Active not recruitingPHASE3INTERVENTIONAL

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

This important study, called HAELO, is looking for people with Hereditary Angioedema (HAE) to test a new medicine named NTLA-2002. HAE is a condition that causes swelling attacks. The main goal is to find out if NTLA-2002 can effectively prevent these attacks and if it's safe to use. Some participants will receive the new medicine, while others will get a placebo (a treatment with no active drug) to help doctors understand the true effects of NTLA-2002. This study is ‘double-blind,’ meaning neither you nor your study doctor will know which treatment you are receiving at first. This helps ensure fair results. The study will involve a single infusion of the medicine and then careful monitoring over a long period.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Intellia Therapeutics

Enrolment target

Start

15 Jan 2025

Estimated completion

01 Sep 2027

Hereditary Angioedema

What is this study about?

This study, called HAELO, is designed to understand if a new medicine, NTLA-2002, can effectively help people who have a condition called Hereditary Angioedema (HAE). HAE causes sudden, unpredictable swelling attacks in different parts of the body, which can be very painful and even dangerous. Scientists believe NTLA-2002 might be able to reduce how often and how severe these attacks are.

The study aims to compare NTLA-2002 with a placebo. A placebo is like a dummy treatment that doesn't contain any active medicine. This comparison is important because it helps researchers work out if any changes in your HAE attacks are truly due to the new medicine or if they might happen anyway. About 60 people with HAE will take part in this study across different countries.

After an initial period, if you started on the dummy medicine, you'll have the option to receive the actual study medicine, and vice-versa. Everyone involved will be monitored for quite a long time – over two years in total – to carefully check both the effects of the medicine and how safe it is. This careful and thorough approach helps ensure that any new treatment is well understood before it becomes widely available.

Key takeaways

This study is testing a new medicine (NTLA-2002) for Hereditary Angioedema (HAE).
It aims to see if NTLA-2002 can reduce HAE attacks and if it's safe.
Participants will receive a single infusion – either the new medicine or a placebo.
The study lasts over two years with regular check-ups.
It's 'double-blind,' meaning you won't know which treatment you get at first.
Only people aged 16 and over with specific types of HAE can join.

Who may be eligible?

To join this study, you need to be at least 16 years old and have a confirmed diagnosis of Hereditary Angioedema (HAE) – specifically Type 1 or Type 2. You’ll also need to be able to show your study doctor that you've been having HAE attacks that meet the study's requirements. It's important that you are able to take your usual emergency medicines if you have an HAE attack during the study, and your doctor must agree it's safe for you to stop your regular long-term preventative HAE medicines for the first few months of the study.

There are a few reasons why you might not be able to join. For example, if you have another type of swelling condition that isn't HAE, or if you've had a known bad reaction to certain fat-based particles (called lipid nanoparticles) that are used in the study medicine. Also, if your doctor believes that taking part in the study might not be safe for you because of another health condition, or if you can't commit to following all the study steps, you wouldn't be able to participate.

All participants must also agree not to join any other studies where you're trying out a new treatment for the whole time you're in this study. If you're 16 or 17, your parent or guardian will need to give their permission for you to join, and you'll also need to agree yourself.

Quick self-check

Are you 16 years old or older?
Do you have Type 1 or Type 2 Hereditary Angioedema?
Are you able to use your emergency HAE medicine if needed?
Can you agree to temporarily stop your regular long-term preventative HAE medicines if your doctor says it's safe?
Are you willing to commit to regular clinic visits and follow study procedures for about 28 months?

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'll first go through a screening period to make sure it's safe and right for you. If you qualify, you'll be randomly assigned to either receive the new medicine, NTLA-2002, or a placebo (a dummy medicine). You will receive this treatment as a single infusion into a vein (like a drip) in your arm.

For the first 28 weeks after your infusion, you'll be closely monitored. During this time, neither you nor your study doctor will know whether you received the active medicine or the placebo. After these 28 weeks, you'll have the option to receive an infusion of the opposite treatment from what you received initially, still without knowing which one you're getting.

Following this, you'll enter a long-term observation period for another 76 weeks. This means the study will involve regular visits to the clinic for assessments, blood tests, and to discuss any HAE attacks you might have. You'll need to use your regular emergency HAE medicines if you have an attack. In total, from the very beginning to the very end, your participation in the study will last for about 28 months (over two years).

Potential risks and benefits

Taking part in any study has potential benefits and risks. You might find that the new medicine helps reduce your HAE attacks, or you might contribute important information to medical science that could help future patients. However, there's also a chance you could receive the placebo and not get the active treatment, or that the medicine might not work for you. There could be side effects from the medicine, or from the infusion itself, which your doctor will explain to you. Your safety and well-being are the most important concerns throughout the study, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (29)

Medical Research of Arizona
Scottsdale, United States
University of California, San Diego (UCSD)
San Diego, United States
Raffi Tachdjian MD, Inc
Santa Monica, United States
Allergy & Asthma Clinical Research
Walnut Creek, United States
IMMUNOe International Research Centers
Centennial, United States
Asthma & Allergy Associates
Colorado Springs, United States
University of South Florida
Tampa, United States
Massachusetts General Hospital
Boston, United States
Washington University in St. Louis
St Louis, United States
NYU Langone Health - Long Island
Mineola, United States
Icahn School of Medicine at Mount Sinai
New York, United States
Bernstein Clinical Research Center, LLC
Cincinnati, United States

+17 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 unpredictable episodes of swelling in different parts of the body, such as hands, feet, face, and airways.

What does 'single IV infusion' mean?

It means the study medicine will be given to you only once, directly into a vein, like a drip you might receive at a hospital.

What is a 'placebo'?

A placebo is an inactive substance or treatment that looks exactly like the real medicine but does not contain any active drug. It helps researchers compare the true effects of the new medicine.

Will I know if I'm getting the real medicine or the placebo?

For the main part of the study, neither you nor your study doctor will know which treatment you are receiving. This is called 'double-blind' and helps keep the study fair.

How long will I be in the study?

If you join, your total time in the study will be about 28 months, which is just over two years.