NTLA-2002 in Adults With Hereditary Angioedema (HAE)

Phase 1

Active, not recruiting

• Conditions: Hereditary Angioedema
• Interventions: Biological: Biological NTLA-2002; Other: Normal Saline IV Administration

• Registration Number: NCT05120830
• Lead Sponsor: Intellia Therapeutics

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-11-03
• Study First Submitted QC: 2021-11-12
• Study First Posted: 2021-11-15
• Study Start: 2021-12-10
• Primary Completion: 2024-04-04
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-10
• Last Update Posted: 2024-07-11
• Study Completion: 2026-03-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 37

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

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Phase 1 Study Arm	Biological NTLA-2002	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.
Phase 2 Experimental Study Arm	Biological NTLA-2002	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.
Phase 2 Placebo Comparator Study Arm	Normal Saline IV Administration	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.
Placebo Crossover and Follow-On Dosing Substudy Arm	Biological NTLA-2002	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.

Primary Outcome Measures

Name	Time	Method
Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)	From NTLA-2002 infusion up to week 104 post-infusion	(Phase 1 only)
Number of HAE attacks per month (Weeks 1-16)	From study drug infusion up to week 16 post-infusion	(Phase 2 only)

Secondary Outcome Measures

Name	Time	Method
Number of HAE attacks per month requiring acute therapy (Weeks 1-16, Weeks 5-16)	From study drug infusion up to week 16 post-infusion	(Phase 2 only)
Change from baseline in total plasma kallikrein protein level	From NTLA-2002 infusion up to week 104 post-infusion	(Phase 1 \& 2)
Number of HAE attacks per month (Weeks 5-16)	From week 6 post-infusion up to week 16 post-infusion	(Phase 2 only)
Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA	From NTLA-2002 infusion up to week 104 post-infusion	(Phase 1 \& 2)
Safety and tolerability of NTLA-2002 as determined by AEs	From study drug infusion up to week 104 post-infusion	(Phase 2 only)

Trial Locations

Locations (1)

Clinical Trial Site; 🇬🇧 Cambridge, United Kingdom