A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1
This research is investigating a new medication, ALE1, for adults with a rare genetic bone condition called Hypophosphatasia (HPP). HPP affects how strong bones are. The study has two main parts. First, it will check if ALE1 is safe and how the body handles it in healthy adult volunteers. Then, it will look at how ALE1 works in adults who have HPP. Researchers want to understand how much of the medicine to give and how well it helps. Some people in the study will receive ALE1, while others will receive a dummy medicine called a placebo. This helps scientists compare the effects of the real medicine accurately. It's a key step in developing new treatments for HPP.
At a glance
What is this study about?
This study is exploring a new potential medicine, called ALE1, for a rare inherited condition called Hypophosphatasia (HPP). If you have HPP, your body struggles to make enough of an important chemical called alkaline phosphatase, which is vital for building strong bones and teeth. This can lead to bones that are softer and more easily broken.
The main goal of this study is to find out if ALE1 is safe for people, how much of it the body uses, and what effects it has. The research is being done in two main steps. First, it will involve healthy adult volunteers to check for safety and how the body handles the medicine. This is a very important first step for any new medicine. Once a good safety profile is established, the study will then look at adults who have been diagnosed with HPP.
In both parts of the study, some participants will receive the new medicine ALE1, and others will receive a 'placebo', which looks like the medicine but doesn't contain any active ingredients. This is a standard way to get accurate results and see if the new medicine really makes a difference compared to no treatment. This type of research, known as a Phase 1/2a study, is crucial for understanding new treatments and eventually making them available to people who need them.
Key takeaways
- This study is testing a new medicine (ALE1) for Hypophosphatasia (HPP).
- It will first check safety in healthy adults, then how it works in adults with HPP.
- Some participants will receive ALE1, others a dummy medicine (placebo).
- Participation involves regular clinic visits for checks and tests.
- You can choose to leave the study at any time.
- Ages 18-50, both sexes, with or without HPP (depending on study part).
Who may be eligible?
To join the first part of this study, you need to be a generally healthy adult. This means you don't have any major ongoing health problems or a significant medical history that would concern the study doctors. Essentially, the doctors will check to make sure you're in good overall health.
For the second part of the study, you must be an adult who has been diagnosed with Hypophosphatasia (HPP). This diagnosis needs to be confirmed by a genetic test showing a fault in the ALPL gene, which is known to cause HPP.
There are also some reasons why you might not be able to join. For instance, if you have had other conditions that affect your bones or how your body handles minerals. Also, if you've recently had a bone fracture (within the last 12 weeks), or if you've previously been treated for HPP or other bone-weakening conditions with certain types of advanced medicines, you might not be suitable.
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.
- Are you between 18 and 50 years old?
- If you have HPP, has it been confirmed by a genetic test?
- Do you have any other significant ongoing health problems?
- Have you had a bone fracture in the last 3 months (12 weeks)?
- Have you been treated for HPP before with enzyme replacement therapy or similar advanced medicines?
- Do you have conditions like hyperparathyroidism or hypoparathyroidism (unless it's due to your HPP)?
What does participation involve?
If you decide to take part in this study, you will be assigned to either receive the new medicine, ALE1, or a 'dummy' medicine called a placebo. You won't know which one you are getting, and neither will your study doctor, which helps to keep the results fair and unbiased. The study will involve a number of visits to the clinic, where you'll have medical checks, blood tests, and other assessments to monitor your health and see how the medicine is working. You'll be given instructions on how to take the study medicine. The total length of your participation in the study and the total number of visits will be explained in detail by the study team, and you will have regular follow-up appointments. You can withdraw from the study at any time.
Potential risks and benefits
Locations (2)
- New Zealand Clinical ResearchVerified postcodeGrafton, New Zealand· Recruiting
- Fortrea Clinical Research UnitVerified postcodeLeeds, United Kingdom· Recruiting
Common questions
What is Hypophosphatasia (HPP)?
HPP is a rare genetic condition that affects how your bones and teeth develop, often making them softer and weaker than they should be.
What is ALE1?
ALE1 is a new medicine that researchers are studying to see if it can help people with Hypophosphatasia.
What does 'placebo-controlled' mean?
It means some people get the new medicine, and others get a 'dummy' medicine (placebo) that looks the same but has no active ingredients. This helps researchers compare the real effects.
Why can't I join if I've had certain other bone treatments?
The researchers want to clearly understand the effects of ALE1 on its own, so previous treatments for HPP or bone conditions could make it harder to see the true results.
How long will the study last?
The exact total duration of your participation, including visits and follow-ups, will be fully explained by the study team if you are eligible and interested.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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