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Trial record 1 of 1 for:    NCT05231785
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A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

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ClinicalTrials.gov Identifier: NCT05231785
Recruitment Status : Recruiting
First Posted : February 9, 2022
Last Update Posted : June 6, 2022
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief 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.

Condition or disease Intervention/treatment Phase
Early-Onset Alzheimer Disease Drug: ALN-APP Drug: Placebo Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Maximum treatment duration for Part A: single dose. Maximum treatment for Part B: 12 months.
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
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)
Actual Study Start Date : February 4, 2022
Estimated Primary Completion Date : July 2025
Estimated Study Completion Date : July 2025

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Part A: ALN-APP
Participants will be administered a single dose of ALN-APP.
Drug: ALN-APP
ALN-APP will be administered intrathecally (IT)

Placebo Comparator: Part A: Placebo
Participants will be administered a single dose of placebo.
Drug: Placebo
Placebo will be administered IT

Experimental: Part B:
Participants will be administered multiple doses of ALN-APP.
Drug: ALN-APP
ALN-APP will be administered intrathecally (IT)




Primary Outcome Measures :
  1. Part A: Frequency of Adverse Events [ Time Frame: Up to 14 months ]
  2. Part B: Frequency of Adverse Events [ Time Frame: Up to 24 months ]

Secondary Outcome Measures :
  1. 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 ]
  2. 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 ]
  3. 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 ]
  4. Part A: Fraction of ALN-APP and Potential Metabolites Excreted in the Urine (fe) [ Time Frame: Up to 14 months ]
  5. 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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05231785


Contacts
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Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526 clinicaltrials@alnylam.com

Locations
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Canada, Ontario
Clinical Trial Site Recruiting
Aurora, Ontario, Canada
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05231785    
Other Study ID Numbers: ALN-APP-001
2021-003198-74 ( EudraCT Number )
First Posted: February 9, 2022    Key Record Dates
Last Update Posted: June 6, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alnylam Pharmaceuticals:
EOAD
Additional relevant MeSH terms:
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Alzheimer Disease
Dementia
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Tauopathies
Neurodegenerative Diseases
Neurocognitive Disorders
Mental Disorders