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

NCT05231785 · tracked via the Priya Life Science UK tracker

Sponsor

Alnylam Pharmaceuticals

Phase

Phase 1

Started

2022-02-04

Last updated

2026-09-14

Condition(s) studied

Early-Onset Alzheimer Disease

Investigational drug(s) / intervention(s)

ALN-APPPlacebo

ALN-APP: ALN-APP will be administered intrathecally (IT)

Placebo: Placebo will be administered IT

Study summary

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose and multiple doses of ALN-APP administered by intrathecal (IT) injection in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose. Maximum treatment duration for Part B: 12 months.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

Inclusion Criteria: * Has mild cognitive impairment or mild dementia due to EOAD * Has Clinical Dementia Rating (CDR) global score 0.5 or 1.0 and Mini Mental State Examination (MMSE) \>20 Exclusion Criteria: * Has Non-Alzheimer's disease dementia * Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2×upper limit of normal (ULN) * Has estimated glomerular filtration rate (eGFR) \<45 mL/min/1.73m\^2 at Screening * Has recently received an investigational agent * Has recent treatment with amyloid-targeting antibody Note: other protocol defined inclusion / exclusion criteria apply

Primary outcome measure(s)

Part A: Frequency of Adverse Events — Up to 12 months
Part B: Frequency of Adverse Events — Up to 42 months

Trial sites (8)

Facility	City	Region	Status
Clinical Trial Site	La Jolla	California	Completed
Clinical Trial Site	Indianapolis	Indiana	Recruiting
Clinical Trial Site	Montreal	Canada	Recruiting
Clinical Trial Site	Toronto	Canada	Recruiting
Clinical Trial Site	Amsterdam	Netherlands	Recruiting
Clinical Trial Site	Groningen	Netherlands	Completed
Clinical Trial Site	Huntley Street	United Kingdom	Recruiting
Clinical Trial Site	Sheffield	United Kingdom	Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05231785 on ClinicalTrials.gov ↗ ← All trials in the UK