ClinicalTrials.gov record NCT07868328
A Study to Evaluate the Safety, Tolerability and Pharmacodynamics of NIO752 in Primary Tauopathies
A Phase II, Randomized, Double Blind, Placebo-controlled, Basket Study to Evaluate the Safety, Tolerability and Pharmacodynamics of NIO752 in Participants With Primary Tauopathies
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase II randomized study evaluates the safety, tolerability, and pharmacodynamics of NIO752 in people with primary tauopathies, including PSP non-RS, CBS non-AD, and MAPT-FTLD. Each of three parallel cohorts (about 16 participants per cohort) is randomized to NIO752 or placebo for a double-blind treatment period, followed by an open-label extension and a final follow-up. The study collects safety data, biomarkers, drug concentrations, and immunogenicity measures.
What it is studying. The study is testing NIO752’s safety and tolerability and measuring how the drug affects tau-related biomarkers (pharmacodynamics). It also measures drug levels over time (pharmacokinetics) and whether participants develop antidrug antibodies.
What the researchers hope to learn. To evaluate the safety and tolerability of NIO752; to assess pharmacodynamic effects on target engagement and neurodegeneration biomarkers; to characterize pharmacokinetics and immunogenicity; and to record adverse events during both the double-blind and open-label periods.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This is a phase II study intended to evaluate the safety, tolerability and pharmacodynamics of NIO752 in participants with primary tauopathies. Eligible participants will be randomized to receive either NIO752 or placebo during the double-blind treatment period followed by an Open-label Extension (OLE).
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- NIO752
- Placebo
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Common across all cohorts:
- Signed informed consent must be obtained prior to participation in the study.
- Participants between the ages of ≥41 and ≤81 years.
- Able to walk at least 10 steps with minimal assistance (stabilization of one arm or use of cane/walker).
- Documented symptom (clinical manifestation) onset in the past ≤5 years prior to screening, defined as the first emergence of disease related motor, cognitive, or behavioral symptoms that led to measurable impairment in instrumental or basic activities of daily living (iADLs/ADLs), based on clinical history or reliable informant report.
- Reliable study partner(s) such as spouse, sibling, close friend or partner, family member or caregiver who are able and willing to provide accurate information (including clinical symptoms and medical history) about the participant and to participate in study visits and informant-based assessments for the duration of the study. A reliable study partner is expected to spend sufficient time (near-daily contact) with the study participant
- If participant is receiving symptomatic medication, the dose must have been stable for at least 8 weeks prior to randomization.
- Cohort specific criteria:
- Inclusion criteria (PSP non-RS): Diagnosis of mild-moderate, probable PSP non-RS as per MDS-PSP 2017 criteria with symptoms onset ≤5 years prior to Screening.
- Inclusion criteria (CBS non-AD): Diagnosis of possible or probable CBD as per the 2013 consensus criteria, with CBS phenotype and symptom onset ≤5 years prior to screening. Individuals meeting criteria for probable CBD with non-CBS phenotypes (e.g., behavioral variant) are excluded to maintain consistency with the CBS non-AD target population.
- Inclusion criteria (MAPT-FTLD): Carriers of a known pathogenic MAPT mutation based on existing evidence of prior genetic testing.
Exclusion criteria
- Diagnosis of other significant neurological or psychiatric disorders including (but not limited to) Parkinson's' Disease (which has not subsequently been revised to a diagnosis of PSP); Alzheimer's disease (AD), dementia with Lewy bodies; prion disease; any psychotic disorders; severe Major Depressive Disorder (MDD); history of epilepsy or seizure disorder (excluding febrile seizures in childhood); brain tumor or other space occupying lesion (e.g., brain tumor, subdural hematoma, abscess, or any intracranial mass causing compression or structural distortion) ; history of clinically significant stroke (e.g., stroke with neurological deficit); history of head injury with loss of consciousness for at least 15 minutes within the past 20 years.
- Diagnosis of amyotrophic lateral sclerosis and/or motor neuron disease.
- Diagnosis of cerebellar ataxia, choreoathetosis, and early symptomatic autonomic dysfunction.
- History of or screening brain Magnetic Resonance Imaging (MRI) scan indicative of significant abnormality, including, but not limited to, prior intracerebral hemorrhage or infarct >1 cm diameter, >3 lacunar infarcts, cerebral contusion, aneurysm, vascular malformation >1 cm diameter, subdural hematoma, hydrocephalus, and space-occupying lesion (e.g., abscess or brain tumor).
