Trial · NCT01347073

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

NCT01347073, Phase 3, Completed, Amgen; results first posted 2015-05-28

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs). Trials, record NCT01347073. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01347073

On this page

Trial

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

Title: Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
NCT number: NCT01347073
Status: Completed
Phases: Phase 3
Enrollment: 23 participants, actual
Lead sponsor: Amgen
Conditions: Urea Cycle Disorders
Interventions: HPN-100
First posted: 2011-05-04
Last updated: 2024-07-11
Results first posted: 2015-05-28
Limitations and caveats: The protocol was designed to capture information important for evaluating safety, pharmacokinetics, and efficacy while recognizing sampling limitations in young children and current standard of care.

Source ClinicalTrials.gov

Registered outcomes5

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

List	Outcome	Time frame	Description
Primary	Adverse Events	2 weeks	Rate of adverse events during the Switch-Over portion of the Protocol
Primary	Adverse Events	12 months	Rate of adverse events during the Safety Extension portion of the protocol ( please note: HPN-100 treatment only during Safety Extension )
Secondary	Blood Ammonia	2 weeks	24-hour ammonia AUC of blood ammonia levels on Days 1 (NaPBA) and 10 (HPN-100) were compared. Ammonia was assessed at Hour 0 (pre-first dose, fasted), Hour 8 (\~2-4 hours after lunch or the second main meal and dose of NaPBA), Hour 12 (\~4 hours after the last main meal) and 24 hours post-first dose (pre-first dose on following day, fasted).
Secondary	Frequency of Ammonia Levels Greater Than the Upper Limit of Normal (ULN) on HPN-100 Compared With NaPBA	2 weeks	Ammonia values were converted to SI units (umol/L) and normalized to a standard ULN of 35 umol/L prior to analysis
Secondary	Hyperammonemic Crisis	1 year	Rate of HAC during pre-enrollment on NaPBA compared to HAC during HPN-100 treatment

Source ClinicalTrials.gov

Outcomes5

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Adverse Events	2 weeks	All patients who received any amount of study medication were included in this population, which is the primary population for all baseline, accountability, demographic and safety analyses.	participants	NUMBER
SECONDARY	Blood Ammonia	2 weeks	All patients who received any amount of both study medications (NaPBA and HPN-100) were included in this population, which is the primary population for analysis of efficacy and pharmacokinetic parameters.	umol/L*hours	MEAN
SECONDARY	Frequency of Ammonia Levels Greater Than the Upper Limit of Normal (ULN) on HPN-100 Compared With NaPBA	2 weeks	All patients who received any amount of both study medications (NaPBA and HPN-100) were included in this population, which is the primary population for analysis of efficacy and pharmacokinetic parameters.	Ammonia Values > ULN	NUMBER
SECONDARY	Hyperammonemic Crisis	1 year		number of crises	NUMBER
PRIMARY	Adverse Events	12 months	All patients that entered the Safety Extension were included in the analysis of adverse events	participants	NUMBER

Source ClinicalTrials.gov

Results by group9

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

Outcome	Group	Value	Spread	Units
Adverse Events	NaPBA	0		participants
Adverse Events	HPN-100	6		participants
Blood Ammonia	NaPBA	914.43	Standard Deviation 630.21	umol/L*hours
Blood Ammonia	HPN-100	647.63	Standard Deviation 379.94	umol/L*hours
Frequency of Ammonia Levels Greater Than the Upper Limit of Normal (ULN) on HPN-100 Compared With NaPBA	NaPBA	22		Ammonia Values > ULN
Frequency of Ammonia Levels Greater Than the Upper Limit of Normal (ULN) on HPN-100 Compared With NaPBA	HPN-100	8		Ammonia Values > ULN
Hyperammonemic Crisis	Pre-enrollment	29		number of crises
Hyperammonemic Crisis	Long-term Phase	12		number of crises
Adverse Events	HPN-100	23		participants

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	23
Overall Study	COMPLETED	HPN-100	21
Overall Study	NOT COMPLETED	HPN-100	2

Source ClinicalTrials.gov

Adverse events31

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	Lymphadenopathy	Blood and lymphatic system disorders	HPN-100	2	23
other	Neutropenia	Blood and lymphatic system disorders	HPN-100	2	23
other	Diarrhea	Gastrointestinal disorders	HPN-100	6	23
other	Gastroesophageal reflux disease	Gastrointestinal disorders	HPN-100	2	23
other	Vomiting	Gastrointestinal disorders	HPN-100	12	23
other	Pyrexia	General disorders	HPN-100	9	23
other	Ear infection	Infections and infestations	HPN-100	2	23
other	Gastroenteritis	Infections and infestations	HPN-100	4	23
other	Otitis media	Infections and infestations	HPN-100	3	23
other	Upper respiratory tract infection	Infections and infestations	HPN-100	14	23

1 to 10 of 31Source ClinicalTrials.gov

See all 31 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	23	23
Total, serious adverse events	HPN-100	11	23

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
23324524	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 comparisons1

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

Comparison
NaPBA vs HPN-100: 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.

GET https://conductscience.org/api/trials/v1/NCT01347073

GET https://conductscience.org/api/trials/v1/NCT01347073/events?page=2

API reference For AI assistants