A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis

NCT06470048 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CVAY736S122012024-511933-36-00
• Study Type: interventional
• Target: 200
• Locations: 25 countries
• Sites: 127

Brief Summary

The purpose of this study is to evaluate efficacy, safety and tolerability of s.c. ianalumab administered in participants with diffuse cutaneous systemic sclerosis relative to placebo

Conditions

Diffuse Cutaneous Systemic Sclerosis

Keywords

Diffuse Cutaneous Systemic Sclerosis (dcSSc); Diffuse Scleroderma; Diffuse Systemic Sclerosis; Scleroderma, Diffuse; Scleroderma, Progressive; Sclerosis, Progressive Systemic; Sudden Onset Scleroderma; B cell depletion; Revised Composite Response Index in Systemic Sclerosis 25 (rCRISS25); modified Rodnan skin score; forced vital capacity

Outcome Measures

Primary Outcomes (1)

• 3/5 rCRISS25 response

  To demonstrate the superiority of ianalumab, compared to placebo, in achieving 3/5 Revised Composite Response Index in Systemic Sclerosis 25 (rCRISS25) response at Week 52

  Week 52

Secondary Outcomes (6)

• Change from baseline in FVC% predicted at Week 52

  Week 52

• Change from baseline in mRSS at Week 52

  Week 52

• Change from baseline in HAQ-DI at Week 52

  Week 52

• Ianalumab concentrations in serum during treatment and Follow-up Period

  Baseline, Week 4, Week 8, Week 12, Week 24, Week 36, Week 52, Week 64, Week 76, Week 88, Week 104, Week 108, Week 112, Week 116, Week 120, Week 124 and Week 208

• Incidence and titer of anti-drug (ianalumab) antibodies (ADAs) in serum over time

  Baseline, Week 4, Week 8, Week 12, Week 24, Week 36, Week 52, Week 64, Week 76, Week 88, Week 104, Week 124, Week 208

Study Arms (2)

VAY736 (Ianalumab)

EXPERIMENTAL

Treatment Period 1: Ianalumab subcutaneous (s.c.) injection as defined in the protocol Treatment Period 2: Open-label (OL) Ianalumab subcutaneous (s.c.) injection as defined in the protocol

Drug: Ianalumab

Placebo

PLACEBO COMPARATOR

Treatment Period 1: Placebo to Ianalumab subcutaneous (s.c.) injection as defined in the protocol Treatment Period 2: Open-label (OL) Ianalumab subcutaneous (s.c.) injection as defined in the protocol

Drug: Placebo; Drug: Ianalumab

Interventions

Placebo DRUG

Ianalumab matching placebo subcutaneous (s.c.) injection as defined in the protocol

Placebo

Ianalumab DRUG

subcutaneous (s.c.) injection as defined in the protocol

Also known as: VAY736

Placebo; VAY736 (Ianalumab)

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female participants \>= 18 and =\< 70 years (at the time of the screening visit).
• Diagnosis of systemic sclerosis, as defined by the 2013 American College of Rheumatology/ European League Against Rheumatism (ACR/EULAR) classification criteria for SSc (van den Hoogen et al 2013) and meet the dcSSc subset classification according to LeRoy (LeRoy 1988)
• Disease duration of =\< 60 months (defined as time from the first non-Raynaud phenomenon manifestation, e.g., puffy hands, scleroderma, digital ulcers, arthralgia, dyspnea)
• mRSS units of \>= 15 and =\< 45 at the time of the screening visit
• Active disease that meets at least one of the following criteria at screening:
• Disease duration of =\< 18 months defined as time from the first non-Raynaud phenomenon manifestation
• Increase in mRSS of \>= 3 units compared with the most recent assessment performed within the previous 6 months
• Involvement of one new body area and an increase in mRSS of \>= 2 units compared with the most recent assessment performed within the previous 6 months
• Involvement of two new body areas within the previous 6 months
• Elevated acute phase reactants (ESR) \>= 30 mm/hr or high-sensitivity C-reactive protein (hsCRP) \>= 6 mg/L)
• Presence of SSc-interstitial lung disease (ILD) and ATA autoantibody positivity
• Modified EUSTAR disease activity index (mDAI) ≥ 2.5
• Participant must be positive for at least one of the following autoantibodies:
• anti-topoisomerase I (ATA) (also known as anti-SCL-70)
• anti-RNA polymerase III (anti-RNAP3)

You may not qualify if:

