Immunosuppressive Therapy for Hypophosphatasia
(RESTORE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores the effectiveness of immunosuppressive therapy for individuals with hypophosphatasia (HPP) who are already receiving asfotase alfa treatment but have experienced worsening due to immune reactions. The study aims to determine if adding this therapy can benefit those who initially responded well to asfotase alfa but now find it less effective. Ideal candidates have experienced a recurrence or worsening of rickets (a condition affecting bone development) for at least three months and have been on asfotase alfa for over six months. As a Phase 4 trial, this study involves an FDA-approved treatment and seeks to understand how it can benefit more patients.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. However, since the study involves immunosuppressive therapy, it's possible that some medications might need to be adjusted. Please consult with the trial coordinators for specific guidance.
What is the safety track record for immunosuppressive therapy?
Research has shown that asfotase alfa, a treatment for hypophosphatasia (HPP), is safe and effective. Patients who received asfotase alfa in studies experienced positive results over several years. These studies examined the long-term effects of the treatment in both adults and teenagers.
Regarding safety, researchers closely monitored any unwanted side effects and found no major safety issues. This indicates that asfotase alfa is generally well-tolerated by patients.
This trial tests the addition of immunosuppressive therapy for patients who have developed an immune reaction to asfotase alfa. The trial is in a later phase, which usually indicates strong safety support. While specific safety details for the immunosuppressive therapy in this context aren't provided, the advanced stage of the trial suggests that researchers have evaluated any risks and consider them manageable.12345Why are researchers enthusiastic about this study treatment?
Unlike the standard treatment for hypophosphatasia, which often involves enzyme replacement therapy like asfotase alfa, immunosuppressive therapy offers a fresh approach by potentially addressing immune-related complications. Researchers are excited about this therapy because it targets immune-mediated loss of efficacy, a challenge some patients face with current treatments. This approach could enhance the effectiveness of existing therapies, offering a new hope for patients with immune issues related to their hypophosphatasia treatment.
What evidence suggests that immunosuppressive therapy might be an effective treatment for hypophosphatasia?
Research shows that immunosuppressive therapy can help when the body's immune response reduces the effectiveness of asfotase alfa. Asfotase alfa improves bone health and growth in people with hypophosphatasia, but sometimes the immune system interferes with its effectiveness. Immunosuppressive therapy, which participants in this trial may receive, may calm the immune system, allowing asfotase alfa to continue working effectively. Previous studies have shown that asfotase alfa improves movement and quality of life for those with hypophosphatasia. This trial aims to determine if immunosuppressive therapy can assist when patients encounter difficulties with this treatment.16789
Are You a Good Fit for This Trial?
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
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive immunosuppressive therapy to mitigate immune-mediated loss of effectiveness to asfotase alfa
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Immunosuppressive Therapy
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alexion Pharmaceuticals, Inc.
Lead Sponsor
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
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