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PHASE IB OPEN LABEL BASKET STUDY OF RAY121

Dermatology
Ali Habib
PHASE IB OPEN LABEL BASKET STUDY OF RAY121 TO INHIBIT COMPLEMENT CLASSICAL PATHWAY IN IMMUNOLOGICAL DISEASES (RAINBOW TRIAL)
Bullous Pemphigoid
Dermatomyositis
Immune Mediated Necrotizing Myopathy
Immunologic Auto Immune

Study Description

Eligibility

Inclusion Criteria:

  1. Signed informed consent form
  2. 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)
  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)

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

  1. 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
  1. 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
  1. 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
  1. 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
  1. 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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