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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Therapy Information
Study Record Dates
Participation Criteria
Locations
Regulatory Information
Resources/Links
Summary
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
NCTID
NCT06634420
(View at clinicaltrials.gov)
Description
This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.
(Show More)
Development Status
Active
Indication
Hereditary Angioedema
Disease Ontology Term
DOID:14735
Compound Name
NTLA-2002
Sponsor
Intellia Therapeutics
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
60
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
50 mg
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase3
Submit Date
2024-10-07
Completion Date
2027-09
Last Update
2025-04-22
Participation Criteria
Eligible Age
>=16 Years
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
27
Locations
New Zealand,Canada,Netherlands,United States,South Africa,United Kingdom,Australia,France,Germany
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
First patient dosed January 2025; BLA submission planned H2 2026; link to NCT05120830
Resources/Links
Clinical Publications
CRISPR-Based Therapy for Hereditary Angioedema
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema
(Corporate Presentation) Results from Phase 2 Study of NTLA-2002 for Hereditary Angioedema - October 2024
News and Press Releases
Intellia Therapeutics Announces First Patient Dosed in the HAELO Phase 3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema
Protocol
Clinical Trial Protocol
Related NCTID
Phase 1/2: NCT05120830