NCT05231785 · Alnylam Pharmaceuticals
A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
What this study is about
The purpose of this study is to evaluate the safety, tolerability, how the drug affects the body (PD) and how the drug moves through the body (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.
View original scientific description
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.
Interventions
DRUG
ALN-APP
ALN-APP will be administered intrathecally (IT)
DRUG
Placebo
Placebo will be administered IT
Primary outcome measures
Part A: Frequency of Adverse Events
Time frame: Up to 12 months
Part B: Frequency of Adverse Events
Time frame: Up to 42 months
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
Where
- La Jolla, California
- Indianapolis, Indiana
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 15, 2026 · Source of record for eligibility and locations