Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

A PHASE I, OPEN-LABEL, RANDOMIZED, ADAPTIVE, CROSS-OVER STUDY TO DETERMINE THE RELATIVE BIOAVAILABILITY OF NIZUBAGLUSTAT CAPSULES COMPARED TO ORALLY DISPERSIBLE TABLETS AND THE FOOD EFFECT IN HEALTHY VOLUNTEERS

This study is trying to understand how a new medicine called nizubaglustat gets into the body. We're comparing two ways to take it: as a regular capsule or as a tablet that dissolves in your mouth. We also want to see if eating food before or after taking the medicine changes how it's absorbed. This research involves healthy volunteers and is an early-stage study, meaning we're mainly focused on how the medicine behaves in the body. The goal is to find the best way for people to take this medicine, especially for conditions called glycosphingolipid lysosomal storage disorders.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Azafaros B.V.

Enrolment target

Start

27 Feb 2024

Glycosphingolipid lysosomal storage disorders

What is this study about?

This study is about a new medicine called nizubaglustat. Think of it like comparing different ways to get fuel into a car – does it work better as a liquid or a solid? In this case, we're looking at whether this new medicine is better absorbed as a capsule or as a special tablet that dissolves in your mouth. Getting the medicine absorbed well is really important so it can do its job in the body.

We're also checking if what you eat affects how the medicine gets into your system. Sometimes, food can help a medicine get absorbed, and other times it can stop it from working as well. Understanding this helps doctors know the best way to tell patients to take their medicine – like whether to take it with food or on an empty stomach.

This early-stage study is a bit like a test drive. We're using healthy volunteers to learn how the medicine acts in the body before it's given to people who are unwell. This helps us gather important information safely and figure out the best way to use this potential new treatment for certain genetic conditions called glycosphingolipid lysosomal storage disorders.

Key takeaways

This study is testing a new medicine called nizubaglustat.
It compares how well capsules and dissolving tablets are absorbed.
It also checks if food affects how the medicine works.
Only healthy adults (18+) can take part.
The goal is to find the best way to give the medicine for rare genetic conditions.
It's an early-stage study to understand the medicine's behaviour in the body.

Who may be eligible?

To join this study, you need to be a healthy adult, aged 18 years or older. This means you generally don't have any major ongoing health problems.

Both men and women can take part in this research. The study team will check your health carefully to make sure it's safe for you 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.

Are you 18 years old or older?
Do you consider yourself generally healthy?
Are you comfortable having regular health checks and blood tests?
Are you able to attend several clinic visits?
Are you not currently taking other strong medications that might interfere with a study drug?

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve several visits to a clinic. You would receive doses of the study medicine, nizubaglustat, in different forms (capsules and dissolving tablets) and sometimes with or without food. You would likely have blood tests taken at different times after taking the medicine to see how it's absorbed. There will also be health checks and monitoring throughout your participation to ensure you are well. The total duration of your involvement, including all visits and follow-up, will be explained fully by the study team.

Potential risks and benefits

Potential benefits of taking part include contributing to medical science and helping develop new treatments for rare genetic conditions. However, there is no direct personal health benefit for you as a healthy volunteer. Potential risks could include side effects from the medication, discomfort from blood tests, or unforeseen reactions. All known risks will be explained in detail, and you have the right to withdraw from the study at any time without giving a reason.

Locations (1)

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

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Unverified
Netherlands

Common questions

What is nizubaglustat?

Nizubaglustat is a new medicine being studied. This research focuses on how your body handles it.

Why is this study only for healthy volunteers?

Using healthy volunteers in early studies helps us understand how the medicine works in the body without other illnesses making it complicated. It's a safer first step.

What does 'orally dispersible tablets' mean?

These are tablets that dissolve quickly in your mouth, which can sometimes make them easier to take than swallowing a pill.

What are 'glycosphingolipid lysosomal storage disorders'?

These are rare, serious genetic conditions where the body can't properly break down certain fats, leading to health problems. This new medicine aims to help treat these.

Will I get paid for taking part?

The study information will confirm if you will be reimbursed for your time and travel expenses. This is common in early-stage studies.