Clinical trial • Phase III • Neurology|Rare Disease

NIO752 for Progressive supranuclear palsy - Richardson syndrome (PSP-RS)

Phase III trial of NIO752 for Progressive supranuclear palsy - Richardson syndrome (PSP-RS).

Overview

Trial Therapeutic Area
Neurology|Rare Disease
Trial Disease
Progressive supranuclear palsy - Richardson syndrome (PSP-RS)
Trial Stage
Phase III
Drug Modality
Oligonucleotide
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
10-02-2026
First CTIS Authorization Date
21-04-2026

Trial design

Randomised, placebo: isotone kochsalz-lösung 0,9 % braun infusionslösung (sodium chloride), intrathecal use; dose and schedule not specified in the record.-controlled Phase III trial across 41 sites in Belgium, Germany, France and others.

Randomised
Yes
Comparator
Placebo: Isotone Kochsalz-Lösung 0,9 % Braun Infusionslösung (sodium chloride), intrathecal use; dose and schedule not specified in the record.
Target Sample Size
149
Trial Duration For Participant
504

Eligibility

Recruits 149 The trial selects a vulnerable population (isVulnerablePopulationSelected = true). Signed informed consent must be obtained prior to participation in the study. A reliable study partner (spouse, sibling, close friend, or caregiver) is required to provide information and participate in informant-based assessments and study visits (expected to spend at least 5 hours per week with participant). Study partner informed consent forms / subject information forms are included in country documents..

Vulnerable Population
The trial selects a vulnerable population (isVulnerablePopulationSelected = true). Signed informed consent must be obtained prior to participation in the study. A reliable study partner (spouse, sibling, close friend, or caregiver) is required to provide information and participate in informant-based assessments and study visits (expected to spend at least 5 hours per week with participant). Study partner informed consent forms / subject information forms are included in country documents.

Inclusion criteria

  • {"criterion_text":"- Signed informed consent must be obtained prior to participation in the study."}
  • {"criterion_text":"- Male or female participants, age between 41-81 years old inclusive."}
  • {"criterion_text":"- Diagnosis of mild-moderate, probable/possible PSP Richardson syndrome as per MDS- PSP 2017 criteria with symptoms onset < 5 years"}
  • {"criterion_text":"- PSPRS total score less than 40 at Baseline"}
  • {"criterion_text":"- 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"}
  • {"criterion_text":"- 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)"}
  • {"criterion_text":"- MMSE score ≥ 20 at Screening."}

Exclusion criteria

  • {"criterion_text":"- 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."}
  • {"criterion_text":"- Diagnosis of amyotrophic lateral sclerosis or other motor neuron diseases."}
  • {"criterion_text":"- Diagnosis of cerebellar ataxia, choreoathetosis, and early symptomatic autonomic dysfunction."}
  • {"criterion_text":"- History of or screening brain MRI scan indicative of significant abnormality, including, but not limited to, prior 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)."}
  • {"criterion_text":"- Contraindications to undergo MRI procedure, including metal (ferromagnetic) implants and/or a cardiac pacemaker that is not compatible with MRI."}
  • {"criterion_text":"- History of deep brain stimulator surgery other than sham surgery for participation in a deep brain stimulation clinical trial."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change from baseline at Week 72 in the rPSPRS-10 score","definition_or_measurement_approach":"Change from baseline at Week 72 measured using the rPSPRS-10 score (comparison of score at Week 72 vs baseline)."}

