Immunosuppressive Therapy for Hypophosphatasia
(RESTORE Trial)
Recruiting in Palo Alto (17 mi)
+4 other locations
Age: < 65
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 4
Waitlist Available
Sponsor: Alexion Pharmaceuticals, Inc.
No Placebo Group
Prior Safety Data
Breakthrough Therapy
Trial Summary
What is the purpose of this trial?
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).
Research Team
Eligibility Criteria
This trial is for people with Hypophosphatasia who initially responded to Asfotase Alfa treatment but are now seeing their condition worsen. They must have been on the treatment for at least 6 months and currently still be receiving it. Participants need proof of specific antibodies, agree to use contraception if they can have children, and sign a consent form.Inclusion Criteria
My rickets got worse in the last 3 months despite taking asfotase alfa.
I am following the study's rules for birth control.
My tests show my cancer is likely to respond to immune therapy.
See 2 more
Treatment Details
Interventions
- Immunosuppressive Therapy (Immunosuppressant)
Trial OverviewThe study tests whether immunosuppressive therapy (IST) using drugs like methotrexate, rituximab, bortezomib, IVIg, and Folic Acid can help patients who've stopped responding well to Asfotase Alfa due to immune system interference.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Pediatric participants with HPPExperimental Treatment5 Interventions
Pediatric participants who have been receiving asfotase alfa treatment for their HPP, and who demonstrate immune-mediated LoE.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alexion Pharmaceuticals, Inc.
Lead Sponsor
Trials
267
Recruited
141,000+
Dr. Alberto R. Martinez
Alexion Pharmaceuticals, Inc.
Chief Medical Officer since 2010
MD from University of Sao Paulo
Marc Dunoyer
Alexion Pharmaceuticals, Inc.
Chief Executive Officer since 2021
PhD in Molecular Biology
Alexion
Lead Sponsor
Trials
247
Recruited
38,600+
Marc Dunoyer
Alexion
Chief Executive Officer since 2021
PhD in Molecular Biology, University of Brussels
Christophe Hotermans
Alexion
Chief Medical Officer since 2021
MD, University of Leuven