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

  • End date
    May 25, 2024
  • participants needed
  • sponsor
    Novartis Pharmaceuticals
Updated on 10 March 2022
screening procedures
gaze palsy
coenzyme q


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


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

Approximately 64 PSP participants in 6 cohorts will be randomized to receive NIO752 or placebo in a ratio of 3:1. Intrathecal ( IT ) injections of NIO752 or placebo will be given 4 times over 3 months. All participants will remain in this study for an additional 9 month follow-up period with 6 scheduled visits.

Cohorts will be enrolled sequentially.

Safety assessments will include physical and neurological examinations, ECGs, vital signs, standard clinical laboratory evaluations (hematology, blood chemistry, and urinalysis), CSF laboratory test, adverse event, and serious adverse event monitoring.

Condition Progressive Supranuclear Palsy (PSP)
Treatment Placebo, antisense oligonucleotide, antisense oligonucleotide
Clinical Study IdentifierNCT04539041
SponsorNovartis Pharmaceuticals
Last Modified on10 March 2022


Yes No Not Sure

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
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
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