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Asfotase alfa in adolescents and adults with HPP
NCT01163149Phase 2
Key facts
- Status
- Completed
- Start
- Jun 2010
- Primary completion
- Jun 2016
- Countries
- 2
- Sponsor
- Alexion Pharmaceuticals
- Modality
- Replacement Enzymes
- Drugs
- asfotase alfa
- Conditions
- hypophosphatasia (HPP)
01
Overview
Phase 2, randomised, open-label trial of asfotase alfa in hypophosphatasia (HPP) (3 arms), sponsored by Alexion Pharmaceuticals. Primary endpoints: Change From Baseline to Week 24 for Plasma Pyridoxal-5' Phosphate, Change From Baseline to Week 24 for Plasma Inorganic Pyrophosphate and 1 more. Started Jun 2010; status: completed.
02
Design
3 arms- Study design
- RandomizedOpen-label
Cohort 1
asfotase alfa 0.3 mg/kg/day SC
Cohort 2
asfotase alfa 0.5 mg/kg/day SC
Concurrent Control
No treatment during primary period, then switches to asfotase alfa in extension
03
Endpoints
Primary
Change From Baseline to Week 24 for Plasma Pyridoxal-5' Phosphate
Change From Baseline to Week 24 for Plasma Inorganic Pyrophosphate
Safety and Tolerability of Asfotase Alfa
Secondary
Change From Baseline in Bone Mineral Content
Change From Baseline in Bone Mineral Density
Change in Walking Ability as Measured by the Six-Minute Walk Test
Change From Baseline in HPP-related Osteomalacia: Osteoid Volume/Bone Volume
Change From Baseline in HPP-related Osteomalacia: Osteoid Thickness
Change From Baseline in HPP-related Osteomalacia: Mineralization Lag Time
04
Results
Not all primary endpoints met
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