CompletedPhase IInterventional

A study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of BCX10013 in healthy participants

This research is looking at a new medicine called BCX10013, taken by mouth, that has never been given to people before. It works by blocking a specific part of the body's defence system, called complement Factor D, which helps fight infections. The main goal of this 'Phase 1' study is to find out if BCX10013 is safe and well-tolerated when healthy volunteers take single or multiple doses. It will also help us understand how the medicine moves through the body and what effects it has. The study is run in the UK and Netherlands and aims to collect important information that might help patients in the future.

At a glance

Status

Completed

Phase

Phase I

Sponsor

BioCryst Pharmaceuticals (United States)

Enrolment target

166

Start

23 Aug 2021

Estimated completion

25 Aug 2023

Healthy participants

What is this study about?

This study is about a brand-new medicine called BCX10013. It's an oral medicine, meaning you take it by mouth, and it's being tested in people for the very first time. The main purpose of this early-stage research is to understand if BCX10013 is safe for people to take, how their bodies handle it, and what effects it has. Researchers hope this information might eventually help people with certain health conditions in the future.

BCX10013 works by targeting a specific part of your body's immune system, which is your natural defence against illness. This part is called the 'complement system', and it helps fight off infections and clear away damaged cells. BCX10013 aims to block a particular protein within this system, called Factor D. By doing this, it might help control certain diseases where this immune pathway is overactive.

Because this is the first time humans are taking BCX10013, the study focuses on healthy volunteers. This allows researchers to carefully monitor the medicine's effects without the complexities of an existing illness. The information gathered here is crucial for deciding if the medicine can be tested further in people who actually have the conditions it aims to treat.

Key takeaways

New medicine BCX10013 is being tested in people for the first time.
The study focuses on safety, how the body uses the medicine, and its effects on the immune system.
Only healthy volunteers aged 18-55 can participate.
Participation involves screening, taking medicine (or a placebo), and regular check-ups.
There are no direct personal health benefits, but the study could help future patients.
Potential risks include side effects, which will be fully explained before you agree to take part.

Who may be eligible?

To join this study, you need to be a healthy man or woman between 18 and 55 years old. Your body mass index (BMI) should be between 18 and 32. Your kidneys need to be working well, and you must be able to give your written permission to take part.

There are also some important rules about contraception. If you are a woman who could get pregnant, or a man with a female partner who could get pregnant, you must agree to use effective contraception from when you start the study until 30 days after you finish.

People will not be able to join if they have any significant long-term health problems, mental health conditions that would make taking part difficult, or certain recent infections. For some parts of the study, you also need to have received a specific vaccination for meningitis (Neisseria meningitidis infection), or for one specific group, be a first-generation Japanese participant.

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 55 years old?
Are you generally healthy with no significant long-term medical conditions?
Do you have a Body Mass Index (BMI) between 18 and 32?
Are you willing and able to use effective contraception if you or your partner could become pregnant?
Are you able to provide written consent to participate?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will first have a screening visit about a month before the study medicine starts, to check if you are suitable. The study has four main parts, and what you do depends on which part you are in.

Some groups will take a single dose of BCX10013 or a dummy medicine (placebo). Others will take the medicine daily for a longer period, from 7 up to 28 days. One group will take a single dose twice, once after not eating and once after a high-fat meal, to see how food affects the medicine. Another group will take two single doses of BCX10013, in a switched order with a week's break in between.

Throughout these periods, you'll have visits for check-ups, blood tests, and other assessments to see how you are doing and how the medicine is working. The study is expected to run from June 2021 to October 2023.

Potential risks and benefits

You won't directly benefit from taking part in this study, though you will receive a meningitis vaccine for certain parts of the study. However, the information gathered could be very helpful in developing new treatments for others in the future. As with any new medicine, there might be unknown risks or side effects, and some known potential effects include severe reactions, infections, kidney or liver problems, or changes in blood cell counts. You will receive a detailed document explaining all the known and potential risks and what to expect. You are free to withdraw from the study at any time.

Locations (2)

Quotient Sciences
Approximate
Nottingham, United Kingdom
ICON
City only
Groningen, Netherlands

Common questions

What is BCX10013?

BCX10013 is a new oral medicine being tested for the first time in people. It aims to block a specific part of the body's immune system.

Why is this study only for healthy people?

Because this is the first time BCX10013 is given to people, testing it in healthy volunteers helps researchers safely understand how the medicine works and if it has any side effects without other health conditions making things complicated.

Will I get the actual medicine or a dummy pill?

Some participants will receive BCX10013, and others will receive a dummy pill (placebo). This helps researchers compare the effects accurately.

Will I be paid to participate?

The information provided does not specify payment. This is a good question to ask the study team if you are interested in taking part.

How long will I be involved in the study?

The duration of your involvement depends on which part of the study you participate in, ranging from single doses with follow-up to taking medicine daily for up to 28 days.

How to find out more

Data Disclosure -

datadisclosure@biocryst.com - Official trial record

Always speak to your GP or specialist before deciding to take part in a study.