ONO-4059 Study in Patients With Steroid-resistant Pemphigus
- Conditions
- PemphigusSteroid-resistant Pemphigus
- Interventions
- Drug: ONO-4059 placebo
- Registration Number
- NCT06696716
- Lead Sponsor
- Ono Pharmaceutical Co. Ltd
- Brief Summary
ONO-4059-10:Multicenter, placebo-controlled, randomized, double-blind, Phase 3 study in patients with steroid-resistant pemphigus
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 34
- Patients with a diagnosis of pemphigus
- Patients with a re-increase in the pemphigus disease area index (PDAI) score before oral corticosteroid tapering to 10mg/day of prednisolone (PSL) equivalent
- Patients with an active infection
- Patients with malignancy
- Patients with past history of serious allergy or anaphylaxis
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ONO-4059 ONO-4059 - Placebo ONO-4059 placebo -
- Primary Outcome Measures
Name Time Method 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. 52 weeks 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.
- Secondary Outcome Measures
Name Time Method Remission rate 52 weeks remission rate \[proportion of subjects who achieve complete or partial remission\], complete remission rate, partial remission rate
Pemphigus Disease Area Index (PDAI) score 52 weeks 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.
Efficacy 1 rate (proportion of subjects for whom the definition of efficacy 1 is applicable) 52 weeks Definition of efficacy 1: Decreases in PDAI score and oral corticosteroid dose from baseline
Efficacy 2 rate (proportion of subjects for whom the definition of efficacy 2 is applicable) 52 weeks Definition of efficacy 2: PDAI score is 0 and oral corticosteroid dose \<= 10 mg/day
Disease control rate (proportion of subjects for whom the definition of disease control is applicable) 52 weeks Definition of disease control: Formation of nascent lesions stops and existing lesions begin to resolve
Rescue therapy rate 52 weeks Proportion of subjects who received rescue therapy
Pemphigus autoantibody titer 52 weeks Anti-desmoglein 1 antibodies, anti-desmoglein 3 antibodies
Oral corticosteroid dose 52 weeks At the equivalent dose of prednisolone
QOL evaluation 52 weeks The Dermatology Life Quality Index (DLQI) score is measured by questionnaire.
Adverse events and adverse drug reactions 52 weeks Clinical laboratory tests 52 weeks Number of participants with clinical laboratory abnormalities (including haematology, clinical chemistry and urinalysis).
12-lead ECG 52 weeks Heart rate, RR interval, PR interval, QRS duration, QT interval and QTcF
Vital signs 52 weeks Body temperature
Immunological tests 52 weeks 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
Plasma ONO-4059 concentration 52 weeks Immunophenotyping 52 weeks Level of naive-B cell, memory-B cell, plasmablast etc.
Cytokines and chemokines 52 weeks Level of tumor necrosis factor-α, interleukin (IL)-8, IL-13, IL-17, C-X-C motif chemokine ligand 13 etc.
RNA sequencing 52 weeks Anti-virus antibody 52 weeks Recurrence rate (the proportion of subjects meeting the definition of recurrence) and time to recurrence 52 weeks 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
Trial Locations
- Locations (20)
Ichinomiya Municipal Hospital
🇯🇵Aichi, Japan
Nagoya City University Hospital
🇯🇵Aichi, Japan
Kurume University Hospital
🇯🇵Fukuoka, Japan
Fukushima Medical University Hospital
🇯🇵Fukushima, Japan
Hokkaido University Hospital
🇯🇵Hokkaido, Japan
Kagoshima University Hospital
🇯🇵Kagoshima, Japan
St.Marianna University School of Medicine Hospital
🇯🇵Kanagawa, Japan
Tokai University Hospital
🇯🇵Kanagawa, Japan
Yokohama City University Hospital
🇯🇵Kanagawa, Japan
University Hospital Kyoto Prefectural University of Medicine
🇯🇵Kyoto, Japan
Niigata University Medical & Dental Hospital
🇯🇵Niigata, Japan
Kawasaki Medical School Hospital
🇯🇵Okayama, Japan
Kindai university hospital
🇯🇵Osaka, Japan
Osaka Metropolitan University Hospital
🇯🇵Osaka, Japan
Saitama Medical Center
🇯🇵Saitama, Japan
Jichi Medical University Hospital
🇯🇵Tochigi, Japan
Keio University Hospital
🇯🇵Tokyo, Japan
Tokyo Medical and Dental University Hospital
🇯🇵Tokyo, Japan
Tokyo Women'ｓ Medical University Hospital
🇯🇵Tokyo, Japan
Yamagata University Hospital
🇯🇵Yamagata, Japan