A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of NIO752 in Participants with Progressive Supranuclear Palsy

Overview

About this study

The main purpose of this multi-center, randomized, placebo-controlled, participant, investigator and sponsor blinded study is to evaluate the safety, tolerability and pharmacokinetics of NIO752 in Progressive Supranuclear Palsy (PSP) participants when administered intrathecally once every 4 weeks (four administrations) in a range of escalating doses.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

1. Signed informed consent

2. Between 40 to 75 years old (inclusive)

3. 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

4. Be able to ambulate independently or able to take at least 5 steps with minimal
assistance

5. At least a 12-month history of postural instability or falls within 3 years from
disease onset as per medical history

6. Vertical supranuclear gaze palsy, or reduced velocity of vertical saccade

7. 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

8. 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:

1. Live in a skilled nursing facility or dementia care facility

2. Evidence of motor neuron disease, or any other neurological disease that could explain
symptoms

3. Clinically significant laboratory abnormality

4. 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.

5. A clear and robust benefit from levodopa by history

6. Use of lithium, methylene blue or other putative disease modifying drugs for PSP
within 30 days of screening

7. Any previous use of experimental therapy within 30 days or 5 half-lives prior to Day
1, whichever is greater

8. Any condition that increases risk of meningitis unless participant is receiving
appropriate prophylactic treatment

9. 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.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Eligibility last updated 3/21/23. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

James Bower, M.D.

Closed for enrollment

Contact information:

Sherry Klingerman CCRP

(507) 284-0451

klingerman.sherry@mayo.edu

More information

Publications

Publications are currently not available