Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy

A Randomized, Participant, Investigator and Sponsor Blinded, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of Intrathecally Administered NIO752 in Participants With Progressive Supranuclear Palsy Identifier: NCT04539041

Novartis Reference Number: CNIO752A02101

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

This is a phase 1, multi-center, double-blind, placebo-controlled, multiple dose escalation study with NIO752 in progressive supranuclear palsy (PSP) participants.

Progressive Supranuclear Palsy (PSP)
Phase 1
Overall status 
Not yet recruiting
Enrollment count 
64 participants
Start date 
Jan 19, 2021
Completion date 
Oct 17, 2023
40 Years - 75 Years (Adult, Older Adult)


antisense oligonucleotide
solution of antisense oligonucleotide injected intrathecally (spine tap) at multiple dose levels
matching placebo for each dose level

Eligibility Criteria

Inclusion Criteria:

Signed informed consent
Between 40 to 75 years old (inclusive)
Have PSP diagnosed for less than 5 years with a current classification of probable PSP Richardson syndrome, a progressive supranuclear palsy rating scale (PSPRS) score < 40 and MOCA score >17 at screening
Be able to ambulate independently or able to take at least 5 steps with minimal assistance
At least a 12-month history of postural instability or falls within 3 years from disease onset as per medical history
Vertical supranuclear gaze palsy, or reduced velocity of vertical saccade

Able and willing to meet all study requirements including:

Have a study partner who is reliable, competent, and at least 18 years of age, and will be able to accompany the participant to study visits, be knowledgeable of the participant's ongoing condition during the study to provide study related information to study site when required both in person and via a phone Reside in a proximity to the study site to allow a timely unscheduled visit if necessary (ideally less than 2 hours) Able to undergo lumbar puncture (LP), CSF draws and blood draws

If the participant is receiving levodopa/carbidopa, levodopa/benserazide, a dopamine agonist, catechol-o-methyltransferase (COMT) inhibitor, rasagiline, CoQ10 or other Parkinson's medications, acetylcholinesterase inhibitors, antipsychotics, memantine, or other non-tau modifying Alzheimer's medication the dose must have been stable for at least 30 days prior to the screening visit and must remain stable for the duration of the study. No such medication can be initiated during the study.

Exclusion Criteria:

Live in a skilled nursing facility or dementia care facility
Evidence of motor neuron disease, or any other neurological disease that could explain symptoms
Clinically significant laboratory abnormality
Attempted suicide, suicidal ideation with a plan that required hospital admission within 12 months prior to Screening. In addition, patients deemed by the Investigator to be at significant risk of suicide, major depressive episode, psychosis, confusion state, or violent behavior should be excluded.
A clear and robust benefit from levodopa by history
Use of lithium, methylene blue or other putative disease modifying drugs for PSP within 30 days of screening
Any previous use of experimental therapy within 30 days or 5 half-lives prior to Day 1, whichever is greater
Any condition that increases risk of meningitis unless participant is receiving appropriate prophylactic treatment
History of post-lumbar-puncture headache of moderate or severe intensity and/or blood patch

11. Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study 12. Unable to undergo magnetic resonance imaging (MRI) due to for example claustrophobia, or presents absolute contraindications to MRI (e.g., metallic implants, metallic foreign bodies, pacemaker, defibrillator) 13. Patients with other significant brain MRI abnormalities by history or at screening.


Novartis Pharmaceuticals
Novartis Pharmaceuticals

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