A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease
A Phase 1, Randomized, Placebo-controlled Study With a Double Blind Period With Open-label Extension Period to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-5288 in Adult Patients With Alzheimer's Disease
Alnylam Pharmaceuticals
50 participants
Oct 15, 2025
INTERVENTIONAL
Conditions
Summary
The purpose of this study is to: * Evaluate the safety and tolerability of intrathecal (IT) ALN-5288 in patients with Alzheimer's Disease (AD) * Evaluate the pharmacodynamic (PD) and pharmacokinetic (PK) effects of ALN-5288 after dose administration
Eligibility
Inclusion Criteria3
- Is able and willing to meet all study requirements in the opinion of the Investigator
- Has a diagnosis of Alzheimer's disease (AD) based on clinical findings supported by cerebrospinal fluid (CSF) biomarkers or positive positron emission tomography (PET) amyloid imaging within 7 years prior to screening
- Has mild cognitive impairment (MCI) or dementia due to AD
Exclusion Criteria12
- Has non-AD dementia
- Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2× upper limit of normal (ULN)
- Has total bilirubin \>1.5×ULN
- Has known human immunodeficiency virus infection
- Has history of hepatitis C virus or current hepatitis B virus infection
- Has systolic blood pressure \>160 mmHg and/or a diastolic blood pressure \>100 mmHg after 10 minutes of rest at screening
- Has an estimated glomerular filtration (eGFR) of \<45 mL/min/1.73 m\^2 at screening
- Has clinically significant ECG abnormalities at screening
- Has uncontrolled psychiatric disease, including patients deemed by the Investigator to be at significant risk of suicide, major depressive episode, psychosis, confusional state, or violent behavior
- Has history of bleeding diathesis or coagulopathy due to chronic conditions
- Has a medical history of brain or spinal disease that would interfere with the IT injection and LP procedures
- Has history of uncontrolled seizures within the last 6 months prior to Screening
Interventions
ALN-5288 will be administered IT.
Placebo will be administered IT.
Locations(6)
View Full Details on ClinicalTrials.gov
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NCT07214727