A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ALN-APP, Placebo.
- Who it may be relevant to
- Registry conditions: Early-Onset Alzheimer Disease. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Canada, Netherlands, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Randomized, Double-blind, Placebo-controlled Single Ascending Dose and Open-label Multi-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ALN-APP in Adult Patients With Early-onset Alzheimer's Disease (EOAD)
Overview
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.
Interventions
- Drug ALN-APP
ALN-APP will be administered intrathecally (IT) - Drug Placebo
Placebo will be administered IT
Primary outcome measures
- Part A: Frequency of Adverse Events [Time frame: Up to 12 months]
- Part B: Frequency of Adverse Events [Time frame: Up to 42 months]
Secondary outcome measures (5)
- Part A and Part B: Change from Baseline in Levels of Cerebrospinal Fluid (CSF) Soluble Amyloid Precursor Protein Alpha (sAPPα) and Soluble Amyloid Precursor Protein Beta (sAPPβ) [Time frame: Part A up to 12 months; Part B up to 24 months]
- Part A and Part B: Area Under the Plasma Concentration-time Curve (AUC) of ALN-APP and of Potential Metabolites [Time frame: Part A up to 12 months; Part B up to 24 months]
- Part A and Part B: Maximum Observed Plasma Concentration (Cmax) of ALN-APP and of Potential Metabolites [Time frame: Part A up to 12 months; Part B up to 24 months]
- Part A: Fraction of ALN-APP and Potential Metabolites Excreted in the Urine (fe) [Time frame: Up to 1 day]
- Part A and Part B: Concentration of ALN-APP and Potential Metabolites at Time 't' (Ct) in Plasma and CSF [Time frame: Part A up to 12 months; Part B up to 24 months]
Eligibility 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
Note: other protocol defined inclusion / exclusion criteria apply
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Triple blind
- Primary purpose
- Treatment
Study locations
United States · 2 centers
- Clinical Trial Site — La Jolla
- Clinical Trial Site — Indianapolis
Canada · 2 centers
- Clinical Trial Site — Montreal
- Clinical Trial Site — Toronto
Netherlands · 2 centers
- Clinical Trial Site — Amsterdam
- Clinical Trial Site — Groningen
United Kingdom · 2 centers
- Clinical Trial Site — Huntley Street
- Clinical Trial Site — Sheffield
Identifiers
NCT: NCT05231785 · ALN-APP-001 · 2023-508363-79-00