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Active, Not Recruiting

NCT Number: NCT06696716

ONO-4059 Study in Patients With Steroid-resistant Pemphigus

ONO-4059-10:Multicenter, placebo-controlled, randomized, double-blind, Phase 3 study in patients with steroid-resistant pemphigus

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Pemphigus Autoimmune Diseases Immune System Diseases Skin Diseases Skin Diseases, Vesiculobullous Skin and Connective Tissue Diseases Steroid-resistant Pemphigus

Age range

20 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Ichinomiya Municipal Hospital, Aichi, Japan

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • Patients with an active infection
  • Patients with malignancy
  • Patients with past history of serious allergy or anaphylaxis

Treatment and study plan

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).

Other names: Tirabrutinib hydrochloride

ONO-4059 placebo

Drug

Placebo will be administered orally once daily after a meal (breakfast, lunch, or dinner).

Primary outcomes

  1. 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.

    Time frame: 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 outcomes

  1. Remission rate

    Time frame: 52 weeks

    remission rate [proportion of subjects who achieve complete or partial remission], complete remission rate, partial remission rate

  2. Pemphigus Disease Area Index (PDAI) score

    Time frame: 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.

  3. Efficacy 1 rate (proportion of subjects for whom the definition of efficacy 1 is applicable)

    Time frame: 52 weeks

    Definition of efficacy 1: Decreases in PDAI score and oral corticosteroid dose from baseline

  4. Efficacy 2 rate (proportion of subjects for whom the definition of efficacy 2 is applicable)

    Time frame: 52 weeks

    Definition of efficacy 2: PDAI score is 0 and oral corticosteroid dose <= 10 mg/day

  5. Disease control rate (proportion of subjects for whom the definition of disease control is applicable)

    Time frame: 52 weeks

    Definition of disease control: Formation of nascent lesions stops and existing lesions begin to resolve

  6. Rescue therapy rate

    Time frame: 52 weeks

    Proportion of subjects who received rescue therapy

  7. Pemphigus autoantibody titer

    Time frame: 52 weeks

    Anti-desmoglein 1 antibodies, anti-desmoglein 3 antibodies

  8. Oral corticosteroid dose

    Time frame: 52 weeks

    At the equivalent dose of prednisolone

  9. QOL evaluation

    Time frame: 52 weeks

    The Dermatology Life Quality Index (DLQI) score is measured by questionnaire.

  10. Adverse events and adverse drug reactions

    Time frame: 52 weeks

  11. Clinical laboratory tests

    Time frame: 52 weeks

    Number of participants with clinical laboratory abnormalities (including haematology, clinical chemistry and urinalysis).

  12. 12-lead ECG

    Time frame: 52 weeks

    Heart rate, RR interval, PR interval, QRS duration, QT interval and QTcF

  13. Vital signs

    Time frame: 52 weeks

    Systolic/diastolic blood pressure

  14. Vital signs

    Time frame: 52 weeks

    Body temperature

  15. Immunological tests

    Time frame: 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

  16. Plasma ONO-4059 concentration

    Time frame: 52 weeks

  17. Immunophenotyping

    Time frame: 52 weeks

    Level of naive-B cell, memory-B cell, plasmablast etc.

  18. Cytokines and chemokines

    Time frame: 52 weeks

    Level of tumor necrosis factor-α, interleukin (IL)-8, IL-13, IL-17, C-X-C motif chemokine ligand 13 etc.

  19. RNA sequencing

    Time frame: 52 weeks

  20. Anti-virus antibody

    Time frame: 52 weeks

  21. Recurrence rate (the proportion of subjects meeting the definition of recurrence) and time to recurrence

    Time frame: 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

Sponsors and collaborators

Lead sponsor

Ono Pharmaceutical Co., Ltd.

Industry

Registry information

Official study title

ONO-4059-10:Multicenter, Placebo-controlled, Randomized, Double-blind, Phase 3 Study in Patients With Steroid-resistant Pemphigus (BRILLIANT Study)

Important dates

Study start
2022
Primary completion
2027
Study completion
2029
First posted
Nov 20, 2024
Registry last updated
Jul 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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