PHASE IB OPEN LABEL BASKET STUDY OF RAY121
Study Description
Eligibility
Inclusion Criteria:
- Signed informed consent form
- Age greater than or equal to 18 and less than or equal to 75 at the time of signing informed consent form (except for BP; Age greater than or equal to 18 and less than or equal to 85 with Karnofsky score greater than or equal to 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)
a. Persistently positive lupus anticoagulant (LA) test
b. Persistently positive anticardiolipin (aCL) immunoglobulin G (IgG) isotype
c. Persistently positive anti-beta-2 glycoprotein-1 (aB2GPI) IgG isotype
- Clinical criteria
a. Livedoid vasculopathy and presence of skin ulcer
b. Acute/chronic aPL nephropathy
- BP cohort:
- 1) Age greater than or equal to 18 and less than or equal to 85 with Karnofsky score greater than or equal to 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 greater than or equal to 20
- 5) Weekly average of daily Peak Pruritus Numerical Rating Score (PP-NRS) greater than or equal to 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 less than 142, with at least one abnormality in the following Core Set Measures:
- Patient Global Activity Visual Analogue Scale (PtGA-VAS) greater than or equal to 2 cm
- Physician Global Activity Visual Analogue Scale (PhGA-VAS) greater than or equal to 2 cm
- Global extra-muscular activity greater than or equal to 2 cm
- At least one muscle enzyme greater than 1.5 times upper limit of normal (ULN)
- Health Assessment Questionnaire (HAQ) greater than or equal to 0.25
- 4) Moderate to severe DM defined as CDASI activity score greater than 14
- IMNM cohort:
- 1) Clinically Diagnosed with IMNM as anti-HMGCR myopathy or anti-SRP myopathy
- 2) Creatine kinase (CK) greater than 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 less than 142
- ITP cohort:
- 1) Confirmed diagnosis of persistent/chronic ITP based on the following criteria:
- ITP defined per the current guidelines
- Platelet count less than or equal to 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)
Note: Other protocol defined Inclusion/Exclusion criteria may apply.

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