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

Study identifier:D8400C00001

ClinicalTrials.gov identifier:NCT06015750

EudraCT identifier:2022-502793-17

CTIS identifier:N/A

Will Be Recruiting

Official Title

An Interventional, Prospective Open-Label Study of Immunosuppressive Therapies to Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia (RESTORE)

Medical condition

Hypophosphatasia

Phase

Phase 4

Healthy volunteers

Study drug

methotrexate, rituximab, bortezomib, IVIg, Folic Acid

Sex

All

Estimated Enrollment

Study type

Interventional

Age

2 Years - 18 Years

Date

Study Start Date: 01 Oct 2024

Estimated Primary Completion Date: 29 Sept 2028

Estimated Study Completion Date: 29 Sept 2028

Study design

Allocation: N/A
Endpoint Classification: -
Intervention Model: Single Group Assignment
Masking: -
Primary Purpose: Treatment

Verification:

Verified 01 Jul 2024 by Alexion Pharmaceuticals, Inc.

Collaborators

Inclusion and exclusion criteria

Inclusion Criteria

- 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 Criteria

- 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.

No locations available

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

Contacts

Contact

Alexion Pharmaceuticals, Inc. (Sponsor)

1-855-752-2356

[email protected]