A Study to Evaluate ALN-XDH in Healthy Subjects and Patients With Gout

• Research type

  Research Study

• Full title

  A Phase 1/2, Randomized, Double-blind, Placebo-controlled, Single Ascending and Multiple Dose Study of the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALN-XDH in Healthy Adult Subjects and Adult Patients with Gout

• IRAS ID

  1004578

• Contact name

  Debi Tran

• Contact email

  detran@alnylam.com

• Sponsor organisation

  Alnylam Pharmaceuticals, Inc.

• Eudract number

  2021-005773-68

• Clinicaltrials.gov Identifier

  NCT05256810

• Research summary

  The purpose of the study is to evaluate if the study drug, ALN-XDH, is safe when administered to healthy volunteers and whether it may be beneficial for patients with gout.
  Gout is the most common form of inflammatory joint disease in adults and includes symptoms such as joint pain and swelling. Gout can occurs when levels of uric acid, a naturally occurring molecule, build up in the blood. Uric acid is produced in the body by a protein called xanthine dehydrogenase (XDH).
  ALN-XDH is an investigational RNAi therapeutic designed to reduce the production of the XDH. This is expected to reduce levels of uric acid and intense joint pain resulting from gout. It has not yet been approved by health authorities, including MHRA, UK for the treatment of gout or other conditions.
  The study is a Phase 1/2, Randomized, Double-blind, Placebo-controlled trial. This means that neither the participants nor the investigator or study team know who is receiving a particular treatment. Patients will be put into separate groups to receive either ALN-XDH or placebo. The placebo will look exactly like the study drug but will be made of saline.
  This Phase 1/2 study will be conducted in 3 parts:
  • Part A: Single Ascending Dose (SAD) phase in healthy volunteers
  • Parts B and C: Single and Multiple Dose (MD) in patients with gout
  The planned enrollment for this study is up to 113 healthy volunteers in Part A and up to 200 patients with gout in Part B and 80 patients in Part C. Part A of the study will be conducted at Richmond Pharmacology in the UK.
  Currently, only part A of the study is being conducted. Parts B and C will be conducted in the future.
  The sponsor of this study is Alnylam Pharmaceuticals, Inc

• REC name

  South Central - Berkshire B Research Ethics Committee

• REC reference

  22/SC/0003

• Date of REC Opinion

  15 Feb 2022

• REC opinion

  Further Information Favourable Opinion