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 groupsLocations
8Canada
Clinical Trial SiteH3A 2B4, Montreal Canada
Clinical Trial SiteM3B 2S7, Toronto Canada
Netherlands
Clinical Trial Site1081 BT, Amsterdam Netherlands
Clinical Trial Site9713 GG, Groningen Netherlands
Sponsors and collaborators
Alnylam Pharmaceuticals
Lead sponsor
Source ClinicalTrials.gov