Hypophosphatasia – European Clinical Trials Information Network

Study of ilofotase alfa effects on biomarkers in adults with hypophosphatasia

Wed, 29 Apr 2026 15:08:27 +0000

This study examines hypophosphatasia, a rare genetic condition that affects bones and teeth due to low levels of an enzyme called alkaline phosphatase. People with this condition have problems with bone development and strength because their bodies cannot properly process certain substances. The treatment being tested is ilofotase alfa, which is given as an infusion into a vein. This medication is designed to replace the missing enzyme and help the body process substances that build up in people with this condition.

The purpose of the study is to see how ilofotase alfa affects certain substances in the blood of adults with hypophosphatasia. Specifically, researchers want to measure changes in two substances called inorganic pyrophosphate and pyridoxal 5′-phosphate after patients receive either a low dose or a high dose of the medication. The study will look at the biochemical profile, which means the levels of various substances in the blood, to understand how the medication works in the body.

During the study, patients will receive a single dose of ilofotase alfa through an intravenous infusion. The medication will be prepared from a concentrate for solution for infusion. Patients will be monitored to see how their blood chemistry changes after receiving the treatment. This is an open-label pilot trial, which means both the patients and doctors will know what treatment is being given, and it involves a small number of participants to gather initial information about how the medication affects adults with this rare condition.

A Study Testing the Safety and Tolerability of ALE1 in Healthy Adults and Adults with Hypophosphatasia

Wed, 29 Apr 2026 15:07:56 +0000

This study is looking at a condition called Hypophosphatasia, which is a rare inherited disorder that affects the development of bones and teeth. The treatment being tested is ALE1, which is given as a hard capsule taken by mouth. Some people in the study will receive ALE1 while others will receive placebo. The study will include both healthy adults and adults who have Hypophosphatasia.

The purpose of the study is to investigate the safety and how well people can tolerate ALE1 when it is given in increasing amounts. The study will also look at how the body processes the medicine by measuring how much of it is found in the blood over time and how it affects certain markers in the blood. Additionally, the study will examine whether taking the medicine with or without food makes any difference in how the body handles it.

The study is divided into two parts. The first part will involve giving single doses of the medicine to healthy adults, starting with lower amounts and gradually increasing them. The second part will involve giving multiple doses over a period of time to both healthy adults and adults with Hypophosphatasia. Throughout the study, participants will be closely monitored for any unwanted effects and will have blood samples taken to measure how the medicine works in the body. Different dose strengths of ALE1 will be tested to help identify which doses are suitable for further research.

Study of ALXN1850 for Children Aged 2 to 12 with Hypophosphatasia Who Have Not Previously Received Asfotase Alfa Treatment

Wed, 29 Apr 2026 14:56:43 +0000

This clinical trial is focused on studying a rare condition called hypophosphatasia (HPP), which affects the development of bones and teeth. The study will evaluate a treatment known as ALXN1850, which is given as a solution for injection under the skin. The trial will compare the effects of ALXN1850 with a placebo in children aged 2 to less than 12 years who have not previously been treated with another medication called asfotase alfa.

The purpose of the study is to assess how well ALXN1850 works in improving bone health in children with HPP. Participants will receive either ALXN1850 or a placebo through subcutaneous injections. The study will last for several months, during which the children will have regular check-ups to monitor their progress and any changes in their condition. The main focus will be on how the treatment affects the bones, as seen in X-ray images, and other health measures.

Throughout the study, the safety and effectiveness of ALXN1850 will be closely monitored. The trial aims to provide valuable information on whether ALXN1850 can be a beneficial treatment option for children with hypophosphatasia, potentially improving their quality of life by addressing the symptoms of this condition.

Study on ALXN1850 for Adolescents and Adults with Hypophosphatasia Not Previously Treated with Asfotase Alfa

Wed, 29 Apr 2026 14:30:38 +0000

This clinical trial is focused on studying a rare condition called Hypophosphatasia (HPP), which affects the development of bones and teeth. The study will evaluate a treatment known as ALXN1850, a type of enzyme therapy, which is given as a solution for injection under the skin. The trial will compare the effects of ALXN1850 to a placebo in adolescents and adults who have not previously been treated with another medication called Asfotase Alfa.

The purpose of the study is to assess how well ALXN1850 works in improving physical function in participants with HPP. Participants will receive either ALXN1850 or a placebo and will be monitored over a period of time to see how their condition changes. The study will involve regular visits to the clinic for assessments and injections, and participants will be asked to perform certain physical tests to measure their progress.

This trial is designed to provide valuable information about the safety and effectiveness of ALXN1850 for people with HPP. By participating, individuals will contribute to research that may help improve treatment options for this condition in the future.

Study on ALXN1850 and Asfotase Alfa for Children Aged 2-12 with Hypophosphatasia Previously Treated with Asfotase Alfa

Wed, 29 Apr 2026 14:30:08 +0000

This clinical trial is focused on studying a rare condition called Hypophosphatasia (HPP), which affects the development of bones and teeth. The study involves children aged 2 to under 12 years who have previously been treated for HPP. The trial will compare two treatments: a new medication known as ALXN1850 and an existing treatment called Asfotase Alfa, which is marketed under the name Strensiq. Both medications are given as a solution for injection under the skin, a method known as subcutaneous injection.

The purpose of the study is to evaluate the safety and how well children tolerate ALXN1850 compared to Asfotase Alfa. Participants will be randomly assigned to receive either ALXN1850 or Asfotase Alfa. The study will monitor the children over a period to observe any side effects and how the treatments affect their condition. The trial will also look at various health measures to understand the impact of the treatments on the children’s overall health and quality of life.

Throughout the study, researchers will collect information on the children’s health and any changes in their condition. This will include regular check-ups and assessments to ensure the safety and effectiveness of the treatments. The study aims to provide valuable insights into the management of Hypophosphatasia in children and potentially improve treatment options for this condition in the future.

Study on Immunosuppressive Therapies with Asfotase Alfa for Patients with Hypophosphatasia Experiencing Reduced Treatment Effectiveness

Wed, 29 Apr 2026 14:25:21 +0000

This clinical trial is focused on studying a rare condition called Hypophosphatasia, which affects the development of bones and teeth. The study aims to explore the use of certain treatments to help patients who have developed an immune response that reduces the effectiveness of their current treatment with Asfotase Alfa, a medication used to manage this condition. The trial will involve the use of immunosuppressive therapies, which are treatments that help reduce the activity of the immune system, to see if they can help restore the effectiveness of Asfotase Alfa.

Participants in the study will receive treatments over a period of time, and their response to the therapies will be monitored. The study will include medications such as Rituximab, Bortezomib, and Methotrexate Disodium, which are known to help manage immune responses. Some participants may also receive a placebo, which is a substance with no active medication, to compare the effects of the treatments. The study will track changes in the participants’ condition and any side effects they may experience.

The purpose of this study is to evaluate whether these immunosuppressive therapies can help patients who have lost the therapeutic benefits of Asfotase Alfa due to an immune response. The trial will last for a set period, during which participants will undergo regular assessments to monitor their health and the effectiveness of the treatments. The goal is to find a way to improve the management of Hypophosphatasia for those affected by this immune-mediated loss of response.