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Looking for participantsPhase1

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

Sponsor: Alnylam Pharmaceuticals

NCT ID: NCT05231785

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
ALN-APP (drug), Placebo (drug)
How long the study runs
Study runs about 86 months (dates as stated)
About the drug or intervention
ALN-APP — drug: ALN-APP will be administered intrathecally (IT) · Placebo — drug: Placebo will be administered IT
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
18 Years and over
Who
All
Number of participants
60
Started
2022-02-04
Last checked
2026-09

Plain English Summary

What is this study?

  • • Testing a new treatment for early-onset alzheimer disease
  • • Phase1 - 60 participants
  • • 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)

Who can take part?

  • • Ages 18 Years and over
  • • Diagnosed with early-onset alzheimer disease

Where?

  • • Huntley Street - Clinical Trial Site
  • • Sheffield - Clinical Trial Site

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

Early-Onset Alzheimer Disease

How this trial compares with your answers

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What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 18 Years and over
  • Who can join: All genders

Biomarkers mentioned

eGFR

What the study is looking for

  • ✓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

Who cannot take part

  • ✗Has Non-Alzheimer's disease dementia
  • ✗Has alanine aminotransferase (liver enzymes) or aspartate aminotransferase (liver enzymes) \>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
See the full criteria
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 Note: other protocol defined inclusion / exclusion criteria apply

Where Is This Study? (2 UK sites)

Clinical Trial Site

Huntley Street WC1E 6AG, United Kingdom

Recruiting

Clinical Trial Site

Sheffield S10 2JF, United Kingdom

Recruiting

How to Get in Touch

Alnylam Clinical Trial Information Line

Sponsor contact

CONTACT

1-877-ALNYLAM clinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

Sponsor contact

CONTACT

1-877-256-9526 clinicaltrials@alnylam.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-09