Recruiting
Phase 1

ALN-APP

Sponsor:

Alnylam Pharmaceuticals

Code:

NCT05231785

Conditions

Early-Onset Alzheimer Disease

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

ALN-APP

Placebo

Study Details

Brief summary:

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

Study ID

NCT05231785

Start date

Feb 4, 2022

Status verified date

Oct, 2026

Completion date

Jan 11, 2029

Anticipated

Primary completion date

Jan 11, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

60 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Part A: ALN-APP

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

placebo comparator: Part A: Placebo

Participants will be administered a single dose of placebo.

experimental: Part B:

Participants will be administered multiple doses of ALN-APP.

Interventions

ALN-APP

ALN-APP will be administered intrathecally (IT)

Placebo

Placebo will be administered IT

Primary outcome measure

  • Part A: Frequency of Adverse Events [ Time Frame: Up to 12 months ]
  • Part B: Frequency of Adverse Events [ Time Frame: Up to 42 months ]

Central Contacts and Locations

Central contacts

Alnylam Clinical Trial Information Line

1-877-ALNYLAMclinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

1-877-256-9526clinicaltrials@alnylam.com

Locations

Clinical Trial Site

Recruiting

Indianapolis, Indiana, United States, 46202

Clinical Trial Site

Recruiting

Montreal, Canada, H3A 2B4

Clinical Trial Site

Recruiting

Toronto, Canada, M3B 2S7

More Information

Sponsor

Alnylam Pharmaceuticals

Last update posted

Oct 20, 2026

Last verified

Oct, 2026

Keywords

  • EOAD

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-22. This information was provided to ClinicalTrials.gov by Alnylam Pharmaceuticals on 2026-10-20. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.