NCT06371417招募中1 期
Phase 1b Open-label Basket Trial of RAY121 to Inhibit Classical Complement Pathway in Immunological Diseases (RAINBOW Trial)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 144
- 试验地点
- 114
- 主要终点
- Adverse events (AEs)
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent form
- •Age ≥ 18 and ≤ 85 at the time of signing informed consent form 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 LA test
- •Persistently positive aCL IgG isotype
- •Persistently positive aβ2GPI IgG isotype
- •Clinical criteria
- •Livedoid vasculopathy and presence of skin ulcer
- •Acute/chronic aPL nephropathy
- •1) Predominant cutaneous lesions 2) Diagnosis with BP with following assessments positive:
- •Positive direct immunofluorescence, and either
- •Positive indirect immunofluorescence, or
- •Positive serology on ELISA for BP180 autoantibody 3) BPDAI score >= 20 4) Weekly average of daily Peak Pruritus NRS >=4 5) Accept to take photograph of bullous lesions
- •8. BS cohort:
- •Diagnosed with BS
- •Oral ulcers that occurred at least 3 times in the previous 12 month period
- •Have at least 2 oral ulcers over the 4 weeks prior to screening
- •Have at least 2 oral ulcers at Week 0
- •Have prior treatment with at least 1 non-biologic BS therapy
- •Patients who need systemic therapy as whose oral or mucocutaneous ulcers cannot be adequately controlled by topical therapy
- •9. DM cohort:
- •Diagnosed with definite or probable inflammatory myopathies and categorized as DM
- •Patients with inadequate response to corticosteroids and/or immune-suppressants or intolerance to DM therapies
- •MMT-8 score < 142, with at least one abnormality in the following Core Set Measures:
- •PtGA-VAS >= 2 cm
- •PhGA-VAS >= 2 cm
- •Global extra-muscular activity >= 2 cm
- •At least one muscle enzyme > 1.5 times ULN
- •HAQ >= 0.25
- •Moderate to severe DM defined as CDASI activity score > 14
- •10. IMNM cohort:
- •Clinically Diagnosed with IMNM as anti-HMGCR myopathy or anti-SRP myopathy
- •CK > 1,000 U/L
- •Patients who have an inadequate response to corticosteroids and/or immunesuppressants or intolerance to IMNM therapies
- •MMT-8 score < 142
- •11. ITP cohort:
- •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
- •Lack of an sustained adequate platelet count response to a thrombopoietin receptor agonist and at least one other ITP treatment or a second TPO-RA
- •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
排除标准
- •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
- •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
- •・ 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
- •・ 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
- •・ 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
- 另有 6 项未显示
研究组 & 干预措施
RAY121
Experimental
All enrolled patients will receive RAY121 multiple dose
干预措施: RAY121 (Drug)
结局指标
主要结局
Adverse events (AEs)
时间窗: Baseline to Week 32
Incidence, severity, and causal relationship of AEs
次要结局
- RAY121 concentration(Baseline to Week 32)
- Cmin(Baseline to Week 32)
- Active C1s(Baseline to Week 32)
- Total C1s(Baseline to Week 32)
- Complement activity (classical pathway)(Baseline to Week 32)
- Anti-RAY121 antibodies(Baseline to Week 32)
- AUCτ(Baseline to Week 32)
- Cmax(Baseline to Week 32)
研究者
Clinical trials information
Scientific
Chugai Pharmaceutical Co. Ltd.
研究点 (114)
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