NTLA-2002 in Adults With Hereditary Angioedema (HAE)

Intellia Therapeutics

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: Hereditary Angioedema

Treatments: Other: Normal Saline IV Administration; Biological: Biological NTLA-2002

Study type: Interventional

Funder types: Industry

Identifiers

NCT05120830

ITL-2002-CL-001

Details and patient eligibility

About

This study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).

Enrollment: 37 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Age >18 years
2. Diagnosis of HAE Types I or II
3. Ability to provide evidence of HAE attacks to meet the screening requirement
4. Subjects must have access to, and the ability to use, ≥ 1 acute medication(s) to treat angioedema attacks.
5. Adequate chemistry and hematology measures at screening
6. Subjects must agree not to participate in another interventional study for the duration of this trial.
7. Subjects must be capable of providing signed informed consent

Exclusion criteria

1. Concurrent diagnosis of any other type of recurrent angioedema
2. Subjects who 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Double Blind

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37 participants in 4 patient groups, including a placebo group

Phase 1 Study Arm

Experimental group

Description:

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.

Treatment:

Biological: Biological NTLA-2002

Phase 2 Experimental Study Arm

Experimental group

Description:

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.

Treatment:

Biological: Biological NTLA-2002

Phase 2 Placebo Comparator Study Arm

Placebo Comparator group

Description:

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.

Treatment:

Other: Normal Saline IV Administration

Placebo Crossover and Follow-On Dosing Substudy Arm

Experimental group

Description:

Participants assigned to this Substudy Arm (participants who previously received either 25mg or placebo only) will have the opportunity to receive a single dose of NTLA-2002 (50mg) and will then be followed for 52 weeks.

Treatment:

Biological: Biological NTLA-2002