- Contraindications to undergo MRI procedure, including metal (ferromagnetic) implants and/or a cardiac pacemaker that is not compatible with MRI.
- Medical conditions that would as per Investigator's judgement prevent the participant from undergoing lumbar puncture (LP), including but not limited to:
- History of/ known allergy to local anesthetic
- History of back surgery (except for microdiscectomy or laminectomy at level one)
- Spinal deformities that would interfere with i.t. administration or CSF flow
- Current dermatological infection at the LP site and/or significant skin alterations at the planned LP site:
- Presence of increased or uncontrolled bleeding and/or risk of bleeding that if not managed optimally could increase a participant's likelihood of procedural bleeding. These could include but are not limited to anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms) and underlying disorders of coagulation, platelet function or platelet count (e.g., abnormal coagulation parameters, hemophilia, Von Willebrand's disease, liver disease).
- Participants on anticoagulants (e.g., warfarin) or antiplatelets therapies [except for low dose aspirin (100 mg/day or lower) and low-dose ibuprofen (600 mg/day or lower) which are allowable], are not eligible to participate, unless temporal suspension of the anticoagulant or antiplatelet therapies for the purpose of the LP is considered safe for the patient, feasible, and guided by a hematologist.
- History of deep brain stimulator surgery other than sham surgery for participation in a deep brain stimulation clinical trial.
- Other protocol defined inclusion/exclusion criteria may apply
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
This is a randomized, double-blind (DB), placebo-controlled, parallel group, basket study to evaluate the safety, tolerability and pharmacodynamics of NIO752 in participants with PSP non-RS (Cohort 1), CBS non-AD (Cohort 2), and MAPT-FTLD (Cohort 3), followed by an OLE. The study participants (16 per cohort) will be randomly allocated to either NIO752 or placebo treatment. The study includes a screening period, a Double Blind treatment period, an Open-Label extension treatment period, and and a follow-up period until End-of-Study (EoS) visit.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will be randomized to receive either NIO752 or placebo during a double-blind treatment period, then may enter an open‑label extension and a final follow‑up/End‑of‑Study visit. The study includes a screening period and regular clinic visits with clinical assessments, questionnaires (including the Columbia Suicide Severity Rating Scale), vital signs, ECGs, and clinical laboratory tests; biomarker, pharmacokinetic, and antidrug‑antibody measurements are scheduled at multiple visits. The registry indicates that a brain MRI is referenced for screening/exclusion and that participants must be able to undergo lumbar puncture (LP); the record does not specify exactly how many MRIs or LPs will be done or their timing. A reliable study partner is expected to attend and provide informant information during study visits; the record does not describe other participant support or compensation.
How the study is structured. Randomized, quadruple-masked (participants, caregivers, investigators, and outcome assessors), placebo-controlled, parallel-group "basket" study with three disease cohorts (PSP non-RS, CBS non-AD, MAPT-FTLD). About 16 participants per cohort are assigned to NIO752 or placebo. The study includes a screening period, a double-blind treatment period, an open-label extension, and a follow-up up to an end-of-study visit.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- Which of the eligibility criteria would need to be checked with tests before I could join?
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Important dates
- Study start
- Oct 23, 2026 (estimated)
- Primary completion
- Mar 26, 2029 (estimated)
- Study completion
- May 20, 2030 (estimated)
- Target enrollment
- 48 participants (estimated)
- Last updated on registry
- Oct 9, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Novartis Pharmaceuticals
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Novartis Pharmaceuticals 📞 1-888-669-6682 ✉ novartis.email@novartis.com
- Novartis Pharmaceuticals 📞 +41613241111
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- pharmacodynamics
- What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- Open-label Extension
- A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- prior to screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- Cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
- Inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Magnetic Resonance Imaging
- A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
- Contraindications
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- lumbar puncture
- A procedure in which a needle is placed between the bones of the lower back to collect spinal fluid or to give a treatment.
- sham
- A procedure designed to resemble the real procedure, for example the same visit and equipment, without its active part. It is the procedure equivalent of a placebo.
- exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- placebo-controlled
- A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
- parallel group
- Participants are divided into two or more groups, and each group receives a different intervention at the same time, so the groups can be compared.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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