NTLA-2002 in Adults With Hereditary Angioedema (HAE) (NTLA-2002)
Hereditary Angioedema
About this trial
This is an interventional treatment trial for Hereditary Angioedema
Eligibility Criteria
Inclusion Criteria:
- Age >18 years
- Diagnosis of HAE Types I or II
- Ability to provide evidence of HAE attacks to meet the screening requirement
- Subjects must have access to, and the ability to use, ≥ 1 acute medication(s) to treat angioedema attacks.
- Adequate chemistry and hematology measures at screening
- Subjects must agree not to participate in another interventional study for the duration of this trial.
- Subjects must be capable of providing signed informed consent
Exclusion Criteria:
- Concurrent diagnosis of any other type of recurrent angioedema
- Subjects who 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.
Sites / Locations
- Clinical Trial SiteRecruiting
- Clinical Trial SiteRecruiting
- Clinical Trial SiteRecruiting
- Clinical Trial SiteRecruiting
- Clinical Trial SiteRecruiting
- Clinical Trial SiteRecruiting
- Clinical Trial SiteRecruiting
- Clinical Trial SiteRecruiting
- Clinical Trial SiteRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Placebo Comparator
Phase 1 Study Arm
Phase 2 Experimental Study Arm
Phase 2 Placebo Comparator Study Arm
Participants assigned to 1 of 3 dose-escalation cohorts will receive a single dose of NTLA-2002 on Day 1 and will then be followed for 104 weeks. Primary observation period is 16 weeks.
Participants randomized to NTLA-2002 (2 dose levels), will receive a single dose of NTLA-2002 on Day 1 and will then be followed for 104 weeks. Primary observation period is 16 weeks.
Participants randomized to placebo will receive IV normal saline on Day 1 and will then be followed for up to 104 weeks. Primary observation period is 16 weeks.