• Positive anti-centromere antibody (ACA+) without positive ATA or anti-RNAP3 autoantibody result at the screening visit
• Previous improvement (decrease) in mRSS \> 10 units
• Pulmonary disease with FVC ≤ 50% of predicted or diffusing capacity of the lung for carbon monoxide (DLCO, corrected for hemoglobin) ≤ 40% of predicted at the screening visit
• WHO Functional Class 3 or higher assessment for pulmonary arterial hypertension (PAH, as defined on right heart catheterization), receiving IV therapy for PAH or evidence of other moderately severe pulmonary disease
• Participants treated with cyclophosphamide within 12 weeks prior to Baseline.
• Prior use of a B-cell depleting therapy other than ianalumab (e.g., rituximab, other anti-CD20 mAb, anti-CD22 mAb, or anti-CD52 mAb) administered within 36 weeks prior to randomization, or as long as B cell count is less than the lower limit of normal or baseline value prior to receipt of B cell-depleting therapy (whichever is lower).
• Treatment with any investigational agent within ≤ 4 weeks (or 5 half-lives of the investigational drug, whichever is longer) of the baseline visit.
• Use of anti-fibrotic agents including colchicine, D-penicillamine, pirfenidone, or tyrosine kinase inhibitors (e.g., nintedanib, nilotinib, imatinib, dasatinib) in the 4 weeks prior to baseline visit. Patients with SSc-ILD requiring antifibrotics for management of ILD during the study, as per investigator judgement, should be excluded.
• Previous treatment with chlorambucil, bone marrow transplantation or total lymphoid irradiation.
• Women of childbearing potential, 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 6 months after stopping study treatment.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (127)

Arizona Arthritis and Rheumatology Research PLLC

Mesa, Arizona, 85202, United States

RECRUITING

UCLA

Los Angeles, California, 90095, United States

RECRUITING

Hoag Hospital

Newport Beach, California, 92663, United States

RECRUITING

Clinical Res Of W Florida

Clearwater, Florida, 33765, United States

RECRUITING

GNP Research

Cooper City, Florida, 33024, United States

RECRUITING

IRIS Research and Development

Plantation, Florida, 33324, United States

RECRUITING

Sarasota Arthritis Res Ctr

Sarasota, Florida, 34239, United States

RECRUITING

University of Chicago Hospitals

Chicago, Illinois, 60637, United States

RECRUITING

UMC New Orleans

New Orleans, Louisiana, 70112, United States

RECRUITING

Uni Of Michigan Health System

Ann Arbor, Michigan, 48109, United States

RECRUITING

Wayne State University

Detroit, Michigan, 48201, United States

RECRUITING

Clinical Research Inst of MI

Saint Clair Shores, Michigan, 48081, United States

RECRUITING

Hospital for Special Surgery

New York, New York, 10021, United States

RECRUITING

West Tennessee Research Institute

Jackson, Tennessee, 38305, United States

RECRUITING

Arthritis and Rheumatology Ins

Allen, Texas, 75013, United States

RECRUITING

Novel Research LLC

Bellaire, Texas, 77401, United States

RECRUITING

Prolato Clinical Research Center

Houston, Texas, 77054, United States

RECRUITING

Novartis Investigative Site

CABA, Buenos Aires, 1280, Argentina

RECRUITING

Novartis Investigative Site

CABA, Buenos Aires, C1221ADC, Argentina

RECRUITING

Novartis Investigative Site

Caba, C1015ABO, Argentina

RECRUITING

Novartis Investigative Site

Caba, C1426, Argentina

RECRUITING

Novartis Investigative Site

San Miguel de Tucumán, 4000, Argentina

RECRUITING

Novartis Investigative Site

Graz, 8036, Austria

RECRUITING

Novartis Investigative Site

Leuven, 3000, Belgium

RECRUITING

Novartis Investigative Site

Salvador, Estado de Bahia, 40150 150, Brazil

RECRUITING

Novartis Investigative Site

Curitiba, Paraná, 80030-110, Brazil

RECRUITING

Novartis Investigative Site

Porto Alegre, Rio Grande do Sul, 90035-074, Brazil

RECRUITING

Novartis Investigative Site

Sao Jose Rio Preto, São Paulo, 15090 000, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 04038-002, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 05403-000, Brazil

