Clinical trial NCT05120830: NTLA-2002 in Adults With Hereditary Angioedema (HAE)
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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Hereditary Angioedema
Lead sponsor: Intellia Therapeutics
Sponsor class: INDUSTRY
First posted: 2021-11-15
Enrollment: 37
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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).


Eligibility 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.

Primary outcomes:
measure: Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
timeFrame: From NTLA-2002 infusion up to week 104 post-infusion
measure: Number of HAE attacks per month (Weeks 1-16)
timeFrame: From study drug infusion up to week 16 post-infusion
Secondary outcomes:
measure: Change from baseline in total plasma kallikrein protein level
timeFrame: From NTLA-2002 infusion up to week 104 post-infusion
measure: Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
timeFrame: From NTLA-2002 infusion up to week 104 post-infusion
measure: Safety and tolerability of NTLA-2002 as determined by AEs
timeFrame: From study drug infusion up to week 104 post-infusion
measure: Number of HAE attacks per month (Weeks 5-16)
timeFrame: From week 6 post-infusion up to week 16 post-infusion
measure: Number of HAE attacks per month requiring acute therapy (Weeks 1-16, Weeks 5-16)
timeFrame: From study drug infusion up to week 16 post-infusion
Other outcomes:
Locations:
city: Campbelltown
country: Australia
facility: Clinical Trial Site
city: Grenoble
country: France
facility: Clinical Trial Site
city: Lille
country: France
facility: Clinical Trial Site
city: Paris
country: France
facility: Clinical Trial Site
city: Berlin
country: Germany
facility: Clinical Trial Site
city: Frankfurt
country: Germany
facility: Clinical Trial Site
city: Amsterdam
country: Netherlands
facility: Clinical Trial Site
city: Auckland
country: New Zealand
facility: Clinical Trial Site
city: Cambridge
country: United Kingdom
facility: Clinical Trial Site
Study officials:
References: