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).
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.
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).
Parallel
NIO752
DrugAntisense Oligonucleotide Solution
Placebo
DrugPlacebo solution
6
Treatment groupsSee each treatment group below.
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.
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.
Change from baseline in clinical laboratory evaluations
Number of participants with laboratory test abnormalities.
Change from baseline in vital signs
Number of participants with clinically notable vital signs.
Change from baseline in ECG parameters
Number of participants with clinically notable ECG abnormalities.
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