Antiphospholipid Syndrome (APS)Phase 1NCT06371417
Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)
A Phase 1 study testing RAY121 in people with Antiphospholipid Syndrome (APS) and Bullous Pemphigoid (BP), run by Chugai Pharmaceutical at 77 sites in 20 countries.
Trial 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)… Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS)
- What is being tested?
- RAY121
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 144 participants
- Where
- 77 sitesin 20 countries
- Study dates
- 19 Aug 2024to 30 Jun 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- History of anaphylaxis or hypersensitivity to a biologic agent
- Active infection requiring systemic antiviral, antibiotics or antifungal
- Planned surgery during the study
- Pregnant or breastfeeding, or intending to become pregnant
- Any serious medical condition or abnormality in clinical laboratory tests that precludes the patient's safe participation in and completion of the study
- Clinically significant ECG abnormalities
Show all 59Show fewer
- Illicit drug or alcohol abuse
- Clinical diagnosis of autoimmune diseases other than the target disease (except for Sjögren's syndrome in DM and IMNM)
- Positive for hepatitis B surface antigen
- Positive for hepatitis C virus antibody
- Positive for human immunodeficiency virus antibody
- Evidence of current infection with tuberculosis
- History of cancer within 5 years
- Treatment with investigational therapy within 28 days or 5 half-lives
- Previous and current treatment with anti-C1s antibody at any time
- Other complement inhibitors within 3 months
- Patients who receive any treatments which fall into the Prohibited Therapy Criteria
- Patients with an elevated alanine aminotransferase or aspartate aminotransferase > 1.5 × ULN in combination with an elevated total bilirubin > 1.5 × ULN
- APS cohort:
- 1) APS associated with other systemic autoimmune disease
- 2) Acute thrombosis (arterial or venous acute thrombosis diagnosis) within 30 days before screening
- 3) Patients with thrombotic APS without any anticoagulation treatment
- 4) Treatment with prohibited medications
- BP cohort:
- ・ 1) Initiation of treatment with or increase in the dose of systemic or topical corticosteroid within 2 weeks
- 2) Current treatment with a drug that may cause or exacerbate BP unless the dose has been stable
- 3) Initiation of treatment with topical calcineurin inhibitor, or topical phosphodiesterase (PDE) 4 inhibitor within 7 days
- 4) Treatment with prohibited medications
- BS cohort:
- ・ 1) BS-related active major organ involvement-ocular lesions requiring immunosuppressive therapy, pulmonary (e.g., pulmonary artery aneurysm), vascular (e.g., thrombophlebitis), gastrointestinal (e.g., ulcers along the gastrointestinal tract), and central nervous systems (e.g., meningoencephalitis) manifestations
- 2) History of venous or arterial thrombosis within 1 year
- 3) Treatment with prohibited medications
- DM cohort:
- ・ 1) PhGA-VAS improvement >= 3, or clinically relevant improvement between screening and baseline
- 2) Overlap myositis (except for overlap with Sjögren's syndrome), connective tissue disease associated DM, inclusion body myositis, polymyositis, IMNM, juvenile DM or drug-induced myopathy
- 3) Cancer-associated myositis
- 4) Significant muscle damage
- 5) Past history of severe Interstitial lung disease flare, severe non-infectious lung inflammation which required active intervention, or multiple episodes of lung disease
- 6) Severe respiratory muscle weakness
- 7) Severe bulbar palsy
- 8) Treatment with prohibited medications
- IMNM cohort:
- ・ 1) PhGA-VAS improvement >= 3, or clinically relevant improvement between screening and baseline
- ・ 2) Overlap myositis (except for overlap with Sjögren's syndrome), connective tissue disease associated DM, inclusion body myositis, polymyositis, juvenile DM or druginduced myopathy
- ・ 3) Cancer-associated myositis
- ・ 4) Significant muscle damage
- ・ 5) Past history of severe Interstitial lung disease (ILD) flare, severe non-infectious lung inflammation which required active intervention, or multiple episodes of lung disease
- ・ 6) Severe respiratory muscle weakness
- ・ 7) Severe bulbar palsy
- ・ 8) Treatment with prohibited medications
- ITP cohort:
- ・ 1) Secondary ITP
- ・ 2) Clinical diagnosis or history of Myelodysplastic Syndrome or autoimmune hemolytic anemia
- ・ 3) History of venous or arterial thrombosis within 12 months
- ・ 4) Patients who experienced major bleeding within 4 weeks
- 5) Treatment with prohibited medications
- 6) Any laboratory test results meet either of the following criteria at screening:
- Hemoglobin <10 g/dL
- Thyroid-stimulating hormone >= 10 μIU/mL
Trial locations and contacts
77 sites in 20 countries. Contact the study team or a site near you to ask about taking part.
