- ongoing, recruitingHuman Pharmacology (Phase I)- Other
Open-Label Pilot Trial to Evaluate the Effects of Ilofotase Alfa on Biomarkers in Adult Patients with Hypophosphatasia
This study is looking at a new medicine called ilofotase alfa for adults with a rare bone condition called hypophosphatasia (HPP). We want to see how different doses of this medicine might change important markers in the body.
Germany - active not recruitingPHASE3
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
This study is looking for children aged 2-11 with a rare bone condition called Hypophosphatasia (HPP) who haven't had previous treatment. It's testing a new medicine, ALXN1850, to see how well it helps their bones compared to a dummy medicine.
United States · Australia · Belgium - active not recruitingPHASE3
Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa
This study is looking into a new medicine, ALXN1850, for teenagers and adults with Hypophosphatasia (HPP). We want to see if it helps with daily activities compared to a dummy medicine (placebo). This is for people who haven't tried a similar treatment called asfotase alfa before.
United States · Argentina · Australia - active not recruitingPHASE3
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa
This study is looking at a new medicine called ALXN1850 for children with a rare bone condition called hypophosphatasia (HPP). It compares ALXN1850 with a current treatment, asfotase alfa, to see if it's safe and works well for children who have already been using asfotase alfa.
United States · Argentina · Australia - authorisedPhase I and Phase II (Integrated)- First administration to humans
A Randomised, Placebo-Controlled, Double-Blind, Single-Ascending Dose and Multiple-Ascending Dose First-In-Human Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Orally Administered ALE1 With or Without Food in Healthy Adult Participants and Adult Patients With Hypophosphatasia
This study is testing a new medicine, ALE1, for a condition called Hypophosphatasia (HPP). We are looking at its safety and how the body handles it in healthy people and those with HPP, starting with small doses.
Germany - recruiting
Characteristics of Hypophosphatasia in Adult Patients in Rheumatology and Their Value in Developing an Algorithm to HPP-diagnosis - the COHIR Multi-center Study
This study looks for a rare bone condition called Hypophosphatasia (HPP) in adults already seeing a rheumatologist. It aims to find out how many people in these clinics might have HPP and create a simple way for doctors to diagnose it earlier.
Germany - ongoing, recruitingTherapeutic use (Phase IV)
An Interventional, Prospective Open-Label Study of Immunosuppressive Therapies to Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia (RESTORE)
This study looks at ways to help a medicine called Asfotase Alfa (STRENSIQ®) work better for people with Hypophosphatasia. Sometimes the body fights this medicine, so the study tests other treatments to stop that and improve how well the medicine works.
France