Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A MULTICENTER, OPEN-LABEL, OUTPATIENT STUDY TO EVALUATE THE SAFE AND EFFECTIVE USE OF A ZILUCOPLAN AUTO INJECTOR COMBINATION PRODUCT FOR SUBCUTANEOUS SELF-ADMINISTRATION BY STUDY PARTICIPANTS WITH GENERALIZED MYASTHENIA GRAVIS

This study is for people in the UK who have a muscle condition called generalized myasthenia gravis. Researchers are looking into a new way for patients to give themselves a medication called zilucoplan, using an auto-injector device. The main goal is to see how well patients can use this device to inject the medicine themselves, checking if it's easy and effective. The study will also carefully monitor any side effects or problems with the device to make sure it's safe. This is a later-stage study (Phase 3), meaning the medication has already shown promise, and now the focus is on confirming its safety and how well people can use this new delivery method at home.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

UCB Biopharma

Enrolment target

Start

15 Jul 2024

Generalized Myasthenia Gravis

What is this study about?

This research study is looking at a new way for people living with a condition called generalized myasthenia gravis to take their medication. Myasthenia gravis is a long-term condition that causes muscle weakness. In this study, the medication being tested is called zilucoplan. Instead of receiving this medicine in a clinic, the study wants to see how well patients can give themselves the medicine at home using a special pre-filled injection device, similar to an EpiPen.

The main purpose of the study is to check if patients can successfully inject themselves with zilucoplan using this auto-injector device throughout the study period. This is important because being able to give yourself medication at home can make managing your condition much easier and more convenient. The study will also be keeping a very close eye on any side effects, whether they are minor or serious, and any issues or problems that might happen specifically with the injection device itself.

This is a 'Phase 3' study, which means the medication has already been tested in earlier stages and shown to be promising. In this phase, the focus is on confirming its safety and effectiveness on a larger group of people, and in this case, specifically how well patients manage using the auto-injector for self-administration. The information gathered from this study will help ensure that if zilucoplan becomes widely available, it can be used safely and easily by patients at home.

Key takeaways

This study evaluates a new way for GMG patients to self-inject zilucoplan.
The main goal is to check if the auto-injector device is easy and effective for home use.
Safety and any device-related issues will be carefully monitored.
It's a Phase 3 study, meaning the treatment has already shown promise.
Participants must be 18 years or older with generalized myasthenia gravis.

Who may be eligible?

To be considered for this study, you need to be an adult, aged 18 years or older. There is no upper age limit, meaning older adults are also welcome to participate.

This study is open to both men and women who have been diagnosed with generalized myasthenia gravis.

There will be other specific health requirements that your doctor will discuss with you to make sure the study is a safe and suitable option for your individual situation.

Quick self-check

Are you 18 years old or older?
Do you have a diagnosis of generalized myasthenia gravis?
Are you able to attend regular clinic visits as required?
Are you willing and able to try giving yourself injections at home?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you will be asked to administer the zilucoplan medication to yourself using an auto-injector device. You will have regular visits to the clinic, which are referred to as Visit 1 to Visit 8. At these visits, the study team will check how well you are using the auto-injector and monitor your health.

The study team will assess if you are successfully giving yourself the injections and will also record any side effects you experience, whether they are minor or more serious. They will also keep track of any issues specifically related to the auto-injector device. The total duration of your participation in the study, including all visits and follow-ups, will depend on the study schedule, which your doctor will explain in detail.

Potential risks and benefits

Taking part in this study could offer several potential benefits, such as gaining early access to a new treatment and playing an active role in managing your health. It might also help improve the way future treatments are delivered for others. However, like all medical studies, there are potential risks, including possible side effects from the medication or issues with the auto-injector device. You will be closely monitored for any problems. You always have the right to withdraw from the study at any time, for any reason, without affecting your standard medical care.

Locations (1)

—
Poland

Common questions

What is generalized myasthenia gravis?

It's a long-term condition that causes weakness in different muscles of the body, making daily activities difficult.

What is an auto-injector?

It's a pre-filled device that allows you to give yourself an injection easily, often by just pushing a button, similar to how some people use EpiPens.

What does 'self-administration' mean?

It means you will be giving yourself the medication at home, rather than needing a healthcare professional to do it.

What does Phase 3 mean for a study?

It means the treatment has already shown promise in earlier tests, and now researchers are looking at its safety and how well it works on a larger group of people, often specifically focusing on how it will be used in real life.

Will I get to keep using the medication after the study?

This study is focused on the research period. Your doctor will discuss what options might be available to you regarding your treatment after the study concludes.