RecruitingPhase 1NCT05231785

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)


Sponsor

Alnylam Pharmaceuticals

Enrollment

60 participants

Start Date

Feb 4, 2022

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study is evaluating the safety and tolerability of an investigational drug called ALN-APP — a gene-silencing therapy designed to reduce levels of amyloid precursor protein (APP) in the brain — in patients with early-onset Alzheimer's disease (EOAD), which affects people before age 65. **You may be eligible if...** - You have mild cognitive impairment or mild dementia believed to be caused by early-onset Alzheimer's disease - Your CDR (Clinical Dementia Rating) score is 0.5 or 1.0 (mild impairment) and MMSE score is above 20 - You meet biological criteria for Alzheimer's disease (e.g., biomarker confirmation) **You may NOT be eligible if...** - Your dementia is caused by something other than Alzheimer's disease - You have abnormal liver enzyme levels - You have significantly reduced kidney function - You are taking certain medications that could interact with the study drug Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGALN-APP

ALN-APP will be administered intrathecally (IT)

DRUGPlacebo

Placebo will be administered IT


Locations(8)

Clinical Trial Site

La Jolla, California, United States

Clinical Trial Site

Indianapolis, Indiana, United States

Clinical Trial Site

Montreal, Canada

Clinical Trial Site

Toronto, Canada

Clinical Trial Site

Amsterdam, Netherlands

Clinical Trial Site

Groningen, Netherlands

Clinical Trial Site

Huntley Street, United Kingdom

Clinical Trial Site

Sheffield, United Kingdom

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

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NCT05231785


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