Trial · NCT00947297

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

NCT00947297, Phase 3, Completed, Amgen; results first posted 2013-08-15

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD). Trials, record NCT00947297. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00947297

On this page

Trial

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

Title: Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)
NCT number: NCT00947297
Status: Completed
Phases: Phase 3
Enrollment: 60 participants, actual
Lead sponsor: Amgen
Conditions: Urea Cycle Disorders
Interventions: HPN-100
First posted: 2009-07-28
Last updated: 2024-07-10
Results first posted: 2013-08-15

Source ClinicalTrials.gov

Registered outcomes4

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

List	Outcome	Time frame	Description
Primary	Rate of Adverse Events (Number of Participants Who Experienced Any AE Considered Related to Study Drug)	1 year
Secondary	Number and Causes of Hyperammonemic Events	1 year	Number of hyperammonemic crises per patient
Secondary	Blood Ammonia Levels	1 Year	Venous Ammonia levels over time
Secondary	Patient Satisfaction With HPN-100	Month 1 post dose	Drug preference will be noted at week 3

Source ClinicalTrials.gov

Outcomes4

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 (Number of Participants Who Experienced Any AE Considered Related to Study Drug)	1 year		participants	NUMBER
SECONDARY	Number and Causes of Hyperammonemic Events	1 year		hyperammonemic events	MEAN
SECONDARY	Blood Ammonia Levels	1 Year		Umol/L	MEAN
SECONDARY	Patient Satisfaction With HPN-100	Month 1 post dose	all available questionnaires	% preferred HPN-100	NUMBER

Source ClinicalTrials.gov

Results by group5

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 (Number of Participants Who Experienced Any AE Considered Related to Study Drug)	HPN-100		33		participants
Number and Causes of Hyperammonemic Events	HPN-100		0.20	Standard Deviation 0.514	hyperammonemic events
Blood Ammonia Levels	HPN-100	Baseline	27.623	Standard Deviation 15.8875	Umol/L
Blood Ammonia Levels	HPN-100	Month 12	24.202	Standard Deviation 20.6124	Umol/L
Patient Satisfaction With HPN-100	HPN-100		90		% preferred HPN-100

Source ClinicalTrials.gov

Participant flow3

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

Period	Milestone	Group	Participants
Overall Study	STARTED	HPN-100	60
Overall Study	COMPLETED	HPN-100	53
Overall Study	NOT COMPLETED	HPN-100	7

Source ClinicalTrials.gov

Adverse events43

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
other	Abdominal discomfort	Gastrointestinal disorders	HPN-100	4	60
other	Abdominal distension	Gastrointestinal disorders	HPN-100	3	60
other	Abdominal pain	General disorders	HPN-100	6	60
other	Constipation	General disorders	HPN-100	4	60
other	Diarrhoea	Gastrointestinal disorders	HPN-100	11	60
other	Dyspepsia	Gastrointestinal disorders	HPN-100	4	60
other	Nausea	Gastrointestinal disorders	HPN-100	13	60
other	Vomiting	Gastrointestinal disorders	HPN-100	19	60
other	Fatigue	General disorders	HPN-100	7	60
other	Pyrexia	General disorders	HPN-100	4	60

1 to 10 of 43Source ClinicalTrials.gov

See all 43 adverse events

Adverse 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	59	60
Total, serious adverse events	HPN-100	12	60

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
22961727	DERIVED	PubMed
24144944	DERIVED	PubMed

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/NCT00947297

GET https://conductscience.org/api/trials/v1/NCT00947297/events?page=2

API reference For AI assistants