ACTIVE_NOT_RECRUITING

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

Conditions

Hereditary Angioedema NTLA-2002 HAE HAE-C1INH-Type1 or -Type 2

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2025-01-15

Study Completion:2027-09

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06634420

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:16 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Age ≥16 years 2. Clinical history consistent with HAE-C1INH-Type 1 or -Type 2 3. Ability to provide evidence of HAE attacks (confirmed by the Investigator) to meet the screening requirement 4. Must agree to refrain from the use of long-term prophylactic therapies from the start of the screening period through the end of the Primary Observation Period. PI must be in agreement that it is medically acceptable for the participant to do so. 5. Must have access to, and the ability to use, on-demand medication(s) to treat potential angioedema attacks 6. Adequate chemistry and hematology measures at screening 7. Must agree not to participate in another interventional study for the duration of this trial. 8. Must be capable of providing signed informed consent. Participants 16 to \< 18 years of age, whose legal guardian provides informed consent, must provide assent. 9. Must agree to follow contraception requirements	1. Concurrent diagnosis of any other type of recurrent angioedema or HAE with normal C1-INH 2. Have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component. 3. Any condition that, in the Investigator's opinion, could adversely affect the safety of the subject. 4. Unwilling to comply with study procedures.

Inclusion Criteria

Exclusion Criteria

1. Age ≥16 years
2. Clinical history consistent with HAE-C1INH-Type 1 or -Type 2
3. Ability to provide evidence of HAE attacks (confirmed by the Investigator) to meet the screening requirement
4. Must agree to refrain from the use of long-term prophylactic therapies from the start of the screening period through the end of the Primary Observation Period. PI must be in agreement that it is medically acceptable for the participant to do so.
5. Must have access to, and the ability to use, on-demand medication(s) to treat potential angioedema attacks
6. Adequate chemistry and hematology measures at screening
7. Must agree not to participate in another interventional study for the duration of this trial.
8. Must be capable of providing signed informed consent. Participants 16 to \< 18 years of age, whose legal guardian provides informed consent, must provide assent.
9. Must agree to follow contraception requirements

1. Concurrent diagnosis of any other type of recurrent angioedema or HAE with normal C1-INH
2. Have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
3. Any condition that, in the Investigator's opinion, could adversely affect the safety of the subject.
4. Unwilling to comply with study procedures.

Contacts and Locations

Study Locations (Sites)

Medical Research of Arizona

Scottsdale, Arizona, 85251

United States

University of California, San Diego (UCSD)

San Diego, California, 92122

United States

Raffi Tachdjian MD, Inc

Santa Monica, California, 90404

United States

Allergy & Asthma Clinical Research

Walnut Creek, California, 94598

United States

IMMUNOe International Research Centers

Centennial, Colorado, 80112

United States

Asthma & Allergy Associates

Colorado Springs, Colorado, 80907

United States

University of South Florida

Tampa, Florida, 33613

United States

Massachusetts General Hospital

Boston, Massachusetts, 02114

United States

Henry Ford Health System

Detriot, Michigan, 48202

United States

Washington University in St. Louis

St. Louis, Missouri, 63141

United States

NYU Langone Health - Long Island

Mineola, New York, 11501

United States

Icahn School of Medicine at Mount Sinai

New York, New York, 10029

United States

Bernstein Clinical Research Center, LLC

Cincinnati, Ohio, 45236

United States

Optimed Research, LTD

Columbus, Ohio, 43235

United States

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033

United States

AARA Research Center

Dallas, Texas, 75231

United States

Collaborators and Investigators

Sponsor: Intellia Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-01-15

Study Completion Date2027-09

Study Record Updates

Study Start Date2025-01-15

Study Completion Date2027-09

Terms related to this study

Keywords Provided by Researchers

Hereditary Angioedema
NTLA-2002
HAE
HAE-C1INH-Type1 or -Type 2

Additional Relevant MeSH Terms

Hereditary Angioedema