Progressive Supranuclear PalsyPhase 2NCT07868328
A Study to Evaluate the Safety, Tolerability and Pharmacodynamics of NIO752 in Primary Tauopathies
A Phase 2 study testing NIO752 in people with Progressive Supranuclear Palsy and Corticobasal Degeneration, run by Novartis Pharmaceuticals.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 41 to 81 years
- Conditions the study is for
- Progressive Supranuclear Palsy, Corticobasal Degeneration, Frontotemporal Lobar Degeneration
- What is being tested?
- NIO752
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- No sites listed yet
- Study dates
- 23 Oct 2026to 20 May 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 41 to 81 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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:
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- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
About this study
Official title: A Phase II, Randomized, Double Blind, Placebo-controlled, Basket Study to Evaluate the Safety, Tolerability and Pharmacodynamics of NIO752 in Participants With Primary Tauopathies
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).
Read the detailed description
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.
- Conditions
- Progressive Supranuclear Palsy, Corticobasal Degeneration, Frontotemporal Lobar Degeneration, Tauopathies
- Being tested
- NIO752, Placebo
- Keywords
- PSP non-Richardson syndrome (PSP non-RS), Corticobasal syndrome, amyloid-negative (CBS non-AD), MAPT mutation carriers (MAPT-FTLD), NIO752, Antisense Oligonucleotide (ASO), Microtubule Associated Protein Tau (MAPT), Double-blind, Intrathecal
- Medical subject headings
- Supranuclear Palsy, Progressive, Corticobasal Degeneration, Frontotemporal Lobar Degeneration, Tauopathies, Pick Disease of the Brain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NIO752Drug
Antisense Oligonucleotide Solution
PlaceboDrug
Placebo solution
| Study group | Role | What they receive |
|---|---|---|
| NIO752 - Cohort 1 (PSP non-RS) | Experimental | NIO752 solution Receives: Drug: NIO752 |
| Placebo - Cohort 1 (PSP non-RS) | Placebo comparator | Placebo solution Receives: Drug: Placebo |
| NIO752 - Cohort 2 (CBS non-AD) | Experimental | NIO752 solution Receives: Drug: NIO752 |
| Placebo - Cohort 2 (CBS non-AD) | Placebo comparator | Placebo solution Receives: Drug: Placebo |
| NIO752 - Cohort 3 (MAPT-FTLD) | Experimental | NIO752 solution Receives: Drug: NIO752 |
| Placebo - Cohort 3 (MAPT-FTLD) | Placebo comparator | Placebo solution Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) in double blind period
Number of participants with TEAEs including SAEs, AESIs, and AEs leading to treatment interruption/discontinuation or dose reduction.
Measured: From first treatment administration up to week 48
Secondary outcomes (9)
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) in open-label extension
Number of participants with TEAE, AESIs, SAEs, AEs leading to treatment interruption/discontinuation or dose reduction in OLE.
Measured: From first treatment administration in OLE period up to week 96
Change from baseline in clinical laboratory evaluations
Number of participants with laboratory test abnormalities.
Measured: From baseline up to week 96
Change from baseline in vital signs
Number of participants with clinically notable vital signs.
Measured: From baseline up to week 96
Change from baseline in ECG parameters
Number of participants with clinically notable ECG abnormalities.
Measured: From baseline up to week 96
Suicidality assessment using the C-SSRS
The number and percentage of participants reporting any suicidal ideation and behavior in Columbia Suicide Severity Rating Scale will be summarized
Measured: From baseline up to week 96
Change from baseline in target engagement biomarker
Change from baseline in biomarker as measure of target engagement.
Measured: Baseline, week 24, week 48 and week 84
Change from baseline in neurodegeneration biomarker
To assess NIO752 effect on neurodegeneration.
