N/A
N=6
Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
Hypophosphatasia · Bone Disease
Bottom Line
View on ClinicalTrials.gov: NCT02235493 ↗Enrolled (actual)
6
Serious AEs
—
Results posted
Sep 2015
Primary outcome: Primary: Modified Performance-Oriented Mobility Assessment-Gait Subtest (MPOMA-G) - Change From Baseline to Last Overall — 1.50 units on a scale — p=0.0625
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Retrospective Case Only (Biological)
- Age
- Pediatric, Adult, Older Adult · 5+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Sep 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Modified Performance-Oriented Mobility Assessment-Gait Subtest (MPOMA-G) - Change From Baseline to Last Overall |
1.50 | 0.0625 |
| SECONDARY Performance-Oriented Mobility Assessment-Gait Subtest (POMA-G) - Change From Baseline to Last Overall |
1.50 | 0.1250 |
Summary
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.
Eligibility Criteria
Inclusion Criteria
- Documented informed consent/assent prior to video collection and data abstraction.
- Documented informed consent/assent in Study ALX-HPP-502.
- Patients with juvenile-onset HPP and was selected as a historical-control for Study ENB-006-09.
- Patients with at least 2 videos of basic mobility that were recorded at 2 different clinic visits when the patient was ≥5 to ≤15 ye
Exclusion Criteria
- There are no exclusion criteria for this study
Data sourced from ClinicalTrials.gov (NCT02235493). 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.