Clinical Trial

Disease: Hereditary Angioedema, HAE, (NCT06262399)

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:
Long-Term Follow-Up of Subjects Treated With NTLA 2002
Who:

Contact

Phone: 1-833-888-0387

Email: clinicalscience@intelliatx.com 

Partners:
Locations:

Grenoble, France
Centre National de Reference - Grenoble, Grenoble, France

Lille, France
Hôpital Claude Huriez, Lille, France

Auckland, New Zealand
New Zealand Clinical Research, Auckland, New Zealand

Cambridge, United Kingdom
Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital, Cambridge, United Kingdom
 

Study start:
Apr. 1, 2024
Enrollment:
100 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Knock-out
Gene:
Kallikrein B1 (KLKB1)
Delivery method:
Lipid nanoparticles (LNPs) - In-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Not yet recruiting

Description

This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.

Last updated: Mar. 11, 2024
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