A 52-week Study of Rilzabrutinib Efficacy and Safety Compared to Placebo in Adults Diagnosed With IgG4-related Disease

NCT07190196 | Recruiting Phase 3 FDA Drug

• 3 other identifiers: EFC17359U1111-1316-02662025-521398-15
• Study Type: interventional
• Target: 124
• Locations: 19 countries
• Sites: 68

Brief Summary

This is a Phase 3, parallel group, 2-arm, randomized, double blind, placebo-controlled, 52-week treatment study to assess the efficacy and safety of rilzabrutinib as a treatment for adult patients with active IgG4-RD. The purpose of this study is to measure time to IgG4-RD clinical disease flare, and other relevant efficacy endpoints including flare-free rate, control of IgG4-RD disease activity, use of GC rescue and safety parameters such as treatment-emergent adverse events, clinical laboratory values and electrocardiograms (ECG) in participants aged 18 years and above, diagnosed with IgG4-RD and treated with rilzabrutinib tablets over a 52-week placebo-controlled period. Study details include: The study duration will be up to 60 weeks, including a 4 to 6-week screening period, a 52-week double blind treatment period, and 2 weeks of follow up (plus an optional OLE of 108 weeks). The number of visits will be 16 (plus an optional 9 visits during the OLE).

Conditions

Immunoglobulin G4 Related Disease

Outcome Measures

Primary Outcomes (1)

• Time to first adjudicated clinical disease flare treated by the investigator during the Blinded Treatment period

  The Adjudication Committee will include internationally recognized independent experts in diagnosis and management of patients with IgG4-RD, blinded to participant, investigator, and site identifiers

  Until Week 52

Secondary Outcomes (11)

• Percentage of participants without IgG4-RD adjudicated clinical disease flare and off glucocorticoids and immunomodulators

  At Week 52

• Percentage of participants without IgG4-RD adjudicated clinical disease flare and off glucocorticoids

  At Week 52

• Annualized rate of clinical disease flares

  At Week 52

• Change in IgG4-RD RI total activity scores from baseline to Week 52

  From baseline to Week 52

• Percent change in IgG4-RD RI total activity scores form baseline to Week 52

  From baseline to Week 52

Study Arms (2)

Rilzabrutinib

EXPERIMENTAL

Rilzabrutinib

Drug: Rilzabrutinib; Drug: Glucocorticoid

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo; Drug: Glucocorticoid

Interventions

Rilzabrutinib DRUG

Pharmaceutical form:Tablet-Route of administration:Oral

Also known as: SAR444671

Rilzabrutinib

Placebo DRUG

Pharmaceutical form:Tablet-Route of administration:Oral

Placebo

Glucocorticoid DRUG

Pharmaceutical form:Tablet, solution, suspension formulations according to local standard practices-Route of administration:Oral

Placebo; Rilzabrutinib

Eligibility Criteria

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

You may qualify if:

• Participants must have an adjudicated clinical diagnosis of IgG4-RD
• Participants with active disease at screening in at least one organ system, excluding lymph nodes, as an IgG4-RD Responder Index total activity score ≥ 2
• Participants with history or current involvement of at least 1 organ/site (excluding lymph nodes) affected with IgG4-RD.
• Participants with active IgG4-RD controlled for at least 2 weeks while on a stable dose of GC.
• Participants willing to taper off GC after starting IMP.
• Participants willing and able to participate in repeated study protocol mandated or clinically indicated imaging procedures to assess IgG4-RD such as computed tomography (CT), magnetic resonance imaging (MRI), positron emission tomography (PET), or ultrasound.
• Participants who have an up-to-date vaccination status as per local guidelines. The last dose of live vaccines should be received at least 30 days before Day 1.
• Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

You may not qualify if:

• History of retroperitoneal fibrosis, sclerosing mesenteritis, fibrosing mediastinitis, or other overwhelmingly fibrotic expression of IgG4-RD that is the sole disease manifestation.
• Active malignancy or history of malignancy within 5 years before Day 1, except completely treated in situ carcinoma of the cervix, completely treated, and resolved nonmetastatic squamous or basal cell carcinoma of the skin.
• Known or suspected immunodeficiency, including history of invasive opportunistic infections (eg, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystosis, and aspergillosis) despite infection resolution, or otherwise recurrent infections of abnormal frequency or prolonged duration suggesting an immune compromised status, as judged by the Investigator.
• History of serious infections with the potential for recurrence (as judged by the Investigator), with less than 4 weeks interval between resolution of serious infection and first dose of study drug, or currently active moderate to severe infection at Screening (Grade 2 or higher).
• Current or chronic history of liver disease unrelated to IgG4-RD.
• Refractory nausea and vomiting, malabsorption, external biliary shunt, bariatric surgery, or significant bowel resection that would preclude adequate rilzabrutinib/placebo absorption.
• History of solid organ transplant.
• Planned major surgical procedure during the participation in this study.
• History of drug abuse within the previous 12 months.
• Alcoholism or excessive alcohol use, defined as regular consumption of more than approximately 3 standard drinks per day.
• Prior participation in any rilzabrutinib studies or other BTK inhibitor studies.
• History of treatment with an investigational drug within 6 months or 5 half-lives of the investigational drug, whichever is longer.
• Laboratory abnormalities at the screening visit identified by the central laboratory The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (68)

San Jose Clinical Trials- Site Number : 8400016

San Jose, California, 95128, United States

RECRUITING

Solace Clinical Research - Tustin- Site Number : 8400020

Tustin, California, 92780, United States

RECRUITING

Life Clinical Trials - Margate - Colonial Drive- Site Number : 8400002

Margate, Florida, 33063, United States

RECRUITING

Schiff Center for Liver Diseases/University of Miami- Site Number : 8400010

Miami, Florida, 33136, United States

RECRUITING

Vitalia Medical Research - Margate- Site Number : 8400025

Palm Beach Gardens, Florida, 33410, United States

RECRUITING

Primeway Clinical Research- Site Number : 8400019

Fayetteville, Georgia, 30214, United States

RECRUITING

Mayo Clinic in Rochester - Minnesota- Site Number : 8400017

Rochester, Minnesota, 55905, United States

RECRUITING

Cleveland Clinic - Cleveland- Site Number : 8400001

Cleveland, Ohio, 44195, United States

RECRUITING

Ohio State University Wexner Medical Center - Site Number : 8400024

Columbus, Ohio, 43210, United States

RECRUITING

Paramount Medical Research and Consulting LLC- Site Number : 8400018

Middleburg Heights, Ohio, 44140, United States

RECRUITING

Stryde Research- Site Number : 8400011

Plano, Texas, 75093, United States

RECRUITING

Epic Medical Research - Red Oak- Site Number : 8400003

Red Oak, Texas, 75154, United States

RECRUITING

Digestive Health Research of North Texas- Site Number : 8400021

Wichita Falls, Texas, 76301, United States

RECRUITING

Velocity Clinical Research - Seattle- Site Number : 8400005

Seattle, Washington, 98105, United States

RECRUITING

Investigational Site Number : 0320005

Berazategui, Buenos Aires, 1886, Argentina

RECRUITING

Investigational Site Number : 0320002

Resistencia, Chaco Province, 3500, Argentina

RECRUITING

Investigational Site Number : 0320004

Buenos Aires, 1015, Argentina

RECRUITING

Investigational Site Number : 0560001

Leuven, 3000, Belgium

RECRUITING

Investigational Site Number : 1240001

Sherbrooke, Quebec, J1G 2E8, Canada

RECRUITING

Investigational Site Number : 1520005

Concepción, Biobio, 4070038, Chile

RECRUITING

Centro de estudios G y C-Site Number : 1520003

Santiago, Reg Metropolitana de Santiago, 7500504, Chile

RECRUITING

Investigational Site Number : 1520006

Santiago, Reg Metropolitana de Santiago, 8207257, Chile

RECRUITING

Investigational Site Number : 1520001

Santiago, Reg Metropolitana de Santiago, 8320325, Chile

RECRUITING

Investigational Site Number : 1520002

Santiago, Reg Metropolitana de Santiago, 8330032, Chile

RECRUITING

Investigational Site Number : 1560008

Beijing, 100034, China

RECRUITING

Investigational Site Number : 1560004

Beijing, 100050, China

RECRUITING

Investigational Site Number : 1560001

Beijing, 100730, China

RECRUITING

Investigational Site Number : 1560012

Chengdu, 610041, China

RECRUITING

Investigational Site Number : 1560010

Hangzhou, 310009, China

RECRUITING

Investigational Site Number : 1560003

Jiazhuang, 050000, China

RECRUITING

Investigational Site Number : 1560013

Taiyuan, 030001, China

RECRUITING

Investigational Site Number : 1560005

Taiyuan, 030032, China

RECRUITING

Investigational Site Number : 1560011

Wuhan, 430030, China

RECRUITING

Investigational Site Number : 1560009

Yantai, 264001, China

RECRUITING

AP-HP - Hôpital Beaujon-Site Number : 2500002

Clichy, 92110, France

ACTIVE NOT RECRUITING

Investigational Site Number : 2500001

Marseille, 13885, France

RECRUITING

CHU Bordeaux - Hôpital Haut Leveque-Site Number : 2500003

Pessac, 33600, France

ACTIVE NOT RECRUITING

Investigational Site Number : 2760001

Berlin, 13353, Germany

RECRUITING

Investigational Site Number : 2760003

Erlangen, 91054, Germany

