Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

NCT06371417 | Recruiting Phase 1 FDA Drug

• 1 other identifier: RAY902CT
• Study Type: interventional
• Target: 144
• Locations: 19 countries
• Sites: 64

Brief Summary

This Phase 1b basket trial will investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary efficacy of RAY121, a inhibitor of classical complement pathway, after multiple dose administration in patients with immunological diseases such as antiphospholipid syndrome (APS), bullous pemphigoid (BP), Behçet's Syndrome (BS), dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM) and immune thrombocytopenia (ITP).

Conditions

Antiphospholipid Syndrome (APS); Bullous Pemphigoid (BP); Behçet's Syndrome (BS); Dermatomyositis (DM); Immune-mediated Necrotizing Myopathy (IMNM); Immune Thrombocytopenia (ITP)

Outcome Measures

Primary Outcomes (1)

• Adverse events (AEs)

  Incidence, severity, and causal relationship of AEs

  Baseline to Week 32

Secondary Outcomes (8)

• RAY121 concentration

  Baseline to Week 32

• AUCτ

  Baseline to Week 32

• Cmax

  Baseline to Week 32

• Cmin

  Baseline to Week 32

• Active C1s

  Baseline to Week 32

Study Arms (1)

RAY121

EXPERIMENTAL

All enrolled patients will receive RAY121 multiple dose

Drug: RAY121

Interventions

RAY121 DRUG

Injection

RAY121

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed informed consent form
• Age \>= 18 and \<=75 at the time of signing informed consent form (except for BP; Age \>=18 and \<= 85 with Karnofsky score \>= 60% at screening)
• Ability to comply with the study protocol
• For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use highly effective contraceptive methods
• For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and agreement to refrain from donating sperm
• APS cohort: Established primary APS defined by the following criteria (at least one of the laboratory criteria and one of the clinical criteria must be met):
• Laboratory criteria (aPL profile)
• Persistently positive lupus anticoagulant (LA) test
• Persistently positive anticardiolipin (aCL) immunoglobulin G (IgG) isotype
• Persistently positive anti-beta-2 glycoprotein-1 (aβ2GPI) IgG isotype
• Clinical criteria
• Livedoid vasculopathy and presence of skin ulcer
• Acute/chronic aPL nephropathy
• BP cohort:
• \) Age \>= 18 and \<= 85 with Karnofsky score \>= 60 %

You may not qualify if:

• History of anaphylaxis or hypersensitivity to a biologic agent
• Active infection requiring systemic antiviral, antibiotics or antifungal
• Planned surgery during the study
• Pregnant or breastfeeding, or intending to become pregnant
• Any serious medical condition or abnormality in clinical laboratory tests that precludes the patient's safe participation in and completion of the study
• Clinically significant ECG abnormalities
• Illicit drug or alcohol abuse
• Clinical diagnosis of autoimmune diseases other than the target disease (except for Sjögren's syndrome in DM and IMNM)
• Positive for hepatitis B surface antigen
• Positive for hepatitis C virus antibody
• Positive for human immunodeficiency virus antibody
• Evidence of current infection with tuberculosis
• History of cancer within 5 years
• Treatment with investigational therapy within 28 days or 5 half-lives
• Previous and current treatment with anti-C1s antibody at any time

Sponsors & Collaborators

• Chugai Pharmaceutical: lead

Study Sites (69)

University of California-Irvine

Orange, California, 92868, United States

RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21224, United States

NOT YET RECRUITING

Northwell Health, LLC PRIME

Lake Success, New York, 11042, United States

ACTIVE NOT RECRUITING

Hospital for Special Surgery

New York, New York, 10021, United States

ACTIVE NOT RECRUITING

Universtity of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

NOT YET RECRUITING

Ohio State University

Columbus, Ohio, 43203, United States

WITHDRAWN

Oregon Health & Science University

Portland, Oregon, 97239, United States

RECRUITING

University of Pennsylvania, Perelman Center for Advanced Medicine

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Amarillo Center for Clinical Research

