Active not recruitingPHASE3INTERVENTIONAL

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

This research study is testing a new medicine called donidalorsen for people with Hereditary Angioedema (HAE). HAE is a condition that causes sudden, painful swelling attacks. The main goal of this study is to check if donidalorsen is safe for long-term use and how well it works to prevent these attacks. Researchers also want to understand if the medicine improves the day-to-day lives of people with HAE. The study involves two groups of participants: those who have been on donidalorsen in a previous study and new participants who have been taking other preventive HAE medicines. Participants will be in the study for up to about 76 weeks, with an option to continue treatment for an additional two years.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Ionis Pharmaceuticals, Inc.

Enrolment target

154

Start

13 Jul 2022

Estimated completion

01 Mar 2027

Hereditary Angioedema

What is this study about?

This study is about a new medicine called donidalorsen, which is being tested as a way to prevent attacks for people living with Hereditary Angioedema (HAE). HAE is a rare genetic condition that causes unpredictable and often painful episodes of severe swelling in different parts of the body, like the face, hands, feet, stomach, or airways. These attacks can be very disruptive and sometimes life-threatening.

The main aim of this research is to find out if donidalorsen is safe and effective when used for a longer period. This means understanding if it helps reduce the number of swelling attacks and if it makes a positive difference to people's quality of life – how they feel physically and mentally, and how well they can carry out daily activities. Finding better ways to prevent HAE attacks could significantly improve the lives of those affected.

This is a 'Phase 3' study, which means the medicine has already been through earlier tests and shown promise. It's an 'open-label' study, so both you and the study doctors will know you are receiving donidalorsen. There are two main groups in the study: people who have already used donidalorsen in an earlier study, and new volunteers who have been managing their HAE with other preventive medicines like lanadelumab, berotralstat, or C1-esterase inhibitor treatments.

Key takeaways

This study is testing a new medicine, donidalorsen, for long-term HAE prevention.
It aims to check safety, reduce HAE attacks, and improve quality of life.
Participants include those previously on donidalorsen and those on other preventive HAE medicines.
Participation involves regular clinic visits and tracking HAE attacks.
The study duration is about 15-18 months, with an option for 2 more years of treatment.
Volunteers can stop participating at any time.

Who may be eligible?

For this study, you must be at least 12 years old and have a confirmed diagnosis of Hereditary Angioedema (either Type 1 or Type 2). You also need to have access to and be able to use your emergency medication for HAE attacks if they occur, such as plasma-derived C1-inhibitor or a bradykinin receptor blocker.

If you've been part of a previous donidalorsen study, you can join if you completed it successfully and tolerated the treatment well. If you are new to donidalorsen, you must have been on a stable dose of another preventive HAE medication (like lanadelumab, berotralstat, or C1-esterase inhibitor) for at least 12 weeks before starting this study.

There are also some reasons why you might not be able to join. For example, if you have other types of angioedema (like acquired angioedema), a recent history of certain cancers, or if you're allergic to donidalorsen or its ingredients. Your study doctor will review all your health information to confirm if this study is right for you.

Quick self-check

Are you 12 years old or older?
Do you have a documented diagnosis of Hereditary Angioedema (Type 1 or Type 2)?
Do you have access to medicine for acute HAE attacks?
If you haven't been in a donidalorsen study before, have you been on a stable dose of another preventive HAE medicine for at least 12 weeks?
Do you have any other conditions or allergies that might prevent you from joining (your doctor will confirm this)?

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

What does participation involve?

If you decide to take part in this study, you will be given the study medicine, donidalorsen. For people new to donidalorsen, the main study period will last about 76 weeks (around 1 year and 5 months). If you were in a previous donidalorsen study, your main participation will be about 70 weeks. After this initial period, you might be able to continue receiving donidalorsen in an extended treatment phase for up to an additional 104 weeks (about 2 years).

During your time in the study, you will have regular visits to the study clinic. These visits will involve health check-ups, blood tests, and discussions about how you are feeling and if you've had any HAE attacks. You will also be asked to keep track of your HAE attacks and any emergency medicine you use. The study team will explain all the details and answer any questions you have about the visits and assessments.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of this study is that donidalorsen might help reduce your HAE attacks and improve your quality of life. However, it's also possible that the medicine may not work for you, or you might experience side effects. The study team will closely monitor your health, and they will fully explain all known and possible risks before you agree to participate. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (50)

Ionis Investigative Site
Paradise Valley, United States
Ionis Investigative Site
La Jolla, United States
Ionis Investigative Site
Santa Monica, United States
Ionis Investigative Site
Walnut Creek, United States
Ionis Investigative Site
Tampa, United States
Ionis Investigative Site
Kansas City, United States
Ionis Investigative Site
Boston, United States
Ionis Investigative Site
Ann Arbor, United States
Ionis Investigative Site
Detroit, United States
Ionis Investigative Site
St Louis, United States
Ionis Investigative Site
Mooresville, United States
Ionis Investigative Site
Cincinnati, United States

+38 more sites — see the official record for the full list.

Common questions

What is Hereditary Angioedema (HAE)?

HAE is a rare genetic condition that causes sudden, unpredictable swelling attacks in different parts of the body.

What is donidalorsen?

Donidalorsen is the new medicine being tested in this study to see if it can help prevent HAE attacks and improve life for people with HAE.

Who can join this study?

You might be able to join if you are 12 years or older, have a confirmed HAE diagnosis, and use an acute HAE medicine. There are also specific criteria depending on previous treatment.

Will I know if I'm getting the study medicine?

Yes, this is an 'open-label' study, which means both you and the study team will know you are receiving donidalorsen.

How long will I be in the study?

The main study lasts around 70-76 weeks, with an option to continue treatment for up to an additional 2 years.