RecruitingPHASE1INTERVENTIONAL

Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Male and Female Subjects and Safety, Tolerability, Pharmacokinetics, and Pilot Efficacy Biomarkers in Subjects With Cold Agglutinin Disease

This study is testing a new drug called GL-0719 for the very first time in people. Initially, it will check how safe the drug is, what side effects it might have, and how your body uses and gets rid of it. This will be done in healthy volunteers. The medicine can be given either into a vein or under the skin. After that, the study will also look at how safe GL-0719 is and how it works in people who have a rare blood disorder called cold agglutinin disease (CAD). For people with CAD, the study will also explore early signs of how well the drug might be working.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Gliknik Inc.

Enrolment target

Start

08 Apr 2022

Estimated completion

30 Jun 2026

First in Man Study to Evaluate Initial Safety

What is this study about?

This study is about a new investigational medicine called GL-0719. It's an important first step because it's the very first time this drug will be given to people. The main goal is to understand if GL-0719 is safe, what side effects it might cause, and how your body processes it. This helps researchers work out the right dose and how often it needs to be given.

Initially, the study will look at healthy adults to gather this safety information. After that, the study will involve people who have a rare blood condition called cold agglutinin disease (CAD). CAD is a condition where your immune system mistakenly attacks and destroys your red blood cells, especially when you're cold. For people with CAD, the study will continue to check safety, but it will also look for early signs that the medicine might be helping their condition.

By taking part, you would be helping doctors and scientists learn more about this new medicine. This is a crucial step in developing potential new treatments that could one day help many people.

Key takeaways

This is a first-time study of a new medicine, GL-0719, in people.
It aims to check the safety and how the body handles the drug.
The study includes healthy volunteers and people with cold agglutinin disease (CAD).
GL-0719 might be given into a vein or under the skin.
You'll need to attend regular clinic visits for monitoring.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To join the first part of this study (for healthy volunteers), you need to be a healthy man or woman between 18 and 65 years old. Your weight and height need to be within a certain range. Women must not be pregnant or breastfeeding, and all participants capable of having children must agree to use contraception during the study. You also need to have had a specific meningitis vaccination in the last five years, or get one at least 14 days before starting the study drug.

For the second part of the study, for people with cold agglutinin disease (CAD), you need to be at least 18 years old and weigh at least 50 kg. Similar to the healthy volunteers, women must not be pregnant or breastfeeding, and contraception is required. You will also need to have a confirmed diagnosis of primary CAD or CAD linked to another blood condition, and your blood test results for haemoglobin and bilirubin will need to be within a specific range.

For both groups, it's really important that you can understand what the study involves, including any possible risks and benefits, and give your permission to take part.

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 65 years old (for healthy volunteers)?
Are you at least 18 years old and weigh at least 50 kg (for CAD patients)?
Are you able to use contraception if you could become pregnant or make someone pregnant?
If healthy, is your weight within a healthy range?
Have you had a meningitis vaccine in the last 5 years, or are you willing to get one?
If you have CAD, is your diagnosis confirmed, and are your blood tests within specific limits?

Answer every question to see your result.

What does participation involve?

If you take part in this study, you will receive the new medicine, GL-0719, or a placebo (a dummy drug with no active medicine) for some healthy volunteers. The medicine might be given into a vein (intravenously) or under the skin (subcutaneously). You'll have several visits to the study centre, where the study team will monitor your health closely, take blood samples, and check for any side effects. You'll also need to return for follow-up appointments to make sure you're recovering well and to continue monitoring your health. The total duration of your involvement will depend on which part of the study you are in, but the team will explain this fully beforehand.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of this study is helping researchers learn more about GL-0719, which could lead to new treatments in the future. For patients with CAD, there's a small chance you could benefit from the drug itself. However, there are also potential risks, such as side effects from the study drug, discomfort from injections or blood tests, or unforeseen reactions. The study team will explain all known risks to you. You have the right to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (1)

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

Fortrea Clinical Research Unit Ltd
Verified postcode
Leeds, United Kingdom· Recruiting

Common questions

What is cold agglutinin disease (CAD)?

CAD is a rare condition where your body's immune system attacks your red blood cells, especially in cold temperatures, causing them to break down too quickly.

What does 'first-in-human (FIH) study' mean?

It means this is the very first time this new medicine is being tested in people. The main goal is to check its safety.

Why do some people get a 'placebo'?

In some parts of the study, a placebo (a dummy drug) is used to compare with the active medicine. This helps researchers understand if any effects are truly from the medicine or just a coincidence.

What is an 'investigational medicine'?

It's a medicine that hasn't been approved for general use yet. It's still being tested to see if it's safe and effective.

Do I have to pay to join the study?

No, you do not have to pay to join the study. All study-related care and medication are provided free of charge.

How to find out more

Gliknik Clinical Trials Group

gliknikclinicaltrialinquiries@gliknik.com 410-665-0662 Official trial record

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