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

NCT ID: NCT05231785

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-02-04

Study Completion Date

2029-03-28

Brief Summary

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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.

Detailed Description

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Conditions

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Early-Onset Alzheimer Disease

Keywords

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EOAD

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Part A: ALN-APP

Participants will be administered a single dose of ALN-APP.

Group Type EXPERIMENTAL

ALN-APP

Intervention Type DRUG

ALN-APP will be administered intrathecally (IT)

Part A: Placebo

Participants will be administered a single dose of placebo.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo will be administered IT

Part B:

Participants will be administered multiple doses of ALN-APP.

Group Type EXPERIMENTAL

ALN-APP

Intervention Type DRUG

ALN-APP will be administered intrathecally (IT)

Interventions

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ALN-APP

ALN-APP will be administered intrathecally (IT)

Intervention Type DRUG

Placebo

Placebo will be administered IT

Intervention Type DRUG

Other Intervention Names

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mivelsiran

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals

Locations

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Clinical Trial Site

San Diego, California, United States

Site Status WITHDRAWN

Clinical Trial Site

San Diego, California, United States

Site Status RECRUITING

Clinical Trial Site

Indianapolis, Indiana, United States

Site Status RECRUITING

Clinical Trial Site

Toronto, Ontario, Canada

Site Status RECRUITING

Clinical Trial Site

Montreal, Quebec, Canada

Site Status RECRUITING

Clinical Trial Site

Amsterdam, , Netherlands

Site Status RECRUITING

Clinical Trial Site

London, , United Kingdom

Site Status RECRUITING

Clinical Trial Site

Sheffield, , United Kingdom

Site Status RECRUITING

Countries

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United States Canada Netherlands United Kingdom

Central Contacts

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Alnylam Clinical Trial Information Line

Role: CONTACT

Phone: 1-877-ALNYLAM

Email: [email protected]

Alnylam Clinical Trial Information Line

Role: CONTACT

Phone: 1-877-256-9526

Email: [email protected]

Other Identifiers

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2023-508363-79-00

Identifier Type: OTHER

Identifier Source: secondary_id

ALN-APP-001

Identifier Type: -

Identifier Source: org_study_id