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Purpose

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

Condition

Eligibility

Eligible Ages
Over 16 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion 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

Exclusion Criteria

  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.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Active Comparator
Arm A: NTLA-2002 		Arm A: NTLA-2002 (50 mg; single IV infusion)
  • Biological: NTLA-2002
    CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration
Placebo Comparator
Arm B: Placebo 		Arm B: Placebo (saline; single IV infusion)
  • Biological: Normal Saline IV Administration
    The administration of intravenous (IV) normal saline

Recruiting Locations

Massachusetts General Hospital
Boston, Massachusetts 02114

More Details

Status
Recruiting
Sponsor
Intellia Therapeutics

Study Contact

Trial Manager at Intellia Therapeutics
1-857-285-6200
medicalinformation@intelliatx.com

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.