A Phase 1 interventional study of NIO752 and NIO752 in Alzheimer Disease and Mild Cognitive Impairment, sponsored by Novartis Pharmaceuticals. Active, not recruiting at 10 sites in 4 countries. Open to participants aged 30 Years to 74 Years. Per ClinicalTrials.gov, last updated 2026-09-18.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 1, Interventional, and Treatment
Phase 1b study to assess the pharmacodynamics, safety, tolerability, and pharmacokinetics of NIO752 in patients with early Alzheimer's disease (AD)
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.
3,965 studies on the registry are indexed under Alzheimer Disease; 926 are open to participants now.
This study's enrollment of 38 is below the median of 70 across 3,019 interventional studies indexed under Alzheimer Disease.
Browse Alzheimer Disease studies →Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Main Inclusion Criteria (placebo-controlled part):
Main Inclusion Criteria (OLE part):
Main Exclusion Criteria (placebo-controlled part):
Main Exclusion Criteria (OLE part):
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A single intrathecal injection of Dose A
Drug: NIO752
A single intrathecal injection of artificial cerebrospinal fluid (CSF)
Drug: Matching placebo
A single intrathecal injection of Dose B
Drug: NIO752
A single intrathecal injection of artificial cerebrospinal fluid (CSF)
Drug: Matching placebo
Multiple intrathecal injections of Dose A
Drug: NIO752
Two intrathecal injections of Dose C
Drug: NIO752
Two intrathecal injections of artificial cerebrospinal fluid (CSF)
Drug: Matching placebo
Single intrathecal injection of Dose C
Drug: NIO752
A single intrathecal (cerebrospinal) injection of NIO752 of Dose A
A single intrathecal (cerebrospinal) injection of NIO752 at dose B
A single intrathecal injection of matching placebo
Multiple intrathecal injections of NIO752 of Dose A
Two intrathecal injections of NIO752 at dose C
Single intrathecal injection of NIO752 at dose C
Two intrathecal injections of matching placebo
Change in cerebrospinal total tau from baseline to Day 85
Total tau protein levels in cerebrospinal fluid. More frequent timepoints might be added as deemed necessary per the site Investigator's judgment.
Time frame: Baseline, Day 85
Number of adverse events and serious adverse events
Adverse events are assessed at each clinic visit. Laboratory values and other safety assessment values considered clinically significant by the investigator and meet the definition of adverse event will be reported.
Time frame: Cohort 1 & 2: Baseline up to 170 days (placebo-controlled part) and up to 589 days (OLE part), Cohort 3: Baseline up to 169 days (placebo-controlled part) and up to 507 days (OLE part)
Concentration of NIO752 in cerebrospinal fluid (CSF)
Concentration of NIO752 in CSF
Time frame: Cohorts 1 & 2: Pre-dose, Days 57, 85, 170 (placebo-controlled part) and Days 252, 420, and 588 (OLE part), Cohort 3: Pre-dose, day 1, 57, 85, 169 (placebo-controlled part) and Days 388 and 506 (OLE part)
Cmax, Ctrough in plasma
Maximum and trough level concentrations of NIO752 in plasma
Time frame: Cohorts 1, 2 & 3: Pre-dose, 0.5, 1, 2, 3 4, 5, 6, 24 hours post first dose. Cohort 1 & 2: Days 14, 57, 85 and 170 (pbo-controlled part) and 252, 253, 420, 421, and 588 (OLE) Cohort 3: Days 14, 57, 85 and 169 (pbo-controlled part) and 338 and 506 (OLE)
Tmax in blood plasma
Time of Cmax in plasma post-IT injection
Time frame: Cohorts 1, 2 & 3: Pre-dose, 0.5, 1, 2, 3 4, 5, 6, 24 hours post first dose. Cohort 1 & 2: Days 14, 57, 85 and 170 (pbo-controlled part) and 252, 253, 420, 421, and 588 (OLE) Cohort 3: Days 14, 57, 85 and 169 (pbo-controlled part) and 338 and 506 (OLE)
AUC-last in blood plasma
Area under curve (AUC) from time zero to the last measurable concentration sampling time (t-last) (mass x time x volume-1)
Time frame: Cohorts 1, 2 & 3: Pre-dose, 0.5, 1, 2, 3 4, 5, 6, 24 hours post first dose. Cohort 1 & 2: Days 14, 57, 85 and 170 (pbo-controlled part) and 252, 253, 420, 421, and 588 (OLE) Cohort 3: Days 14, 57, 85 and 169 (pbo-controlled part) and 338 and 506 (OLE)
AUC-inf in blood plasma
The AUC from time zero to infinity (mass x time x volume-1)
Time frame: Cohorts 1, 2 & 3: Pre-dose, 0.5, 1, 2, 3 4, 5, 6, 24 hours post first dose. Cohort 1 & 2: Days 14, 57, 85 and 170 (pbo-controlled part) and 252, 253, 420, 421, and 588 (OLE) Cohort 3: Days 14, 57, 85 and 169 (pbo-controlled part) and 338 and 506 (OLE)
Change in CSF total Tau
Evaluating the ability of NIO752 to lower CSF total tau in participants with early AD when administered as multiple doses
Time frame: Cohorts 1 & 2: Pre-dose, Days 57, 85, 170 (placebo-controlled part) and Days 252, 420, and 588 (OLE part), Cohort 3: Pre-dose, day 1, 57, 85, 169 (placebo-controlled part) and Days 388 and 506 (OLE part)
Plan to share: No
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This study is active, not recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: NIO752