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

Status: Recruiting
Location: See all (66) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
SUMMARY

This Phase 1b basket trial will investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary efficacy of RAY121, a inhibitor of classical complement pathway, after multiple dose administration 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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 85
Healthy Volunteers: f
View:

• Signed informed consent form

• Age \>= 18 and \<=75 at the time of signing informed consent form (except for BP; Age \>=18 and \<= 85 with Karnofsky score \>= 60% at screening)

• Ability to comply with the study protocol

• For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use highly effective contraceptive methods

• For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and agreement to refrain from donating sperm

• APS cohort: Established primary APS defined by the following criteria (at least one of the laboratory criteria and one of the clinical criteria must be met):

‣ Laboratory criteria (aPL profile)

• Persistently positive lupus anticoagulant (LA) test

∙ Persistently positive anticardiolipin (aCL) immunoglobulin G (IgG) isotype

∙ Persistently positive anti-beta-2 glycoprotein-1 (aβ2GPI) IgG isotype

⁃ Clinical criteria

• Livedoid vasculopathy and presence of skin ulcer

∙ Acute/chronic aPL nephropathy

• BP cohort:

‣ 1\) Age \>= 18 and \<= 85 with Karnofsky score \>= 60 %

⁃ 2\) Predominant cutaneous lesions

⁃ 3\) Diagnosis with BP with following assessments positive:

⁃ a Positive direct immunofluorescence, and either

⁃ b Positive indirect immunofluorescence, or

⁃ c Positive serology on ELISA for BP180 autoantibody

⁃ 4\) Bullous Pemphigoid Disease Area Index (BPDAI) score \>= 20

⁃ 5\) Weekly average of daily Peak Pruritus Numerical Rating Score (PP-NRS) \>=4

⁃ 6\) Accept to take photograph of bullous lesions

• BS cohort:

‣ 1\) Diagnosed with BS

⁃ 2\) Oral ulcers that occurred at least 3 times in the previous 12 month period

⁃ 3\) Have at least 2 oral ulcers over the 4 weeks prior to screening

⁃ 4\) Have at least 2 oral ulcers at Week 0

⁃ 5\) Have prior treatment with at least 1 non-biologic BS therapy

⁃ 6\) Patients who need systemic therapy as whose oral or mucocutaneous ulcers cannot be adequately controlled by topical therapy

• DM cohort:

‣ 1\) Diagnosed with definite or probable inflammatory myopathies and categorized as DM

⁃ 2\) Patients with inadequate response to corticosteroids and/or immune-suppressants or intolerance to DM therapies

⁃ 3\) Manual Muscle Test-8 (MMT-8) score \< 142, with at least one abnormality in the following Core Set Measures:

• Patient Global Activity Visual Analogue Scale (PtGA-VAS) \>= 2 cm

∙ Physician Global Activity Visual Analogue Scale (PhGA-VAS) \>= 2 cm

∙ Global extra-muscular activity \>= 2 cm

∙ At least one muscle enzyme \> 1.5 times upper limit of normal (ULN)

∙ Health Assessment Questionnaire (HAQ) \>= 0.25

⁃ 4\) Moderate to severe DM defined as CDASI activity score \> 14

⁃ IMNM cohort:

∙ 1\) Clinically Diagnosed with IMNM as anti-HMGCR myopathy or anti-SRP myopathy

‣ 2\) Creatine kinase (CK) \> 1,000 U/L

‣ 3\) Patients who have an inadequate response to corticosteroids and/or immunosuppressants or intolerance to IMNM therapies

‣ 4\) MMT-8 score \< 142

⁃ ITP cohort:

∙ 1\) Confirmed diagnosis of persistent/chronic ITP based on the following criteria:

⁃ ITP defined per the current guidelines

• Platelet count \<= 30 × 10\^9/L on 2 consecutive occasions

‣ 2\) Lack of an sustained adequate platelet count response to a thrombopoietin receptor agonist and at least one other ITP treatment or a second thrombopoietin receptor agonist (TPO-RA)

‣ 3\) A history of response with an platelet counts increase more than 20 × 10\^9/L from baseline by at least one prior line of therapy

