A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

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.

Conditions

Early-Onset Alzheimer Disease

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Study Overview

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Study Details

Study overview

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.

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)

A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

Condition
Early-Onset Alzheimer Disease
Intervention / Treatment

-

Contacts and Locations

San Diego

Clinical Trial Site, San Diego, California, United States, 92037

San Diego

Clinical Trial Site, San Diego, California, United States, 92103

Indianapolis

Clinical Trial Site, Indianapolis, Indiana, United States, 46202

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Alnylam Pharmaceuticals,

Medical Director, STUDY_DIRECTOR, Alnylam Pharmaceuticals

Study Record Dates

2027-07-01