NTLA-2002 in Adults with Hereditary Angioedema (HAE)
Research type
Research Study
Full title
Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults with Hereditary Angioedema (HAE)
IRAS ID
1003771
Contact name
Dahua Pan
Contact email
Sponsor organisation
Intellia Therapeutics, Inc.
Eudract number
2021-001693-33
Clinicaltrials.gov Identifier
Research summary
NTLA-2002 is an investigational drug being developed as a potential new treatment for patients with Hereditary
Angioedema (HAE). HAE is a rare, genetic disorder that is characterised by severe recurring and unpredictable
inflammatory attacks in various organs and tissues of the body which can be painful, debilitating, and lifethreatening.
NTLA-2002 consists of a CRISPR/Cas9 gene editing system, which is able to potentially treat HAE through editing of the KLKB1 gene.
This first-in-human (FIH) study will aim to assess the safety and tolerability of NTLA-2002 in patients with HAE. The
study is comprised of 2 phases, referred to as Part 1 and Part 2 (approx. 30 patients in Part 1 and approx. 25
patients in Part 2). Part 1 will evaluate different dose levels of NTLA-2002 and assess how the body responds. If
Part 1 identifies a safe and effective dose (defined by blood results and an observed reduction in HAE attacks) then
the study will proceed to Part 2. The doses selected for Part 2 will be determined to be safe, effective and well
tolerated based on the information from Part 1.
Patients will be enrolled in the study for approximately 2 years, in which there will be a screening and treatment
period, followed by a long-term follow up period. Depending on which part of the study and what group they are
enrolled in, they will either receive a single dose of NTLA-2002 or placebo (if randomised to placebo arm in Part 2).
The results from this study will be used to inform further clinical development of NTLA-2002.REC name
North East - York Research Ethics Committee
REC reference
21/NE/0116
Date of REC Opinion
13 Oct 2021
REC opinion
Further Information Favourable Opinion