Asfotase Alfa for Hypophosphatasia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial observes individuals with pediatric-onset hypophosphatasia (HPP) who are receiving or will soon begin treatment with asfotase alfa, a medication designed to manage this rare bone disease. The researchers aim to assess how the treatment affects various aspects of health and quality of life over at least five years. Individuals who showed symptoms of HPP before age 18 and are either currently on asfotase alfa or plan to start it soon are eligible. Participants must also have low levels of a specific enzyme related to HPP. As a Phase 4 trial, this study focuses on understanding how the already FDA-approved and effective treatment benefits more patients, providing valuable insights into long-term health improvements.
Do I have to stop taking my current medications for this trial?
The trial information does not specify whether you need to stop taking your current medications. However, you must be receiving or planning to start asfotase alfa treatment.
What prior data suggests that Asfotase Alfa is safe for pediatric-onset hypophosphatasia?
Research has shown that Asfotase Alfa is usually well-tolerated. Most patients experience only mild side effects, such as redness or swelling at the injection site. Some may also experience headaches or a fever.
The FDA has already approved the treatment for a different condition, indicating that its safety is well understood. This approval helps doctors understand its effects on the body and potential side effects. However, individual responses can vary, so discussing any concerns with a doctor is important.
Why are researchers excited about this trial?
Unlike traditional treatments for hypophosphatasia (HPP), which often focus on managing symptoms, Asfotase Alfa targets the root cause of the disease. It is an enzyme replacement therapy designed to address the enzyme deficiency that leads to HPP. Researchers are excited about Asfotase Alfa because it has the potential to significantly improve bone mineralization and overall quality of life for patients, particularly those with pediatric-onset HPP. This targeted approach distinguishes it from standard treatments and offers new hope for a more effective management strategy.
What evidence suggests that Asfotase Alfa might be an effective treatment for hypophosphatasia?
Research has shown that asfotase alfa helps manage symptoms of hypophosphatasia (HPP), a rare bone disease. Studies have found that children taking asfotase alfa experience better growth, with improvements in height and weight. For adults with childhood-onset HPP, asfotase alfa enhances physical abilities and quality of life. Long-term evidence supports that this treatment manages various HPP symptoms over many years. Overall, asfotase alfa has a strong record of improving life for those with childhood-onset HPP.12345
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Observational Follow-up
Participants with pediatric-onset hypophosphatasia are followed for a minimum of 5 years to assess biochemical, clinical, imaging, and functional/quality of life outcomes
Follow-up
Participants are monitored for safety and effectiveness after the observational period
What Are the Treatments Tested in This Trial?
Interventions
- Asfotase Alfa
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Each participant will be followed for a minimum of 5 years or, if applicable, until early withdrawal. Biochemical, clinical, imaging (if clinically indicated), and functional/quality of life outcomes relevant to HPP will be assessed.
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
Alexion Pharmaceuticals
Lead Sponsor
Marc Dunoyer
Alexion Pharmaceuticals
Chief Executive Officer since 2021
B.A. in Psychology from the University of New Hampshire
Christophe Hotermans
Alexion Pharmaceuticals
Chief Medical Officer since 2023
MD
Citations
Mobility and Quality of Life in Children with Paediatric ...
Children who took the drug had improved physical development through up to 5 years of follow-up. They had improvements in height and weight, ...
2.
clinicaltrials.gov
clinicaltrials.gov/study/NCT04195763?term=Hypophosphatasia&viewType=Table&rank=9Patient Reported Outcomes in Adults With Pediatric-onset ...
This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with ...
Improvement in quality of life after asfotase alfa treatment in ...
This study of a real-world cohort of adults with pediatric-onset HPP suggests that asfotase alfa is effective in improving physical functioning, fatigue, sleep, ...
10 Years Post-Approval | Advances in Therapy
The findings from the past decade show that asfotase alfa is effective in managing the wide range of symptoms associated with hypophosphatasia, ...
Long-term effectiveness of asfotase alfa in adults with ...
These data suggest adults who have pediatric-onset HPP treated with asfotase alfa in real-world settings have marked improvements in clinical and functional ...
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