Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia

Sponsor
Alexion Pharmaceuticals, Inc.
Study ID
NCT06015750
Phase
PHASE4
Status
Withdrawn

Conditions

  • Hypophosphatasia

Eligibility Criteria

Sex
ALL
Age
2 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • methotrexate — DRUG
    Methotrexate will be administered SC or orally weekly for 104 weeks.
  • rituximab — DRUG
    Rituximab will be administered intravenously (IV) continuously weekly, for up to 74 weeks.
  • bortezomib — DRUG
    Bortezomib will be administered via IV bolus or SC, as needed.
  • IVIg — DRUG
    IVIg will be administered via IV monthly through initial 74 weeks.
  • Folic Acid — DRUG
    Folic acid will be given orally as long as methotrexate is being dosed.

Study Details

The primary objective of this study is to evaluate the effect of immunosuppressive therapy (IST) in participants treated with asfotase alfa who demonstrate immune-mediated loss of effectiveness (LoE).

Key Dates

Start date
Jul 29, 2026
Status verified
Oct 2025
Primary completion
Mar 13, 2030
Completion
Mar 13, 2030

Study Design

Enrollment
0 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Pediatric participants with HPP
    Pediatric participants who have been receiving asfotase alfa treatment for their HPP, and who demonstrate immune-mediated LoE.

Primary Outcome Measure

Number of Participants Who Achieve Immunosuppressive Therapy (IST) Complete Response at Week 100 [ Time Frame: Week 100 ]

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