition disorders | HPN-100 | 2 | 17 |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | HPN-100 | ||
| Total, other adverse events | HPN-100 | 4 | 17 |
| Total, serious adverse events | HPN-100 | 3 | 17 |
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| HPN-100 vs NaPBA: Rate of Adverse Events During the Switchover Part of the Study Rate of Adverse Events (Number of Participants Showing 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.
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/NCT00947544
GET https://conductscience.org/api/trials/v1/NCT00947544/results?page=2