Active not recruitingPHASE3INTERVENTIONAL

Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa

This research study is about finding better treatments for children with a rare bone condition called hypophosphatasia (HPP). HPP affects how bones and teeth develop, making them weaker. The study is testing a new medicine called ALXN1850. Researchers want to see if ALXN1850 is safe and well-tolerated compared to a medicine that's already used for HPP, called asfotase alfa. This study is specifically for children aged 2 to 11 years old who have already been receiving treatment with asfotase alfa. The aim is to understand if this new treatment could be a good option for managing HPP in children, helping them to have stronger bones and healthier development. It's a 'Phase 3' study, which means it’s one of the final steps before a new medicine might become widely available.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Alexion Pharmaceuticals, Inc.

Enrolment target

Start

02 Apr 2024

Estimated completion

29 Feb 2028

Hypophosphatasia

What is this study about?

Hypophosphatasia, often shortened to HPP, is a rare condition that affects how your bones and teeth grow and develop. It can make bones softer and weaker, which can lead to problems like bowed legs, easily broken bones, and issues with teeth. Currently, one of the treatments available for HPP is a medicine called asfotase alfa, which helps to improve bone health.

This study is investigating a new medication called ALXN1850. The main goal is to compare ALXN1850 with asfotase alfa to see if the new medicine is safe and well-tolerated in children who have HPP. Researchers are especially interested in children who are already being treated with asfotase alfa. This comparison will help them understand if ALXN1850 could be another effective treatment option for children with HPP, potentially offering similar or even improved outcomes.

This is a Phase 3 study, which means it’s a big step towards potentially making a new medicine available. In this phase, a larger group of people takes part to confirm the safety and effectiveness seen in earlier studies. The findings from this research will help doctors and patients make informed decisions about the best ways to manage HPP in children.

Key takeaways

The study is investigating a new medicine, ALXN1850, for children with HPP.
It compares ALXN1850 to the commonly used medicine, asfotase alfa.
Children aged 2 to 11 who are already on asfotase alfa might be suitable.
The main goal is to check the safety and how well the new medicine is tolerated.
Participation involves regular clinic visits for checks, injections, and tests.
You can stop participating at any time.

Who may be eligible?

To join this study, children need to be between 2 and 11 years old and have a confirmed diagnosis of HPP. They also need to have 'open growth plates,' which means their bones are still growing, and be at an early stage of puberty (Tanner stage 2 or less).

An important requirement is that they must have been taking asfotase alfa for at least six months before starting this study. Children who weigh less than 10 kilograms (about 22 pounds) cannot take part. Also, if a child has certain other serious health problems, like heart, kidney, or nervous system disorders, or has had a recent broken bone, they unfortunately won't be able to join. If they're allergic to asfotase alfa or ALXN1850, or are planning to have surgery that might affect the study results, they also wouldn't be able to participate.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is your child between 2 and 11 years old?
Does your child have a confirmed diagnosis of HPP?
Has your child been taking asfotase alfa for at least 6 months?
Are your child's bones still growing (have open growth plates)?
Does your child weigh at least 10 kilograms (about 22 lbs)?
Does your child have any other serious ongoing health conditions or recent broken bones?

Answer every question to see your result.

What does participation involve?

If your child takes part, they will receive either the new medicine (ALXN1850) or the existing medicine (asfotase alfa). Both medicines are given by injections under the skin. You and your child will have regular visits to the clinic so doctors can check on their health, give them the study medicine, and perform different tests. These tests might include blood and urine samples, X-rays to look at their bones, and other checks to see how they are growing and developing.

The research team will closely monitor your child’s health and any effects of the medicine. The total length of time your child would be in the study hasn't been specified, but clinical trials usually involve a period of treatment followed by careful follow-up. You will be told the exact schedule of visits and tests if your child is considered for the study.

Potential risks and benefits

Participating in this study might offer your child access to a new experimental medicine for HPP that isn't widely available yet, which could potentially help manage their condition. However, as with any medicine, there could be side effects or risks that are currently unknown or not fully understood. The research team will carefully monitor your child for any problems, but there’s no guarantee that the new medicine will be better than the existing treatment. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their regular medical care.

Locations (21)

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Hartford, United States
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Baltimore, United States
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Minneapolis, United States
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Kansas City, United States
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Durham, United States
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Nashville, United States
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Mar del Plata, Argentina
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South Brisbane, Australia
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Ottawa, Canada
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Bunkyō City, Japan
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Minatoku, Japan
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Suita-shi, Japan

Common questions

What is hypophosphatasia (HPP)?

HPP is a rare condition that affects bone and teeth development, making them weaker and more prone to problems.

What is ALXN1850?

ALXN1850 is a new medicine being tested for HPP to see if it is safe and works well, similar to or better than current treatments.

Who is funding this study?

The study is sponsored by a pharmaceutical company, which is common for research into new medications.

Will my child definitely get the new medicine?

Not necessarily. This study compares the new medicine with the existing one, so your child might receive either ALXN1850 or asfotase alfa.

What if we decide to stop participating?

You can withdraw your child from the study at any time without needing a reason, and it won't affect their ongoing medical care.