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NTLA-2002 in Adults With Hereditary Angioedema (HAE) (NTLA-2002)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05120830
Recruitment Status : Active, not recruiting
First Posted : November 15, 2021
Last Update Posted : March 12, 2024
Sponsor:
Information provided by (Responsible Party):
Intellia Therapeutics

Study Description
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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).

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Biological: Biological NTLA-2002 Other: Normal Saline IV Administration Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 37 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double (Participant, Investigator)
Masking Description: Phase 1 is an open label non-randomized study Phase 2 is a randomized, double-blind, placebo-controlled study
Primary Purpose: Treatment
Official Title: Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Actual Study Start Date : December 10, 2021
Estimated Primary Completion Date : April 15, 2024
Estimated Study Completion Date : December 15, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 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.
 Biological: Biological NTLA-2002
CRISPR/Cas9 gene editing system delivered by LNP for IV administration
Experimental: 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.
 Biological: Biological NTLA-2002
CRISPR/Cas9 gene editing system delivered by LNP for IV administration
Placebo Comparator: 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.
 Other: Normal Saline IV Administration
The administration of IV normal saline


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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)


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05120830


Locations
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Australia
Clinical Trial Site
Campbelltown, Australia
France
Clinical Trial Site
Grenoble, France
Clinical Trial Site
Lille, France
Clinical Trial Site
Paris, France
Germany
Clinical Trial Site
Berlin, Germany
Clinical Trial Site
Frankfurt, Germany
Netherlands
Clinical Trial Site
Amsterdam, Netherlands
New Zealand
Clinical Trial Site
Auckland, New Zealand
United Kingdom
Clinical Trial Site
Cambridge, United Kingdom
Sponsors and Collaborators
Intellia Therapeutics
More Information
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Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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Responsible Party: Intellia Therapeutics
ClinicalTrials.gov Identifier: NCT05120830    
Other Study ID Numbers: ITL-2002-CL-001
First Posted: November 15, 2021    Key Record Dates
Last Update Posted: March 12, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
 Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Hereditary Complement Deficiency Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes