HAELO clinical study | HAE clinical study

What is HAE?

What is hereditary angioedema (HAE)?

HAE is a rare genetic disorder caused by changes in the body’s instructions (genes). These instructions make proteins that act as workers, carrying out the instructions that allow your body to be built and function. When the instructions are wrong, proteins cannot do their job properly which can impact your health. HAE causes painful, potentially life-threatening, dangerous swelling attacks that can occur without warning and last for several days. These attacks can affect the abdomen, hands and feet, face, genitals, and upper airway.

What causes HAE?

Most people with HAE have changes in the SERPING1 gene. This gene is responsible for producing C1 esterase inhibitor (C1-INH), a protein which plays a critical role in regulating blood vessel swelling.
C1-INH controls the activity of other proteins, mainly produced in the liver, which are involved in the inflammatory response.
When C1-INH is not functioning properly, the kallikrein protein produced by the KLKB1 gene becomes overactive, directly promoting inflammation. The disruption to the body’s normal inflammatory control processes then leads to the swelling that is a hallmark symptom of HAE.

Where can I learn more?

To learn more about HAE, visit US Hereditary Angioedema Association or HAE International.

HAELO

What is the HAELO study?

The HAELO study is a Phase 3 clinical study for adults living with Type 1 or Type 2 hereditary angioedema (HAE). The purpose of this study is to test an experimental treatment, NTLA-2002, to evaluate its efficacy and safety in reducing HAE attacks versus placebo (inactive substance).

This study is double-blind, which means you, your study doctor and the study team will not know what study intervention you will be receiving.

Study participants may be able to join the study if they meet the following requirements:

Must be 18 years of age or older

Confirmed diagnosis of Type 1 or Type 2 HAE

Participants must be willing to stop taking long-term preventative HAE medication, if applicable (check with study doctor). On-demand HAE treatment will still be permissible at no cost to you.

Find a Study Clinic

Additional study requirements will apply. Contact a study clinic for more information on study criteria.

What will happen during the study?

Participation in this study can last up to approximately 2 years and 4 months. Study participants can expect the following:

Review & Sign the Informed Consent Form

Sign the Informed Consent Form (ICF). The ICF contains information about the study including its goals, duration, benefits, risks, tests and procedures.

Screening Period
(up to 4 months)

Receive study health assessments to confirm if you qualify for the HAELO study.

Dosing and Primary Observation Period
Part 1 (about 7 months)

Participants will be assigned at random to receive NTLA-2002 or a placebo. Participants will receive 1 blinded, intravenous infusion (IV) of NTLA-2002 or placebo over about 4 hours. Participants will also have up to 7 visits (including the infusion visit) during this period. Two of these visits can be done via telemedicine. Each visit will include study assessments, such as physical exams, blood tests, heart tests and questionnaires.

Long-Term Observation Period
Part 2 (about 19 months)

Participants will have the option to receive a second, blinded intravenous infusion (IV) of NTLA-2002 or placebo. If you choose to receive a second dose, you will be assigned to receive the opposite of what was administered previously. All study participants will continue to visit their assigned study clinic regularly for more study assessments. You will have up to 11 more visits during this period. Three of these visits can be done via telemedicine.

Separate Long-Term Follow-up Study
(up to 15 years)

You will be asked to participate in a separate follow-up study to help determine the long-term safety and effectiveness of NTLA-2002.

Participation in a clinical study is voluntary. You can ask any questions you have and may leave the study at any time, for any reason.

What is NTLA-2002?

What is the experimental treatment NTLA-2002?

NTLA-2002 is an experimental treatment which consists of a CRISPR/Cas9 gene editing system designed to precisely edit the KLKB1 gene. NTLA-2002 permanently changes the DNA in the liver cells by inactivating the KLKB1 gene so less plasma kallikrein is produced. The hope is that by decreasing the production of plasma kallikrein, NTLA-2002 may reduce the frequency of HAE attacks in people with Type 1 or Type 2 HAE. In HAELO, NTLA-2002 will be administered over about 4 hours as an intravenous infusion (IV) into the arm or hand.

Will I receive the experimental treatment?

Study participants will be assigned at random by a computer to receive NTLA-2002 or a placebo. You have a 67% (2 in 3) chance of receiving NTLA-2002 and 33% (1 in 3) chance of receiving placebo. All participants will have the opportunity to receive NTLA-2002 during this study.

What is an “experimental treatment"?

Experimental means the treatment is not approved by regulatory authorities like the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA) and cannot be prescribed to you. This treatment can only be used in clinical research studies like HAELO.

Overview of Clinical Studies

Here are some common questions and answers about study participation.

What are clinical studies?

Clinical studies help scientists and doctors explore whether a medication, device or procedure is safe and effective for people. Before any medication can be approved and made available to the public, it must go through several phases of clinical research.

What is informed consent?

Before enrolling in a clinical study, you should understand what is involved and have the opportunity for your questions to be answered by a medical professional. The Informed Consent Form (ICF) contains information about the study, including study goals, how long the study will last, benefits and risks, and the tests and procedures you will receive. Information in the ICF will be reviewed with you as part of the consent process, and the ICF must be signed before enrolling in a clinical study.

What does study participation involve?

Study participation involves visiting a clinic to receive a one-time infusion of the experimental treatment, after which you will continue to regularly visit the clinic. During these visits, your health will be assessed, and you’ll need to complete an electronic HAE attack diary as well as electronic questionnaires. You can continue seeing your regular doctor, but you should inform them of your involvement in this study.

Participation in a clinical study is your choice, and you may stop at any time.

If you have additional questions about participating in a clinical study, contact a study clinic near you.

Study Clinic Locations

Find a study clinic near you!

Use the filters, map, and listing to find the study clinic closest to you. Contact the study clinic to learn more.

Are you currently living with hereditary angioedema (HAE)?

If so, consider joining the HAELO study, a clinical study for people with Type 1 or Type 2 hereditary angioedema that will investigate a new experimental treatment option for adults living with HAE.