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

• Status: Not yet recruiting
• Trial end date: 2025-07-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single intrathecal (IT) doses of ALN-APP in adult patients with early-onset Alzheimer's Disease (EOAD). Maximum treatment duration for Part A: single dose. Maximum treatment for Part B: 12 months.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

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