Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
This trial is for children aged 2 to 11 who have a rare genetic bone condition called Hypophosphatasia (HPP) and haven't been treated with a specific medicine before. The main aim is to find out if a new medicine, ALXN1850, can help improve bone health better than a placebo (a dummy medicine with no active ingredients). Researchers will check X-rays to see the medicine's effects. Half of the children will receive the new medicine and the other half will get the placebo. This study is important for understanding new potential ways to treat HPP in children.
At a glance
What is this study about?
This study is for children who have a rare bone condition called Hypophosphatasia, often shortened to HPP. HPP makes bones weaker and can cause problems with how children grow. If your child has HPP and hasn't been treated with a medicine called asfotase alfa before, then this study might be relevant.
The main goal of the study is to test a new medicine, ALXN1850. The researchers want to see if this new medicine can help children's bones get stronger compared to a 'placebo', which looks exactly like the real medicine but has no active ingredients. They will look at X-rays to measure any improvements in bone health over time. This type of research, where some people get the new medicine and others get a placebo, helps doctors understand if the medicine genuinely works.
Taking part in a study like this helps doctors learn more about HPP and find better ways to treat it in children. It's a way for families to contribute to medical science while potentially getting access to a new treatment. All studies are carefully planned and monitored to ensure the safety and well-being of the participants.
Key takeaways
- Targets children aged 2-11 with Hypophosphatasia (HPP).
- Tests a new medicine, ALXN1850, against a placebo.
- Main goal is to see if it improves bone health on X-rays.
- Children must not have been previously treated with asfotase alfa.
- Participation involves regular clinic visits for assessments.
- Helps advance understanding and treatment of HPP.
Who may be eligible?
This study is looking for children between 2 and 11 years old. To be considered, your child must have a clear diagnosis of Hypophosphatasia (HPP) and show signs of it, like changes on bone X-rays and specific lower-than-normal levels of a substance in their blood called alkaline phosphatase (ALP).
Additionally, there needs to be genetic evidence of HPP or high levels of another substance called PLP in their blood. Your child must also be in early stages of puberty (Tanner stage 2 or less). This helps ensure that the study results are clear and not affected by changes that happen during puberty.
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 my child between 2 and 11 years old?
- Does my child have a confirmed diagnosis of HPP?
- Do their X-rays show signs of HPP affecting their bones?
- Are their blood tests showing typical HPP markers?
- Has my child not been treated with asfotase alfa before?
- Is my child in the early stages of puberty (Tanner stage 2 or less)?
What does participation involve?
If your child joins this study, they will either receive the new medicine, ALXN1850, or a placebo (a dummy medicine). Which one they receive is decided by chance, like flipping a coin. You won't know which one they are getting, nor will the study doctors. They will have regular visits to the clinic for check-ups, blood tests, and X-rays to see how their bones are doing. The doctors will monitor their health closely throughout the study. The total length of the study will be explained by the study team, and you'll know upfront how long your child would be involved.
Potential risks and benefits
Locations (32)
- Research SiteVerified postcodeBaltimore, United States
- Research SiteVerified postcodeKansas City, United States
- Research SiteVerified postcodeDurham, United States
- Research SiteVerified postcodeNedlands, Australia
- Research SiteVerified postcodeParkville, Australia
- Research SiteVerified postcodeBrussels, Belgium
- Research SiteVerified postcodeBrasília, Brazil
- Research SiteVerified postcodePorto Alegre, Brazil
- Research SiteVerified postcodeRecife, Brazil
- Research SiteVerified postcodeSalvador, Brazil
- Research SiteVerified postcodeSão Paulo, Brazil
- Research SiteVerified postcodeSão Paulo, Brazil
Common questions
What is Hypophosphatasia (HPP)?
HPP is a rare genetic condition that affects a child's bones, making them softer and weaker than they should be.
What is 'Phase 3' of a study?
Phase 3 means the new medicine has already been studied in smaller groups and is now being tested in a larger group of people to confirm its safety and effectiveness.
What does 'placebo' mean?
A placebo is a dummy treatment. It looks like the real medicine but contains no active ingredients. It helps doctors see if the new medicine truly works better than nothing.
Will my child know if they are getting the real medicine or the placebo?
No, neither you nor the study doctors will know whether your child is receiving the active medicine or the placebo. This helps make the study results as fair and accurate as possible.
Can I take my child out of the study once it starts?
Yes, you have the right to withdraw your child from the study at any time, for any reason, without it affecting their medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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