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NCT Number: NCT07214727

A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease

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

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Key information

Conditions

Alzheimer's Disease Alzheimer Disease Brain Diseases Central Nervous System Diseases Dementia Mental Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases Tauopathies

Age range

40 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Clinical Trial Site, Montreal, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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 criteria

  • 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

Treatment and study plan

ALN-5288

Drug

ALN-5288 will be administered IT.

Placebo

Drug

Placebo will be administered IT.

Primary outcomes

  1. Frequency of Adverse Events (AEs)

    Time frame: Up to 32 months

  2. Severity of AEs

    Time frame: Up to 32 months

Secondary outcomes

  1. Concentration of Total Tau (t-Tau) Protein in CSF Over Time

    Time frame: Up to 32 months

  2. Change from Baseline in Concentration of t-Tau Protein in CSF Over Time

    Time frame: Up to 32 months

  3. Concentration of ALN-5288 in Plasma Over Time (Ct)

    Time frame: Up to 32 months

  4. Change from Baseline in Concentration of ALN-5288 CSF Ct

    Time frame: Up to 32 months

  5. Concentration of ALN-5288 in Plasma

    Time frame: Up to 32 months

  6. Concentration of ALN-5288 in Urine

    Time frame: Up to 32 months

  7. Concentration of ALN-5288 in CSF

    Time frame: Up to 32 months

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Trial Information Line

CONTACT

[email protected]

1-877-ALNYLAM

Clinical Trial Information Line

CONTACT

[email protected]

1-877-256-9526

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Collaborators

  • Regeneron Pharmaceuticals

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2030
Study completion
2030
First posted
Oct 9, 2025
Registry last updated
Jul 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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