NCT06634420

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

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)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Intellia Therapeutics · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.

Detailed description

This is a multinational, multicenter, double-blind, placebo-controlled study in which approximately 60 participants will be randomized in a 2:1 ratio to receive a single IV infusion of NTLA-2002 or placebo. After the Primary Observation Period (Week 1 through Week 28), participants will have the option to receive a blinded, single IV infusion of the opposite treatment. Following the Primary Observation Period, participants will enter the Long-Term Observation Period (76 weeks), for a total of 104 weeks. Including the Screening and Run-In Period, prior to the first blinded dosing, the total study duration is approximately 28 months.

Conditions

• Hereditary Angioedema

Interventions

Type	Name	Description
BIOLOGICAL	NTLA-2002	CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration
BIOLOGICAL	Normal Saline IV Administration	The administration of intravenous (IV) normal saline

Timeline

• Start date: 2025-01-15
• Primary completion: 2026-04-01
• Completion: 2027-09-01
• First posted: 2024-10-09
• Last updated: 2025-12-18

Locations

29 sites across 9 countries: United States, Australia, Canada, France, Germany, Netherlands, New Zealand, South Africa, United Kingdom

Regulatory

• FDA-regulated drug study