Secondary endpoints

  • {"endpoint_text":"- Change from baseline at Week 72 in the PSPRS-28","definition_or_measurement_approach":"Change from baseline at Week 72 measured using the PSPRS-28 score."}
  • {"endpoint_text":"- Changes from baseline at Week 72 in activities of daily living on the XXX","definition_or_measurement_approach":"Change from baseline at Week 72 in activities of daily living measured by the specified XXX instrument."}
  • {"endpoint_text":"- Change from baseline at Week 72 in the Progressive Supranuclear Palsy Quality of Life scale (PSP-ShoQoL)","definition_or_measurement_approach":"Change from baseline at Week 72 measured using the PSP-ShoQoL scale."}
  • {"endpoint_text":"- Change from baseline at Week 72 in the Category Fluency, Phonemic Fluency, Symbol Digit Modality Test (SDMT), and Letter-Number- Sequencing (LNS)","definition_or_measurement_approach":"Change from baseline at Week 72 measured using Category Fluency, Phonemic Fluency, SDMT and LNS cognitive tests."}
  • {"endpoint_text":"- Changes from baseline in volumes of ventricles, whole brain, midbrain, pons, superior cerebellar peduncle, third ventricle, frontal lobe as measured by MRI until Week 72.","definition_or_measurement_approach":"MRI-assessed volumetric changes from baseline to Week 72 in listed brain regions."}
  • {"endpoint_text":"- Safety and tolerability parameters including AEs, AESIs, SAEs (including SAEs with fatal outcomes), AEs leading to XXX and treatment interruptions, AEs leading to dose discontinuation, clinical laboratory evaluations, vital signs, electrocardiogram (ECG), XXX","definition_or_measurement_approach":"Standard safety and tolerability assessments including adverse events (AEs), adverse events of special interest (AESIs), serious adverse events (SAEs), laboratory tests, vital signs and ECGs collected throughout the study."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
149
Recruitment Window Months
57
Consent Approach
Signed informed consent must be obtained prior to participation. Adult main ICFs are provided (country-specific ICF documents present). Study partner informed consent / information sheets are provided for study partners; study partner involvement is required. Country ICFs and information sheets available in multiple languages as provided in documents (examples include English, Dutch, French, German, Spanish, Italian in the document list). Follow-up information sheets for pregnant participants and female partners are also provided where applicable.

Methods

  • Patient recruitment material and activities provided by third parties (e.g. Jumo Health USA Inc. listed with role 'Patient Recruitment material').
  • Site-level and country-level advertisements and recruitment arrangements (documents K1/K2 recruitment and advertisement files listed for multiple countries).
  • Patient reimbursement and transportation support provided by Greenphire LLC (listed in third parties).

Geography

Total Number Of Sites
41
Total Number Of Participants
138

Belgium

Earliest CTIS Part Ii Submission Date
01-04-2026
Latest Decision Or Authorization Date
22-04-2026
Processing Time Days
21
Number Of Sites
2
Number Of Participants
9

Sites

Site Name
AZ ST-JAN Brugge A.V.
Department Name
2050 : Neurology
Contact Person Name
Bruno Bergmans
Contact Person Email
bruno.bergmans@azsintjan.be
Site Name
UZ Leuven
Department Name
2050 : Neurology
Contact Person Name
Wim Vandenberghe
Contact Person Email
wim.vandenberghe@uzleuven.be

Germany

Earliest CTIS Part Ii Submission Date
30-03-2026
Latest Decision Or Authorization Date
23-04-2026
Processing Time Days
24
Number Of Sites
11
Number Of Participants
38

Sites

Site Name
LMU Klinikum Muenchen AöR
Department Name
3002 : Neurologische Klinik und Poliklinik
Contact Person Name
Guenter Hoeglinger
Site Name
Universitaetsklinikum Tuebingen AöR
Department Name
3014 : Neurologische Universitätsklinik
Contact Person Name
Benjamin Roeben
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
3008 : Klinik fuer Neurologie
Contact Person Name
Patricia Krause
Contact Person Email
patricia.krause@charite.de
Site Name
Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.
Department Name
3005 : NA
Contact Person Name
Eva Annika Spottke
Contact Person Email
Annika.spottke@dzne.de
Site Name
Asklepios Fachklinikum Stadtroda GmbH
Department Name
3011 : Klinik fuer Neurologie und Schmerztherapie
Contact Person Name
David Weise
Contact Person Email
Da.weise@asklepios.com
Site Name
Kliniken Beelitz GmbH
Department Name
3007 : Klinik fuer Neurologie
Contact Person Name
Katarina Rukavina
Contact Person Email
rukavina@kliniken-beelitz.de
Site Name
Universitaetsklinikum Duesseldorf AöR
Department Name
3006 : Zentrum für Bewegungsstörungen und Neuromodulation
Contact Person Name
Alfons Schnitzler
Contact Person Email
Alfons.schnitzler@hhu.de
Site Name
Universitaetsklinikum Leipzig AöR
Department Name
3013 : Klinik und Poliklinik fuer Neurologie
Contact Person Name
Jost-Julian Rumpf
Site Name
Universitaetsklinikum Wuerzburg AöR
Department Name
3012 : Neurologische Klinik und Poliklinik
Contact Person Name
Jens Volkmann
Contact Person Email
volkmann_j@ukw.de
Site Name
Universitaetsklinikum Ulm AöR
Department Name
3004 : Klinik für Neurologie
Contact Person Name
Olga Vintonyak
Contact Person Email
olga.vintonyak@uni-ulm.de
Site Name
Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
Department Name
3001 : Klinik und Poliklinik fuer Neurologie
Contact Person Name
Elisabeth Dinter

