search
Back to results

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

Primary Purpose

Hereditary Angioedema

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Biological NTLA-2002
Normal Saline IV Administration
Sponsored by
Intellia Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Angioedema

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Clinical Trial SiteRecruiting
  • Clinical Trial SiteRecruiting
  • Clinical Trial SiteRecruiting
  • Clinical Trial SiteRecruiting
  • Clinical Trial SiteRecruiting
  • Clinical Trial SiteRecruiting
  • Clinical Trial SiteRecruiting
  • Clinical Trial SiteRecruiting
  • Clinical Trial SiteRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

Phase 1 Study Arm

Phase 2 Experimental Study Arm

Phase 2 Placebo Comparator Study Arm

Arm Description

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.

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.

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.

Outcomes

Primary Outcome Measures

Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
(Phase 1 only)
Number of HAE attacks per month (Weeks 1-16)
(Phase 2 only)

Secondary Outcome Measures

Change from baseline in total plasma kallikrein protein level
(Phase 1 & 2)
Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
(Phase 1 & 2)
Safety and tolerability of NTLA-2002 as determined by AEs
(Phase 2 only)
Number of HAE attacks per month (Weeks 5-16)
(Phase 2 only)
Number of HAE attacks per month requiring acute therapy (Weeks 1-16, Weeks 5-16)
(Phase 2 only)

Full Information

First Posted
November 3, 2021
Last Updated
September 20, 2023
Sponsor
Intellia Therapeutics
search

1. Study Identification

Unique Protocol Identification Number
NCT05120830
Brief Title
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Acronym
NTLA-2002
Official Title
Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
December 10, 2021 (Actual)
Primary Completion Date
April 15, 2024 (Anticipated)
Study Completion Date
December 15, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Intellia Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
55 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Phase 1 Study Arm
Arm Type
Experimental
Arm Description
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.
Arm Title
Phase 2 Experimental Study Arm
Arm Type
Experimental
Arm Description
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.
Arm Title
Phase 2 Placebo Comparator Study Arm
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Biological
Intervention Name(s)
Biological NTLA-2002
Intervention Description
CRISPR/Cas9 gene editing system delivered by LNP for IV administration
Intervention Type
Other
Intervention Name(s)
Normal Saline IV Administration
Intervention Description
The administration of IV normal saline
Primary Outcome Measure Information:
Title
Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
Description
(Phase 1 only)
Time Frame
From NTLA-2002 infusion up to week 104 post-infusion
Title
Number of HAE attacks per month (Weeks 1-16)
Description
(Phase 2 only)
Time Frame
From study drug infusion up to week 16 post-infusion
Secondary Outcome Measure Information:
Title
Change from baseline in total plasma kallikrein protein level
Description
(Phase 1 & 2)
Time Frame
From NTLA-2002 infusion up to week 104 post-infusion
Title
Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
Description
(Phase 1 & 2)
Time Frame
From NTLA-2002 infusion up to week 104 post-infusion
Title
Safety and tolerability of NTLA-2002 as determined by AEs
Description
(Phase 2 only)
Time Frame
From study drug infusion up to week 104 post-infusion
Title
Number of HAE attacks per month (Weeks 5-16)
Description
(Phase 2 only)
Time Frame
From week 6 post-infusion up to week 16 post-infusion
Title
Number of HAE attacks per month requiring acute therapy (Weeks 1-16, Weeks 5-16)
Description
(Phase 2 only)
Time Frame
From study drug infusion up to week 16 post-infusion

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Trial Manager at Intellia
Phone
833-888-0387
Email
clinicalscience@intelliatx.com
Facility Information:
Facility Name
Clinical Trial Site
City
Campbelltown
Country
Australia
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Grenoble
Country
France
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Lille
Country
France
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Paris
Country
France
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Berlin
Country
Germany
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Frankfurt
Country
Germany
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Amsterdam
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Auckland
Country
New Zealand
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Cambridge
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

We'll reach out to this number within 24 hrs