Active not recruitingPHASE3INTERVENTIONAL

Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa

This research study is testing a new medicine called ALXN1850 for teenagers and adults who have a rare bone condition called Hypophosphatasia (HPP). The main goal is to find out if this new medicine can help people perform better in their daily activities compared to taking a placebo (a dummy medicine with no active ingredients). This is a 'Phase 3' study, which means it’s a big step before a medicine might become widely available. Importantly, this study is for individuals who have not previously taken a different HPP medicine called asfotase alfa. We aim to understand if ALXN1850 is an effective and safe option for managing HPP.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Alexion Pharmaceuticals, Inc.

Enrolment target

124

Start

03 Jan 2024

Estimated completion

29 Mar 2028

Hypophosphatasia

What is this study about?

Hypophosphatasia, often called HPP, is a rare inherited condition that affects how bones and teeth develop. It can lead to weak bones, frequent fractures, and problems with movement. This study is testing a new medicine, ALXN1850, which scientists hope might help improve bone health and how people with HPP can move and carry out their daily lives.

Currently, there are limited treatments for HPP. This study is important because it could offer a new option for patients. Researchers want to compare ALXN1850 against a 'placebo', which is a treatment that looks the same but contains no active medicine. This allows them to clearly see if any improvements are due to the study medicine or other factors. For ethical reasons, the study design ensures that participants who receive a placebo will still receive appropriate care for their HPP.

By carefully studying how participants respond to ALXN1850, doctors can learn if it's a safe and effective treatment. This research is a crucial step in potentially making new HPP medicines available to more people in the future.

Key takeaways

This study tests a new medicine (ALXN1850) for teenage and adult HPP patients.
It compares the new medicine to a dummy pill (placebo) to see if it helps with daily activities.
Participation is for those who haven't used asfotase alfa before.
You would attend clinic visits for assessments and blood tests.
There are specific health criteria for joining, including age and HPP diagnosis details.
You can stop participating at any time.

Who may be eligible?

This study is looking for teenagers and adults aged 12 to 130 years old who have Hypophosphatasia (HPP). To join, you need to have a confirmed diagnosis of HPP, either proven by a genetic test or by certain blood tests showing specific levels that are typical for HPP. You also need to have low alkaline phosphatase (ALP) levels in your blood, which is another sign of HPP.

We would also check your walking ability. You'd need to complete a walking test where you walk as far as you can in six minutes, and your results would need to be below what's expected for someone of your age, sex, weight, and height. It's important that any problems you have with walking are due to your HPP and not another cause.

There are also some reasons why you might not be able to join. For instance, if you have other serious health problems affecting your heart, lungs, kidneys, or other major organs, or if you've had a new bone fracture (not including some specific types of fractures) in the 12 weeks before starting the study. If you're planning surgery that might affect the study results, or if you're allergic to any ingredients in the study medicine, you wouldn't be able to take part. Also, anyone with certain specific bone or hormone conditions would not be eligible.

Quick self-check

Are you between 12 and 130 years old?
Do you have a confirmed diagnosis of Hypophosphatasia (HPP)?
Have you *not* been treated with a medicine called asfotase alfa before?
Can you complete a walking test that might show your walking ability is below average for your age and sex?
Do you have low alkaline phosphatase (ALP) blood levels?
Do you have generally stable health without other serious ongoing medical conditions?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, you would receive either the new medicine ALXN1850 or a placebo (a dummy medicine). You wouldn't know which one you're getting, and neither would your study doctor. Over the course of the study, you would attend several appointments at the clinic. These visits would involve various assessments like blood tests, physical examinations, and walking tests to see how you are responding to the treatment. The research team will track your progress carefully. The total length of your participation would be explained by the study doctor, including how long you'd be taking the medicine and any follow-up visits after you stop the treatment.

Potential risks and benefits

Taking part in any clinical trial has potential benefits and risks. You might benefit from receiving a new medicine before it's widely available, and your health could improve. However, there's also a chance you could receive a placebo and not benefit directly from the active treatment. All medicines can have side effects, and some might be serious. The study team will monitor you closely for any side effects and explain all known risks. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (65)

Research Site
Indianapolis, United States
Research Site
Garden City, United States
Research Site
Durham, United States
Research Site
Columbus, United States
Research Site
Nashville, United States
Research Site
Ciudad de Buenos Aires, Argentina
Research Site
Clayton, Australia
Research Site
Herston, Australia
Research Site
Parkville, Australia
Research Site
St Leonards, Australia
Research Site
Belo Horizonte, Brazil
Research Site
Brasília, Brazil

+53 more sites — see the official record for the full list.

Common questions

What is Hypophosphatasia (HPP)?

HPP is a rare genetic condition that affects how your bones and teeth develop, making them weak or fragile.

What is a placebo?

A placebo is a substance that looks like the real medicine but contains no active ingredients. It helps researchers compare the new medicine's effects.

Will I know if I'm getting the real medicine or the placebo?

No, this is a 'blinded' study, meaning neither you nor your study doctor will know which treatment you are receiving until after the study ends.

How long will the study last?

The total duration of the study, including treatment and follow-up, will be explained in detail by the study team.

Can I leave the study at any time?

Yes, your participation is voluntary, and you can withdraw from the study at any point without it affecting your medical care.