RECRUITING

Rabin MC, Outpatient Clinics Building-Site Number : 3760001

Petah Tikva, Central District, 4941492, Israel

RECRUITING

Sourasky Rheumatology Dept, Rishonim Building-Site Number : 3760005

Tel Aviv, Central District, 6423906, Israel

RECRUITING

AOU Meyer IRCCS-Site Number : 3800003

Florence, Firenze, 50139, Italy

RECRUITING

IRCCS Ospedale San Raffaele-Site Number : 3800001

Milan, Lombardy, 20132, Italy

RECRUITING

Centro Ricerche Cliniche di Verona s.r.l. c/o Policlinico G.B.Rossi-Site Number : 3800002

Verona, Veneto, 37134, Italy

RECRUITING

Hiroshima University Hospital-Site Number : 3920007

Hiroshima, Hiroshima, 734-8551, Japan

RECRUITING

Investigational Site Number : 3920011

Sapporo, Hokkaido, 060-8543, Japan

RECRUITING

Kanazawa Medical University Hospital-Site Number : 3920002

Kahoku, Uchinada, Ishikawa-ken, 920-0293, Japan

RECRUITING

Investigational Site Number : 3920001

Kanazawa, Ishikawa-ken, 920-8641, Japan

RECRUITING

Investigational Site Number : 3920003

Hirakata, Osaka, 573-1191, Japan

RECRUITING

Investigational Site Number : 3920008

Bunkyo-ku, Tokyo, 113-8677, Japan

RECRUITING

The Institute of Medical Science, The University of Tokyo-Site Number : 3920006

Minato-ku, Tokyo, 108-8639, Japan

RECRUITING

Erasmus Medisch Centrum-Site Number : 5280001

Rotterdam, South Holland, 3015 GD, Netherlands

ACTIVE NOT RECRUITING

UMCG-Site Number : 5280002

Groningen, 9713 GZ, Netherlands

ACTIVE NOT RECRUITING

Malopolskie Badania Kliniczne sp. z. o.o.-Site Number : 6160001

Krakow, Lesser Poland Voivodeship, 30-002, Poland

RECRUITING

Investigational Site Number : 6160002

Wroclaw, Lower Silesian Voivodeship, 50-556, Poland

RECRUITING

Uniwersyteckie Centrum Kliniczne, Centrum Wsparcia Badan Klinicznych UCK Osrodek Badan Wczesnych Faz-Site Number : 6160003

Gdansk, Pomeranian Voivodeship, 80-214, Poland

RECRUITING

Investigational Site Number : 6820002

Riyadh, 12372, Saudi Arabia

RECRUITING

Investigational Site Number : 6820001

Riyadh, 12713, Saudi Arabia

RECRUITING

Investigational Site Number : 4100002

Suwon, Gyeonggi-do, 16499, South Korea

RECRUITING

Investigational Site Number : 4100001

Seoul, Seoul-teukbyeolsi, 03080, South Korea

RECRUITING

Investigational Site Number : 7240001

Barcelona, Barcelona [Barcelona], 08035, Spain

RECRUITING

Investigational Site Number : 7240002

Barcelona, Catalunya [Cataluña], 08041, Spain

RECRUITING

Investigational Site Number : 7240004

Madrid, 28034, Spain

RECRUITING

Investigational Site Number : 7520001

Huddinge, 141 57, Sweden

RECRUITING

Investigational Site Number : 7520002

Uppsala, 751 85, Sweden

RECRUITING

Investigational Site Number : 1580001

Taipei, 10016, Taiwan

RECRUITING

Investigational Site Number : 1580002

Taoyuan City, 333, Taiwan

RECRUITING

Investigational Site Number : 8260004

Norwich, Norfolk, NR4 7UY, United Kingdom

RECRUITING

Related Links

• EFC17359 Plain Language Results Summary

MeSH Terms

Conditions

Immunoglobulin G4-Related Disease

Interventions

Glucocorticoids

Condition Hierarchy (Ancestors)

Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses

Central Study Contacts

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

800-633-1610; contact-us@sanofi.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 17, 2025
• First Posted: September 24, 2025
• Study Start: September 26, 2025
• Primary Completion (Estimated): June 28, 2028
• Study Completion (Estimated): December 25, 2030
• Last Updated: May 4, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

KR (2); US (14); AR (3); GB (1); IT (3); IL (2); CN (10); SA (2); ES (3); DE (2); SE (2); CL (5); CA (1); JP (7); NL (2); PL (3); BE (1); TW (2); FR (3)