France

Earliest CTIS Part Ii Submission Date
10-04-2026
Latest Decision Or Authorization Date
27-04-2026
Processing Time Days
17
Number Of Sites
12
Number Of Participants
30

Sites

Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
4002: Neurologie
Contact Person Name
Mathieu ANHEIM
Site Name
Centre Hospitalier Universitaire De Rennes
Department Name
4006 : Neurologie
Contact Person Name
Sophie DRAPIER
Contact Person Email
Sophie.drapier@chu-rennes.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
4010: Neurologie
Contact Person Name
Alice DORMEUIL
Contact Person Email
Alice.dormeuil@aphp.fr
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
4005 : Neurologie et pathologies du mouvement
Contact Person Name
Luc DEFEBVRE
Contact Person Email
luc.defebvre@chru-lille.fr
Site Name
Centre Hospitalier Regional De Marseille
Department Name
4007: Neurologie et pathologies du mouvement
Contact Person Name
Alexandre EUSEBIO
Contact Person Email
Alexandre.eusbio@ap-hm.fr
Site Name
Assistance Publique Hopitaux De Paris (Paris site)
Department Name
4001: Neurologie
Contact Person Name
Jean-Christophe CORVOL
Contact Person Email
Jean-christophe.corvol@aphp.fr
Site Name
Hospices Civils De Lyon
Department Name
4012: Neurologie
Contact Person Name
Stephane THOBOIS
Contact Person Email
Stephane.thobois@chu-lyon.fr
Site Name
Centre Hospitalier Universitaire De Caen Normandie
Department Name
4011: Neurologie
Contact Person Name
Claire THIRIEZ
Contact Person Email
thiriez-c@chu-caen.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
4008: Neurologie
Contact Person Name
Fabienne ORY-MAGNE
Contact Person Email
ory.f@chu-toulouse.fr
Site Name
Centre Hospitalier Universitaire De Nantes
Department Name
4003 : Neurologie
Contact Person Name
Philippe DAMIER
Contact Person Email
Philippe.damier@chu-nantes.fr
Site Name
Centre Hospitalier Universitaire De Nimes
Department Name
4004 : Neurologie
Contact Person Name
Marie De VERDAL
Contact Person Email
Marie.deverdal@chu-nimes.fr
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
4009: Neurologie
Contact Person Name
Pauline PRIN
Contact Person Email
p-prin@chu-montpellier.fr

Italy

Earliest CTIS Part Ii Submission Date
01-04-2026
Latest Decision Or Authorization Date
24-04-2026
Processing Time Days
23
Number Of Sites
5
Number Of Participants
20

Sites

Site Name
IRCCS Ospedale Policlinico San Martino
Department Name
6002: Clinica Neurologica
Contact Person Name
Roberta Marchese
Site Name
Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d'Aragona
Department Name
6005: Neurologia
Contact Person Name
Marina Picillo
Contact Person Email
mpicillo@unisa.it
Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
6003: U.O. Neurologia Dip. Neuroscienze
Contact Person Name
Anna Rita Bentivoglio
Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
6001: SC Neurologia 1 - Parkinson e Disturbi del Movimento
Contact Person Name
Roberto Eleopra
Site Name
Azienda Ospedaliera di Padova
Department Name
6004: Dipartimento di Neuroscienze
Contact Person Name
Angelo Antonini
Contact Person Email
angelo.antonini@unipd.it

