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Clinical Trial Report
Gene Therapy Trial Report
Summary
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
NCTID
NCT05120830
(View at clinicaltrials.gov)
Description
This study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).
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Development Status
Active
Indication
Hereditary Angioedema
Disease Ontology Term
DOID:14735
Compound Name
NTLA-2002
Sponsor
Intellia Therapeutics
Funder Type
Industry
Recruitment Status
Active not recruiting
Enrollment Count
37
Results Posted
Not Available
Therapy Information
Target Gene/Variant
KLKB1
Therapy Type
Gene editing
Therapy Route
In-vivo
Mechanism of Action
Gene inactivation
Route of Administration
Intravenous
Drug Product Type
MRNA, LNP
Target Tissue/Cell
Hepatocyte
Delivery System
Lipid encapsulation
Vector Type
LDLR
Editor Type
Cas9 mRNA
Dose 1
25 mg
Dose 2
50 mg (pivotal dose)
Dose 3
75 mg
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2021-11-03
Completion Date
2026-03-31
Last Update
2024-09-19
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
9
Locations
New Zealand,Netherlands,United Kingdom,Australia,France,Germany
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
BLA submission planned 2026; Phase II study met all primary and secondary endpoints, selected pivotal dose
Resources/Links
Clinical Publications
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema
News and Press Releases
(Corporate Presentation) NTLA-2002 Long-Term Phase 1 Data Update from the Ongoing Phase 1/2 Study
Intellia Therapeutics Announces Second Quarter 2024 Financial Results and Highlights Recent Company Progress
Protocol
Clinical Trial Protocol