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IST for Loss of Response to Strensiq in HPP
NCT06015750Phase 4
Key facts
- Status
- Withdrawn
- Start
- Jul 2026
- Primary completion
- Mar 2030
- Sponsor
- Alexion Pharmaceuticals
- Modality
- Monoclonal Antibodies, Modified Peptides, Recombinant Proteins and Enzymes
- Conditions
- hypophosphatasia (HPP)
01
Overview
Phase 4, open-label, single-arm trial of methotrexate, rituximab, bortezomib and intravenous immunoglobulin G in hypophosphatasia (HPP), sponsored by Alexion Pharmaceuticals. Primary endpoint: Number of Participants Who Achieve Immunosuppressive Therapy (IST) Complete Response at Week 100. Planned start Jul 2026; status: withdrawn.
02
Design
1 arm- Study design
- Non-randomizedOpen-labelSingle group
Pediatric participants with HPP
Pediatric participants who have been receiving asfotase alfa treatment for their HPP, and who demonstrate immune-mediated loss of effectiveness.
03
Who it enrols
Pediatric patients with HPP who have lost response to asfotase alfa due to anti-drug antibodies.
04
Endpoints
Primary
Number of Participants Who Achieve Immunosuppressive Therapy (IST) Complete Response at Week 100
Secondary
Number Participants with Antidrug Antibodies (ADAs) and Neutralizing Antibodies (NAbs)
ADA and NAb Titer Levels
Serum Concentration of Asofatase Alfa (Measured as Enzyme Activity)
Plasma Concentration of Pyridoxal-5ˈ-Phosphate (PLP)
Plasma Concentration of Inorganic Pyrophosphates (PPi)
Number of Participants with Treatment-emergent Adverse Events and Treatment-emergent Serious Adverse Events
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