Phase 2
Completed N=10
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
Hypophosphatasia
Source: ClinicalTrials.gov NCT01205152 ↗
Enrolled (actual)
10
Serious AEs
90.0%
Results posted
Nov 2017
Primary outcomePrimary: Long-term Tolerability of Subcutaneous (SC) Asfotase Alfa — 10 Participants
Summary
This clinical trial studied the long term safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP who completed study ENB-002-08 (NCT00744042).
Partial funding for this study was provided by the Office of Orphan Product Development (OOPD).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Long-term Tolerability of Subcutaneous (SC) Asfotase Alfa |
10 | — |
| PRIMARY Long-term Efficacy of Asfotase Alfa in Treating Rickets in Infants and Young Children With Hypophosphatasia (HPP). |
2.00 | 0.0020 sig |
| SECONDARY Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Plasma Inorganic Pyrophosphate (PPi) Levels |
-2.460 | — |
| SECONDARY Long-term Pharmacodynamics (PD) of SC Asfotase Alfa: Pyridoxal-5-phosphate (PLP) Levels |
-266.200 | — |
| SECONDARY Effect of SC Asfotase Alfa on Growth: Weight Z-scores |
2.43 | — |
| SECONDARY Effect of SC Asfotase Alfa on Growth: Height/Length Z-scores |
1.93 | — |
| SECONDARY Effect of SC Asfotase Alfa on Respiratory Function |
6; 9; 0; 0; 1; 0 | — |
Eligibility Criteria
Inclusion Criteria
- Patient completed participation in ENB-002-08 (NCT00744042)
- 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
- History of sensitivity to any of the constituents of the study drug
- Clinically significant disease that precludes study participation
- Enrollment in any study (other than ENB-002-08) involving an investigational drug, device, or treatment for HPP (e.g., bone marrow transplantation)
Data sourced from ClinicalTrials.gov (NCT01205152). 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. Informational only — not medical advice.