ALXN1850 for Hypophosphatasia
(HICKORY Trial)
Trial Summary
What is the purpose of this trial?
This trial is testing a new treatment called ALXN1850 to see if it helps adolescents and adults with HPP improve their ability to perform daily activities. The participants have not been treated with another common medication for HPP.
Do I have to stop taking my current medications for this trial?
The trial protocol does not specify if you need to stop taking your current medications. However, if you have certain health conditions that affect drug absorption, metabolism, or elimination, you may be excluded from the trial.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
What data supports the idea that ALXN1850 for Hypophosphatasia is an effective treatment?
The available research shows that ALXN1850, also known as asfotase alfa, is effective for treating hypophosphatasia. It is the only approved treatment that can normalize mineralization in patients, which is crucial for bone health. Studies have shown that it improves survival in severely affected infants and enhances the quality of life in children and adults. Additionally, when treatment was stopped in a group of adults, their condition worsened, but improved again once the treatment was restarted. This suggests that ALXN1850 is effective in managing the symptoms of hypophosphatasia.12345
What data supports the effectiveness of the drug ALXN1850 for treating hypophosphatasia?
Research shows that asfotase alfa, a component of ALXN1850, is effective in improving bone and dental health in people with hypophosphatasia, a condition where bones and teeth don't harden properly. This drug has been transformative in improving survival and quality of life for patients, especially in severe cases.12346
What safety data is available for ALXN1850 treatment in hypophosphatasia?
The safety data for ALXN1850, also known as asfotase alfa, includes findings from several studies. A five-year study (NCT01163149) evaluated the efficacy and safety of asfotase alfa in adults and adolescents with hypophosphatasia, showing significant changes in plasma PLP and PPi concentrations. Another study reported altered thyroid function tests in patients treated with asfotase alfa, indicating potential interference in ALP-dependent immunoassays. Additionally, a case series highlighted clinical deterioration upon discontinuation of asfotase alfa, suggesting the need for close monitoring if treatment is stopped. Overall, these studies provide insights into the safety profile of asfotase alfa in treating hypophosphatasia.12357
Is ALXN1850 (Efzimfotase alfa) safe for humans?
Is the drug ALXN1850, also known as asfotase alfa, a promising treatment for hypophosphatasia?
Yes, ALXN1850 (asfotase alfa) is a promising treatment for hypophosphatasia. It is the only approved drug that can improve bone and dental health in patients with this condition. It has been shown to enhance survival in severely affected infants and improve the quality of life in children and adults. The drug works by replacing a missing enzyme, helping to normalize mineralization in the body.13478
How is the drug ALXN1850 different from other treatments for hypophosphatasia?
ALXN1850, also known as asfotase alfa, is unique because it is an enzyme replacement therapy specifically designed to address the underlying cause of hypophosphatasia by replacing the deficient enzyme, tissue-nonspecific alkaline phosphatase (TNSALP). This approach is different from previous supportive care methods, as it directly targets the enzyme deficiency to improve bone mineralization and overall quality of life.13478
Eligibility Criteria
This trial is for adolescents and adults with Hypophosphatasia (HPP) who have a documented ALPL gene variant, low serum alkaline phosphatase activity without other causes than HPP, high plasma PLP levels, and reduced walking capacity. They must not have been treated with asfotase alfa before. People with hypoparathyroidism unrelated to HPP, recent fractures or surgeries that could affect the study, allergies to ingredients in the treatment or placebo, or significant health issues affecting drug absorption or posing risks are excluded.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Randomized Evaluation Period
Participants receive either ALXN1850 or placebo via subcutaneous injection every 2 weeks for 24 weeks
Open-label Extension (OLE) Period
Participants receive ALXN1850 via subcutaneous injection every 2 weeks for up to 132 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- ALXN1850
- Placebo
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