Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease Participants

Alzheimer Disease Clinical Trial

Official title: A Randomized, Participant and Investigator Blinded, Placebo-Controlled Study to Evaluate the Ability of a Single Intrathecally Administered Dose of NIO752 to Lower Cerebrospinal Fluid Total Tau Levels in Participants With Early Alzheimer's Disease

Status Active, not recruiting

Clinical Trial Summary

Phase 1b study to assess the pharmacodynamics, safety, tolerability, and pharmacokinetics of NIO752 in patients with early Alzheimer's disease (AD)

Clinical Trial Description

This is a phase 1b, randomized, double-blind, placebo-controlled study, in which patients with early AD will receive a single intrathecal (IT) dose of NIO752 in the placebo-controlled part of the study and multiple administrations of NIO752 in the open-label extension (OLE) part of the study.

A total of 24 participants will be enrolled into one of two cohorts (each with 12 participants) and randomized into receiving one dose of NIO752 or placebo in 2:1 ratio. Participants will remain in this study for a 170-day follow-up period including approximately 3 in-clinic visits.

Cohorts will be enrolled sequentially.

Participants who complete Day 170 visit of the placebo-controlled part of the study will be eligible to continue in an OLE part of the study regardless of randomization assignment in the placebo-controlled part. Participants in the OLE part will receive NIO752 IT injections at approximately 252 and 420 days after Day 1 (first IT injection).

Study assessments will include pharmacokinetics (PK), physical and neurological examinations, ECGs, vital signs, standard clinical laboratory evaluations (hematology, blood chemistry, and urinalysis), CSF routine laboratory test, adverse event, and serious adverse event monitoring. ;

Related Conditions & MeSH terms

• Alzheimer Disease
• Cognitive Dysfunction
• Mild Cognitive Impairment

• NCT number: NCT05469360
• Study type: Interventional
• Source: Novartis
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: February 23, 2023
• Completion date: October 21, 2025