A clinical trial to find out how well LOU064 (remibrutinib) works and how safe it is for people with secondary progressive multiple sclerosis

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]

Trial duration

21 Apr 2026 → ongoing

Decision date (initial)

2026-03-09

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2025-521546-23-00

WHO UTN

U1111-1326-4880

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacodynamic, Therapy, Pharmacokinetic, Pharmacogenetic, Efficacy, Others

To demonstrate efficacy of remibrutinib compared to placebo in delaying disability progression based on EDSS

Secondary objectives 2

1. To assess whether remibrutinib is superior to placebo in: o Delaying disability progression based on EDSS o Other clinical and MRI measures
2. To assess the safety and tolerability of remibrutinib

Conditions and MedDRA coding

Secondary progressive multiple sclerosis

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Food And Drug Administration

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 6

1. Signed informed consent must be obtained prior to any assessment performed.
2. Male or female participants aged 18-65 (inclusive) at Screening.
3. Diagnosis of SPMS according to the 2017 revised McDonald criteria (Thompson et al 2018) at Screening.
4. Absence of documented clinical relapses in the 24 months before Screening and randomization.
5. EDSS score of 3.0 to 6.0 (inclusive) at Screening.
6. Documented evidence of disability progression in the 12 months before Screening.

Exclusion criteria 7

1. Unwilling or unable to undergo MRI scans as per protocol (for example claustrophobia, or presents absolute contraindications to MRI (e.g., metallic implants, metallic foreign bodies, pacemaker, defibrillator)).
2. History of clinically significant CNS disease (e.g. stroke, traumatic brain or spinal injury, history or presence of myelopathy) or neurological disorders which may mimic MS at Screening.
3. Ongoing substance abuse (drug or alcohol) or any other factor (e.g. serious psychiatric condition) that may interfere with the participant’s ability to cooperate and comply with the study procedures.
4. Participants with history of confirmed Progressive Multifocal Leukoencephalopathy (PML) or neurological symptoms consistent with PML
5. Women of childbearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they are using highly effective methods of contraception (failure rate < 1% per year) while taking study treatment and for at least 1 week after stopping study treatment
6. Significant bleeding risk or coagulation disorders, at Screening.
7. Use of exclusionary medication prior to Screening/randomization as listed in the protocol.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Time to 6-month confirmed disability progression (6mCDP) on Expanded Disability Status Scale (EDSS)

Secondary endpoints 8

1. Time to 3-month confirmed disability progression (3mCDP) on EDSS (key secondary)
2. Time to 6-month confirmed disability improvement (6mCDI) on EDSS (key secondary)
3. Time to 3-month worsening by at least 20% in Timed 25-Foot Walk (T25FW) (key secondary)
4. Time to 3-month worsening by at least 20% in 9-Hole Peg Test (9-HPT) (key secondary)
5. Annualized rate of new or enlarging T2 lesions (key secondary)
6. Percentage of participants with annualized rate of brain atrophy > XX (key secondary)
7. Time to 6-month worsening by at least 4 points in Symbol Digit Modalities Test (SDMT)
8. Adverse events, laboratory data, vital signs, electrocardiogram (ECG), Columbia Suicide Severity Rating Scale (C-SSRS)

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 15

Locations

23 EU/EEA countries · 164 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 281 files

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

Application history

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