A Phase 1 Study to Evaluate ALN-5288 in Patients with Alzheimer’s Disease

• Research type

  Research Study

• Full title

  A Phase 1, Randomized, Placebo-controlled Study with a Double-blind Period with Open-label Extension Period to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-5288 in Adult Patients with Alzheimer’s Disease

• IRAS ID

  1012935

• Contact name

  Andrew Slugg

• Contact email

  aslugg@alnylam.com

• Sponsor organisation

  Alnylam Pharmaceuticals, Inc

• Clinicaltrials.gov Identifier

  NCT07214727

• Research summary

  Alzheimer’s Disease is the leading cause of cognitive impairment and dementia in older individuals throughout the world. Alzheimer’s Disease is a degenerative disease progressing from symptoms of mild cognitive impairment (MCI), to dementia. One of the contributors of disease progression in Alzheimer’s is thought to be the depositing and clumping together of ‘tau’ proteins in the brain.
  The new drug being investigated in this study, ALN-5288, works by interfering with messages from a gene that makes ‘tau’ protein which is then thought to reduce the amount of ‘tau’ protein being made.
  This research study is testing the safety and effectiveness of ALN-5288 to find out what effects, if any, ALN-5288 has on people with Alzheimer’s Disease.
  Participants are expected to be in the study for up to 14 months with the option to extend for up to 32 months and they will be randomised (chosen by chance, like rolling a dice) to receive either the study drug ALN-5288 or a placebo.
  The study will be separated into 3 periods:
  • Double-blind treatment period (neither the participant or the study doctor will know if the participant is receiving the study drug or a placebo)
  • Recovery period – During this period participants will not receive any study medications (ALN-5288 or placebo) and will just have their health monitored
  • After the recovery period, eligible participants will progress to an open-label extension period where all participants will receive the study drug 50 people are expected to take part in several countries across the world.
  Study procedures will include: Blood, urine and cerebrospinal fluid (CSF) collections, physical and neurological examinations, MRI scans, PET scans, questionnaires and cognitive assessments.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  25/LO/0767

• Date of REC Opinion

  18 Nov 2025

• REC opinion

  Further Information Favourable Opinion