ALN-APP-001 - A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients with EOAD

• Research type

  Research Study

• Full title

  A Randomized, Double-blind, Placebo-controlled Single Ascending Dose and Open-label Multi-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-APP in Adult Patients with Early-onset Alzheimer’s Disease (EOAD)

• IRAS ID

  1004512

• Contact name

  Kerry Ward

• Contact email

  kerry.ward@iqvia.com

• Sponsor organisation

  IQVIA Ltd

• Eudract number

  2021-003198-74

• Clinicaltrials.gov Identifier

  NCT05231785

• Research summary

  This study is investigating the safety, tolerability and effects of a new drug called ALN-APP in participants with Early-onset Alzheimer’s disease (EOAD). The study is being conducted to understand if ALN-APP is safe and well tolerated in people with EOAD and to collect initial data on how ALN-APP affects the body. Approximately 60 study participants will participate worldwide.

  Potential participants may be able to take part if they:
  • are 18 years or older
  • have experienced symptoms of EOAD at 65 years or younger (either mild cognitive impairment or mild dementia)

  There are two parts to this study, Part A and Part B:
  • Part A includes 4 planned groups: 2 groups (1 and 2) will enrol 6 participants who will be randomised, with 4 participants receiving a single dose of ALN-APP and 2 participants receiving placebo; 2 groups (3 and 4) will enrol 8 participants who will be randomised, with 6 participants receiving a single dose of ALN-APP and 2 participants receiving placebo. Up to 4 optional groups each with 8 participants randomised in a 6:2 ratio to receive a single dose of ALN-APP or placebo may be added.
  • Part B: All participants in this part of the study will receive ALN-APP.
  Participants may be able to enter Part B after completing Part A.

  A single intrathecal administration (injection into the fluid around spinal cord) of ALN-APP or placebo will be given to participants in Part A. Multiple intrathecal administrations of ALN-APP will be given to participants in Part B:
  Every 3 months (5 injections)
  Every 4 months (4 injections)
  Every 6 months (3 injections)

  The estimated duration of Part A for each participant is up to 14 months.
  The estimated duration of Part B for each participant is up to 24 months (26 months for replacement participants)

• REC name

  Yorkshire & The Humber - Leeds East Research Ethics Committee

• REC reference

  22/YH/0019

• Date of REC Opinion

  9 Mar 2022

• REC opinion

  Further Information Favourable Opinion