NCT05231785RecruitingPhase 1
A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
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)
Source: ClinicalTrials.gov · Last updated 20 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 60 (estimated)
- First submitted
- 30 Jan 2022
- Start date
- 4 Feb 2022
- Primary completion date
- 11 Jan 2029
- Lead sponsor
- Alnylam Pharmaceuticals
- Diseases
- Alzheimer disease
- NCT number
- NCT05231785
- EU CT number
- 2023-508363-79-00
- EudraCT number
- 2021-003198-74
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 60
- From 4 Feb 2022 to the primary completion date, 11 Jan 2029
- 7 sites in 4 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Has recently received an investigational agent
- Has recent treatment with amyloid-targeting antibody
- Note: other protocol defined inclusion / exclusion criteria apply
Other illness
- Has Non-Alzheimer's disease dementia
Lab values
- 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
Who the study is looking for
- 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
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - Coordinator of this site? Confirm its status | Montreal | Canada | Recruiting |
| - Coordinator of this site? Confirm its status | Toronto | Canada | Recruiting |
| - Coordinator of this site? Confirm its status | Huntley Street | United Kingdom | Recruiting |
| - Coordinator of this site? Confirm its status | Sheffield | United Kingdom | Recruiting |
| Brain Research Center Amsterdam B.V. Coordinator of this site? Confirm its status | Amsterdam | Netherlands | Recruiting |
| - Coordinator of this site? Confirm its status | Indianapolis | United States | Recruiting |
| - Coordinator of this site? Confirm its status | La Jolla | United States | Completed |
This page is information, not medical advice. Discuss any trial with your doctor.