This Phase III study is intended to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy (PSP). Eligible participants will be randomized to receive either NIO752 or placebo followed by an open-label extension.
Eligibility
Sex
ALL
Min age
41 Years
Max age
81 Years
Healthy volunteers
No
Inclusion Criteria:
1. Signed informed consent must be obtained prior to participation in the study.
2. Male or female participants, age between 41-81 yrs inclusive.
3. Diagnosis of mild-moderate, probable/possible PSP Richardson syndrome as per MDS-PSP 2017 criteria with symptoms onset \< 5 years.
4. PSPRS total score less than 40 at Baseline.
5. Reliable study partner such as spouse, sibling, close friend, or caregiver 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 enough time (at least 5 hours per week) with the study participant.
6. Participant is able to ambulate defined as the ability to take at least 10 steps independently or with minimal assistance (stabilization of one arm to minimize fall risk).
7. Mini Mental State Examination (MMSE) score ≥ 20 at Screening.
Exclusion Criteria:
1. Diagnosis of other significant neurological or psychiatric disorders including (but not limited to) Parkinsons' 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; seizure; brain tumor or other space-occupying lesion; history of clinically significant stroke (e.g., stroke with permanent neurological deficit); history of head injury with loss of consciousness for at least 15 minutes within the past 20 years.
2. Diagnosis of amyotrophic lateral sclerosis or other motor neuron diseases.
3. Diagnosis of cerebellar ataxia, choreoathetosis, and early symptomatic autonomic dysfunction.
4. History of or screening brain MRI scan indicative of significant abnormality, including, but not limited to, prior hemorrhage or infarct \>1 cm3, \>3 lacunar infarcts, cerebral contusion, aneurysm, vascular malformation \>1 cm3, subdural hematoma, hydrocephalus, and space-occupying lesion (e.g., abscess or brain tumor).
5. Contraindications to undergo MRI procedure, including metal (ferromagnetic) implants and/or a cardiac pacemaker that is not compatible with MRI.
6. Medical conditions that would, as per Investigator's judgement, prevent the participant from undergoing lumbar puncture, including but not limited to:
1. Known allergy to local anesthetic
2. History of back surgery (with the exception of microdiscectomy or laminectomy over 1 level)
3. Spinal deformities
4. Current dermatological infection at the lumbar puncture spot and/or significant skin alterations at the planned puncture place
5. Risk of increased or uncontrolled bleeding and/or risk of bleeding that if not managed optimally, could place a participant at an increased risk for 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).
7. History of deep brain stimulator surgery other than sham surgery for participation in a deep brain stimulation clinical trial.
Other protocol-defined inclusion/exclusion criteria may apply
Primary outcome measure(s)
Change from baseline in the mPSPRS-10 score — Baseline, Week 72 The 10-item Progressive Supranuclear Palsy Rating Scale (mPSPRS-10) is a modified version of the original 28 item PSPRS developed to improve clinical meaningfulness and statistical performance. The 10 items measure three key motor domains: gait, limb function, and bulbar. The mPSPRS-10 ranges between 0 and 30, with higher scores indicating greater disability.
Trial sites (42)
Facility
City
Region
Status
Mayo Clinic Arizona
Scottsdale
Arizona
Recruiting
Univ of California San Francisco
San Francisco
California
Recruiting
CenExcel Rocky Mtn Clin Research
Englewood
Colorado
Recruiting
Mayo Jacksonville
Jacksonville
Florida
Recruiting
Mayo Clinic
Rochester
Minnesota
Recruiting
Novartis Investigative Site
Westmead
New South Wales
Recruiting
Novartis Investigative Site
Melbourne
Victoria
Recruiting
Novartis Investigative Site
Shanghai
China
Recruiting
Novartis Investigative Site
Suzhou
China
Recruiting
Novartis Investigative Site
Caen
France
Recruiting
Novartis Investigative Site
Créteil
France
Recruiting
Novartis Investigative Site
Lille
France
Recruiting
Novartis Investigative Site
Marseille
France
Recruiting
Novartis Investigative Site
Nantes
France
Recruiting
Novartis Investigative Site
Paris
France
Recruiting
Novartis Investigative Site
Rennes
France
Recruiting
Novartis Investigative Site
Toulouse
France
Recruiting
Novartis Investigative Site
Munich
Bavaria
Recruiting
Novartis Investigative Site
Würzburg
Bavaria
Recruiting
Novartis Investigative Site
Bonn
North Rhine-Westphalia
Recruiting
Novartis Investigative Site
Düsseldorf
North Rhine-Westphalia
Recruiting
Novartis Investigative Site
Dresden
Saxony
Recruiting
Novartis Investigative Site
Leipzig
Saxony
Recruiting
Novartis Investigative Site
Beelitz
Germany
Recruiting
Novartis Investigative Site
Berlin
Germany
Recruiting
Novartis Investigative Site
Stadtroda
Germany
Recruiting
Novartis Investigative Site
Tübingen
Germany
Recruiting
Klinik für Neurologie (Schwerpunkt Neurodegeneration)
Ulm
Germany
Recruiting
Novartis Investigative Site
Milan
MI
Recruiting
Novartis Investigative Site
Roma
RM
Recruiting
Novartis Investigative Site
Kodaira
Tokyo
Recruiting
Novartis Investigative Site
Rotterdam
South Holland
Recruiting
Novartis Investigative Site
Nijmegen
Netherlands
Recruiting
Novartis Investigative Site
Seoul
South Korea
Recruiting
Novartis Investigative Site
Pozuelo de Alarcón
Madrid
Recruiting
Novartis Investigative Site
Barcelona
Spain
Recruiting
Novartis Investigative Site
Barcelona
Spain
Recruiting
Novartis Investigative Site
Las Palmas GC
Spain
Recruiting
Novartis Investigative Site
Madrid
Spain
Recruiting
Novartis Investigative Site
Madrid
Spain
Recruiting
+ 2 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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