Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

Open-Label Pilot Trial to Evaluate the Effects of Ilofotase Alfa on Biomarkers in Adult Patients with Hypophosphatasia

This is an early-stage study testing a new medicine called ilofotase alfa for adults who have hypophosphatasia (HPP). HPP is a rare condition that affects bones and teeth. The main goal is to understand how this medicine affects certain natural substances in the body, called biomarkers, that can tell us more about the condition. We will be looking at two different doses of ilofotase alfa – a low dose and a high dose – to see if and how they change these biomarkers. This is a "Human Pharmacology" study, meaning it's one of the first steps in testing a new medicine in people to learn how it works and what effects it has on the body.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

AM-Pharma B.V.

Enrolment target

Start

02 May 2023

Hypophosphatasia

What is this study about?

You might have come across information about a new research study. This study is for adults living with a rare bone condition called hypophosphatasia, or HPP. HPP stops the body from making enough of a certain enzyme, which is a tiny protein that helps with important body processes, especially for strong bones and teeth. Because HPP is quite rare, finding new ways to help manage it is really important.

The study is testing an experimental medicine called ilofotase alfa. This medicine is designed to help the body in a similar way to the missing enzyme in HPP. Our main goal in this early study is to understand how ilofotase alfa works inside the body, specifically looking at how it changes something called 'biomarkers'. Biomarkers are like internal signals that can tell us about your health and how your body is responding to treatments.

We will be giving adult patients with HPP either a low dose or a high dose of ilofotase alfa. By doing this, we can compare the effects of different amounts of the medicine on these important biomarkers. This type of study is often one of the first steps in developing a new medicine, helping us learn key information about its safety and how it affects the body.

Key takeaways

This study is for adults with hypophosphatasia (HPP).
It's testing a new medicine called ilofotase alfa.
The main aim is to see how the medicine affects body markers (biomarkers).
Both low and high doses of the medicine will be studied.
It's an early-stage study to learn more about the medicine's effects.
Participation is voluntary, and you can withdraw anytime.

Who may be eligible?

To be considered for this study, you need to be an adult, meaning you are 18 years old or older. There is no upper age limit for taking part.

Both men and women are welcome to participate in this research. The study needs people from all backgrounds to make sure the results are as widely applicable as possible.

Beyond age and sex, there will be other specific health checks and medical history questions to make sure the study is a good fit for you and that taking part would be safe. These details would be discussed with you by the study team.

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?
Do you have hypophosphatasia (HPP)?
Are you able to attend all required clinic visits?
Are you willing to have blood tests as part of the study?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would first have a screening visit to check if you're suitable. Once confirmed, you would receive the study medicine, ilofotase alfa, which would likely be given as an injection. The study team would explain exactly how and when you would receive it.

You would have several follow-up visits to the clinic. During these visits, doctors and nurses would take blood samples, measure your vital signs (like blood pressure and heart rate), and ask you how you are feeling. These tests help us understand how your body is reacting to the medicine and if there are any changes in the biomarkers we are interested in. The total duration of your participation, including all visits and follow-ups, would be explained in full by the study team.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit of this study could be that you receive a new medicine that might help with your hypophosphatasia, though this is not guaranteed as it's an early-stage trial. We hope to gain valuable information that could lead to new treatments for HPP. Potential risks could include side effects from the study medicine, or discomfort from procedures like blood tests. You will be closely monitored for any adverse reactions. Remember, taking part is completely your choice, and you have the right to withdraw from 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 condition that affects how your bones and teeth develop and stay strong.

What is ilofotase alfa?

It's a new, experimental medicine being tested in this study for HPP.

What does 'biomarkers' mean?

Biomarkers are natural substances in your body that can show how your body is working and reacting to treatments.

Is this medicine approved for HPP?

No, it's an experimental medicine, meaning it's still being tested to see if it's safe and effective.

Will I get the actual medicine or a dummy medicine?

This study is giving everyone the active medicine, ilofotase alfa, at either a low or high dose.