AuthorisedPhase I and Phase II (Integrated)- First administration to humansInterventional

A Randomised, Placebo-Controlled, Double-Blind, Single-Ascending Dose and Multiple-Ascending Dose First-In-Human Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Orally Administered ALE1 With or Without Food in Healthy Adult Participants and Adult Patients With Hypophosphatasia

This research is looking into a new medicine called ALE1. It's designed for a condition called Hypophosphatasia (HPP). We want to understand if it's safe and how well people tolerate it, both in healthy volunteers and in patients living with HPP. The study starts with very small doses of the medicine and gradually increases them, carefully watching for any side effects. We are also looking at how the body absorbs, uses, and gets rid of ALE1, and whether eating food affects how it works. This is one of the first times this medicine is being given to people.

At a glance

Status

Authorised

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Alesta Therapeutics B.V.

Enrolment target

Start

19 Nov 2025

Hypophosphatasia

What is this study about?

This study is about a new medicine called ALE1. It's being developed to help people with a rare condition called Hypophosphatasia (HPP). HPP affects bones and teeth, making them weaker.

The main goal of this research is to see if ALE1 is safe for people to take and to check how well their bodies cope with it. We will be giving the medicine in different amounts, starting with very small doses, to both healthy adults and adults who have HPP. We’ll be looking very closely for any unwanted effects.

We also want to understand what happens to ALE1 inside the body. This includes how much of the medicine gets into the bloodstream, how long it stays there, and whether having food with it changes how the body handles it. This information is important because it helps doctors figure out the right dose for future use. This is a very early stage study, meaning it's one of the first times this medicine is being tested in humans.

Key takeaways

New medicine (ALE1) for Hypophosphatasia (HPP) is being tested.
Study is looking at safety and how the body handles the medicine.
Involves both healthy adult volunteers and adults with HPP.
Participants will receive either ALE1 or a dummy medicine (placebo).
Close monitoring for any side effects.
Early stage research (first time in humans).

Who may be eligible?

To join this study, you need to be an adult, aged 18 years or older. There is no upper age limit, meaning older adults can also take part.

Both men and women are welcome to participate in this research. The study needs both healthy volunteers and people who have been diagnosed with Hypophosphatasia (HPP).

More specific details about your health, any other medicines you take, or other medical conditions will be discussed with the study team to make sure this study is right for you.

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 18 years old or older?
Are you willing to potentially receive a dummy medicine?
Are you able to attend regular clinic visits?
Do you have Hypophosphatasia, or are you a healthy adult?
Are you able to take medicine by mouth?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will be given the study medicine, ALE1, or a placebo (a dummy medicine that looks the same but has no active drug). You won't know if you're getting ALE1 or the placebo, and neither will the study team. You will take the medicine by mouth.

During the study, you'll have several visits to the clinic. At these visits, doctors and nurses will regularly check your health, collect blood samples, and ask you specific questions to see how you're feeling and whether you're experiencing any effects from the medicine. Some visits might involve staying at the clinic for a short period.

The total amount of time you spend in the study will depend on whether you are taking single doses or multiple doses of the medicine, and this will be explained fully before you join.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. You might not directly benefit from taking part in this early study; however, the information we learn could help people with Hypophosphatasia in the future. As with any new medicine, there's always a chance of experiencing side effects, some of which might not be known yet. The study team will monitor you closely for any unwanted effects and will explain all known risks before you decide to join. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Germany

Common questions

What is Hypophosphatasia (HPP)?

HPP is a rare genetic condition that affects how bones and teeth develop, making them weaker and more prone to problems.

What is a 'new medicine' or 'investigational drug'?

It's a medicine that scientists are still studying. It hasn't been fully approved for everyday use yet, and we are still learning about how it works.

What does 'placebo-controlled' mean?

It means some participants will receive the active medicine, while others will receive a dummy medicine (placebo) that looks identical but contains no drug. This helps us see the real effect of the new medicine.

What does 'double-blind' mean?

It means neither you nor the doctors and nurses involved in your care will know whether you are receiving the active medicine or the placebo. This helps prevent bias in the results.

What is the 'first-in-human' part of the study?

This means it's one of the first times this new medicine is being given to people, after extensive testing in laboratories and animals. It allows us to carefully check its safety in humans.