Phase 1b Long-term Extension Trial of RAY121 in Immunological Diseases (RAINBOW-LTE Trial)

NCT06723106 | Enrolling By Invitation Phase 1

• 1 other identifier: RAY903CT
• Study Type: interventional
• Target: 144
• Locations: 18 countries
• Sites: 57

Brief Summary

This is a long-term extension trial of RAY121 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 52

Secondary Outcomes (5)

• RAY121 concentration

  Baseline to Week 52

• Change from baseline in active C1s

  Baseline to Week 52

• Change from baseline in total C1s

  Baseline to Week 52

• Change from baseline in complement activity (classical pathway)

  Baseline to Week 52

• Anti-RAY121 antibodies

  Baseline to Week 52

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
• Have completed 4 doses of RAY121 administrations in RAY902CT trial and shown clinical responses
• Ability to comply with the study protocol, in the investigator's judgment
• For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use highly effective contraceptive methods during the treatment period and 20 weeks (140 days) after the last dose of RAY121
• For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and agreement to refrain from donating sperm

You may not qualify if:

• History of anaphylaxis or hypersensitivity to a biologic agent
• Known active infection with encapsuled bacteria
• History of Neisseria meningitidis infection
• Planned surgery
• Pregnant or breastfeeding, or intending to become pregnant
• Clinically significant electrocardiogram abnormalities
• Illicit drug or alcohol abuse
• Known or suspected immune deficiency
• Treatment with investigational therapy other than RAY121
• Vaccination with a live vaccine within 4 weeks

Sponsors & Collaborators

• Chugai Pharmaceutical: lead

Study Sites (58)

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

Location

Westmead Hospital

Sydney, New South Wales, 2145, Australia

Location

Campbelltown Public Hospital

Sydney, New South Wales, 2560, Australia

Location

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

Location

Box Hill Hospital

Melbourne, Victoria, 3128, Australia

Location

AKH - Medizinische Universitaet Wien, Abteilung fuer Klinische Pharmakologie

Vienna, 1090, Austria

Location

Diagnostic Consultation Center CONVEX EOOD

Sofia, Sofia City Province, 1202, Bulgaria

Location

"SHATHD" EAD Sofia

Sofia, Sofia City Province, 1756, Bulgaria

Location

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

Stara Zagora, Stara Zagora Province, 6003, Bulgaria

Location

DiIEX Recherche Sherbrooke, Inc

Sherbrooke, Quebec, J1L 0H8, Canada

Location

Clinical Hospital Center "Sestre Milosrdnice"

Zagreb, City of Zagreb, 10000, Croatia

Location

University hospital centre Zagreb

Zagreb, City of Zagreb, 10000, Croatia

Location

Specialty Hospital Medico

Rijeka, Primorje-Gorski Kotar County, 51000, Croatia

Location

Sanatorium Profesora Arenbergera

Prague, Prague, 11000, Czechia

Location

Hopital Lapeyronie,Service d'Immuno Rhumatologie

Montpellier, Occitanie, 34295, France

Location

AP-HP Hôpital Universitaire Pitié Salpêtrière

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

Location

Universitaetsklinikum Tuebingen

Tübingen, Baden-Wurttemberg, 72076, Germany

Location

Universitaetsklinikum Erlangen

Erlangen, Bavaria, 91054, Germany

Location

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

Sachsen, Bundesländer, 1307, Germany

Location

Universitaetsmedizin Goettingen

Göttingen, Göttingen District, 3075, Germany

Location

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

Lübeck, Schleswig-Holstein, 23538, Germany

Location

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont

Szeged, Csongrád-Csanád County, 6720, Hungary

Location

Semmelweis Egyetem

Budapest, 1083, Hungary

Location

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

Meldola, Forlì-Cesena Province, 47014, Italy

Location

Istituto Clinico Humanitas

Milan, Milan Province, 20089, Italy

Location

Ospedale San Giovanni Bosco

Torino, Turin Province, 10154, Italy

Location

Hokkaido University Hospital

Sapporo, Hokkaido, 060-0808, Japan

Location

Tohoku University Hospital

Sendai, Miyagi, 980-8574, Japan

Location

National Hospital Organization Osaka Minami Medical Center

Kawachi-Nagano, Osaka, 586-8521, Japan

Location

Kindai University Hospital

Sakai, Osaka, 589-8511, Japan

Location

Osaka University Hospital

Suita, Osaka, 565-0871, Japan

Location

Hamamatsu University Hospital

Hamamatsu, Shizuoka, 431-3192, Japan

Location

Institute of Science Tokyo Hospital

Bunkyo-ku, Tokyo, 113-8519, Japan

Location

National Center of Neurology and Psychiatry

Kodaira, Tokyo, 187-8551, Japan

Location

National Hospital Organization Tokyo Medical Center

Meguro-ku, Tokyo, 152-8902, Japan

Location

Toho University Omori Medical Center

Ōta-ku, Tokyo, 134-8540, Japan

Location

National Hospital Organization Kyushu Medical Center

Fukuoka, 810-8563, Japan

Location

Okayama University Hospital

Okayama, 700-8558, Japan

Location

University Medical Centre Groningen UMCG

Groningen, Groningen Province, 9713 GZ, Netherlands

Location

UMC Utrecht

Utrecht, Utrecht Province, 3584 CX, Netherlands

Location

Sorlandet sykehus Kristiansand

Kristiansand, Agder County, 4604, Norway

Location

Stavanger Universitetssjukehus

Stavanger, Rogaland County, 4011, Norway

Location

Institute Reumatologii I'm. Eleonory Reicher

Warsaw, Masovian Voivodeship, 02-637, Poland

Location

Centro Clinico Academico Braga

Braga, Braga District, 471-243, Portugal

Location

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

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

Location

Centrul Medical Monza SRL

Bucharest, Bucharest Municipality, 400015, Romania

Location

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

Cluj-Napoca, Cluj, 11658, Romania

Location

Clinica Universidad de Navarra

Pamplona, Community of Madrid & Navarre, 28027, Spain

Location

Hospital Universitario Ramon y Cajal, Servicio de Reumatologia

Madrid, Madrid, 28034, Spain

Location

Hospital Universitario 12 de October

Madrid, Madrid, 28041, Spain

Location

Hospital Universitari Vall d'Hebron, Internal Medicine Dept.

Barcelona, Province of Barcelona and Catalonia, 08035, Spain

Location

Hospital Universitario Reina Sofia

Córdoba, Province Of Córdoba, 14004, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, Province Of Seville, 41013, Spain

Location

Hospital Universitari i Politecnic La Fe

Valencia, Valencia, 46026, Spain

Location

National Taiwan University Hospital

Taipei, 100229, Taiwan

Location

Taichung Veterans General Hospital

Taipei, 407219, Taiwan

Location

Dr. Abdurrahman Yurtaslan Oncology Teaching and Research Hospital

Ankara, Ankara, 06200, Turkey (Türkiye)

Location

Istanbul University Istanbul Medical Faculty

Istanbul, Istanbul, 34093, Turkey (Türkiye)

Location

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

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: November 27, 2024
• First Posted: December 9, 2024
• Study Start: December 9, 2024
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): April 30, 2027
• Last Updated: April 2, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

HU (2); NL (2); CA (1); IT (3); CR (3); BU (3); CZ (1); RO (2); AU (5); TU (2); DE (5); FR (2); PL (1); NO (2); ES (7); TW (2); JP (12); PT (2)