NCT05469360

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

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: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 30 Years – 74 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

This is a phase 1b, randomized, double-blind, placebo-controlled study, in which 36 patients with early AD will be enrolled in one of three cohorts. Cohorts 1 \& 2 will receive a single intrathecal (IT) dose of NIO752 or placebo in the placebo-controlled part of the study, and multiple administrations of NIO752 in the open-label extension (OLE) part of the study. Cohort 3 will receive two single IT doses of NIO752 or placebo in the placebo-controlled part of the study, and a single administration of NIO752 in the OLE part of the study. Each cohort will enroll 12 participants, and they will be randomized into receiving NIO752 or placebo in 2:1 ratio. Participants in cohorts 1\& 2 will remain in this study for approximately \~19 months, including \~18 in-clinic follow up visits during that period of time. In cohort 3, participants will remain in this study for a follow up period of approximately \~18 months including \~10 in-clinic follow up visits. Cohorts will be enrolled sequentially. Participants who complete 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. All OLE participants will receive either two (cohorts 1 \& 2) or one (cohort 3) dose of NIO752. Study assessments will include physical and neurological examinations, ECGs, vital signs, standard clinical laboratory evaluations (hematology, blood chemistry, and urinalysis), routine laboratory evaluation of CSF collected through lumbar puncture, adverse event, and serious adverse event monitoring. Cohort 3 participation will also require 3 MRI scans and 3 PET scans throughout the 18 months of the study.

Conditions

• Alzheimer Disease
• Mild Cognitive Impairment

Interventions

Type	Name	Description
DRUG	NIO752	A single intrathecal (cerebrospinal) injection of NIO752 of Dose A
DRUG	NIO752	A single intrathecal (cerebrospinal) injection of NIO752 at dose B
DRUG	Matching placebo	A single intrathecal injection of matching placebo
DRUG	NIO752	Multiple intrathecal injections of NIO752 of Dose A
DRUG	NIO752	Two intrathecal injections of NIO752 at dose C
DRUG	NIO752	Single intrathecal injection of NIO752 at dose C
DRUG	Matching placebo	Two intrathecal injections of matching placebo

Timeline

• Start date: 2023-02-23
• Primary completion: 2026-09-11
• Completion: 2028-01-02
• First posted: 2022-07-21
• Last updated: 2025-08-28

Locations

10 sites across 4 countries: Finland, France, Spain, Sweden