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

Phase 1b Open-label, Long-term Extension Basket Trial of RAY121 to Inhibit Classical Complement Pathway in Immunological Diseases (RAINBOW-LTE Trial)

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).

Study Overview

• Status: Enrolling by invitation
• Conditions: Antiphospholipid Syndrome (APS); Immune Thrombocytopenia (ITP); Dermatomyositis (DM); Bullous Pemphigoid (BP); Behçet's Syndrome (BS); Immune-mediated Necrotizing Myopathy (IMNM)
• Intervention / Treatment: Drug: RAY121

• Study Type: Interventional
• Enrollment (Estimated): 144
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Royal Prince Alfred Hospital
    • Sydney, New South Wales, Australia, 2145
      • Westmead Hospital
    • Sydney, New South Wales, Australia, 2560
      • Campbelltown Public Hospital
  • Victoria
    • Melbourne, Victoria, Australia, 3004
      • The Alfred Hospital
    • Melbourne, Victoria, Australia, 3128
      • Box Hill Hospital
• Austria
  • Vienna, Austria, 1090
    • AKH - Medizinische Universitaet Wien, Abteilung fuer Klinische Pharmakologie
• Bulgaria
  • Sofia City Province
    • Sofia, Sofia City Province, Bulgaria, 1202
      • Diagnostic Consultation Center CONVEX EOOD
    • Sofia, Sofia City Province, Bulgaria, 1756
      • "SHATHD" EAD Sofia
  • Stara Zagora Province
    • Stara Zagora, Stara Zagora Province, Bulgaria, 6003
      • UMHAT "Prof. Dr. St. Kirkovich", AD
• Canada
  • Quebec
    • Sherbrooke, Quebec, Canada, J1L 0H8
      • DiIEX Recherche Sherbrooke, Inc
• Croatia
  • City of Zagreb
    • Zagreb, City of Zagreb, Croatia, 10000
      • University hospital centre Zagreb
    • Zagreb, City of Zagreb, Croatia, 10000
      • Clinical Hospital Center "Sestre Milosrdnice"
  • Primorje-Gorski Kotar County
    • Rijeka, Primorje-Gorski Kotar County, Croatia, 51000
      • Specialty Hospital Medico
• Czechia
  • Prague
    • Prague, Prague, Czechia, 11000
      • Sanatorium Profesora Arenbergera
• France
  • Occitanie
    • Montpellier, Occitanie, France, 34295
      • Hopital Lapeyronie,Service d'Immuno Rhumatologie
  • Île-de-France Region
    • Paris, Île-de-France Region, France, 75013
      • AP-HP Hôpital Universitaire Pitié Salpêtrière
• Germany
  • Baden-Wurttemberg
    • Tübingen, Baden-Wurttemberg, Germany, 72076
      • Universitaetsklinikum Tuebingen
  • Bavaria
    • Erlangen, Bavaria, Germany, 91054
      • Universitaetsklinikum Erlangen
  • Bundesländer
    • Sachsen, Bundesländer, Germany, 1307
      • Universitaetsklinikum Carl Gustav Carus TU Dresden, Klinik und Poliklinik f. Dermatologie
  • Göttingen District
    • Göttingen, Göttingen District, Germany, 3075
      • Universitaetsmedizin Goettingen
  • Schleswig-Holstein
    • Lübeck, Schleswig-Holstein, Germany, 23538
      • Universitaetsklinikum Schleswig Holstein - Campus Luebeck, Klinik f Dermatologie, Allergologie u Venerologie
• Hungary
  • Budapest, Hungary, 1083
    • Semmelweis Egyetem
  • Csongrád-Csanád County
    • Szeged, Csongrád-Csanád County, Hungary, 6720
      • Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont
• Italy
  • Forlì-Cesena Province
    • Meldola, Forlì-Cesena Province, Italy, 47014
      • IRCCS Istituto Scientifico Romagnolo Per Lo Studio Dei Tumori "Dino Amadori" - IRST
  • Milan Province
    • Milan, Milan Province, Italy, 20089
      • Istituto Clinico Humanitas
  • Turin Province
    • Torino, Turin Province, Italy, 10154
      • Ospedale San Giovanni Bosco
• Japan
  • Fukuoka, Japan, 810-8563
    • National Hospital Organization Kyushu Medical Center
  • Okayama, Japan, 700-8558
    • Okayama University Hospital
  • Hokkaido
    • Sapporo, Hokkaido, Japan, 060-0808
      • Hokkaido University Hospital
  • Miyagi
    • Sendai, Miyagi, Japan, 980-8574
      • Tohoku University Hospital
  • Osaka
    • Kawachi-Nagano, Osaka, Japan, 586-8521
      • National Hospital Organization Osaka Minami Medical Center
    • Sakai, Osaka, Japan, 589-8511
      • Kindai University Hospital
    • Suita, Osaka, Japan, 565-0871
      • Osaka University Hospital
  • Shizuoka
    • Hamamatsu, Shizuoka, Japan, 431-3192
      • Hamamatsu University Hospital
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan, 113-8519
      • Institute of Science Tokyo Hospital
    • Kodaira, Tokyo, Japan, 187-8551
      • National Center of Neurology and Psychiatry
    • Meguro-ku, Tokyo, Japan, 152-8902
      • National Hospital Organization Tokyo Medical Center
    • Ōta-ku, Tokyo, Japan, 134-8540
      • Toho University Omori Medical Center
• Netherlands
  • Groningen Province
    • Groningen, Groningen Province, Netherlands, 9713 GZ
      • University Medical Centre Groningen UMCG
  • Utrecht Province
    • Utrecht, Utrecht Province, Netherlands, 3584 CX
      • UMC Utrecht
• Norway
  • Agder County
    • Kristiansand, Agder County, Norway, 4604
      • Sorlandet sykehus Kristiansand
  • Rogaland County
    • Stavanger, Rogaland County, Norway, 4011
      • Stavanger Universitetssjukehus
• Poland
  • Masovian Voivodeship
    • Warsaw, Masovian Voivodeship, Poland, 02-637
      • Institute Reumatologii I'm. Eleonory Reicher
• Portugal
  • Braga District
    • Braga, Braga District, Portugal, 471-243
      • Centro Clinico Academico Braga
  • Porto District
    • Vila Nova de Gaia, Porto District, Portugal, 4434-502
      • Centro Hospitalar de Vila Nova de Gaia/Espinho, E.P.E.
• Romania
  • Bucharest Municipality
    • Bucharest, Bucharest Municipality, Romania, 400015
      • Centrul Medical Monza SRL
  • Cluj
    • Cluj-Napoca, Cluj, Romania, 11658
      • Oncology Institute Prof. Dr. Ion Chiricuta I.O.C.N.
• Spain
  • Community of Madrid & Navarre
    • Pamplona, Community of Madrid & Navarre, Spain, 28027
      • Clinica Universidad de Navarra
  • Madrid
    • Madrid, Madrid, Spain, 28034
      • Hospital Universitario Ramon y Cajal, Servicio de Reumatologia
    • Madrid, Madrid, Spain, 28041
      • Hospital Universitario 12 de October
  • Province Of Córdoba
    • Córdoba, Province Of Córdoba, Spain, 14004
      • Hospital Universitario Reina Sofia
  • Province Of Seville
    • Seville, Province Of Seville, Spain, 41013
      • Hospital Universitario Virgen del Rocio
  • Province of Barcelona and Catalonia
    • Barcelona, Province of Barcelona and Catalonia, Spain, 08035
      • Hospital Universitari Vall d'Hebron, Internal Medicine Dept.
  • Valencia
    • Valencia, Valencia, Spain, 46026
      • Hospital Universitari i Politecnic La Fe
• Taiwan
  • Taipei, Taiwan, 100229
    • National Taiwan University Hospital
  • Taipei, Taiwan, 407219
    • Taichung Veterans General Hospital
• Turkey (Türkiye)
  • Ankara
    • Ankara, Ankara, Turkey (Türkiye), 06200
      • Dr. Abdurrahman Yurtaslan Oncology Teaching and Research Hospital
  • Istanbul
    • Istanbul, Istanbul, Turkey (Türkiye), 34093
      • Istanbul University Istanbul Medical Faculty