77 sites in 20 countries
Japan13 sites, 13 recruiting
- Recruiting
Institute of Science Tokyo Hospital
Bunkyō-Ku, Tokyo, 113-8519
- Recruiting
Hamamatsu University Hospital
Hamamatsu, Shizuoka, 431-3192
- Recruiting
Hyogo Prefectural Harima-Himeji General Medical Center
Himeji, Hyōgo, 670-8560
- Recruiting
Tokai University Hospital
Isehara, Kanagawa, 259-1193
- Recruiting
National Center of Neurology and Psychiatry
Kodaira, Tokyo, 187-8551
- Recruiting
Okayama University Hospital
Okayama, 700-8558
- Recruiting
Toho University Omori Medical Center
Ōta-ku, Tokyo, 143-8540
- Recruiting
Kindai University Hospital
Sakai, Osaka, 590-0197
- Recruiting
Hokkaido University Hospital
Sapporo, Hokkaido, 060-0808
- Recruiting
Tohoku University Hospital
Sendai, Miyagi, 980-8574
- Recruiting
Tohoku Medical and Pharmaceutical University Hospital
Sendai, Miyagi, ９８３-８５１２
- Recruiting
The University of Osaka Hospital
Suita, Osaka, 565-0871
- Not yet recruiting
Toyama University Hospital
Toyama, 930-0194
United States11 sites, 7 recruiting
- Withdrawn
Amarillo Center for Clinical Research
Amarillo, Texas, 79124
- Recruiting
Austin Neuromuscular Center
Austin, Texas, 78759
- Recruiting
Johns Hopkins University
Baltimore, Maryland, 21224
- Recruiting
Universtity of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
- Withdrawn
Ohio State University
Columbus, Ohio, 43203
- Recruiting
Nerve and Muscle Center of Texas
Houston, Texas, 77030
- Withdrawn
Northwell Health, LLC PRIME
Lake Success, New York, 11042
- Withdrawn
Hospital for Special Surgery
New York, New York, 10021
- Recruiting
University of California-Irvine
Orange, California, 92868
- Recruiting
University of Pennsylvania, Perelman Center for Advanced Medicine
Philadelphia, Pennsylvania, 19104
- Recruiting
Oregon Health & Science University
Portland, Oregon, 97239
Spain7 sites, 6 recruiting
- Recruiting
Hospital Universitari Vall d'Hebron, Internal Medicine Dept.
