Phase 2
N=29
Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
Hypophosphatasia (HPP)
Bottom Line
View on ClinicalTrials.gov: NCT00952484 ↗Enrolled (actual)
29
Serious AEs
0.0%
Results posted
Jul 2011
Primary outcome: Primary: Change in Rickets Severity on Skeletal Radiographs From Baseline to Week 24 as Measured by the Radiographic Global Impression of Change (RGI-C) Scale — 0; 2.00 units on a scale — p=0.0007
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
- Jul 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Rickets Severity on Skeletal Radiographs From Baseline to Week 24 as Measured by the Radiographic Global Impression of Change (RGI-C) Scale |
0; 2.00 | 0.0007 sig |
| SECONDARY Change in Osteomalacia - Osteoid Thickness (as Measured by Trans-iliac Crest Bone Biopsy) |
-3.858 | 0.0097 sig |
| SECONDARY Change in Osteomalacia - Osteoid Volume/Bone Volume (as Measured by Trans-iliac Crest Bone Biopsy) |
-4.225 | 0.0789 |
| SECONDARY Change in Osteomalacia - Mineralization Lag Time (as Measured by Trans-iliac Crest Bone Biopsy) |
20.392 | 0.7417 |
| SECONDARY Change in Height (Z-scores) |
0.01 | 0.7570 |
| SECONDARY Change in Biomarkers of Asfotase Alfa Activity as Measured by Plasma Inorganic Pyrophosphate (PPi) |
-1.883 | <0.0001 sig |
| SECONDARY Change in Biomarkers of Asfotase Alfa Activity as Measured by Pyridoxal-5'-Phosphate (PLP) |
-164.533 | 0.0007 sig |
| SECONDARY Maximum Serum Concentration of Asfotase Alfa (Cmax). |
1780; 2280 | — |
| SECONDARY Time at Maximum Serum Concentration of Asfotase Alfa (Tmax) |
37.2; 34.3 | — |
| SECONDARY Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt) |
76000; 94200 | — |
| SECONDARY Maximum Serum Concentration of Asfotase Alfa (Cmax). |
1780; 2280 | — |
| SECONDARY Time at Maximum Serum Concentration of Asfotase Alfa (Tmax). |
20.8; 17.3 | — |
| SECONDARY Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt) |
76000; 94200 | — |
Summary
This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.
Eligibility Criteria
Inclusion Criteria
- Written informed consent from parent or legal guardian prior to participation
- Patients > 5 and 20 ng/mL
- Ability of patient and parent/guardian to comply with study requirements
Exclusion Criteria
- Serum calcium or phosphorus below age-adjusted normal range
- History of sensitivity to any study drug constituent
- Medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities
- Treatment with an investigational drug within 1 month before start of study drug
- Current enrollment in any other study involving an investigational new drug, device, or treatment for HPP (e.g., bone marrow transplantation)
- Current evidence of a treatable form of rickets
- Prior treatment with bisphosphonates
- Bone fracture or orthopedic surgery within the past 12 months that, in the opinion of the Investigator would interfere with the ability of study patient to comply with study protocol
- Major congenital abnormality other than those associated with HPP
Data sourced from ClinicalTrials.gov (NCT00952484). 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.