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Signed informed consent form
2. Have completed 4 doses of RAY121 administrations in RAY902CT trial and shown clinical responses
3. Ability to comply with the study protocol, in the investigator's judgment
4. 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
5. For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and agreement to refrain from donating sperm

Exclusion Criteria:

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RAY121; All enrolled patients will receive RAY121 multiple dose	Drug: RAY121; Injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse events (AEs)	Incidence, severity, and causal relationship of AEs	Baseline to Week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
RAY121 concentration	Serum RAY121 concentration	Baseline to Week 52
Change from baseline in active C1s	Concentration of active C1s will be measured and assessed against baseline values	Baseline to Week 52
Change from baseline in total C1s	Concentration of total C1s will be measured and assessed against baseline values	Baseline to Week 52
Change from baseline in complement activity (classical pathway)	Level of complement activity (classical pathway) will be measured and assessed against baseline values	Baseline to Week 52
Anti-RAY121 antibodies	Titer of anti-RAY121 antibodies	Baseline to Week 52

Collaborators and Investigators

• Sponsor: Chugai Pharmaceutical
• Investigators: Study Director: Sponsor Chugai Pharmaceutical Co.Ltd, clinical-trials@chugai-pharm.co.jp

Study record dates

Study Major Dates

• Study Start (Actual): December 9, 2024
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): April 30, 2027

Study Registration Dates

• First Submitted: November 27, 2024
• First Submitted That Met QC Criteria: December 6, 2024
• First Posted (Actual): December 9, 2024

Study Record Updates

• Last Update Posted (Actual): April 2, 2026
• Last Update Submitted That Met QC Criteria: April 1, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cytopenia; Musculoskeletal Diseases; Mouth Diseases; Stomatognathic Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Muscular Diseases; Pathologic Processes; Neuromuscular Diseases; Genetic Diseases, Inborn; Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Eye Diseases; Hematologic Diseases; Skin Diseases; Blood Coagulation Disorders; Skin Diseases, Vascular; Hemorrhagic Disorders; Skin Diseases, Genetic; Blood Platelet Disorders; Thrombotic Microangiopathies; Uveal Diseases; Vasculitis; Skin Diseases, Vesiculobullous; Myositis; Panuveitis; Purpura, Thrombocytopenic; Purpura; Polymyositis; Thrombocytopenia; Uveitis, Anterior; Uveitis; Hereditary Autoinflammatory Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathological Conditions, Signs and Symptoms; Skin and Connective Tissue Diseases; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic; Dermatomyositis; Behcet Syndrome; Antiphospholipid Syndrome; Pemphigoid, Bullous
• Other Study ID Numbers: RAY903CT

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to individual patient level data through the clinical study data request platform. For further details on Chugai's Data Sharing Policy and how to request access to related clinical study documents, see here (www.chugai-pharm.co.jp/english/profile/rd/ctds_request.html).

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No