Amarillo, Texas, 79124, United States

ACTIVE NOT RECRUITING

Austin Neuromuscular Center

Austin, Texas, 78759, United States

RECRUITING

Nerve and Muscle Center of Texas

Houston, Texas, 77030, United States

NOT YET RECRUITING

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

RECRUITING

Westmead Hospital

Sydney, New South Wales, 2145, Australia

RECRUITING

Campbelltown Public Hospital

Sydney, New South Wales, 2560, Australia

RECRUITING

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

RECRUITING

Box Hill Hospital

Melbourne, Victoria, 3128, Australia

RECRUITING

AKH - Medizinische Universitaet Wien, Abteilung fuer Klinische Pharmakologie

Vienna, 1090, Austria

NOT YET RECRUITING

Diagnostic Consultation Center CONVEX EOOD

Sofia, Sofia City Province, 1202, Bulgaria

NOT YET RECRUITING

"SHATHD" EAD Sofia

Sofia, Sofia City Province, 1756, Bulgaria

NOT YET RECRUITING

UMHAT "Prof. Dr. St. Kirkovich", AD

Stara Zagora, Stara Zagora Province, 6003, Bulgaria

NOT YET RECRUITING

University of Alberta Hospital - Department of Anesthesiology and Pain Medicine

Edmonton, Alberta, T6G 2B7, Canada

RECRUITING

University of Alberta Hospital - Dermatology

Edmonton, Alberta, T6G 2G3, Canada

RECRUITING

The Royal Institution for the Advancement of Learning/McGill University

Montreal, Quebec, H3A 0G4, Canada

RECRUITING

Centre de Rhumatologie de l'Est du Quebec

Rimouski, Quebec, G5L 8W1, Canada

RECRUITING

DIEX Recherche Sherbrooke Inc.

Sherbrooke, Quebec, J1L 0H8, Canada

RECRUITING

Clinical Hospital Center "Sestre Milosrdnice"

Zagreb, City of Zagreb, 10000, Croatia

NOT YET RECRUITING

University hospital centre Zagreb

Zagreb, City of Zagreb, 10000, Croatia

NOT YET RECRUITING

Specialty Hospital Medico

Rijeka, Primorje-Gorski Kotar County, 51000, Croatia

NOT YET RECRUITING

Sanatorium Profesora Arenbergera

Prague, Prague, 11000, Czechia

NOT YET RECRUITING

Hopital Lapeyronie,Service d'Immuno Rhumatologie

Montpellier, Occitanie, 34295, France

NOT YET RECRUITING

AP-HP Hôpital Universitaire Pitié Salpêtrière

Paris, Île-de-France Region, 75013, France

RECRUITING

Universitaetsklinikum Tuebingen

Tübingen, Baden-Wurttemberg, 72076, Germany

NOT YET RECRUITING

Universitaetsklinikum Erlangen

Erlangen, Bavaria, 91054, Germany

NOT YET RECRUITING

Universitaetsklinikum Carl Gustav Carus TU Dresden, Klinik und Poliklinik f. Dermatologie

Sachsen, Bundesländer, 1307, Germany

NOT YET RECRUITING

Universitaetsmedizin Goettingen

Göttingen, Göttingen District, 3075, Germany

NOT YET RECRUITING

Universitaetsklinikum Schleswig Holstein - Campus Luebeck, Klinik f Dermatologie, Allergologie u Venerologie

Lübeck, Schleswig-Holstein, 23538, Germany

NOT YET RECRUITING

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont

Szeged, Csongrád-Csanád County, 6720, Hungary

NOT YET RECRUITING

Semmelweis Egyetem

Budapest, 1083, Hungary

NOT YET RECRUITING

IRCCS Istituto Scientifico Romagnolo Per Lo Studio Dei Tumori "Dino Amadori" - IRST

