Prowadzone metodą otwartej próby badanie koszykowe fazy Ib dotyczące stosowania RAY121 w celu hamowania klasycznej drogi aktywacji układu dopełniacza w chorobach immunologicznych (Badanie RAINBOW)

Kryteria wyłączenia kto nie może wziąć udziału (24)

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