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Active, Not Recruiting

NCT Number: NCT05120830

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Hereditary Angioedema Angioedema Angioedemas, Hereditary Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Complement Deficiency Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Immunologic Deficiency Syndromes Primary Immunodeficiency Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Clinical Trial Site, Campbelltown, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • Concurrent diagnosis of any other type of recurrent angioedema
  • Subjects who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
  • Any condition that, in the Investigator's opinion, could adversely affect the safety of the subject.
  • Unwilling to comply with study procedures.

Treatment and study plan

Biological NTLA-2002

Biological

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

Normal Saline IV Administration

Other

The administration of IV normal saline

Primary outcomes

  1. Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)

    Time frame: From NTLA-2002 infusion up to week 104 post-infusion

    (Phase 1 only)

  2. Number of HAE attacks per month (Weeks 1-16)

    Time frame: From study drug infusion up to week 16 post-infusion

    (Phase 2 only)

Secondary outcomes

  1. Change from baseline in total plasma kallikrein protein level

    Time frame: From NTLA-2002 infusion up to week 104 post-infusion

    (Phase 1 & 2)

  2. Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA

    Time frame: From NTLA-2002 infusion up to week 104 post-infusion

    (Phase 1 & 2)

  3. Safety and tolerability of NTLA-2002 as determined by AEs

    Time frame: From study drug infusion up to week 104 post-infusion

    (Phase 2 only)

  4. Number of HAE attacks per month (Weeks 5-16)

    Time frame: From week 6 post-infusion up to week 16 post-infusion

    (Phase 2 only)

  5. Number of HAE attacks per month requiring acute therapy (Weeks 1-16, Weeks 5-16)

    Time frame: From study drug infusion up to week 16 post-infusion

    (Phase 2 only)

Sponsors and collaborators

Lead sponsor

Intellia Therapeutics

Industry

Registry information

Official study title

Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)

Acronym: NTLA-2002

Important dates

Study start
2021
Primary completion
2024
Study completion
2026
First posted
Nov 15, 2021
Registry last updated
Mar 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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