HAELO
Research type
Research Study
Full title
HAELO: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2002 in Participants With Hereditary Angioedema (HAE)
IRAS ID
1010722
Contact name
Surbhi Goel
Contact email
Sponsor organisation
Intellia Therapeutics, Inc
Research summary
This is a study of the experimental treatment called NTLA-2002 in people with hereditary angioedema (HAE), a rare genetic disorder. HAE causes attacks of swelling in organs and tissues of the body that can be unpredictable, painful, and even life-threatening. Most people with HAE have a mutation in the SERPING1 gene. This gene produces C1 esterase inhibitor (C1INH), which helps control blood vessel permeability, and thus swelling in organs and tissues, through its ability to affect the activity of plasma kallikrein. The production of kallikrein itself is controlled by the KLKB1 gene. Current treatments to prevent attacks must be taken regularly to help control disease in people with HAE, but they may still experience breakthrough attacks. NTLA-2002 is a CRISPR-based gene editing therapy administered as a one-time dose that targets the KLKB1 gene and disables it so it can no longer make kallikrein protein. Lower levels of plasma kallikrein protein may result in fewer HAE attacks in people with HAE. The purpose of this study is to investigate the effects of NTLA-2002 on adults with HAE to see if NTLA-2002 can reduce the number of HAE attacks per month when compared to placebo (no active ingredients), and to see how safe NTLA-2002 is when given to people with HAE. This is a double-blind study, which means that neither the study participants, nor the doctors will know who has received which study intervention. Participants will be randomly assigned (by chance) to receive either NTLA-2002 or placebo. They will have a 2 in 3 chance of receiving NTLA-2002 and a 1 in 3 chance of receiving placebo. Both NTLA-2002 and placebo will be given as an intravenous infusion. Participants will have tests such as blood and urine tests, physical exams, and electrocardiograms and will answer questionnaires about their quality of life. All participants will have to complete an electronic diary to record HAE attacks, symptoms, treatments, and other related information.
REC name
North East - York Research Ethics Committee
REC reference
24/NE/0176
Date of REC Opinion
26 Nov 2024
REC opinion
Further Information Favourable Opinion