N/A
N=32
Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
Hypophosphatasia (HPP)
Bottom Line
View on ClinicalTrials.gov: NCT02104219 ↗Enrolled (actual)
32
Serious AEs
—
Results posted
Dec 2015
Primary outcome: Primary: Radiographic Global Impression of Change - RGI-C — 0.33 units on a scale — p=0.0755
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- —
- Age
- Pediatric, Adult, Older Adult · 5+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Sep 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Radiographic Global Impression of Change - RGI-C |
0.33 | 0.0755 |
| SECONDARY Change in Height Z-score From Baseline to Last Assessment |
-0.05 | 0.6344 |
| SECONDARY Change in Weight Z-score From Baseline to Last Assessment |
0.12 | 0.4520 |
Summary
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.
Eligibility Criteria
Inclusion Criteria
- Documented informed consent unless patient is deceased
- Patients with Juvenile-onset HPP, defined as documented onset of first signs/symptoms at ≥ 6 months to ˂18 years
- Documented diagnosis of HPP as indicated by skeletal manifestations and low alkaline phosphatase or genotyping
Exclusion Criteria
- Received treatment with asfotase alfa in the ENB-006-09 study and/or currently enrolled in the ENB-008-10 study
- Received other treatment and/or intervention to treat HPP up to 15 years old
- Other clinically significant disease
Data sourced from ClinicalTrials.gov (NCT02104219). 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.