NCT05120830: NTLA-2002

Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)

Active, enrolled Phase 1/Phase 2 Last updated 23 March 2026

What this trial tests

Phase 1/Phase 2 trial testing Biological NTLA-2002 in Hereditary Angioedema in 37 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

10 December 2021

Primary endpoint
4 April 2024

1 July 2026

Quick facts

Lead sponsor	Intellia Therapeutics
Phase	Phase 1/Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	randomized
Design	sequential
Masking	double
Primary purpose	treatment
Enrollment	37
Start date	10 December 2021
Primary completion	4 April 2024
Estimated completion	1 July 2026
Sites	9 locations across Australia, France, Germany, Netherlands, New Zealand, United Kingdom

Drugs / interventions tested

• Biological NTLA-2002 — full drug profile →
• Normal Saline IV Administration — full drug profile →

Conditions studied

• Hereditary Angioedema — all drugs for Hereditary Angioedema →

Sponsor

Intellia Therapeutics — full company profile →

Who can join

18 and older, any sex, with Hereditary Angioedema. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
  Time frame: From NTLA-2002 infusion up to week 104 post-infusion
  (Phase 1 only)
• Number of HAE attacks per month (Weeks 1-16)
  Time frame: From study drug infusion up to week 16 post-infusion
  (Phase 2 only)

Sponsor's own description

This study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Passive, active and endogenous organ-targeted lipid and polymer nanoparticles for delivery of genetic drugs.
   Dilliard SA, Siegwart DJ. · · 2023 · cited 384× · PMID 36691401 · DOI 10.1038/s41578-022-00529-7
2. PEGylated therapeutics in the clinic.
   Gao Y, Joshi M, Zhao Z, Mitragotri S. · · 2024 · cited 133× · PMID 38193121 · DOI 10.1002/btm2.10600
3. CRISPR-Cas9 In Vivo Gene Editing of <i>KLKB1</i> for Hereditary Angioedema.
   Longhurst HJ, Lindsay K, Petersen RS, Fijen LM, et al · · 2024 · cited 103× · PMID 38294975 · DOI 10.1056/nejmoa2309149
4. Combinatorial design of ionizable lipid nanoparticles for muscle-selective mRNA delivery with minimized off-target effects.
   Chen J, Xu Y, Zhou M, Xu S, et al · · 2023 · cited 101× · PMID 38060560 · DOI 10.1073/pnas.2309472120
5. Nucleic acid drugs: recent progress and future perspectives.
   Sun X, Setrerrahmane S, Li C, Hu J, et al · · 2024 · cited 72× · PMID 39609384 · DOI 10.1038/s41392-024-02035-4
6. Recent advances in nanoparticulate RNA delivery systems.
   Witten J, Hu Y, Langer R, Anderson DG. · · 2024 · cited 70× · PMID 38437569 · DOI 10.1073/pnas.2307798120
7. Carrier strategies boost the application of CRISPR/Cas system in gene therapy.
   Xu Z, Wang Q, Zhong H, Jiang Y, et al · · 2022 · cited 69× · PMID 37323878 · DOI 10.1002/exp.20210081
8. Progress and prospects of mRNA-based drugs in pre-clinical and clinical applications.
   Shi Y, Shi M, Wang Y, You J. · · 2024 · cited 67× · PMID 39543114 · DOI 10.1038/s41392-024-02002-z