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Evaluating NTLA-2002 for treating Hereditary Angioedema

HAELO: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2002 in Participants With Hereditary Angioedema (HAE)

PHASE3 · Intellia Therapeutics · NCT06634420

This study is testing a new treatment called NTLA-2002 to see if it can help people with Hereditary Angioedema feel better and prevent attacks.

Quick facts

PhasePHASE3
Study typeInterventional
Enrollment60 (estimated)
Ages16 Years and up
SexAll
SponsorIntellia Therapeutics (industry)
Locations30 sites (Scottsdale, Arizona and 29 other locations)
Trial IDNCT06634420 on ClinicalTrials.gov

What this trial studies

This Phase 3 study assesses the efficacy and safety of NTLA-2002 in individuals with Hereditary Angioedema (HAE). Approximately 60 participants will be randomly assigned to receive either a single intravenous infusion of NTLA-2002 or a placebo in a double-blind manner. The study includes a Primary Observation Period lasting 28 weeks, followed by a Long-Term Observation Period of 76 weeks, totaling around 104 weeks of participation. Participants must refrain from long-term prophylactic therapies during the study and will have access to on-demand medications for HAE attacks.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 16 and older with a clinical history of HAE-C1INH-Type 1 or -Type 2.

Not a fit: Patients with concurrent diagnoses that may interfere with the study or those unable to provide informed consent may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the frequency and severity of HAE attacks for patients.

How similar studies have performed: Other studies have shown promise in treating HAE with novel therapies, suggesting potential success for this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Age ≥16 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. Participants 16 to \< 18 years of age, whose legal guardian provides informed consent, must provide assent.
9. Must agree to follow contraception requirements

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.

Where this trial is running

Scottsdale, Arizona and 29 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Hereditary Angioedema, NTLA-2002, HAE, HAE-C1INH-Type1 or -Type 2

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.