Barcelona, Province of Barcelona and Catalonia, 08035
- Not yet recruiting
Hospital Universitario Reina Sofia
Córdoba, Province Of Córdoba, 14004
- Not yet recruiting
Hospital Universitario Ramon y Cajal, Servicio de Reumatologia
Madrid, Madrid, 28034
- Not yet recruiting
Hospital Universitario 12 de October
Madrid, Madrid, 28041
- Recruiting
Clinica Universidad de Navarra
Pamplona, Community of Madrid & Navarre, 28027
- Recruiting
Hospital Universitario Virgen del Rocio
Seville, Province Of Seville, 41013
- Withdrawn
Hospital Universitari i Politecnic La Fe
Valencia, Valencia, 46026
Germany6 sites, 4 recruiting
- Not yet recruiting
Universitaetsklinikum Erlangen
Erlangen, Bavaria, 91054
- Withdrawn
Universitaetsmedizin Goettingen
Göttingen, Göttingen District, 3075
- Withdrawn
Universitaetsklinikum Schleswig Holstein - Campus Luebeck, Klinik f Dermatologie, Allergologie u Venerologie
Lübeck, Schleswig-Holstein, 23538
- Recruiting
Immanuel Klinik Rudersdorf
Rüdersdorf, Brandenburg, 15622
- Not yet recruiting
Universitaetsklinikum Carl Gustav Carus TU Dresden, Klinik und Poliklinik f. Dermatologie
Sachsen, Bundesländer, 1307
- Not yet recruiting
Universitaetsklinikum Tuebingen
Tübingen, Baden-Wurttemberg, 72076
Australia5 sites, 5 recruiting
- Recruiting
Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050
- Recruiting
The Alfred Hospital
Melbourne, Victoria, 3004
- Recruiting
Box Hill Hospital
Melbourne, Victoria, 3128
- Recruiting
Westmead Hospital
Sydney, New South Wales, 2145
- Recruiting
Campbelltown Public Hospital
Sydney, New South Wales, 2560
Canada5 sites, 5 recruiting
- Recruiting
University of Alberta Hospital - Department of Anesthesiology and Pain Medicine
Edmonton, Alberta, T6G 2B7
- Recruiting
University of Alberta Hospital - Dermatology
Edmonton, Alberta, T6G 2G3
- Recruiting
The Royal Institution for the Advancement of Learning/McGill University
Montreal, Quebec, H3A 0G4
- Recruiting
Centre de Rhumatologie de l'Est du Quebec
Rimouski, Quebec, G5L 8W1
- Recruiting
DIEX Recherche Sherbrooke Inc.
Sherbrooke, Quebec, J1L 0H8
Bulgaria4 sites, 3 recruiting
- Withdrawn
Diagnostic Consultation Center CONVEX EOOD
Sofia, Sofia City Province, 1202
- Recruiting
UMHAT Sv Ivan Rilski EAD
Sofia, Sofia City Province, 1612
- Not yet recruiting
"SHATHD" EAD Sofia
Sofia, Sofia City Province, 1756
- Not yet recruiting
UMHAT "Prof. Dr. St. Kirkovich", AD
Stara Zagora, Stara Zagora Province, 6003
Croatia3 sites, 2 recruiting
- Withdrawn
Specialty Hospital Medico
Rijeka, Primorje-Gorski Kotar County, 51000
- Recruiting
Clinical Hospital Center "Sestre Milosrdnice"
Zagreb, City of Zagreb, 10000
- Not yet recruiting
University hospital centre Zagreb
Zagreb, City of Zagreb, 10000
Italy3 sites, 2 recruiting
- Recruiting
IRCCS Istituto Scientifico Romagnolo Per Lo Studio Dei Tumori "Dino Amadori" - IRST
Meldola, Forlì-Cesena Province, 47014
- Not yet recruiting
Istituto Clinico Humanitas
Milan, Milan Province, 20089
- Withdrawn
Ospedale San Giovanni Bosco
Torino, Turin Province, 10154
Norway3 sites, 3 recruiting
- Not yet recruiting
Sykehuset Ostfold Fredrikstad
Fredrikstad, Østfold fylke, 1603
- Recruiting
Sorlandet sykehus Kristiansand
Kristiansand, Agder County, 4604
- Recruiting
Stavanger Universitetssjukehus
Stavanger, Rogaland County, 4011
France2 sites, 2 recruiting
- Recruiting
Hopital Lapeyronie,Service d'Immuno Rhumatologie
Montpellier, Occitanie, 34295
- Recruiting
AP-HP Hôpital Universitaire Pitié Salpêtrière
Paris, Île-de-France Region, 75013
Hungary2 sites, 2 recruiting
- Recruiting
Semmelweis Egyetem
Budapest, 1083
- Not yet recruiting
Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont
Szeged, Csongrád-Csanád County, 6720
Netherlands2 sites, 2 recruiting
- Recruiting
University Medical Centre Groningen UMCG
Groningen, Groningen Province, 9713 GZ
- Recruiting
UMC Utrecht
Utrecht, Utrecht Province, 3584 CX
Portugal2 sites, 2 recruiting
- Not yet recruiting
Centro Clinico Academico Braga
Braga, Braga District, 471-243
- Not yet recruiting
Centro Hospitalar de Vila Nova de Gaia/Espinho, E.P.E.
