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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06371417




Registration number
NCT06371417
Ethics application status
Date submitted
4/04/2024
Date registered
17/04/2024

Titles & IDs
Public title
Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)
Scientific title
Phase 1b Open-label Basket Trial of RAY121 to Inhibit Classical Complement Pathway in Immunological Diseases (RAINBOW Trial)
Secondary ID [1] 0 0
RAY902CT
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Antiphospholipid Syndrome (APS) 0 0
Bullous Pemphigoid (BP) 0 0
Behçet's Syndrome (BS) 0 0
Dermatomyositis (DM) 0 0
Immune-mediated Necrotizing Myopathy (IMNM) 0 0
Immune Thrombocytopenia (ITP) 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Other neurological disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Other cardiovascular diseases
Skin 0 0 0 0
Other skin conditions
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Skin 0 0 0 0
Dermatological conditions
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - RAY121

Experimental: RAY121 - All enrolled patients will receive RAY121 multiple dose


Treatment: Drugs: RAY121
Injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Adverse events (AEs)
Timepoint [1] 0 0
Baseline to Week 32
Secondary outcome [1] 0 0
RAY121 concentration
Timepoint [1] 0 0
Baseline to Week 32
Secondary outcome [2] 0 0
AUCt
Timepoint [2] 0 0
Baseline to Week 32
Secondary outcome [3] 0 0
Cmax
Timepoint [3] 0 0
Baseline to Week 32
Secondary outcome [4] 0 0
Cmin
Timepoint [4] 0 0
Baseline to Week 32
Secondary outcome [5] 0 0
Active C1s
Timepoint [5] 0 0
Baseline to Week 32
Secondary outcome [6] 0 0
Total C1s
Timepoint [6] 0 0
Baseline to Week 32
Secondary outcome [7] 0 0
Complement activity (classical pathway)
Timepoint [7] 0 0
Baseline to Week 32
Secondary outcome [8] 0 0
Anti-RAY121 antibodies
Timepoint [8] 0 0
Baseline to Week 32

Eligibility
Key inclusion criteria
1. Signed informed consent form
2. 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)
3. Ability to comply with the study protocol
4. For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use highly effective contraceptive methods
5. For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and agreement to refrain from donating sperm
6. 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 (aß2GPI) IgG isotype
* Clinical criteria

