NTLA-2002 in Adults With Hereditary Angioedema (HAE)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

37 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-12-10

Study Completion Date

2026-03-31

Brief Summary

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

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Detailed Description

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Conditions

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Hereditary Angioedema

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Phase 1 is an open label non-randomized study Phase 2 is a randomized, double-blind, placebo-controlled study

Study Groups

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Phase 1 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.

Group Type EXPERIMENTAL

Biological NTLA-2002

Intervention Type BIOLOGICAL

CRISPR/Cas9 gene editing system delivered by LNP for IV administration

Phase 2 Experimental Study Arm

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.

Group Type EXPERIMENTAL

Biological NTLA-2002

Intervention Type BIOLOGICAL

CRISPR/Cas9 gene editing system delivered by LNP for IV administration

Phase 2 Placebo Comparator Study Arm

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.

Group Type PLACEBO_COMPARATOR

Normal Saline IV Administration

Intervention Type OTHER

The administration of IV normal saline

Placebo Crossover and Follow-On Dosing Substudy Arm

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.

Group Type EXPERIMENTAL

Biological NTLA-2002

Intervention Type BIOLOGICAL

CRISPR/Cas9 gene editing system delivered by LNP for IV administration

Interventions

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Biological NTLA-2002

CRISPR/Cas9 gene editing system delivered by LNP for IV administration

Intervention Type BIOLOGICAL

Normal Saline IV Administration

The administration of IV normal saline

Intervention Type OTHER

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No