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

Official title

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

Overview

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).

Interventions

• Drug RAY121
  Injection

Primary outcome measures

• Adverse events (AEs) [Time frame: Baseline to Week 52]

Secondary outcome measures (5)

• RAY121 concentration [Time frame: Baseline to Week 52]
• Change from baseline in active C1s [Time frame: Baseline to Week 52]
• Change from baseline in total C1s [Time frame: Baseline to Week 52]
• Change from baseline in complement activity (classical pathway) [Time frame: Baseline to Week 52]
• Anti-RAY121 antibodies [Time frame: Baseline to Week 52]

Eligibility criteria

Inclusion criteria

• 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

Exclusion criteria

• 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

Japan · 16 centers

• Hokkaido University Hospital — Sapporo
• Hyogo Prefectural Harima-Himeji General Medical Center — Himeji
• Tokai University Hospital — Isehara
• Tohoku University Hospital — Sendai
• Tohoku Medical and Pharmaceutical University Hospital — Sendai
• National Hospital Organization Osaka Minami Medical Center — Kawachi-Nagano
• Kindai University Hospital — Sakai
• The University of Osaka Hospital — Suita
• … and 8 more centers

Spain · 7 centers

• Clinica Universidad de Navarra — Pamplona
• Hospital Universitario Ramon y Cajal, Servicio de Reumatologia — Madrid
• Hospital Universitario 12 de October — Madrid
• Hospital Universitari Vall d'Hebron, Internal Medicine Dept. — Barcelona
• Hospital Universitario Reina Sofia — Córdoba
• Hospital Universitario Virgen del Rocio — Seville
• Hospital Universitari i Politecnic La Fe — Valencia

Australia · 5 centers

• Royal Prince Alfred Hospital — Camperdown
• Westmead Hospital — Sydney
• Campbelltown Public Hospital — Sydney
• The Alfred Hospital — Melbourne
• Box Hill Hospital — Melbourne

Germany · 5 centers

• Universitaetsklinikum Tuebingen — Tübingen
• Universitaetsklinikum Erlangen — Erlangen
• Universitaetsklinikum Carl Gustav Carus TU Dresden, Klinik und Poliklinik f. Dermatologie — Sachsen
• Universitaetsmedizin Goettingen — Göttingen
• Universitaetsklinikum Schleswig Holstein - Campus Luebeck, Klinik f Dermatologie, Allergol — Lübeck

Bulgaria · 3 centers

• Diagnostic Consultation Center CONVEX EOOD — Sofia
• "SHATHD" EAD Sofia — Sofia
• UMHAT "Prof. Dr. St. Kirkovich", AD — Stara Zagora

Croatia · 3 centers

• Clinical Hospital Center "Sestre Milosrdnice" — Zagreb
• University hospital centre Zagreb — Zagreb
• Specialty Hospital Medico — Rijeka

Italy · 3 centers

• IRCCS Istituto Scientifico Romagnolo Per Lo Studio Dei Tumori "Dino Amadori" - IRST — Meldola
• Istituto Clinico Humanitas — Milan
• Ospedale San Giovanni Bosco — Torino

France · 2 centers

• Hopital Lapeyronie,Service d'Immuno Rhumatologie — Montpellier
• AP-HP Hôpital Universitaire Pitié Salpêtrière — Paris

Hungary · 2 centers

• Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont — Szeged
• Semmelweis Egyetem — Budapest

Netherlands · 2 centers

• University Medical Centre Groningen UMCG — Groningen
• UMC Utrecht — Utrecht

Norway · 2 centers

• Sorlandet sykehus Kristiansand — Kristiansand
• Stavanger Universitetssjukehus — Stavanger

Portugal · 2 centers

• Centro Clinico Academico Braga — Braga
• Centro Hospitalar de Vila Nova de Gaia/Espinho, E.P.E. — Vila Nova de Gaia

Romania · 2 centers

• Centrul Medical Monza SRL — Bucharest
• Oncology Institute Prof. Dr. Ion Chiricuta I.O.C.N. — Cluj-Napoca

Taiwan · 2 centers

• National Taiwan University Hospital — Taipei
• Taichung Veterans General Hospital — Taipei

Turkey (Türkiye) · 2 centers

• Dr. Abdurrahman Yurtaslan Oncology Teaching and Research Hospital — Ankara
• Istanbul University Istanbul Medical Faculty — Istanbul

Austria · 1 center

• AKH - Medizinische Universitaet Wien, Abteilung fuer Klinische Pharmakologie — Vienna

Canada · 1 center

• DiIEX Recherche Sherbrooke, Inc — Sherbrooke

Czechia · 1 center

• Sanatorium Profesora Arenbergera — Prague

Poland · 1 center

• Institute Reumatologii I'm. Eleonory Reicher — Warsaw

Identifiers

NCT: NCT06723106 · RAY903CT

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