Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| jRCT2031220043 | Registry Identifier | Japan Registry of Clinical Trials |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
ONO-4059-10:Multicenter, placebo-controlled, randomized, double-blind, Phase 3 study in patients with steroid-resistant pemphigus
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ONO-4059 | Active Comparator |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ONO-4059 | Drug | 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). |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Remission rate | remission rate [proportion of subjects who achieve complete or partial remission], complete remission rate, partial remission rate | 52 weeks |
| Pemphigus Disease Area Index (PDAI) score |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| North America Clinical Trial Support Desk | Contact | +18665877745(Toll-Free) | clinical_trial@ono-pharma.com | |
| International Clinical Trial Support Desk | Contact | +17162141777(Standard) | clinical_trial@ono-pharma.com |
| Name | Affiliation | Role |
|---|---|---|
| Project Leader | Ono Pharmaceutical Co., Ltd. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ichinomiya Municipal Hospital | Recruiting | Aichi | Japan | |||
| Nagoya City University Hospital |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| ONO-4059 placebo | Drug | Placebo will be administered orally once daily after a meal (breakfast, lunch, or dinner). |
|
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.
| 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 | 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 | 52 weeks |
| Disease control rate (proportion of subjects for whom the definition of disease control is applicable) | Definition of disease control: Formation of nascent lesions stops and existing lesions begin to resolve | 52 weeks |
| Rescue therapy rate | Proportion of subjects who received rescue therapy | 52 weeks |
| Pemphigus autoantibody titer | Anti-desmoglein 1 antibodies, anti-desmoglein 3 antibodies | 52 weeks |
| Oral corticosteroid dose | At the equivalent dose of prednisolone | 52 weeks |
| QOL evaluation | The Dermatology Life Quality Index (DLQI) score is measured by questionnaire. | 52 weeks |
| Adverse events and adverse drug reactions | 52 weeks |
| Clinical laboratory tests | Number of participants with clinical laboratory abnormalities (including haematology, clinical chemistry and urinalysis). | 52 weeks |
| 12-lead ECG | Heart rate, RR interval, PR interval, QRS duration, QT interval and QTcF | 52 weeks |
| Vital signs | Systolic/diastolic blood pressure | 52 weeks |
| Vital signs | Body temperature | 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 | 52 weeks |
| Plasma ONO-4059 concentration | 52 weeks |
| Immunophenotyping | Level of naive-B cell, memory-B cell, plasmablast etc. | 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. | 52 weeks |
| RNA sequencing | 52 weeks |
| Anti-virus antibody | 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 | 52 weeks |
| Recruiting |
| Aichi |
| Japan |
| Ehime University Hospital | Recruiting | Ehime | Japan |
| Kurume University Hospital | Recruiting | Fukuoka | Japan |
| Fukushima Medical University Hospital | Recruiting | Fukushima | Japan |
| Gunma University Hospital | Recruiting | Gunma | Japan |
| Hokkaido University Hospital | Recruiting | Hokkaido | Japan |
| Kagoshima University Hospital | Recruiting | Kagoshima | Japan |
| St.Marianna University School of Medicine Hospital | Recruiting | Kanagawa | Japan |
| Tokai University Hospital | Recruiting | Kanagawa | Japan |
| Yokohama City University Hospital | Recruiting | Kanagawa | Japan |
| Kumamoto University Hospital | Recruiting | Kumamoto | Japan |
| University Hospital Kyoto Prefectural University of Medicine | Recruiting | Kyoto | Japan |
| Niigata University Medical & Dental Hospital | Recruiting | Niigata | Japan |
| Kawasaki Medical School Hospital | Recruiting | Okayama | Japan |
| University of the Ryukyus Hospital | Recruiting | Okinawa | Japan |
| Kindai university hospital | Recruiting | Osaka | Japan |
| Osaka Metropolitan University Hospital | Recruiting | Osaka | Japan |
| Saitama Medical Center | Recruiting | Saitama | Japan |
| Shiga University of Medical Science Hospital | Recruiting | Shiga | Japan |
| Jichi Medical University Hospital | Recruiting | Tochigi | Japan |
| Keio University Hospital | Recruiting | Tokyo | Japan |
| Tokyo Medical and Dental University Hospital | Recruiting | Tokyo | Japan |
| Tokyo Women'ｓ Medical University Hospital | Recruiting | Tokyo | Japan |
| Yamagata University Hospital | Not yet recruiting | Yamagata | Japan |
| ID | Term |
|---|---|
| D010392 | Pemphigus |
| ID | Term |
|---|---|
| D012872 | Skin Diseases, Vesiculobullous |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000608238 | tirabrutinib |
Not provided
Not provided
Not provided