A Phase 3 study to assess efficacy, safety and tolerability of remibrutinib in adult and adolescent patients with moderate to severe hidradenitis suppurativa

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

28 May 2025 → ongoing

Decision date (initial)

2025-05-13

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Others, Safety

To demonstrate the efficacy of remibrutinib compared to placebo with respect to HiSCR50 after 16 weeks of treatment

Secondary objectives 2

1. To demonstrate the efficacy of remibrutinib compared to placebo after 16 weeks (unless specified otherwise) of treatment with respect to: ● proportion of participants with AN50 response ● percentage change from baseline in IHS4 ● proportion of participants with HiSCR75 response ● proportion of participants experiencing HS flares ● proportion of participants with HiSCR50 response at Week 8 ● proportion of participants with HiSCR90 response ● proportion of participants with clinical response in HS related skin pain (NRS 30), at worst
2. To demonstrate the safety and tolerability of remibrutinib

Conditions and MedDRA coding

Hidradenitis Suppurativa (HS)

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 4

1. Signed informed consent must be obtained prior to participation in the study. For participants aged ≥ 12 to < 18 years: parent’s or legal guardian’s signed written informed consent and child’s assent, if appropriate, must be obtained before any assessment is performed. Of note, if the participant reaches age of consent (age as per local law) during the study, they will also need to sign the corresponding study Informed Consent Form (ICF) at the next study visit.
2. Male and female participants ≥ 12 years of age at the time of signing of the informed consent forms.
3. Diagnosis of Hidradenitis Suppurativa (HS) based on clinical history and physical examination for at least 6 months prior to the Baseline visit.
4. Participants with moderate to severe HS at baseline defined as: • A total of at least 5 AN, i.e. abscesses and/or inflammatory nodules AND • Inflammatory lesions should affect at least 2 distinct anatomic areas (e.g., left and right axillae)

Exclusion criteria 13

1. Presence of more than 20 fistulae/tunnels (both draining and non-draining) in total at baseline.
2. Any active skin disease or conditions that may interfere with the assessment of HS.
3. Previous exposure to remibrutinib or other BTK inhibitors.
4. Use of other investigational drugs within 5 half-lives, or within 30 days (for small molecules) prior to randomization, or until the pharmacodynamic effect has returned to baseline (for biologics), whichever is longer.
5. Significant bleeding risk or coagulation disorders
6. History of gastrointestinal bleeding.
7. Requirement for anti-platelet (except for acetylsalicylic acid up to 100 mg/d or clopidogrel up to 75 mg/d) or anti-coagulant medication.
8. History or current hepatic disease.
9. Evidence of clinically significant cardiovascular, neurological, psychiatric, pulmonary, renal, hepatic, endocrine, metabolic, hematological disorders, gastrointestinal disease or immunodeficiency that, in the Investigator's opinion, would compromise the safety of the participant, interfere with the interpretation of the study results or otherwise preclude participation or protocol adherence of the participant.
10. History of hypersensitivity to any of the study drug constituents.
11. Known or suspected infectious disease that is active, chronic or recurrent which precludes the participant from participating in the trial as per investigator's assessment. These infectious diseases include and are not limited to opportunistic infections (e.g., tuberculosis, atypical mycobacterioses, listeriosis or aspergillosis) and/or known or suspected Human Immunodeficiency Virus (HIV) infection. Should it be required by local regulations and/or considered appropriate by the investigator, an HIV test can be performed to confirm eligibility.
12. History of live attenuated vaccine administration within 6 weeks prior to randomization or requirement to receive these vaccinations at any time while on study treatment
13. Major surgery within 8 weeks prior to screening or planned surgery for the duration of the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Achievement of HiSCR50 at Week 16. HiSCR50 is defined as at least a 50% decrease in Abscess and Inflammatory Nodule (AN) count with no increase in the number of abscesses and in the number of draining tunnels/fistulae compared to baseline.

Secondary endpoints 8

1. Achievement of AN50 at Week 16. AN50 is defined as at least a 50% decrease in Abscess and Inflammatory Nodule (AN) count compared to baseline
2. Percentage change from baseline in IHS4 at Week 16
3. Achievement of HiSCR75 at Week 16. HiSCR75 is defined as at least a 75% decrease in Abscess and Inflammatory Nodule (AN) count with no increase in the number of abscesses and in the number of draining tunnels/fistulae compared to baseline.
4. Flaring up to Week 16. Flare is defined as at least a 25% increase in AN count with a minimum increase of 2 AN relative to baseline.
5. Achievement of HiSCR50 at Week 8.
6. Achievement of HiSCR90 at Week 16. HiSCR90 is defined as at least a 90% decrease in Abscess and Inflammatory Nodule (AN) count with no increase in the number of abscesses and in the number of draining tunnels/fistulae compared to baseline.
7. Achievement of NRS30 at Week 16, among participants with baseline NRS ≥ 3 (pooled data from studies CLOU064J12301 and CLOU064J12302). NRS30 is defined as at least a 30% reduction and at least 2-unit reduction from baseline in Patient's Global Assessment of Skin Pain - at worst over the past 7 days
8. Occurrence of treatment emergent adverse events and serious adverse events during the study

Investigational products

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

Test 2

Placebo 2

Auxiliary 3

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 20

Locations

9 EU/EEA countries · 60 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 152 files

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

Application history

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