A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
ConditionEarly-Onset Alzheimer Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorAlnylam Pharmaceuticals
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.
Eligibility criteria
Qualifiers
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
Disqualifiers
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/1.73m² at Screening
Has recently received an investigational agent
Trial design
Treatments tested in this trial
- ALN-APP
- Placebo
Treatment groups
60 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov