HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
- Conditions
- Hereditary Angioedema
- Interventions
- Biological: Normal Saline IV Administration
- Registration Number
- NCT06634420
- Lead Sponsor
- Intellia Therapeutics
- Brief 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 60
- Age ≥16 years
- Clinical history consistent with HAE-C1INH-Type 1 or -Type 2
- Ability to provide evidence of HAE attacks (confirmed by the Investigator) to meet the screening requirement
- 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.
- Must have access to, and the ability to use, on-demand medication(s) to treat potential angioedema attacks
- Adequate chemistry and hematology measures at screening
- Must agree not to participate in another interventional study for the duration of this trial.
- Must be capable of providing signed informed consent. Participants 16 to < 18 years of age, whose legal guardian provides informed consent, must provide assent.
- Must agree to follow contraception requirements
- Concurrent diagnosis of any other type of recurrent angioedema or HAE with normal C1-INH
- Have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
- Any condition that, in the Investigator's opinion, could adversely affect the safety of the subject.
- Unwilling to comply with study procedures.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Arm B: Placebo Normal Saline IV Administration Arm B: Placebo (saline; single IV infusion) Arm A: NTLA-2002 NTLA-2002 Arm A: NTLA-2002 (50 mg; single IV infusion)
- Primary Outcome Measures
Name Time Method Time-normalized number of Investigator-confirmed HAE attacks From Week 5 through Week 28
- Secondary Outcome Measures
Name Time Method Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment from Week 1 through Week 28 Time-normalized number of moderate or severe Investigator-confirmed HAE attacks From Week 1 through Week 28 Investigator-confirmed HAE attack-free status From Week 1 through Week 28 Change from baseline to Week 28 in AE-QoL Questionnaire total score From baseline to Week 28 Responder status, defined as reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks From Week 5 through Week 28 Time-normalized number of Investigator-confirmed HAE attacks From Week 1 through Week 28 Reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks From Week 1 through Week 28 Long-term prophylaxis-free status From Week 5 through Week 104 HAE treatment-free status, defined as no usage of long-term prophylaxis or on-demand medication From Week 5 through Week 104 Complete responder status, defined as zero Investigator-confirmed attacks with no long-term prophylaxis From Week 5 through Week 104 Reduction in the time-normalized number of Investigator-confirmed HAE attacks after crossover treatment (Week 33 through Week 104) compared to before crossover treatment (Screening through Week 28) in participants randomized to placebo From Week 33 through Week 104 and from Screening through Week 28
Trial Locations
- Locations (30)
CHU de Lille - Hopital Claude Huriez
🇫🇷Lille, France
AP-HM - Hopital de la Timone
🇫🇷Marseille, France
Charite Universitaetsmedizin Berlin
🇩🇪Berlin, Germany
Amsterdam UMC - Locatie AMC
🇳🇱Amsterdam, Netherlands
Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
🇩🇪Frankfurt, Germany
Medizinische Hochschule Hannover (MHH)
🇩🇪Hannover, Germany
Medical Research of Arizona
🇺🇸Scottsdale, Arizona, United States
Raffi Tachdjian MD, Inc
🇺🇸Santa Monica, California, United States
Allergy & Asthma Clinical Research
🇺🇸Walnut Creek, California, United States
IMMUNOe International Research Centers
🇺🇸Centennial, Colorado, United States
Asthma & Allergy Associates
🇺🇸Colorado Springs, Colorado, United States
University of South Florida
🇺🇸Tampa, Florida, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Henry Ford Health System
🇺🇸Detriot, Michigan, United States
Washington University in St. Louis
🇺🇸St. Louis, Missouri, United States
NYU Langone Health - Long Island
🇺🇸Mineola, New York, United States
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
Bernstein Clinical Research Center, LLC
🇺🇸Cincinnati, Ohio, United States
Optimed Research, LTD
🇺🇸Columbus, Ohio, United States
Penn State Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
AARA Research Center
🇺🇸Dallas, Texas, United States
Campbelltown Hospital
🇦🇺Campbelltown, New South Wales, Australia
The Alfred Hospital
🇦🇺Melbourne, Victoria, Australia
Royal Melbourne Hospital
🇦🇺Parkville, Victoria, Australia
University of Alberta
🇨🇦Edmonton, Alberta, Canada
Ottawa Allergy Research Corporation
🇨🇦Ottawa, Ontario, Canada
CHU Grenoble-Alpes - Hopital Michallon
🇫🇷Grenoble, France
New Zealand Clinical Research (NZCR)
🇳🇿Auckland, New Zealand
University of Cape Town - Lung Institute - Lung Clinical Research Unit
🇿🇦Cape Town, South Africa
Cambridge University Hospitals NHS Foundation Trust
🇬🇧Cambridge, United Kingdom