NTLA-2002 in Adults With Hereditary Angioedema (HAE)

Hereditary Angioedema Clinical Trial

— NTLA-2002  

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05120830
• Other study ID #: ITL-2002-CL-001
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: December 10, 2021
• Est. completion date: December 15, 2025

Study information

• Verified date: March 2024
• Source: Intellia Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 37
• Est. completion date: December 15, 2025
• Est. primary completion date: April 15, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema

Intervention

Biological:
• Biological NTLA-2002
CRISPR/Cas9 gene editing system delivered by LNP for IV administration

Other:
• Normal Saline IV Administration
The administration of IV normal saline

Locations

Country	Name	City	State
Australia	Clinical Trial Site	Campbelltown
France	Clinical Trial Site	Grenoble
France	Clinical Trial Site	Lille
France	Clinical Trial Site	Paris
Germany	Clinical Trial Site	Berlin
Germany	Clinical Trial Site	Frankfurt
Netherlands	Clinical Trial Site	Amsterdam
New Zealand	Clinical Trial Site	Auckland
United Kingdom	Clinical Trial Site	Cambridge

Sponsors (1)

Lead Sponsor	Collaborator
Intellia Therapeutics

Countries where clinical trial is conducted

Australia,  France,  Germany,  Netherlands,  New Zealand,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)	(Phase 1 only)	From NTLA-2002 infusion up to week 104 post-infusion
Primary	Number of HAE attacks per month (Weeks 1-16)	(Phase 2 only)	From study drug infusion up to week 16 post-infusion
Secondary	Change from baseline in total plasma kallikrein protein level	(Phase 1 & 2)	From NTLA-2002 infusion up to week 104 post-infusion
Secondary	Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA	(Phase 1 & 2)	From NTLA-2002 infusion up to week 104 post-infusion
Secondary	Safety and tolerability of NTLA-2002 as determined by AEs	(Phase 2 only)	From study drug infusion up to week 104 post-infusion
Secondary	Number of HAE attacks per month (Weeks 5-16)	(Phase 2 only)	From week 6 post-infusion up to week 16 post-infusion
Secondary	Number of HAE attacks per month requiring acute therapy (Weeks 1-16, Weeks 5-16)	(Phase 2 only)	From study drug infusion up to week 16 post-infusion