Meldola, Forlì-Cesena Province, 47014, Italy

NOT YET RECRUITING

Istituto Clinico Humanitas

Milan, Milan Province, 20089, Italy

NOT YET RECRUITING

Ospedale San Giovanni Bosco

Torino, Turin Province, 10154, Italy

NOT YET RECRUITING

Hokkaido University Hospital

Sapporo, Hokkaido, 060-0808, Japan

RECRUITING

Tohoku University Hospital

Sendai, Miyagi, 980-8574, Japan

RECRUITING

Kindai University Hospital

Sayama, Osaka, 589-8511, Japan

NOT YET RECRUITING

Osaka University Hospital

Suita, Osaka, 565-0871, Japan

RECRUITING

Hamamatsu University Hospital

Hamamatsu, Shizuoka, 431-3192, Japan

RECRUITING

National Center of Neurology and Psychiatry

Kodaira, Tokyo, 187-8551, Japan

RECRUITING

Toho University Omori Medical Center

Ōta-ku, Tokyo, 143-8540, Japan

RECRUITING

Okayama University Hospital

Okayama, 700-8558, Japan

RECRUITING

University Medical Centre Groningen UMCG

Groningen, Groningen Province, 9713 GZ, Netherlands

NOT YET RECRUITING

UMC Utrecht

Utrecht, Utrecht Province, 3584 CX, Netherlands

NOT YET RECRUITING

Sorlandet sykehus Kristiansand

Kristiansand, Agder County, 4604, Norway

NOT YET RECRUITING

Stavanger Universitetssjukehus

Stavanger, Rogaland County, 4011, Norway

NOT YET RECRUITING

Institute Reumatologii I'm. Eleonory Reicher

Warsaw, Masovian Voivodeship, 02-637, Poland

RECRUITING

Centro Clinico Academico Braga

Braga, Braga District, 471-243, Portugal

NOT YET RECRUITING

Centro Hospitalar de Vila Nova de Gaia/Espinho, E.P.E.

Vila Nova de Gaia, Porto District, 4434-502, Portugal

NOT YET RECRUITING

Centrul Medical Monza SRL

Bucharest, Bucharest Municipality, 400015, Romania

RECRUITING

Oncology Institute Prof. Dr. Ion Chiricuta I.O.C.N.

Cluj-Napoca, Cluj, 11658, Romania

RECRUITING

Clinica Universidad de Navarra

Pamplona, Community of Madrid & Navarre, 28027, Spain

NOT YET RECRUITING

Hospital Universitario Ramon y Cajal, Servicio de Reumatologia

Madrid, Madrid, 28034, Spain

NOT YET RECRUITING

Hospital Universitario 12 de October

Madrid, Madrid, 28041, Spain

NOT YET RECRUITING

Hospital Universitari Vall d'Hebron, Internal Medicine Dept.

Barcelona, Province of Barcelona and Catalonia, 08035, Spain

NOT YET RECRUITING

Hospital Universitario Reina Sofia

Córdoba, Province Of Córdoba, 14004, Spain

NOT YET RECRUITING

Hospital Universitario Virgen del Rocio

Seville, Province Of Seville, 41013, Spain

RECRUITING

Hospital Universitari i Politecnic La Fe

Valencia, Valencia, 46026, Spain

NOT YET RECRUITING

National Taiwan University Hospital

Taipei, 100229, Taiwan

RECRUITING

Taichung Veterans General Hospital

Taipei, 407219, Taiwan

NOT YET RECRUITING

Dr. Abdurrahman Yurtaslan Oncology Teaching and Research Hospital

Ankara, Ankara, 06200, Turkey (Türkiye)

NOT YET RECRUITING

Istanbul University Istanbul Medical Faculty

Istanbul, Istanbul, 34093, Turkey (Türkiye)

NOT YET RECRUITING

MeSH Terms

Conditions

Antiphospholipid Syndrome; Pemphigoid, Bullous; Behcet Syndrome; Dermatomyositis; Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Autoimmune Diseases; Immune System Diseases; Skin Diseases, Vesiculobullous; Skin Diseases; Skin and Connective Tissue Diseases; Mouth Diseases; Stomatognathic Diseases; Uveitis, Anterior; Panuveitis; Uveitis; Uveal Diseases; Eye Diseases; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases, Vascular; Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Sponsor Chugai Pharmaceutical Co.Ltd

  clinical-trials@chugai-pharm.co.jp

  STUDY DIRECTOR

Central Study Contacts

Clinical trials information

CONTACT

only use Email; clinical-trials@chugai-pharm.co.jp

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2024
• First Posted: April 17, 2024
• Study Start: August 19, 2024
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2026
• Last Updated: April 2, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

PL (1); HU (2); AU (1); CA (5); IT (3); NL (2); CZ (1); CR (3); US (11); FR (2); RO (2); ES (7); JP (8); BU (3); TW (2); PT (2); TU (2); NO (2); DE (5)