A Study of the Natural History of ALECT2 Disease

• Research type

  Research Study

• Full title

  A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis(ALECT2) Disease

• IRAS ID

  259612

• Contact name

  Julian Gillmore

• Contact email

  j.gillmore@ucl.ac.uk

• Sponsor organisation

  Alnylam Pharmaceuticals

• Duration of Study in the UK

  5 years, 2 months, 1 days

• Research summary

  Amyloidosis refers to a group of medical conditions caused by proteins that fold abnormally, this generates piles of proteins called fibrils that can accumulate in different organs. Overtime however, the fibrils can pile on top of each other into amyloid plaques. These can cause damage or failure of the tissue or organs.
  
  Leukocyte Chemotactic Factor 2, or LECT2, is the name of the protein that is produced in the Liver which abnormally fold, causing fibrils in Leukocyte Chemotactic Factor 2 Amyloidosis, also called ALECT2. The LECT2 fibrils build up in the kidney, reducing kidney function and cause chronic kidney disease (CKD) and can eventually lead to kidney failure. We currently do not know why some people have LECT2 that fold abnormally causing fibril build up and damage while other people have normal LECT2.

  Alnylam Pharmaceutical, Inc., want to better understand why ALECT2 develops, and what happens to people with ALECT2 over time. They have therefore designed a natural history study to observe and collected information on people who have ALECT2. Once ALECT2 is better understood, we hope to discover better methods of diagnosis and eventual therapies to treat individuals with ALECT2.

  This is a global, natural history study with a planned enrolment of 150 participants including approximately 75 participants who are not End-Stage Renal Disease (ESRD), who will be followed prospectively. The study consists of 2 parts:
  - Screening (3 months)
  - Observational Period (approximately 4 years)

  The participant is expected to be in the study for approximately 51 months in total. During this time the participant will attend study visits every 6 months at the hospital. No study participants will be administered an investigational medicine during the study.

  This natural history study aims to better understand why ALECT2 develops, and what happens to people with ALECT2 over time.

• REC name

  London - Queen Square Research Ethics Committee

• REC reference

  19/LO/0760

• Date of REC Opinion

  24 Jun 2019

• REC opinion

  Further Information Favourable Opinion