In short
Clinical trials are investigating Alxn1850 in people with hypophosphatasia (HPP), a rare bone disease. These studies are looking at whether Alxn1850 is effective and safe in children, teenagers, and adults, including people who have not used asfotase alfa before and those previously treated with it.
Key points
- Clinical trials of Alxn1850 are being done in hypophosphatasia, a rare bone disease. All three listed studies are Phase 3 trials, which means they are later-stage studies in larger groups. One study is in pediatric participants who have not been treated before, one is in pediatric participants previously treated with asfotase alfa, and one is in adolescent and adult participants who have not been treated before. The trials are looking at both effectiveness and safety, using outcomes such as X-ray-based scores, the 6-minute walk test, and treatment-related safety events. Some studies compare Alxn1850 with placebo, while one compares it with asfotase alfa.
Trial overview
Alxn1850 is being studied in hypophosphatasia, also called HPP, which is the condition named in all of the listed trials. All three studies are Phase 3 and are interventional trials, meaning researchers are giving a study treatment and measuring what happens.
The trials are focused on different age groups and treatment histories. Two studies are for participants who have not previously been treated with asfotase alfa, and one study is for pediatric participants who were previously treated with asfotase alfa.
Who is being studied
One study includes treatment-naïve pediatric participants, which means children who have not been treated before with asfotase alfa. Another study includes pediatric participants with HPP who were already treated with asfotase alfa.
The third study includes adolescent and adult participants with HPP who have not previously been treated with asfotase alfa. This shows that the research program is looking at Alxn1850 across different ages and different treatment backgrounds.
What the trials measure
The pediatric study in treatment-naïve participants measures the RGI-C score at the end of the randomized evaluation period on Day 169. This is a radiographic outcome, which means it looks at changes seen on imaging such as X-rays.
The adolescent and adult study measures change from baseline in the 6-minute walk test at Day 169. This test shows how far a person can walk in 6 minutes and is used here as a functional outcome, meaning it helps measure how well the body works in daily life.
The pediatric study in participants previously treated with asfotase alfa focuses on safety and tolerability, using outcomes such as the incidence of TEAEs, TESAEs, AESIs, and adverse events that lead to stopping or pausing study treatment. These terms describe different kinds of unwanted medical events during the study, from general side effects to serious events and events of special interest.
What the studies compare
Two trials compare Alxn1850 with placebo, which is an inactive product used to make the comparison fair. This helps researchers see whether changes are due to Alxn1850 rather than to chance or to the trial setting.
The pediatric safety study compares Alxn1850 with asfotase alfa, which is listed in the trial record as Strensiq. This comparison is used to assess safety and tolerability in children who have already received asfotase alfa.
Trial status and size
The listed study statuses are Authorised for two trials and Ongoing for one trial. The planned enrollment is 47 participants in the treatment-naïve pediatric study, 40 in the pediatric safety study, and 122 in the adolescent and adult study.
Together, these trials show that Alxn1850 is being tested in a staged clinical research program for people with HPP, with separate studies for different ages and prior treatment histories.