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NCT00947544 · ClinicalTrials.gov registry record · Phase 2
Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
A Phase 2 study, sponsored by Amgen.
- Completed
- Registry status
- Phase 2
- Development phase
- 17
- Enrollment target
NCT00947544: Completed Phase 2 study, sponsored by Amgen.
NCT00947544 is a Phase 2 study that has completed, run by Amgen. The registered enrollment target is 17 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (87% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00947544, a Phase 2 study, has completed, sponsored by Amgen.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 17 participants
- Enrollment target
Study Summary
Protocol HPN-100-005 was the first study of HPN-100 in pediatric subjects with urea cycle disorders (UCDs) and was a fixed-sequence, open-label, switch over study of HPN-100 with a long-term (12 month) safety extension designed to assess the safety of HPN-100 and to prospectively assess its ability to control blood ammonia as compared with Sodium Phenylbutyrate (NaPBA). Upon DSMB review of the first ten subjects who completed the switch over part of the study, and with DSMB approval, up to an additional 20 subjects were enrolled into the safety extension part of the study. HPN-100 is a triglyceride that has a similar mechanism of action as NaPBA. It is a liquid with minimal taste and odor. Three teaspoons of HPN-100 (\~17.4mL) delivers an equivalent amount of PBA to 40 tablets of NaPBA.
Primary Outcome
To evaluate the safety and PK characteristics of HPN-100 compared with sodium phenylbutyrate (NaPBA) in pediatric patients with urea cycle disorders (UCDs)
Interventions
- DRUG HPN-100
- DRUG NaPBA
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 17 participants |
| Start Date | 2010-03 |
| Est. Completion | 2011-08 |
| Phase | Phase 2 |
What the finished NCT00947544 record still lists
NCT00947544 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 17 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (87% lower).
The record links to 0 conditions, and to 2 interventions - of which HPN-100 is the first listed.
NCT00947544 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00947544 about?
NCT00947544 is a clinical study titled "Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders". Protocol HPN-100-005 was the first study of HPN-100 in pediatric subjects with urea cycle disorders (UCDs) and was a fixed-sequence, open-label, switch over study of HPN-100 with a long-term (12 month) safety extension designed to assess the safety of HPN-100 and to prospectively assess its ability ...
What is the current status of trial NCT00947544?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 17 participants. The study started on 2010-03. Estimated completion is 2011-08.
What interventions are being tested in trial NCT00947544?
The interventions under investigation include: HPN-100 (DRUG), NaPBA (DRUG).
Who is sponsoring clinical trial NCT00947544?
This trial is sponsored by Amgen, which has 558 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00947544's enrollment target sits among peer trials
17 1903rd of 2000 higher than 93 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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