A Phase III, randomized, placebo-controlled, parallel group, double-blind study to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy followed by an Open Label Extension

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2026-04-22

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2025-523481-24-00

WHO UTN

U1111-1331-9092

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To evaluate the efficacy of NIO752 compared to placebo on disease progression using rPSPRS-10 at week 72

Secondary objectives 8

1. To evaluate the efficacy of NIO752 compared to placebo on disease progression using PSPRS-28 at week 72
2. To assess the effect of NIO752 compared to placebo on activities of daily living
3. To assess the effect of NIO752 compared to placebo on Quality of life (QOL)
4. To assess the effect of NIO752 compared to placebo on cognition
5. To assess the effect of NIO752 compared to placebo on XXX
6. To assess the effect of NIO752 compared to placebo on XXX of target engagement
7. To assess the effect of NIO752 compared to placebo on specific brain region volumes
8. To assess the safety and tolerability of NIO752 compared to placebo

Conditions and MedDRA coding

Participants with Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration, European Medicines Agency

Plan to share IPD

Yes

IPD plan description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Signed informed consent must be obtained prior to participation in the study.
2. Male or female participants, age between 41-81 years old 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. MMSE score ≥ 20 at Screening.

Exclusion criteria 6

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 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).
5. Contraindications to undergo MRI procedure, including metal (ferromagnetic) implants and/or a cardiac pacemaker that is not compatible with MRI.
6. History of deep brain stimulator surgery other than sham surgery for participation in a deep brain stimulation clinical trial.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline at Week 72 in the rPSPRS-10 score

Secondary endpoints 6

1. Change from baseline at Week 72 in the PSPRS-28
2. Changes from baseline at Week 72 in activities of daily living on the XXX
3. Change from baseline at Week 72 in the Progressive Supranuclear Palsy Quality of Life scale (PSP-ShoQoL)
4. Change from baseline at Week 72 in the Category Fluency, Phonemic Fluency, Symbol Digit Modality Test (SDMT), and Letter-Number- Sequencing (LNS)
5. 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.
6. 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

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

Sponsor organisation

Novartis Pharma AG

Address

Lichtstrasse 35

City

Basel

Postcode

4056

Country

Switzerland

Scientific contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Public contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Third parties 11

Locations

6 EU/EEA countries · 41 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 76 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications