Clinical Trial

Disease: Hereditary Angioedema, HAE, (NCT06634420)

Disease info:

Hereditary Angioedema (HAE) is a rare, genetic disorder characterised by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body. Currently available treatment options include life-long therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week, or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. 

Frequency:
It is estimated that one in 50,000 people are affected by HAE.
Official 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)
Who:

Conntact: Trial Manager at Intellia Therapeutics

Phone: 1-857-285-6200 ext 6

Email: medicalinformation@intelliatx.com

Sponsor:

Intellia Therapeutics

Partners:
Locations:

United States, California

Allergy & Asthma Clinical Research, Walnut Creek, California, United States, 94598

Study start:
Oct. 1, 2024
Enrollment:
60 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene knockout
Gene:
kallikrein B1 (KLKB1)
Delivery method:
Lipid nanoparticles (LNP) - In-vivo
Trial link:
https://clinicaltrials.gov/study/NCT06634420?spons=Intellia%20Therapeutics
Safety updates:

(24-10-2024) CRISPR-Based Therapy for Hereditary Angioedema

(07-10-2024) Intellia Therapeutics Announces Initiation of HAELO Phase 3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema (HAE)

Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

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

Last updated: Nov. 3, 2024
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