Vila Nova de Gaia, Porto District, 4434-502
Romania2 sites, 2 recruiting
- Recruiting
Centrul Medical Monza SRL
Bucharest, Bucharest Municipality, 400015
- Recruiting
Oncology Institute Prof. Dr. Ion Chiricuta I.O.C.N.
Cluj-Napoca, Cluj, 11658
Taiwan2 sites, 2 recruiting
- Recruiting
National Taiwan University Hospital
Taipei, 100229
- Recruiting
Taichung Veterans General Hospital
Taipei, 407219
Turkey (Türkiye)2 sites
- Withdrawn
Dr. Abdurrahman Yurtaslan Oncology Teaching and Research Hospital
Ankara, Ankara, 06200
- Withdrawn
Istanbul University Istanbul Medical Faculty
Istanbul, Istanbul, 34093
Austria1 site
- Withdrawn
AKH - Medizinische Universitaet Wien, Abteilung fuer Klinische Pharmakologie
Vienna, 1090
Poland1 site, 1 recruiting
- Recruiting
Institute Reumatologii I'm. Eleonory Reicher
Warsaw, Masovian Voivodeship, 02-637
About this study
Official title: Phase 1b Open-label Basket Trial of RAY121 to Inhibit Classical Complement Pathway in Immunological Diseases (RAINBOW Trial)
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).
- Conditions
- Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM), Immune-mediated Necrotizing Myopathy (IMNM), Immune Thrombocytopenia (ITP)
- Being tested
- RAY121
- Medical subject headings
- Antiphospholipid Syndrome, Pemphigoid, Bullous, Behcet Syndrome, Dermatomyositis, Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RAY121Drug
Injection
| Study group | Role | What they receive |
|---|---|---|
| RAY121 | Experimental | All enrolled patients will receive RAY121 multiple dose Receives: Drug: RAY121 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events (AEs)
Incidence, severity, and causal relationship of AEs
Measured: Baseline to Week 32
Secondary outcomes (8)
RAY121 concentration
Serum RAY121 concentration
Measured: Baseline to Week 32
AUCτ
Area under the concentration-time curve over a dosing interval (AUCτ)
Measured: Baseline to Week 32
Cmax
Maximum observed serum concentration (Cmax)
Measured: Baseline to Week 32
Cmin
Minimum observed serum concentration (Cmin)
Measured: Baseline to Week 32
Active C1s
Active C1s
Measured: Baseline to Week 32
Total C1s
Total C1s
Measured: Baseline to Week 32
Complement activity (classical pathway)
Complement activity (classical pathway)
Measured: Baseline to Week 32
Anti-RAY121 antibodies
Titer of anti-RAY121 antibodies
Measured: Baseline to Week 32
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2024
- Study start19 Aug 2024
- Primary completion31 Mar 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Antiphospholipid Syndrome (APS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 144 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chugai Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sponsor Chugai Pharmaceutical Co.Ltd, Study director, clinical-trials@chugai-pharm.co.jp
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06371417
- Sponsor's protocol ID
- RAY902CT
- Status last verified
- Jul 2026
- First submitted
- 4 Apr 2024
- Last updated
- 22 Jul 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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).