Clinical trial NCT06723106: 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)
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE1
Conditions: Antiphospholipid Syndrome (APS)
Bullous Pemphigoid (BP)
Behçet's Syndrome (BS)
Dermatomyositis (DM)
Immune-mediated Necrotizing Myopathy (IMNM)
Immune Thrombocytopenia (ITP)
Lead sponsor: Chugai Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2024-12-09
Enrollment: 144
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility criteria: 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

Primary outcomes:
measure: Adverse events (AEs)
timeFrame: Baseline to Week 52
Secondary outcomes:
measure: RAY121 concentration
timeFrame: Baseline to Week 52
measure: Change from baseline in active C1s
timeFrame: Baseline to Week 52
measure: Change from baseline in total C1s
timeFrame: Baseline to Week 52
measure: Change from baseline in complement activity (classical pathway)
timeFrame: Baseline to Week 52
measure: Anti-RAY121 antibodies
timeFrame: Baseline to Week 52
Other outcomes:
Locations:
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
state: New South Wales
city: Sydney
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Sydney
country: Australia
facility: Campbelltown Public Hospital
state: New South Wales
city: Melbourne
country: Australia
facility: The Alfred Hospital
state: Victoria
city: Melbourne
country: Australia
facility: Box Hill Hospital
state: Victoria
city: Vienna
country: Austria
facility: AKH - Medizinische Universitaet Wien, Abteilung fuer Klinische Pharmakologie
city: Sofia
country: Bulgaria
facility: Diagnostic Consultation Center CONVEX EOOD
state: Sofia City Province
city: Sofia
country: Bulgaria
facility: "SHATHD" EAD Sofia
state: Sofia City Province
city: Stara Zagora
country: Bulgaria
facility: UMHAT "Prof. Dr. St. Kirkovich", AD
state: Stara Zagora Province
city: Sherbrooke
country: Canada
facility: DiIEX Recherche Sherbrooke, Inc
state: Quebec
city: Zagreb
country: Croatia
facility: Clinical Hospital Center "Sestre Milosrdnice"
state: City Of Zagreb
city: Zagreb
country: Croatia
facility: University hospital centre Zagreb
state: City Of Zagreb
city: Rijeka
country: Croatia
facility: Specialty Hospital Medico
state: Primorje-Gorski Kotar County
city: Prague
country: Czechia
facility: Sanatorium Profesora Arenbergera
state: City Of Prague
city: Montpellier
country: France
facility: Hopital Lapeyronie,Service d'Immuno Rhumatologie
state: Occitanie
city: Paris
country: France
facility: AP-HP Hôpital Universitaire Pitié Salpêtrière
state: Île-de-France
city: Tuebingen
country: Germany
facility: Universitaetsklinikum Tuebingen
state: Baden-Württemberg
city: Erlangen
country: Germany
facility: Universitaetsklinikum Erlangen
state: Bavaria
city: Sachsen
country: Germany
facility: Universitaetsklinikum Carl Gustav Carus TU Dresden, Klinik und Poliklinik f. Dermatologie
state: Bundesländer
city: Göttingen
country: Germany
facility: Universitaetsmedizin Goettingen
state: Göttingen District
city: Luebeck
country: Germany
facility: Universitaetsklinikum Schleswig Holstein - Campus Luebeck, Klinik f Dermatologie, Allergologie u Venerologie
state: Schleswig-Holstein
city: Szeged
country: Hungary
facility: Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont
state: Csongrád-Csanád County
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Meldola
country: Italy
facility: IRCCS Istituto Scientifico Romagnolo Per Lo Studio Dei Tumori "Dino Amadori" - IRST
state: Forlì-Cesena Province
city: Milano
country: Italy
facility: Istituto Clinico Humanitas
state: Milan Province
city: Torino
country: Italy
facility: Ospedale San Giovanni Bosco
state: Turin Province
city: Sapporo
country: Japan
facility: Hokkaido University Hospital
state: Hokkaido
city: Sendai
country: Japan
facility: Tohoku University Hospital
state: Miyagi
city: Osakasayama
country: Japan
facility: Kindai University Hospital
state: Osaka
city: Suita
country: Japan
facility: Osaka University Hospital
state: Osaka
city: Hamamatsu
country: Japan
facility: Hamamatsu University Hospital
state: Shizuoka
city: Kodaira
country: Japan
facility: National Center of Neurology and Psychiatry
state: Tokyo
city: Ota-ku
country: Japan
facility: Toho University Omori Medical Center
state: Tokyo
city: Okayama
country: Japan
facility: Okayama University Hospital
city: Groningen
country: Netherlands
facility: University Medical Centre Groningen UMCG
state: Groningen Province
city: Utrecht
country: Netherlands
facility: UMC Utrecht
state: Utrecht Province
city: Kristiansand
country: Norway
facility: Sorlandet sykehus Kristiansand
state: Agder County
city: Stavanger
country: Norway
facility: Stavanger Universitetssjukehus
state: Rogaland County
city: Warszawa
country: Poland
facility: Institute Reumatologii I'm. Eleonory Reicher
state: Masovian Voivodeship
city: Braga
country: Portugal
facility: Centro Clinico Academico Braga
state: Braga District
city: Vila Nova de Gaia
country: Portugal
facility: Centro Hospitalar de Vila Nova de Gaia/Espinho, E.P.E.
state: Porto District
city: Bucharest
country: Romania
facility: Centrul Medical Monza SRL
state: Bucharest Municipality
city: Cluj-Napoca
country: Romania
facility: Oncology Institute Prof. Dr. Ion Chiricuta I.O.C.N.
state: Cluj County
city: Pamplona
country: Spain
facility: Clinica Universidad de Navarra
state: Community Of Madrid & Navarre
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal, Servicio de Reumatologia
state: Community Of Madrid
city: Madrid
country: Spain
facility: Hospital Universitario 12 de October
state: Community Of Madrid
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron, Internal Medicine Dept.
state: Province Of Barcelona And Catalonia
city: Cordoba
country: Spain
facility: Hospital Universitario Reina Sofia
state: Province Of Córdoba
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
state: Province Of Seville
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe
state: Valencian Community
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Taipei
country: Taiwan
facility: Taichung Veterans General Hospital
city: Ankara
country: Turkey
facility: Dr. Abdurrahman Yurtaslan Oncology Teaching and Research Hospital
state: Ankara Province
city: Istanbul
country: Turkey
facility: Istanbul University Istanbul Medical Faculty
state: Istanbul Province
Study officials:
affiliation: clinical-trials@chugai-pharm.co.jp
name: Sponsor Chugai Pharmaceutical Co.Ltd
role: STUDY_DIRECTOR
References: