HAELO: a Phase 3 Study to Evaluate NTLA-2002 in Participants with Hereditary Angioedema (HAE)

Phase 3

Recruiting

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

• Registration Number: NCT06634420
• Lead Sponsor: Intellia Therapeutics

Brief Summary

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

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.

Study Timeline

• Study First Submitted: 2024-10-07
• Study First Submitted QC: 2024-10-08
• Study First Posted: 2024-10-09
• Status Verified: 2024-12
• Study Start: 2024-12
• Last Update Submitted: 2024-12-16
• Last Update Posted: 2024-12-19
• Primary Completion: 2026-04
• Study Completion: 2027-09

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

1. Age ≥18 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
9. Must agree to follow contraception requirements

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Arm B: Placebo	Normal Saline IV Administration	Arm B: Placebo (saline; single IV infusion)
Arm A: NTLA-2002	NTLA-2002	Arm A: NTLA-2002 (50 mg; single IV infusion)

Primary Outcome Measures

Name	Time	Method
Time-normalized number of Investigator-confirmed HAE attacks	From Week 5 through Week 28

Secondary Outcome Measures

Name	Time	Method
Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment	from Week 1 through Week 28
Time-normalized number of moderate or severe Investigator-confirmed HAE attacks	From Week 1 through Week 28
Investigator-confirmed HAE attack-free status	From Week 1 through Week 28
Change from baseline to Week 28 in AE-QoL Questionnaire total score	From baseline to Week 28
Responder status, defined as reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks	From Week 5 through Week 28
Time-normalized number of Investigator-confirmed HAE attacks	From Week 1 through Week 28
Reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks	From Week 1 through Week 28
Long-term prophylaxis-free status	From Week 5 through Week 104
HAE treatment-free status, defined as no usage of long-term prophylaxis or on-demand medication	From Week 5 through Week 104
Complete responder status, defined as zero Investigator-confirmed attacks with no long-term prophylaxis	From Week 5 through Week 104
Reduction in the time-normalized number of Investigator-confirmed HAE attacks after crossover treatment (Week 33 through Week 104) compared to before crossover treatment (Screening through Week 28) in participants randomized to placebo	From Week 33 through Week 104 and from Screening through Week 28

Trial Locations

Locations (3)

Asthma & Allergy Associates; 🇺🇸 Colorado Springs, Colorado, United States

Optimed Research, LTD; 🇺🇸 Columbus, Ohio, United States

Allergy & Asthma Clinical Research; 🇺🇸 Walnut Creek, California, United States