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NCT00744042 · ClinicalTrials.gov registry record · Phase 1
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
A Phase 1 study, sponsored by Alexion Pharmaceuticals.
- Completed
- Registry status
- Phase 1
- Development phase
- 11
- Enrollment target
NCT00744042: Completed Phase 1 study, sponsored by Alexion Pharmaceuticals.
NCT00744042 is a Phase 1 study that has completed, run by Alexion Pharmaceuticals. The registered enrollment target is 11 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (82% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00744042, a Phase 1 study, has completed, sponsored by Alexion Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 11 participants
- Enrollment target
Study Summary
This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.
Primary Outcome
A 7-point RGI-C (Radiographic Global Impression of Change) score was used to rate change in rickets severity. Scores ranged from -3 (severe worsening of rickets) to +3 (complete healing of rickets). Only those patients with a minimum score of +2 indicating substantial healing of rickets) were considered "responders". Three pediatric radiologists not affiliated with the conduct of the study performed the ratings. Average scores were derived for each patient at each assessment.
Interventions
- BIOLOGICAL asfotase alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 11 participants |
| Start Date | 2008-09 |
| Est. Completion | 2010-05 |
| Phase | Phase 1 |
What the finished NCT00744042 record still lists
NCT00744042 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 11 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (82% lower).
The record links to 0 conditions, and to 1 intervention - of which asfotase alfa is the first listed.
NCT00744042 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00744042 about?
NCT00744042 is a clinical study titled "Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)". This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.
What is the current status of trial NCT00744042?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 11 participants. The study started on 2008-09. Estimated completion is 2010-05.
What interventions are being tested in trial NCT00744042?
The interventions under investigation include: asfotase alfa (BIOLOGICAL).
Who is sponsoring clinical trial NCT00744042?
This trial is sponsored by Alexion Pharmaceuticals, which has 162 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00744042's enrollment target sits among peer trials
11 1911th of 2000 higher than 88 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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