Clinical Trial

Disease: Hereditary Angioedema, HAE, (NCT05120830)

Disease info:

Hereditary Angioedema (HAE) is a rare, genetic disorder characterised by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body. Currently available treatment options include life-long therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week, or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. 
 

Frequency:
It is estimated that one in 50,000 people are affected by HAE.
Official title:
Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Who:

Contact

Phone: 833-888-0387 

Email: clinicalscience@intelliatx.com

Partners:
Locations:

Australia
Campbelltown, Australia, Clinical Trial Site


France
Grenoble, France, Clinical Trial Site

Lille, France, Clinical Trial Site

Paris, France, Clinical Trial Site


Germany
Berlin, Germany, Clinical Trial Site

Frankfurt, Germany, Clinical Trial Site

 

Netherlands

Academic Medical Center - University of Amsterdam, Amsterdam, Netherlands

 

New Zealand

Study Site, Auckland, New Zealand

 

United Kingdom

Cambridge University Hospitals NHS Foundation Trust Addenbrookes Hospital, Cambridge, United Kingdom

Study start:
Dec. 10, 2021
Enrollment:
55
Gene editing method:
CRISPR-Cas9
Type of edit:
Knock-out
Gene:
Kallikrein B1 (KLKB1)
Delivery method:
Lipid Nano Particles (LNPs) - In-vivo
Safety updates:

(01-02-2024) CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema

(14-11-2023) Intellia Therapeutics Receives European Union Orphan Drug Designation for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema

(13-10-2023) Intellia Therapeutics Receives Priority Medicines (PRIME) Designation From the European Medicines Agency for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema

(11-06-2023) Intellia Therapeutics Announces New Positive Clinical Data from Phase 1 Study of NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema (HAE)

(21-03-2023) Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to NTLA-2002 for the Treatment of Hereditary Angioedema

(15-03-2023) Intellia Therapeutics Gets FDA Green Light to Initiate U.S. CRISPR Trial for Hereditary Angioedema

(02-03-2023) Intellia Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for NTLA-2002, an In Vivo CRISPR-Based Investigational Therapy for the Treatment of Hereditary Angioedema (HAE)

(12-11-2022) Intellia Therapeutics Presents New Interim Data from First-in-Human Study of NTLA-2002 for the Treatment of Hereditary Angioedema (HAE) at the American College of Allergy, Asthma & Immunology 2022 Annual Scientific Meeting

(13-12-2021) Intellia Therapeutics Announces First Patient Dosed in Phase 1/2 Clinical Trial of NTLA-2002 for the Treatment of Hereditary Angioedema

(6-10-2021) Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-2002 for the Treatment of Hereditary Angioedema

IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Last updated: Feb. 21, 2024
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