* Livedoid vasculopathy and presence of skin ulcer
* Acute/chronic aPL nephropathy
7. 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
8. 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
9. 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
10. 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
11. 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
Minimum age
18 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. History of anaphylaxis or hypersensitivity to a biologic agent
2. Active infection requiring systemic antiviral, antibiotics or antifungal
3. Planned surgery during the study
4. Pregnant or breastfeeding, or intending to become pregnant
5. Any serious medical condition or abnormality in clinical laboratory tests that precludes the patient's safe participation in and completion of the study
6. Clinically significant ECG abnormalities
7. Illicit drug or alcohol abuse
8. Clinical diagnosis of autoimmune diseases other than the target disease (except for Sjögren's syndrome in DM and IMNM)
9. Positive for hepatitis B surface antigen
10. Positive for hepatitis C virus antibody
11. Positive for human immunodeficiency virus antibody
12. Evidence of current infection with tuberculosis
13. History of cancer within 5 years
14. Treatment with investigational therapy within 28 days or 5 half-lives
15. Previous and current treatment with anti-C1s antibody at any time
16. Other complement inhibitors within 3 months
17. Patients who receive any treatments which fall into the Prohibited Therapy Criteria
18. Patients with an elevated alanine aminotransferase or aspartate aminotransferase > 1.5 × ULN in combination with an elevated total bilirubin > 1.5 × ULN
19. 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
20. 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
21. 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
22. 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
23. 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
24. 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/06/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/06/2026
Actual
Sample size
Target
144
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Westmead Hospital - Sydney
Recruitment hospital [3] 0 0
Campbelltown Public Hospital - Sydney
Recruitment hospital [4] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [5] 0 0
Box Hill Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2145 - Sydney
Recruitment postcode(s) [3] 0 0
2560 - Sydney
Recruitment postcode(s) [4] 0 0
3004 - Melbourne
Recruitment postcode(s) [5] 0 0
3128 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
New York
Country [3] 0 0
United States of America
State/province [3] 0 0
Ohio
Country [4] 0 0
United States of America
State/province [4] 0 0
Oregon
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
United States of America
State/province [6] 0 0
Texas
Country [7] 0 0
Austria
State/province [7] 0 0
Vienna
Country [8] 0 0
Bulgaria
State/province [8] 0 0
Sofia City Province
Country [9] 0 0
Bulgaria
State/province [9] 0 0
Stara Zagora Province
Country [10] 0 0
Canada
State/province [10] 0 0
Alberta
Country [11] 0 0
Canada
State/province [11] 0 0
Quebec
Country [12] 0 0
Croatia
State/province [12] 0 0
City Of Zagreb
Country [13] 0 0
Croatia
State/province [13] 0 0
Primorje-Gorski Kotar County
Country [14] 0 0
Czechia
State/province [14] 0 0
City Of Prague
Country [15] 0 0
France
State/province [15] 0 0
Occitanie
Country [16] 0 0
France
State/province [16] 0 0
Île-de-France
Country [17] 0 0
Germany
State/province [17] 0 0
Baden-Württemberg
Country [18] 0 0
Germany
State/province [18] 0 0
Bavaria
Country [19] 0 0
Germany
State/province [19] 0 0
Bundesländer
Country [20] 0 0
Germany
State/province [20] 0 0
Göttingen District
Country [21] 0 0
Germany
State/province [21] 0 0
Schleswig-Holstein
Country [22] 0 0
Hungary
State/province [22] 0 0
Csongrád-Csanád County
Country [23] 0 0
Hungary
State/province [23] 0 0
Budapest
Country [24] 0 0
Italy
State/province [24] 0 0
Forlì-Cesena Province
Country [25] 0 0
Italy
State/province [25] 0 0
Milan Province
Country [26] 0 0
Italy
State/province [26] 0 0
Turin Province
Country [27] 0 0
Japan
State/province [27] 0 0
Hokkaido
Country [28] 0 0
Japan
State/province [28] 0 0
Miyagi
Country [29] 0 0
Japan
State/province [29] 0 0
Osaka
Country [30] 0 0
Japan
State/province [30] 0 0
Shizuoka
Country [31] 0 0
Japan
State/province [31] 0 0
Tokyo
Country [32] 0 0
Netherlands
State/province [32] 0 0
Groningen Province
Country [33] 0 0
Netherlands
State/province [33] 0 0
Utrecht Province
Country [34] 0 0
Norway
State/province [34] 0 0
Agder County
Country [35] 0 0
Norway
State/province [35] 0 0
Rogaland County
Country [36] 0 0
Poland
State/province [36] 0 0
Masovian Voivodeship
Country [37] 0 0
Portugal
State/province [37] 0 0
Braga District
Country [38] 0 0
Portugal
State/province [38] 0 0
Porto District
Country [39] 0 0
Romania
State/province [39] 0 0
Bucharest Municipality
Country [40] 0 0
Romania
State/province [40] 0 0
Cluj County
Country [41] 0 0
Spain
State/province [41] 0 0
Community Of Madrid & Navarre
Country [42] 0 0
Spain
State/province [42] 0 0
Community Of Madrid
Country [43] 0 0
Spain
State/province [43] 0 0
Province Of Barcelona And Catalonia
Country [44] 0 0
Spain
State/province [44] 0 0
Province Of Córdoba
Country [45] 0 0
Spain
State/province [45] 0 0
Province Of Seville
Country [46] 0 0
Spain
State/province [46] 0 0
Valencian Community
Country [47] 0 0
Taiwan
State/province [47] 0 0
Taipei
Country [48] 0 0
Turkey
State/province [48] 0 0
Ankara Province
Country [49] 0 0
Turkey
State/province [49] 0 0
Istanbul Province

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Chugai Pharmaceutical
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Sponsor Chugai Pharmaceutical Co.Ltd
Address 0 0
clinical-trials@chugai-pharm.co.jp
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical trials information
Address 0 0
Country 0 0
Phone 0 0
only use Email
Fax 0 0
Email 0 0
clinical-trials@chugai-pharm.co.jp
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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).
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06371417