Phase 2
N=12
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
Hypophosphatasia (HPP)
Bottom Line
View on ClinicalTrials.gov: NCT01203826 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Jul 2017
Primary outcome: Primary: Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09. — 2.83 units on a scale — p=0.0005
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Asfotase Alfa (Biological)
- Age
- Pediatric · 5+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Jun 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Skeletal Radiograph Evaluation Using a Qualitative Radiographic Global Impression of Change (RGI-C) Scale Compared to Baseline (Pre-treatment) in Study ENB-006-09. |
2.83 | 0.0005 sig |
Summary
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).
Eligibility Criteria
Inclusion Criteria
- Compliant and satisfactory completion of Enobia-sponsored clinical trial ENB-006-09
- Written informed consent by parent or other legal guardian prior to any study procedures being performed
- Parent or other legal guardian willing to comply with study requirements
Exclusion Criteria
- Clinically significant disease that precludes study participation, in the Investigator's opinion
- Treatment with an investigational drug other than asfotase alfa
- Enrollment in any study (other than ENB-006-09) involving an investigational drug, device, or treatment for HPP
- Prior treatment with bisphosphonates
Data sourced from ClinicalTrials.gov (NCT01203826). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.