Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)
a study on Antiphospholipid Syndrome Bullous Pemphigoid (BP) Behçet's Disease Diabetes Myopathy Immune Thrombocytopenia
Summary
- Eligibility
- for people ages 18-85 (full criteria)
- Location
- at UC Irvine
- Dates
- study startedstudy ends around
Description
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).
Official Title
Phase 1b Open-label Basket Trial of RAY121 to Inhibit Classical Complement Pathway in Immunological Diseases (RAINBOW Trial)
Keywords
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM), Immune-mediated Necrotizing Myopathy (IMNM), Immune Thrombocytopenia (ITP), Antiphospholipid Syndrome, Bullous Pemphigoid, Behcet Syndrome, Dermatomyositis, Purpura, Thrombocytopenic, Idiopathic, RAY121
Eligibility
You can join if…
Open to people ages 18-85
- 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
- Laboratory criteria (aPL profile)
- 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 × 109/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 × 109/L from baseline by at least one prior line of therapy
- 1) Confirmed diagnosis of persistent/chronic ITP based on the following criteria:
You CAN'T join if...
- 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
- 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
Locations
- University of California-Irvine
accepting new patients
Orange 5379513 California 5332921 92868 United States - Oregon Health & Science University
accepting new patients
Portland 5746545 Oregon 5744337 97239 United States
Details
- Status
- accepting new patients
- Start Date
- Completion Date
- (estimated)
- Sponsor
- Chugai Pharmaceutical
- ID
- NCT06371417
- Phase
- Phase 1 research study
- Study Type
- Interventional
- Participants
- Expecting 144 study participants
- Last Updated