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

NCT01347073 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2024-07-11

Study results available
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Summary

This non-randomized, open-label study was approximately one year in duration and consisted of a short term NaPBA to HPN-100 switchover part involving two overnight stays followed by a 12-month long term treatment period involving monthly visits.

Conditions

  • Urea Cycle Disorders

Interventions

DRUG

HPN-100

HPN-100 is a pro-drug of PAA that combines with glutamine to provide an alternative vehicle for waste nitrogen elimination. It is a liquid with minimal taste and odor. Approximately three teaspoons of HPN-100 (\~17.4 mL) delivers an equivalent amount as PBA that 40 tablets of NaPBA.

Sponsors & Collaborators

Principal Investigators

  • F MD · Amgen

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
29 Days
Max Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-07-31
Primary Completion
2013-02-28
Completion
2013-03-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01347073 on ClinicalTrials.gov