RECRUITING

Novartis Investigative Site

Nanning, Guangxi, 530021, China

RECRUITING

Novartis Investigative Site

Zhengzhou, Henan, 450003, China

RECRUITING

Novartis Investigative Site

Changchun, Jilin, 130021, China

RECRUITING

Novartis Investigative Site

Chengdu, Sichuan, 610041, China

RECRUITING

Novartis Investigative Site

Ningbo, Zhejiang, 315016, China

RECRUITING

Novartis Investigative Site

Beijing, 100191, China

RECRUITING

Novartis Investigative Site

Beijing, 100730, China

RECRUITING

Novartis Investigative Site

Tianjin, 300052, China

RECRUITING

Novartis Investigative Site

Medellín, Antioquia, 050001, Colombia

RECRUITING

Novartis Investigative Site

Bogota, Cundinamarca, 110111, Colombia

RECRUITING

Novartis Investigative Site

Bogota, Cundinamarca, 110221, Colombia

RECRUITING

Novartis Investigative Site

Cali, Valle del Cauca Department, 760012, Colombia

RECRUITING

Novartis Investigative Site

Bogotá, 110231, Colombia

RECRUITING

Novartis Investigative Site

Medellín, 050034, Colombia

RECRUITING

Novartis Investigative Site

Dijon, 21000, France

RECRUITING

Novartis Investigative Site

Le Mans, 72000, France

RECRUITING

Novartis Investigative Site

Lille, 59037, France

RECRUITING

Novartis Investigative Site

Montpellier, 34295, France

RECRUITING

Novartis Investigative Site

Paris, 75014, France

RECRUITING

Novartis Investigative Site

Rennes, 35033, France

RECRUITING

Novartis Investigative Site

Strasbourg, 67000, France

RECRUITING

Novartis Investigative Site

Toulouse, 31054, France

RECRUITING

Novartis Investigative Site

Würzburg, Bavaria, 97080, Germany

RECRUITING

Novartis Investigative Site

Jena, Thuringia, 07740, Germany

RECRUITING

Novartis Investigative Site

Berlin, 13353, Germany

RECRUITING

Novartis Investigative Site

Alexandroupoli, 681 00, Greece

RECRUITING

Novartis Investigative Site

Athens, 115 21, Greece

RECRUITING

Novartis Investigative Site

Athens, 115 27, Greece

RECRUITING

Novartis Investigative Site

Pécs, Baranya, 7623, Hungary

RECRUITING

Novartis Investigative Site

Debrecen, Hajdu Bihar Megye, 4032, Hungary

RECRUITING

Novartis Investigative Site

Budapest, H-1083, Hungary

RECRUITING

Novartis Investigative Site

Kochi, Kerala, 682018, India

RECRUITING

Novartis Investigative Site

Mumbai, Maharashtra, 400013, India

RECRUITING

Novartis Investigative Site

Mumbai, Maharashtra, 400078, India

RECRUITING

Novartis Investigative Site

New Delhi, National Capital Territory of Delhi, 110060, India

RECRUITING

Novartis Investigative Site

Jaipur, Rajasthan, 302004, India

RECRUITING

Novartis Investigative Site

New Delhi, 110029, India

RECRUITING

Novartis Investigative Site

Ancona, AN, 60126, Italy

RECRUITING

Novartis Investigative Site

Milan, MI, 20100, Italy

RECRUITING

Novartis Investigative Site

Rozzano, MI, 20089, Italy

RECRUITING

Novartis Investigative Site

Modena, MO, 41124, Italy

RECRUITING

Novartis Investigative Site

Palermo, PA, 90127, Italy

RECRUITING

Novartis Investigative Site

Pavia, PV, 27100, Italy

RECRUITING

Novartis Investigative Site

Roma, RM, 00168, Italy

RECRUITING

Novartis Investigative Site

Verona, VR, 37134, Italy

RECRUITING

Novartis Investigative Site

Nagoya, Aichi-ken, 4578510, Japan

RECRUITING

Novartis Investigative Site

Kitakyushu, Fukuoka, 8078556, Japan

RECRUITING

Novartis Investigative Site

Maebashi, Gunma, 371 8511, Japan

RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 0608543, Japan

RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 0608648, Japan

RECRUITING

Novartis Investigative Site

Kanazawa, Ishikawa-ken, 920 8641, Japan

RECRUITING

Novartis Investigative Site

Yokohama, Kanagawa-ku, 236-0004, Japan

RECRUITING

Novartis Investigative Site

Suita, Osaka, 5650871, Japan

RECRUITING

Novartis Investigative Site

