Long-Term Follow-Up of Subjects Treated With NTLA-2001

• Research type

  Research Study

• Full title

  Long-Term Follow-Up of Subjects Treated With NTLA 2001

• IRAS ID

  1006472

• Contact name

  Robert Fairweather Graham

• Contact email

  uk-regulatory@medpace.com

• Sponsor organisation

  Intellia Therapeutics, Inc.

• Eudract number

  2022-003405-30

• Research summary

  Intellia Therapeutics, Inc. is developing NTLA-2001, an investigational CRISPR/Cas9-based genome editing therapy as a potential new treatment for ATTR with polyneuropathy (ATTR-PN) and cardiomyopathy (ATTR-CM). The primary objective of this long-term follow-up (LTFU) study (ITL-2001-CL-999) is to evaluate long-term safety of NTLA-2001 in previously treated subjects with ATTR-PN and/or ATTR-CM. Subjects will be followed for up to 15 years after the last NTLA-2001 infusion (inclusive of follow-up period from the primary/parent study) or for as long as required by national health authorities. No investigational product will be administered in this study.

• REC name

  Wales REC 1

• REC reference

  23/WA/0101

• Date of REC Opinion

  21 Aug 2023

• REC opinion

  Further Information Favourable Opinion