ONO-4059-10:Multicenter, placebo-controlled, randomized, double-blind, Phase 3 study in patients with steroid-resistant pemphigus
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
34
Tirabrutinib is a Bruton's Tyrosine Kinase Inhibitor. Tirabrutinib will be administered orally at a dose of 80 mg once daily after a meal (breakfast, lunch, or dinner).
Placebo will be administered orally once daily after a meal (breakfast, lunch, or dinner).
Ichinomiya Municipal Hospital
Aichi, Japan
The proportion of patients with a Complete response (CR) or Partial response (PR) at 52 weeks, and maintaining complete/partial remission at 52 weeks from initial remission achievement.
CR: Absence of pemphigus-related blisters or new erythema that lasts for 8 weeks during treatment with ONO-4059 plus an oral corticosteroid (≤10 mg/day prednisolone-equivalent) or minimal adjunctive therapy. PR: Occurrence of only a transient lesion that resolves within 1 week without an increased dose of an oral corticosteroids, without treatment, or with a topical corticosteroid, etc., during treatment with ONO-4059 plus an oral corticosteroid (≤10 mg/day prednisolone-equivalent) or minimal adjunctive therapy for 8 weeks.
Time frame: 52 weeks
Remission rate
remission rate \[proportion of subjects who achieve complete or partial remission\], complete remission rate, partial remission rate
Time frame: 52 weeks
Pemphigus Disease Area Index (PDAI) score
Pemphigus Disease Area Index (PDAI). PDAI activity score cutoffs were defined as 0 to 8 for mild, 9 to 24 for moderate, and 25 or higher for severe disease.
Time frame: 52 weeks
Efficacy 1 rate (proportion of subjects for whom the definition of efficacy 1 is applicable)
Definition of efficacy 1: Decreases in PDAI score and oral corticosteroid dose from baseline
Time frame: 52 weeks
Efficacy 2 rate (proportion of subjects for whom the definition of efficacy 2 is applicable)
Definition of efficacy 2: PDAI score is 0 and oral corticosteroid dose \<= 10 mg/day
Time frame: 52 weeks
Disease control rate (proportion of subjects for whom the definition of disease control is applicable)
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Nagoya City University Hospital
Aichi, Japan
RECRUITINGEhime University Hospital
Ehime, Japan
RECRUITINGKurume University Hospital
Fukuoka, Japan
RECRUITINGFukushima Medical University Hospital
Fukushima, Japan
RECRUITINGGunma University Hospital
Gunma, Japan
RECRUITINGHokkaido University Hospital
Hokkaido, Japan
RECRUITINGKagoshima University Hospital
Kagoshima, Japan
RECRUITINGSt.Marianna University School of Medicine Hospital
Kanagawa, Japan
RECRUITINGTokai University Hospital
Kanagawa, Japan
RECRUITING...and 15 more locations
Definition of disease control: Formation of nascent lesions stops and existing lesions begin to resolve
Time frame: 52 weeks
Rescue therapy rate
Proportion of subjects who received rescue therapy
Time frame: 52 weeks
Pemphigus autoantibody titer
Anti-desmoglein 1 antibodies, anti-desmoglein 3 antibodies
Time frame: 52 weeks
Oral corticosteroid dose
At the equivalent dose of prednisolone
Time frame: 52 weeks
QOL evaluation
The Dermatology Life Quality Index (DLQI) score is measured by questionnaire.
Time frame: 52 weeks
Adverse events and adverse drug reactions
Time frame: 52 weeks
Clinical laboratory tests
Number of participants with clinical laboratory abnormalities (including haematology, clinical chemistry and urinalysis).
Time frame: 52 weeks
12-lead ECG
Heart rate, RR interval, PR interval, QRS duration, QT interval and QTcF
Time frame: 52 weeks
Vital signs
Systolic/diastolic blood pressure
Time frame: 52 weeks
Vital signs
Body temperature
Time frame: 52 weeks
Immunological tests
Immunoglobulin G and its subclasses (IgG 1, 2, 3, and 4), immunoglobulin M, immunoglobulin A, peripheral blood B-cell count, and peripheral blood T-cell count
Time frame: 52 weeks
Plasma ONO-4059 concentration
Time frame: 52 weeks
Immunophenotyping
Level of naive-B cell, memory-B cell, plasmablast etc.
Time frame: 52 weeks
Cytokines and chemokines
Level of tumor necrosis factor-α, interleukin (IL)-8, IL-13, IL-17, C-X-C motif chemokine ligand 13 etc.
Time frame: 52 weeks
RNA sequencing
Time frame: 52 weeks
Anti-virus antibody
Time frame: 52 weeks
Recurrence rate (the proportion of subjects meeting the definition of recurrence) and time to recurrence
Definition of recurrence: In patients with controlled disease, the presence of three or more new lesions in a month that do not spontaneously resolve within a week, or the expansion of existing lesions Definition of time to recurrence: The period from achieving disease control to recurrence
Time frame: 52 weeks