Iruma-gun, Saitama, 3500495, Japan

RECRUITING

Novartis Investigative Site

Bunkyo-ku, Tokyo, 113-8603, Japan

RECRUITING

Novartis Investigative Site

Chuo Ku, Tokyo, 1048560, Japan

RECRUITING

Novartis Investigative Site

Shinjuku Ku, Tokyo, 1628666, Japan

RECRUITING

Novartis Investigative Site

Shinjuku-ku, Tokyo, 1608582, Japan

RECRUITING

Novartis Investigative Site

Fukuoka, 8128582, Japan

RECRUITING

Novartis Investigative Site

Hiroshima, 7348551, Japan

RECRUITING

Novartis Investigative Site

Kyoto, 6068507, Japan

RECRUITING

Novartis Investigative Site

Toyama, 9300194, Japan

RECRUITING

Novartis Investigative Site

Cheras, Kuala Lumpur, 56000, Malaysia

RECRUITING

Novartis Investigative Site

Kuala Lumpur, 59100, Malaysia

RECRUITING

Novartis Investigative Site

Guadalajara, Jalisco, 44650, Mexico

RECRUITING

Novartis Investigative Site

Mexico City, Mexico City, 06700, Mexico

RECRUITING

Novartis Investigative Site

Mexico City, Mexico City, 11850, Mexico

RECRUITING

Novartis Investigative Site

Monterrey, Nuevo León, 64460, Mexico

RECRUITING

Novartis Investigative Site

Chihuahua City, 31000, Mexico

RECRUITING

Novartis Investigative Site

Bydgoszcz, 85-168, Poland

RECRUITING

Novartis Investigative Site

Warsaw, 00-874, Poland

RECRUITING

Novartis Investigative Site

Coimbra, 3004-561, Portugal

RECRUITING

Novartis Investigative Site

Lisbon, 1649-035, Portugal

RECRUITING

Novartis Investigative Site

Vila Nova de Gaia, 4434 502, Portugal

RECRUITING

Novartis Investigative Site

Seoul, Korea, 03080, South Korea

ACTIVE NOT RECRUITING

Novartis Investigative Site

Busan, 49241, South Korea

RECRUITING

Novartis Investigative Site

Seoul, 04763, South Korea

RECRUITING

Novartis Investigative Site

Seoul, 06273, South Korea

RECRUITING

Novartis Investigative Site

Málaga, Andalusia, 29009, Spain

RECRUITING

Novartis Investigative Site

Valencia, Valencia, 46017, Spain

RECRUITING

Novartis Investigative Site

Barcelona, 08041, Spain

RECRUITING

Novartis Investigative Site

Madrid, 28009, Spain

RECRUITING

Novartis Investigative Site

Salamanca, 37007, Spain

RECRUITING

Novartis Investigative Site

Changhua, 50006, Taiwan

RECRUITING

Novartis Investigative Site

Kaohsiung City, 83301, Taiwan

RECRUITING

Novartis Investigative Site

Taichung, 40447, Taiwan

RECRUITING

Novartis Investigative Site

Taoyuan District, 33305, Taiwan

RECRUITING

Novartis Investigative Site

Khon Kaen, THA, 40002, Thailand

RECRUITING

Novartis Investigative Site

Bangkok, 10400, Thailand

RECRUITING

Novartis Investigative Site

Istanbul, Fatih, 34093, Turkey (Türkiye)

RECRUITING

Novartis Investigative Site

Ankara, Sihhiye-Altindag, 06230, Turkey (Türkiye)

RECRUITING

Novartis Investigative Site

Ankara, Yenimahalle, 06500, Turkey (Türkiye)

RECRUITING

Novartis Investigative Site

Basingstoke, Hampshire, RG24 9NA, United Kingdom

RECRUITING

Novartis Investigative Site

Bristol, BS10 5NB, United Kingdom

RECRUITING

Novartis Investigative Site

London, NW3 2QG, United Kingdom

RECRUITING

Novartis Investigative Site

Ho Chi Minh City, VNM, 700000, Vietnam

RECRUITING

Novartis Investigative Site

Ho Chi Minh City, 700000, Vietnam

RECRUITING

MeSH Terms

Conditions

Scleroderma, Diffuse

Interventions

ianalumab

Condition Hierarchy (Ancestors)

Scleroderma, Systemic; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: June 17, 2024
• First Posted: June 24, 2024
• Study Start: October 9, 2024
• Primary Completion (Estimated): July 6, 2027
• Study Completion (Estimated): July 15, 2030
• Last Updated: March 11, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

PL (2); FR (8); ME (5); BE (1); PT (3); AU (1); CN (8); GR (3); CO (6); JP (17); VN (2); TU (3); DE (3); TW (4); HU (3); GB (3); IN (6); AR (5); US (17); TH (2); KR (4); BR (6); MY (2); ES (5); IT (8)