This study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
37
CRISPR/Cas9 gene editing system delivered by LNP for IV administration
The administration of IV normal saline
Clinical Trial Site
Campbelltown, Australia
Clinical Trial Site
Grenoble, France
Clinical Trial Site
Lille, France
Clinical Trial Site
Paris, France
Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
(Phase 1 only)
Time frame: From NTLA-2002 infusion up to week 104 post-infusion
Number of HAE attacks per month (Weeks 1-16)
(Phase 2 only)
Time frame: From study drug infusion up to week 16 post-infusion
Change from baseline in total plasma kallikrein protein level
(Phase 1 \& 2)
Time frame: From NTLA-2002 infusion up to week 104 post-infusion
Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
(Phase 1 \& 2)
Time frame: From NTLA-2002 infusion up to week 104 post-infusion
Safety and tolerability of NTLA-2002 as determined by AEs
(Phase 2 only)
Time frame: From study drug infusion up to week 104 post-infusion
Number of HAE attacks per month (Weeks 5-16)
(Phase 2 only)
Time frame: From week 6 post-infusion up to week 16 post-infusion
Number of HAE attacks per month requiring acute therapy (Weeks 1-16, Weeks 5-16)
(Phase 2 only)
Time frame: From study drug infusion up to week 16 post-infusion
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Clinical Trial Site
Berlin, Germany
Clinical Trial Site
Frankfurt, Germany
Clinical Trial Site
Amsterdam, Netherlands
Clinical Trial Site
Auckland, New Zealand
Clinical Trial Site
Cambridge, United Kingdom