Spain

Earliest CTIS Part Ii Submission Date
02-04-2026
Latest Decision Or Authorization Date
27-04-2026
Processing Time Days
25
Number Of Sites
9
Number Of Participants
32

Sites

Site Name
Hospital Universitario Quironsalud Madrid
Department Name
9006: Neurology
Contact Person Name
Rafael Arroyo Gonzalez
Contact Person Email
rafaelarroyo09@gmail.com
Site Name
Hospital Universitario 12 De Octubre
Department Name
9008: Neurology
Contact Person Name
Antonio José Méndez Guerrero
Site Name
Hospital Clinic De Barcelona
Department Name
9004: Neurology
Contact Person Name
Yaroslau Compta Hirnyj
Contact Person Email
YCOMPTA@clinic.cat
Site Name
Hospital General Universitario Gregorio Maranon
Department Name
9005: Neurology
Contact Person Name
Francisco Javier Grandas Perez
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
9003: Neurology
Contact Person Name
Laura Munoz Delgado
Contact Person Email
lmunoz-ibis@us.es
Site Name
El Hospital Universitario De Gran Canaria Dr. Negrin
Department Name
9009: Neurology
Contact Person Name
Ayoze Nauzet González Hernández
Contact Person Email
ayozegonzalez@hotmail.com
Site Name
Hospital Universitari General De Catalunya
Department Name
9007: Neurology
Contact Person Name
Ernest Balaguer Martinez
Contact Person Email
ebalaguer@quironsalud.es
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
9001: Neurology
Contact Person Name
Jose Luis Lopez-Sendon Moreno
Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
9002: Neurology
Contact Person Name
Javier Pagonabarraga Mora
Contact Person Email
JPagonabarraga@santpau.cat

Netherlands

Earliest CTIS Part Ii Submission Date
01-04-2026
Latest Decision Or Authorization Date
21-04-2026
Processing Time Days
20
Number Of Sites
2
Number Of Participants
9

Sites

Site Name
Radboud universitair medisch centrum Stichting
Department Name
2060 : Neurology
Contact Person Name
Bas Bloem
Contact Person Email
bas.bloem@radboudumc.nl
Site Name
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department Name
2061 : Neurology
Contact Person Name
Agnita Boon
Contact Person Email
a.j.w.boon@erasmusmc.nl

Sponsor

Primary sponsor

Full Name
Novartis Pharma AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
IQVIA Limited
Responsibilities
ECG vendor (sponsorDuties entry includes value 'ECG vendor'); other contract research services (sponsorDuties codes present).
Name
Icon Clinical Research Limited
Responsibilities
Sponsor duties present (sponsorDuties code 1) - specific responsibility text not provided in record.
Name
Parexel International (IRL) Limited
Responsibilities
Sponsor duties present (sponsorDuties code 12) - specific responsibility text not provided in record.
Name
Syneos Health Inc.
Responsibilities
Sponsor duties present (sponsorDuties code 1) - specific responsibility text not provided in record.
Name
Eresearchtechnology Inc.
Responsibilities
PRO management, PRO licensing, formatting and translations, e-data capture

Third parties

  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"ECG vendor","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"Patient Recruitment material","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"DATAMAP-Gesellschaft fuer Datenmanagement Datenanalyse und Datenpraesentation mbH","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"PRO management, PRO licensing, formatting and translations, e-data capture","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient reimbursement and transportation support","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}

Investigational products

Investigational Product Name
NIO752
Active Substance
NIO752
Modality
Oligonucleotide
Routes Of Administration
Intrathecal use
Route
Intrathecal
Authorisation Status
Not authorised (prodAuthStatus = 1)
Orphan Designation
Yes
Investigational Product Name
Isotone Kochsalz-Lösung 0,9 % Braun Infusionslösung
Active Substance
SODIUM CHLORIDE
Modality
Small molecule
Routes Of Administration
Intrathecal use
Route
Intrathecal
Authorisation Status
Authorised/marketed (marketingAuthNumber present, prodAuthStatus = 2)

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