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

Phase 1

• Conditions: Antiphospholipid Syndrome (APS); Bullous Pemphigoid (BP); Dermatomyositis (DM); Immune-mediated Necrotizing Myopathy (IMNM); Behçet's Syndrome (BS); Immune Thrombocytopenia (ITP)
• Interventions: Drug: RAY121

• Registration Number: NCT06723106
• Lead Sponsor: Chugai Pharmaceutical

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-11-27
• Study First Submitted QC: 2024-12-06
• Study Start: 2024-12-09
• Study First Posted: 2024-12-09
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-30
• Last Update Posted: 2025-04-01
• Primary Completion: 2027-04-30
• Study Completion: 2027-04-30

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 144

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
RAY121	RAY121	All enrolled patients will receive RAY121 multiple dose

Primary Outcome Measures

Name	Time	Method
Adverse events (AEs)	Baseline to Week 52	Incidence, severity, and causal relationship of AEs

Secondary Outcome Measures

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

Trial Locations

Locations (52)

Royal Prince Alfred Hospital; 🇦🇺 Camperdown, New South Wales, Australia

Westmead Hospital; 🇦🇺 Sydney, New South Wales, Australia

Campbelltown Public Hospital; 🇦🇺 Sydney, New South Wales, Australia

The Alfred Hospital; 🇦🇺 Melbourne, Victoria, Australia

Box Hill Hospital; 🇦🇺 Melbourne, Victoria, Australia

AKH - Medizinische Universitaet Wien, Abteilung fuer Klinische Pharmakologie; 🇦🇹 Vienna, Austria

Diagnostic Consultation Center CONVEX EOOD; 🇧🇬 Sofia, Sofia City Province, Bulgaria

"SHATHD" EAD Sofia; 🇧🇬 Sofia, Sofia City Province, Bulgaria

UMHAT "Prof. Dr. St. Kirkovich", AD; 🇧🇬 Stara Zagora, Stara Zagora Province, Bulgaria

Clinical Hospital Center "Sestre Milosrdnice"; 🇭🇷 Zagreb, City Of Zagreb, Croatia

University hospital centre Zagreb; 🇭🇷 Zagreb, City Of Zagreb, Croatia

Specialty Hospital Medico; 🇭🇷 Rijeka, Primorje-Gorski Kotar County, Croatia

Sanatorium Profesora Arenbergera; 🇨🇿 Prague, City Of Prague, Czechia

Hopital Lapeyronie,Service d'Immuno Rhumatologie; 🇫🇷 Montpellier, Occitanie, France

AP-HP Hôpital Universitaire Pitié Salpêtrière; 🇫🇷 Paris, Île-de-France, France

Universitaetsklinikum Tuebingen; 🇩🇪 Tuebingen, Baden-Württemberg, Germany

Universitaetsklinikum Erlangen; 🇩🇪 Erlangen, Bavaria, Germany

Universitaetsklinikum Carl Gustav Carus TU Dresden, Klinik und Poliklinik f. Dermatologie; 🇩🇪 Sachsen, Bundesländer, Germany

Universitaetsmedizin Goettingen; 🇩🇪 Göttingen, Göttingen District, Germany

Universitaetsklinikum Schleswig Holstein - Campus Luebeck, Klinik f Dermatologie, Allergologie u Venerologie; 🇩🇪 Luebeck, Schleswig-Holstein, Germany

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont; 🇭🇺 Szeged, Csongrád-Csanád County, Hungary

Semmelweis Egyetem; 🇭🇺 Budapest, Hungary

IRCCS Istituto Scientifico Romagnolo Per Lo Studio Dei Tumori "Dino Amadori" - IRST; 🇮🇹 Meldola, Forlì-Cesena Province, Italy

Istituto Clinico Humanitas; 🇮🇹 Milano, Milan Province, Italy

Ospedale San Giovanni Bosco; 🇮🇹 Torino, Turin Province, Italy

Hokkaido University Hospital; 🇯🇵 Sapporo, Hokkaido, Japan

Tohoku University Hospital; 🇯🇵 Sendai, Miyagi, Japan

Kindai University Hospital; 🇯🇵 Osakasayama, Osaka, Japan

Osaka University Hospital; 🇯🇵 Suita, Osaka, Japan

Hamamatsu University Hospital; 🇯🇵 Hamamatsu, Shizuoka, Japan

National Center of Neurology and Psychiatry; 🇯🇵 Kodaira, Tokyo, Japan

Toho University Omori Medical Center; 🇯🇵 Ota-ku, Tokyo, Japan

University Medical Centre Groningen UMCG; 🇳🇱 Groningen, Groningen Province, Netherlands

UMC Utrecht; 🇳🇱 Utrecht, Utrecht Province, Netherlands

Sorlandet sykehus Kristiansand; 🇳🇴 Kristiansand, Agder County, Norway

Stavanger Universitetssjukehus; 🇳🇴 Stavanger, Rogaland County, Norway

Institute Reumatologii I'm. Eleonory Reicher; 🇵🇱 Warszawa, Masovian Voivodeship, Poland

Centro Clinico Academico Braga; 🇵🇹 Braga, Braga District, Portugal

Centro Hospitalar de Vila Nova de Gaia/Espinho, E.P.E.; 🇵🇹 Vila Nova de Gaia, Porto District, Portugal

Centrul Medical Monza SRL; 🇷🇴 Bucharest, Bucharest Municipality, Romania

Oncology Institute Prof. Dr. Ion Chiricuta I.O.C.N.; 🇷🇴 Cluj-Napoca, Cluj County, Romania

Clinica Universidad de Navarra; 🇪🇸 Pamplona, Community Of Madrid & Navarre, Spain

Hospital Universitario Ramon y Cajal, Servicio de Reumatologia; 🇪🇸 Madrid, Community Of Madrid, Spain

Hospital Universitario 12 de October; 🇪🇸 Madrid, Community Of Madrid, Spain

Hospital Universitari Vall d'Hebron, Internal Medicine Dept.; 🇪🇸 Barcelona, Province Of Barcelona And Catalonia, Spain

Hospital Universitario Reina Sofia; 🇪🇸 Cordoba, Province Of Córdoba, Spain

Hospital Universitario Virgen del Rocio; 🇪🇸 Sevilla, Province Of Seville, Spain

Hospital Universitari i Politecnic La Fe; 🇪🇸 Valencia, Valencian Community, Spain

National Taiwan University Hospital; 🇨🇳 Taipei, Taiwan

Taichung Veterans General Hospital; 🇨🇳 Taipei, Taiwan

Dr. Abdurrahman Yurtaslan Oncology Teaching and Research Hospital; 🇹🇷 Ankara, Ankara Province, Turkey

Istanbul University Istanbul Medical Faculty; 🇹🇷 Istanbul, Istanbul Province, Turkey