Status:
WITHDRAWN
Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Hypophosphatasia
Eligibility:
All Genders
2-18 years
Phase:
PHASE4
Brief Summary
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).
Detailed Description
The administration of biological drugs to patients, especially for chronic conditions, carries a risk of eliciting anti-drug antibodies. Neutralizing antibodies can neutralize the clinical benefit of ...
Eligibility Criteria
Inclusion
- Reoccurrence or worsening of rickets for at least the past 3 months in participants who showed an initial efficacy response to asfotase alfa after at least 6 months of continuous treatment and currently receiving asfotase alfa. RSS will be used to determine severity at Baseline.
- Presence of ADAs, with or without NAbs, irrespective of their titers.
- Confirmation by the TMB that both the clinical evidence and immunogenicity-mediated association noted above are present.
- Female participants of childbearing potential and male participants with partners of childbearing potential must follow protocol-specified contraception guidance as described in Section 10.5.
- Participant, or participant's legal guardian, is capable of signing informed consent or assent as described in Section 10.1.3, which includes compliance with the requirements and restrictions listed in the informed consent or assent form and in this protocol.
Exclusion
- Known history of human immunodeficiency virus (HIV) infection (evidenced by HIV type 1 or type 2 \[HIV 1, HIV 2\] antibody) or hepatitis B or C viral infection.
- Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to Screening.
- Inability of the participant, or the participant's legal guardian, to provide informed consent.
- Pregnant, breastfeeding, or intending to conceive during the course of the study.
- Inability to travel to the clinic for specified visits during the Treatment Period caused by disease per se or logistics (does not apply to external travel restrictions).
- The participant is at risk of reactivation or has an active significant viral infection such as hepatitis B, cytomegalovirus, herpes simplex, human polyomavirus (also known as John Cunningham \[JC\] virus), parvovirus, or Epstein Barr virus.
- The participant is at risk of reactivation of tuberculosis or has regular contact (eg, in the household) with individuals who are being actively treated for tuberculosis.
- The participant has had or is required to have any live vaccination within 1 month prior to enrollment.
Key Trial Info
Start Date :
July 29 2026
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 13 2030
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT06015750
Start Date
July 29 2026
End Date
March 13 2030
Last Update
December 18 2025
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