Health TrialsbyHealthPanel

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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

  • ALN-APP

    Drug

    ALN-APP will be administered intrathecally (IT)

  • Placebo

    Drug

    Placebo will be administered IT

Treatment groups

60 Participants
are divided into 3 treatment groups
Group A: Part A: ALN-APPExperimental treatment 1 intervention
Group B: Part A: PlaceboPlacebo comparator 1 intervention
Group C: Part B:Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Part A: Frequency of Adverse Events

Time frame
Up to 12 months
2

Part B: Frequency of Adverse Events

Time frame
Up to 42 months

Secondary outcomes

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 12 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 12 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 12 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 1 day

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Alnylam Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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