Measured: Baseline, week 24, week 48 and week 84
Pharmacokinetic concentrations of NIO752
Observed concentration of NIO752
Measured: Baseline, week 12, 24, 36, 48, 60, 72 and 84
NIO752 antidrug antibodies (ADA)
To assess immunogenicity of NIO752
Measured: Baseline, week 12, 24, 36, 48, 60, 72 and 84
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2026
- Study start23 Oct 2026 (estimated)
- Primary completion26 Mar 2029 (estimated)
- Study completion20 May 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Progressive Supranuclear PalsyStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Parallel arm basket trial with 3 cohorts for the double blind period
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07868328
- Sponsor's protocol ID
- CNIO752A12201
- Status last verified
- Oct 2026
- First submitted
- 5 Oct 2026
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Randomized, Double Blind, Placebo-controlled, Basket Study to Evaluate the Safety, Tolerability and Pharmacodynamics of NIO752 in Participants With Primary Tauopathies
- ClinicalTrials.gov ID
- NCT07868328
- Acronym / sponsor ID
- CNIO752A12201
- Conditions
- Progressive Supranuclear Palsy, Corticobasal Degeneration, Frontotemporal Lobar Degeneration, Tauopathies
- Keywords
- PSP non-Richardson syndrome (PSP non-RS), Corticobasal syndrome, amyloid-negative (CBS non-AD), MAPT mutation carriers (MAPT-FTLD), NIO752, Antisense Oligonucleotide (ASO), Microtubule Associated Protein Tau (MAPT), Double-blind, Intrathecal
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 23 Oct 2026 (estimated)
- Primary completion
- 26 Mar 2029 (estimated)
- Study completion
- 20 May 2030 (estimated)
- First posted
- 9 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 6
- Model description
- Parallel arm basket trial with 3 cohorts for the double blind period
| Arm | Type | Receives |
|---|---|---|
| NIO752 - Cohort 1 (PSP non-RS) | Experimental | Drug: NIO752NIO752 solution |
| Placebo - Cohort 1 (PSP non-RS) | Placebo comparator | Drug: PlaceboPlacebo solution |
| NIO752 - Cohort 2 (CBS non-AD) | Experimental | Drug: NIO752NIO752 solution |
| Placebo - Cohort 2 (CBS non-AD) | Placebo comparator | Drug: PlaceboPlacebo solution |
| NIO752 - Cohort 3 (MAPT-FTLD) | Experimental | Drug: NIO752NIO752 solution |
| Placebo - Cohort 3 (MAPT-FTLD) | Placebo comparator | Drug: PlaceboPlacebo solution |
Who may qualify
- Ages
- 41 to 81 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (14 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NIO752Antisense Oligonucleotide Solution | Drug | NIO752 - Cohort 1 (PSP non-RS), NIO752 - Cohort 2 (CBS non-AD), NIO752 - Cohort 3 (MAPT-FTLD) |
| PlaceboPlacebo solution | Drug | Placebo - Cohort 1 (PSP non-RS), Placebo - Cohort 2 (CBS non-AD), Placebo - Cohort 3 (MAPT-FTLD) |
Primary outcome
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) in double blind period
Time frame: From first treatment administration up to week 48
Number of participants with TEAEs including SAEs, AESIs, and AEs leading to treatment interruption/discontinuation or dose reduction.
9 secondary outcomes
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) in open-label extension
Time frame: From first treatment administration in OLE period up to week 96
Change from baseline in clinical laboratory evaluations
Time frame: From baseline up to week 96
Change from baseline in vital signs
Time frame: From baseline up to week 96
Change from baseline in ECG parameters
Time frame: From baseline up to week 96
Suicidality assessment using the C-SSRS
Time frame: From baseline up to week 96
Change from baseline in target engagement biomarker
Time frame: Baseline, week 24, week 48 and week 84
Change from baseline in neurodegeneration biomarker
Time frame: Baseline, week 24, week 48 and week 84
Pharmacokinetic concentrations of NIO752
Time frame: Baseline, week 12, 24, 36, 48, 60, 72 and 84
NIO752 antidrug antibodies (ADA)
Time frame: Baseline, week 12, 24, 36, 48, 60, 72 and 84
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2026.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals contact
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NIO752 in people with Progressive Supranuclear Palsy and Corticobasal Degeneration, run by Novartis Pharmaceuticals.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs) in double blind period (time frame: From first treatment administration up to week 48). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2026.