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Immunosuppressant

Immunosuppressive Therapy for Hypophosphatasia (RESTORE Trial)

Phase 4
Waitlist Available
Research Sponsored by Alexion Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Presence of ADAs, with or without NAbs, irrespective of their titers.
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Drug Has Already Been Approved
No Placebo-Only Group
Pivotal Trial

Summary

This trial aims to see if using immunosuppressive therapy can help patients who are not responding well to treatment with asfotase alfa due to immune-related issues.

Who is the study for?
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.
What is being tested?
The 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.
What are the potential side effects?
Possible side effects include weakened immunity leading to infections, reactions at injection sites, potential liver issues from methotrexate or rituximab; nerve damage from bortezomib; headaches or nausea from IVIg; and stomach upset from Folic Acid.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have antibodies against the drug I was given.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Drug Has Already Been Approved
The FDA has already approved this drug, and is just seeking more data.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
methotrexate
1997
Completed Phase 3
~9960
rituximab
2000
Completed Phase 3
~2760
bortezomib
2011
Completed Phase 3
~850
IVIg
2013
Completed Phase 2
~140
Folic Acid
2008
Completed Phase 4
~7610

Find a Location

Who is running the clinical trial?

Alexion Pharmaceuticals, Inc.Lead Sponsor
261 Previous Clinical Trials
140,476 Total Patients Enrolled
25 Trials studying Hypophosphatasia
1,544 Patients Enrolled for Hypophosphatasia
AlexionLead Sponsor
246 Previous Clinical Trials
38,509 Total Patients Enrolled
25 Trials studying Hypophosphatasia
1,544 Patients Enrolled for Hypophosphatasia
~5 spots leftby Jan 2029
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