Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

ISIS 721744-CS3: An Open-Label Extension Study of ISIS 721744 in Patients with Hereditary Angioedema

This research is an ongoing study for a new medicine, ISIS 721744, specifically for individuals who have Hereditary Angioedema (HAE) and have already taken part in a previous related study. The main goal is to keep an eye on how safe the medicine is by tracking any side effects people might experience. Researchers also want to understand if the medicine helps reduce the number of HAE attacks, how often people need other medicines to treat attacks, and if it improves their overall quality of life. This study is an important step in learning more about this potential new treatment for HAE.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Ionis Pharmaceuticals Inc.

Enrolment target

Start

04 Nov 2024

Hereditary Angioedema

What is this study about?

This study is an extension of previous research into a new investigational medicine called ISIS 721744, which is being developed to help people with Hereditary Angioedema (HAE). HAE is a rare genetic condition that causes unpredictable and often painful swelling in different parts of the body, such as the hands, feet, face, and airway. These swelling attacks can be very hard to live with and can sometimes be dangerous.

The main purpose of this particular study is to continue making sure the new medicine is safe for people to use. Researchers will be carefully monitoring participants for any side effects, big or small, to understand the full safety profile of ISIS 721744 over a longer period. This information is really important for deciding if the medicine should be made widely available in the future.

Beyond safety, the study also aims to learn more about how well ISIS 721744 works. This includes looking at how many HAE attacks people have while taking the medicine, if they need fewer other treatments for sudden attacks, and how their general quality of life changes. Understanding these aspects helps researchers see if the medicine can truly make a positive difference in the lives of people with HAE.

Key takeaways

This study is testing a new medicine for Hereditary Angioedema (HAE).
It's an ongoing study for people who have taken part in a previous trial.
The main goals are to check the medicine's safety and how well it works.
It aims to see if the medicine reduces HAE attacks and improves life quality.
Participation requires being aged 18 or over and having HAE.

Who may be eligible?

To join this study, you must have been part of an earlier trial testing the same medicine, ISIS 721744. This is because this study is designed to continue gathering information from people who have already started treatment.

Participants need to be 18 years old or older. The study is open to both men and women. Your study doctor will check other specific health requirements to make sure it's safe for you to take part.

Quick self-check

I have Hereditary Angioedema (HAE).
I have previously participated in a study of ISIS 721744.
I am 18 years old or older.
I am able and willing to attend regular clinic visits.

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 continue to receive the study medicine, ISIS 721744, as directed by the research team. You will have regular visits to the clinic so the doctors can monitor your health, check for any side effects, and see how the medicine is affecting your HAE. These visits may include physical exams, blood tests, and questionnaires to track your symptoms and how you're feeling. You'll also be asked to keep a record of any HAE attacks you experience and any other medicines you take for them. The total duration of your participation will depend on the study's design and your individual progress, but it allows for ongoing treatment and monitoring.

Potential risks and benefits

Taking part in any clinical trial involves potential benefits and potential risks. A potential benefit could be access to a new treatment for HAE that might reduce your attacks or improve your quality of life, which may not yet be available outside of a study. However, the medicine may not work for everyone, and there's a chance you could experience side effects, some of which might be serious or unexpected. The research team will carefully explain all known risks before you decide to join. Remember, participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

—
Netherlands

Common questions

What is Hereditary Angioedema (HAE)?

HAE is a rare genetic condition that causes sudden and painful swelling in various parts of the body, like the hands, feet, face, or throat. These attacks can be unpredictable and severely impact quality of life.

What is ISIS 721744?

ISIS 721744 is a new medicine being tested as a potential treatment for Hereditary Angioedema. It's an 'investigational' medicine, meaning it's still in development and not yet approved for general use.

Why is this study needed if I've been in a previous one?

This study is an 'extension' of earlier research. It allows doctors to gather more information about the long-term safety and effectiveness of ISIS 721744 in people who have already started taking it.

Will I receive new treatment in this study?

Yes, if you qualify and choose to participate, you will continue to receive the investigational medicine ISIS 721744 as part of the study plan.

Is there a cost to participate?

Generally, clinical trial participation, including study medicine and related appointments, is covered by the study sponsor and should not incur cost to you. Always confirm this with the study team.