NTLA-2001 in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)

• Research type

  Research Study

• Full title

  Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN) and Patients with Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

• IRAS ID

  1003342

• Contact name

  Julian Gillmore

• Contact email

  j.gillmore@ucl.ac.uk

• Eudract number

  2020-002034-32

• Research summary

  This is a study of NTLA-2001 in patients with hereditary transthyretin amyloidosis with
  polyneuropathy (ATTRv-PN). NTLA-2001 is a highly specific genome editing genome
  editing therapeutic that is anticipated to substantially deplete production of misfolded
  TTR protein thereby decreasing disease progression due to amyloidosis. Patients aged 18
  to 80 years with a confirmed diagnosis of polyneuropathy due to ATTR amyloidosis are
  eligible to participate subsequent to meeting defined eligibility criteria. Patients shall be
  followed for a period of 24 months. The study shall be conducted in a non-NHS centre
  with patients identified by the chief investigator at the National Amyloidosis Centre
  (NAC).

• REC name

  North East - York Research Ethics Committee

• REC reference

  20/NE/0191

• Date of REC Opinion

  13 Oct 2020

• REC opinion

  Further Information Favourable Opinion