- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05231785
A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
February 12, 2024 updated by: Alnylam Pharmaceuticals
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)
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.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
60
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Alnylam Clinical Trial Information Line
- Phone Number: 1-877-ALNYLAM
- Email: clinicaltrials@alnylam.com
Study Contact Backup
- Name: Alnylam Clinical Trial Information Line
- Phone Number: 1-877-256-9526
- Email: clinicaltrials@alnylam.com
Study Locations
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Ontario
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Toronto, Ontario, Canada
- Recruiting
- Clinical Trial Site
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Quebec
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Montréal, Quebec, Canada
- Recruiting
- Clinical Trial Site
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Amsterdam, Netherlands
- Recruiting
- Clinical Trial Site
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London, United Kingdom
- Recruiting
- Clinical Trial Site
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Indiana
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Indianapolis, Indiana, United States, 46202
- Recruiting
- Clinical Trial Site
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Part A: ALN-APP
Participants will be administered a single dose of ALN-APP.
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ALN-APP will be administered intrathecally (IT)
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Placebo Comparator: Part A: Placebo
Participants will be administered a single dose of placebo.
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Placebo will be administered IT
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Experimental: Part B:
Participants will be administered multiple doses of ALN-APP.
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ALN-APP will be administered intrathecally (IT)
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Part A: Frequency of Adverse Events
Time Frame: Up to 14 months
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Up to 14 months
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Part B: Frequency of Adverse Events
Time Frame: Up to 24 months
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Up to 24 months
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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 14 months; Part B up to 24 months
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Part A up to 14 months; Part B up to 24 months
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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 14 months; Part B up to 24 months
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Part A up to 14 months; Part B up to 24 months
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Part A and Part B: Maximum Observed Plasma Concentration (Cmax) of ALN-APP and of Potential Metabolites
Time Frame: Part A up to 14 months; Part B up to 24 months
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Part A up to 14 months; Part B up to 24 months
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Part A: Fraction of ALN-APP and Potential Metabolites Excreted in the Urine (fe)
Time Frame: Up to 14 months
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Up to 14 months
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Part A and Part B: Concentration at Time 't' (Ct) of ALN-APP and Potential Metabolites
Time Frame: Part A up to 14 months; Part B up to 24 months
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Part A up to 14 months; Part B up to 24 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Medical Director, Alnylam Pharmaceuticals
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
February 4, 2022
Primary Completion (Estimated)
July 1, 2025
Study Completion (Estimated)
July 1, 2025
Study Registration Dates
First Submitted
January 30, 2022
First Submitted That Met QC Criteria
January 30, 2022
First Posted (Actual)
February 9, 2022
Study Record Updates
Last Update Posted (Actual)
February 13, 2024
Last Update Submitted That Met QC Criteria
February 12, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- ALN-APP-001
- 2021-003198-74 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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