An open-label phase II trial to evaluate the clinical efficacy, safety and tolerability of MAS825 in pediatric and adult participants with Still’s disease

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

15 Jan 2026 → ongoing

Decision date (initial)

2025-11-03

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2024-520208-25-00

WHO UTN

U1111-1321-5579

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

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

To evaluate the efficacy of MAS825 on disease activity in participants with Still’s Disease

Secondary objectives 6

1. To evaluate the safety and tolerability of MAS825
2. To evaluate the efficacy of MAS825 in reducing clinical disease activity
3. To assess the effect of MAS825 on macrophage activation syndrome
4. To evaluate the efficacy of MAS825 as a glucocorticoid-sparing agent
5. To evaluate the efficacy of MAS825 in improving participant’s quality of life (QoL)
6. To evaluate the effect of MAS825 in maintaining clinically inactive disease without the requirement of treatment-dose corticosteroids

Conditions and MedDRA coding

Still’s disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Age ≥ 1 with a diagnosis of Still’s disease
2. Active disease
3. Need for glucocorticoids (prednisone or equivalent)

Exclusion criteria 9

1. Ongoing or previous treatment with immunomodulatory drugs as outlined in the protocol’s Prohibited therapies section, except a limited number of patients that have previously received MAS825 through a managed access program.
2. Glucocorticoid dose exceeding a certain threshold or requiring IV pulsed glucocorticoid therapy
3. Any conditions or significant medical problems which, in the opinion of the investigator, places the patient at unacceptable risk for MAS825 therapy.
4. History of ongoing, chronic or possibly recurrent infection(s) (e.g. HIV, TB, HCV, HBV) and/or symptoms and signs, in the judgement of the investigator, of clinically significant active bacterial, fungal, or viral infections.
5. Live vaccinations within a certain period prior to MAS825 treatment.
6. History of malignancy of any organ system, including post-transplant lymphoproliferative disorder (other than adequately treated localized basal cell carcinoma of the skin or in situ cervical cancer), treated or untreated, within a certain period, regardless of whether there is evidence of local recurrence or metastases.
7. History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients.
8. Pregnant or breastfeeding women
9. Women of child-bearing potential who do not agree to comply with required contraceptive use.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Clinical response defined by achieving a composite endpoint

Secondary endpoints 6

1. Adverse events and serious adverse events. Safety measures including vital signs, ECG and laboratory assessments throughout the study
2. Clinical response defined by achieving a composite endpoint
3. Change in physician´s assessment. Change in laboratory features of Macrophage Activation Syndrome
4. Glucocorticoid dose compared to baseline
5. Change from baseline in patient/parent assessment of physical function
6. Clinically inactive disease whilst on non‑treatment dose corticosteroid at any time during the study

Investigational products

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

Test 1

Auxiliary 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 16

Locations

5 EU/EEA countries · 17 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 88 files

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

Application history

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