Locations
United States
California
University of California-Irvine
RECRUITING
Orange
Maryland
Johns Hopkins University
NOT_YET_RECRUITING
Baltimore
New York
Northwell Health, LLC PRIME
NOT_YET_RECRUITING
Lake Success
Hospital for Special Surgery
RECRUITING
New York
Ohio
Ohio State University
WITHDRAWN
Columbus
Oregon
Oregon Health & Science University
RECRUITING
Portland
Pennsylvania
University of Pennsylvania, Perelman Center for Advanced Medicine
NOT_YET_RECRUITING
Philadelphia
Texas
Amarillo Center for Clinical Research
RECRUITING
Amarillo
Nerve and Muscle Center of Texas
NOT_YET_RECRUITING
Houston
Other Locations
Australia
Royal Prince Alfred Hospital
RECRUITING
Camperdown
Box Hill Hospital
RECRUITING
Melbourne
The Alfred Hospital
RECRUITING
Melbourne
Campbelltown Public Hospital
RECRUITING
Sydney
Westmead Hospital
RECRUITING
Sydney
Austria
AKH - Medizinische Universitaet Wien, Abteilung fuer Klinische Pharmakologie
NOT_YET_RECRUITING
Vienna
Bulgaria
Diagnostic Consultation Center CONVEX EOOD
NOT_YET_RECRUITING
Sofia
SHATHD EAD Sofia
NOT_YET_RECRUITING
Sofia
UMHAT Prof. Dr. St. Kirkovich, AD
NOT_YET_RECRUITING
Stara Zagora
Canada
University of Alberta Hospital - Department of Anesthesiology and Pain Medicine
NOT_YET_RECRUITING
Edmonton
University of Alberta Hospital - Dermatology
NOT_YET_RECRUITING
Edmonton
The Royal Institution for the Advancement of Learning/McGill University
NOT_YET_RECRUITING
Montreal
Centre de Rhumatologie de l'Est du Quebec
NOT_YET_RECRUITING
Rimouski
DIEX Recherche Sherbrooke Inc.
RECRUITING
Sherbrooke
Croatia
Specialty Hospital Medico
NOT_YET_RECRUITING
Rijeka
Clinical Hospital Center Sestre Milosrdnice
NOT_YET_RECRUITING
Zagreb
University hospital centre Zagreb
NOT_YET_RECRUITING
Zagreb
France
Hopital Lapeyronie,Service d'Immuno Rhumatologie
NOT_YET_RECRUITING
Montpellier
AP-HP Hôpital Universitaire Pitié Salpêtrière
RECRUITING
Paris
Germany
Universitaetsklinikum Erlangen
NOT_YET_RECRUITING
Erlangen
Universitaetsmedizin Goettingen
NOT_YET_RECRUITING
Göttingen
Universitaetsklinikum Schleswig Holstein - Campus Luebeck, Klinik f Dermatologie, Allergologie u Venerologie
NOT_YET_RECRUITING
Lübeck
Universitaetsklinikum Carl Gustav Carus TU Dresden, Klinik und Poliklinik f. Dermatologie
NOT_YET_RECRUITING
Sachsen
Universitaetsklinikum Tuebingen
NOT_YET_RECRUITING
Tübingen
Hungary
Semmelweis Egyetem
NOT_YET_RECRUITING
Budapest
Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont
NOT_YET_RECRUITING
Szeged
Italy
IRCCS Istituto Scientifico Romagnolo Per Lo Studio Dei Tumori Dino Amadori - IRST
NOT_YET_RECRUITING
Meldola
Istituto Clinico Humanitas
NOT_YET_RECRUITING
Milan
Ospedale San Giovanni Bosco
NOT_YET_RECRUITING
Torino
Japan
Hamamatsu University Hospital
RECRUITING
Hamamatsu
National Center of Neurology and Psychiatry
RECRUITING
Kodaira
Okayama University Hospital
RECRUITING
Okayama
Toho University Omori Medical Center
RECRUITING
Ōta-ku
Hokkaido University Hospital
RECRUITING
Sapporo
Kindai University Hospital
NOT_YET_RECRUITING
Sayama
Tohoku University Hospital
RECRUITING
Sendai
Osaka University Hospital
RECRUITING
Suita
Netherlands
University Medical Centre Groningen UMCG
NOT_YET_RECRUITING
Groningen
UMC Utrecht
NOT_YET_RECRUITING
Utrecht
Norway
Sorlandet sykehus Kristiansand
NOT_YET_RECRUITING
Kristiansand
Stavanger Universitetssjukehus
NOT_YET_RECRUITING
Stavanger
Poland
Institute Reumatologii I'm. Eleonory Reicher
RECRUITING
Warsaw
Portugal
Centro Clinico Academico Braga
NOT_YET_RECRUITING
Braga
Centro Hospitalar de Vila Nova de Gaia/Espinho, E.P.E.
NOT_YET_RECRUITING
Vila Nova De Gaia
Romania
Centrul Medical Monza SRL
RECRUITING
Bucharest
Oncology Institute Prof. Dr. Ion Chiricuta I.O.C.N.
RECRUITING
Cluj-napoca
Spain
Hospital Universitari Vall d'Hebron, Internal Medicine Dept.
NOT_YET_RECRUITING
Barcelona
Hospital Universitario Reina Sofia
NOT_YET_RECRUITING
Córdoba
Hospital Universitario 12 de October
NOT_YET_RECRUITING
Madrid
Hospital Universitario Ramon y Cajal, Servicio de Reumatologia
NOT_YET_RECRUITING
Madrid
Clinica Universidad de Navarra
NOT_YET_RECRUITING
Pamplona
Hospital Universitario Virgen del Rocio
RECRUITING
Seville
Hospital Universitari i Politecnic La Fe
NOT_YET_RECRUITING
Valencia
Taiwan
National Taiwan University Hospital
RECRUITING
Taipei
Taichung Veterans General Hospital
NOT_YET_RECRUITING
Taipei
Turkey
Dr. Abdurrahman Yurtaslan Oncology Teaching and Research Hospital
NOT_YET_RECRUITING
Ankara
Istanbul University Istanbul Medical Faculty
NOT_YET_RECRUITING
Istanbul
Contact Information
Primary
Clinical trials information
clinical-trials@chugai-pharm.co.jp
only use Email
Time Frame
Start Date: 2024-08-19
Estimated Completion Date: 2026-06-30
Participants
Target number of participants: 144
Treatments
Experimental: RAY121
All enrolled patients will receive RAY121 multiple dose
Sponsors
Leads: Chugai Pharmaceutical

This content was sourced from clinicaltrials.gov