RecruitingPhase 1

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

United States, Canada, Netherlands60 participantsStarted 2022-02-04

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Part A: Frequency of Adverse Events

Timeframe: Up to 12 months

Part B: Frequency of Adverse Events

Timeframe: Up to 42 months

Trial details

NCT IDNCT05231785

SponsorAlnylam Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-04-20

Contact for this trial

Alnylam Clinical Trial Information Line

1-877-ALNYLAM clinicaltrials@alnylam.com

View on ClinicalTrials.gov More Early-Onset Alzheimer Disease trials