[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100659627":3},{"organization":4,"outcomesModule":7,"designInfo":46,"detailedDescription":59,"studyPopulation":45,"armGroups":60,"interventions":85,"overallOfficials":45,"centralContacts":94,"locations":45,"responsibleParty":102,"collaborators":45,"id":104,"slug":105,"hasResults":106,"nctId":107,"briefTitle":108,"officialTitle":109,"acronym":45,"eligibilityCriteria":110,"healthyVolunteers":106,"sex":111,"minAge":112,"maxAge":113,"enrollmentInfo":114,"targetDuration":45,"studyType":117,"phases":118,"briefSummary":120,"conditions":121,"keywords":126,"overallStatus":134,"whyStopped":45,"lastUpdateSubmitDate":135,"lastUpdatePostDateStruct":136,"startDateStruct":139,"completionDateStruct":141,"leadSponsor":143,"locationsCount":45},{"fullName":5,"class":6},"Novartis","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":45},[9],{"measure":10,"description":11,"timeFrame":12},"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\u002Fdiscontinuation or dose reduction.","From first treatment administration up to week 48",[14,18,22,25,28,31,35,38,42],{"measure":15,"description":16,"timeFrame":17},"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\u002Fdiscontinuation or dose reduction in OLE.","From first treatment administration in OLE period up to week 96",{"measure":19,"description":20,"timeFrame":21},"Change from baseline in clinical laboratory evaluations","Number of participants with laboratory test abnormalities.","From baseline up to week 96",{"measure":23,"description":24,"timeFrame":21},"Change from baseline in vital signs","Number of participants with clinically notable vital signs.",{"measure":26,"description":27,"timeFrame":21},"Change from baseline in ECG parameters","Number of participants with clinically notable ECG abnormalities.",{"measure":29,"description":30,"timeFrame":21},"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",{"measure":32,"description":33,"timeFrame":34},"Change from baseline in target engagement biomarker","Change from baseline in biomarker as measure of target engagement.","Baseline, week 24, week 48 and week 84",{"measure":36,"description":37,"timeFrame":34},"Change from baseline in neurodegeneration biomarker","To assess NIO752 effect on neurodegeneration.",{"measure":39,"description":40,"timeFrame":41},"Pharmacokinetic concentrations of NIO752","Observed concentration of NIO752","Baseline, week 12, 24, 36, 48, 60, 72 and 84",{"measure":43,"description":44,"timeFrame":41},"NIO752 antidrug antibodies (ADA)","To assess immunogenicity of NIO752",null,{"allocation":47,"interventionModel":48,"interventionModelDescription":49,"primaryPurpose":50,"observationalModel":45,"timePerspective":45,"maskingInfo":51},"RANDOMIZED","PARALLEL","Parallel arm basket trial with 3 cohorts for the double blind period","TREATMENT",{"masking":52,"maskingDescription":53,"whoMasked":54},"QUADRUPLE","Double-blind for the core treatment period; open-label NIO752 for all participants in the extension; raters blinded to safety data.",[55,56,57,58],"PARTICIPANT","CARE_PROVIDER","INVESTIGATOR","OUTCOMES_ASSESSOR","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.\n\nThe study participants (16 per cohort) will be randomly allocated to either NIO752 or placebo treatment.\n\nThe 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.",[61,67,73,76,79,82],{"label":62,"type":63,"description":64,"interventionNames":65},"NIO752 - Cohort 1 (PSP non-RS)","EXPERIMENTAL","NIO752 solution",[66],"Drug: NIO752",{"label":68,"type":69,"description":70,"interventionNames":71},"Placebo - Cohort 1 (PSP non-RS)","PLACEBO_COMPARATOR","Placebo solution",[72],"Drug: Placebo",{"label":74,"type":63,"description":64,"interventionNames":75},"NIO752 - Cohort 2 (CBS non-AD)",[66],{"label":77,"type":69,"description":70,"interventionNames":78},"Placebo - Cohort 2 (CBS non-AD)",[72],{"label":80,"type":63,"description":64,"interventionNames":81},"NIO752 - Cohort 3 (MAPT-FTLD)",[66],{"label":83,"type":69,"description":70,"interventionNames":84},"Placebo - Cohort 3 (MAPT-FTLD)",[72],[86,91],{"type":87,"name":88,"description":89,"armGroupLabels":90,"otherNames":45},"DRUG","NIO752","Antisense Oligonucleotide Solution",[62,74,80],{"type":87,"name":92,"description":70,"armGroupLabels":93,"otherNames":45},"Placebo",[68,77,83],[95,100],{"name":96,"role":97,"phone":98,"phoneExt":45,"email":99},"Novartis Pharmaceuticals","CONTACT","1-888-669-6682","novartis.email@novartis.com",{"name":96,"role":97,"phone":101,"phoneExt":45,"email":45},"+41613241111",{"type":103,"investigatorFullName":45,"investigatorTitle":45,"investigatorAffiliation":45,"oldNameTitle":45,"oldOrganization":45},"SPONSOR","100659627","phase-2-a-study-to-evaluate-the-safety-tolerability-and-pharmacodynamics-of-nio752-in-primary-tauopathies-100659627",false,"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","Inclusion Criteria:\n\nCommon across all cohorts:\n\n1. Signed informed consent must be obtained prior to participation in the study.\n2. Participants between the ages of ≥41 and ≤81 years.\n3. Able to walk at least 10 steps with minimal assistance (stabilization of one arm or use of cane\u002Fwalker).\n4. 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\u002FADLs), based on clinical history or reliable informant report.\n5. 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\n6. If participant is receiving symptomatic medication, the dose must have been stable for at least 8 weeks prior to randomization.\n\nCohort specific criteria:\n\nInclusion 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.\n\nInclusion 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.\n\nInclusion criteria (MAPT-FTLD): Carriers of a known pathogenic MAPT mutation based on existing evidence of prior genetic testing.\n\nExclusion Criteria:\n\n1. 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.\n2. Diagnosis of amyotrophic lateral sclerosis and\u002For motor neuron disease.\n3. Diagnosis of cerebellar ataxia, choreoathetosis, and early symptomatic autonomic dysfunction.\n4. 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).\n5. Contraindications to undergo MRI procedure, including metal (ferromagnetic) implants and\u002For a cardiac pacemaker that is not compatible with MRI.\n6. Medical conditions that would as per Investigator's judgement prevent the participant from undergoing lumbar puncture (LP), including but not limited to:\n\n   * History of\u002F known allergy to local anesthetic\n   * History of back surgery (except for microdiscectomy or laminectomy at level one)\n   * Spinal deformities that would interfere with i.t. administration or CSF flow\n   * Current dermatological infection at the LP site and\u002For significant skin alterations at the planned LP site:\n\n     * Presence of increased or uncontrolled bleeding and\u002For 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).\n     * Participants on anticoagulants (e.g., warfarin) or antiplatelets therapies \\[except for low dose aspirin (100 mg\u002Fday or lower) and low-dose ibuprofen (600 mg\u002Fday 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.\n7. History of deep brain stimulator surgery other than sham surgery for participation in a deep brain stimulation clinical trial.\n\nOther protocol defined inclusion\u002Fexclusion criteria may apply","ALL","41 Years","81 Years",{"count":115,"type":116},48,"ESTIMATED","INTERVENTIONAL",[119],"PHASE2","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).",[122,123,124,125],"Progressive Supranuclear Palsy","Corticobasal Degeneration","Frontotemporal Lobar Degeneration","Tauopathies",[127,128,129,88,130,131,132,133],"PSP non-Richardson syndrome (PSP non-RS)","Corticobasal syndrome, amyloid-negative (CBS non-AD)","MAPT mutation carriers (MAPT-FTLD)","Antisense Oligonucleotide (ASO)","Microtubule Associated Protein Tau (MAPT)","Double-blind","Intrathecal","NOT_YET_RECRUITING","2026-10-05",{"date":137,"type":138},"2026-10-09","ACTUAL",{"date":140,"type":116},"2026-10-23",{"date":142,"type":116},"2030-05-20",{"name":96,"class":6}]