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PemphigusPhase 3NCT06696716

ONO-4059 Study in Patients With Steroid-resistant Pemphigus

A Phase 3 study testing ONO-4059 in people with Pemphigus and Steroid-resistant Pemphigus, run by Ono Pharmaceutical Co., Ltd. at 25 sites in Japan.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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

Who is it for?
Ages 20 years and older
Conditions the study is for
Pemphigus, Steroid-resistant Pemphigus
What is being tested?
ONO-4059
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
34 participants
Where
25 sitesJapan
Study dates
15 Aug 2022to 31 Mar 2029
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 20 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

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

Trial locations and contacts

25 sites in 1 country.

25 sites in 1 country

Japan25 sites

  • Ichinomiya Municipal Hospital

    Aichi

  • Nagoya City University Hospital

    Aichi

  • Ehime University Hospital

    Ehime

  • Kurume University Hospital

    Fukuoka

  • Fukushima Medical University Hospital

    Fukushima

  • Gunma University Hospital

    Gunma

  • Hokkaido University Hospital

    Hokkaido

  • Kagoshima University Hospital

    Kagoshima

  • St.Marianna University School of Medicine Hospital

    Kanagawa

  • Tokai University Hospital

    Kanagawa

  • Yokohama City University Hospital

    Kanagawa

  • Kumamoto University Hospital

    Kumamoto

  • University Hospital Kyoto Prefectural University of Medicine

    Kyoto

  • Niigata University Medical & Dental Hospital

    Niigata

  • Kawasaki Medical School Hospital

    Okayama

  • University of the Ryukyus Hospital

    Okinawa

  • Kindai university hospital

    Osaka

  • Osaka Metropolitan University Hospital

    Osaka

  • Saitama Medical Center

    Saitama

  • Shiga University of Medical Science Hospital

    Shiga

  • Jichi Medical University Hospital

    Tochigi

  • Keio University Hospital

    Tokyo

  • Tokyo Medical and Dental University Hospital

    Tokyo

  • Tokyo Women'ｓ Medical University Hospital

    Tokyo

  • Yamagata University Hospital

    Yamagata

Other trials in Japan

About this study

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

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

Being tested
ONO-4059, ONO-4059 placebo
Medical subject headings
Pemphigus

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ONO-4059Drug

    Also called Tirabrutinib hydrochloride

    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).

  • ONO-4059 placeboDrug

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

Study groups in NCT06696716
Study groupRoleWhat they receive
ONO-4059Active comparatorNot described
Receives: Drug: ONO-4059
PlaceboPlacebo comparatorNot described
Receives: Drug: ONO-4059 placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    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.

    Measured: 52 weeks

Secondary outcomes (21)

  1. Remission rate

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

    Measured: 52 weeks

  2. 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.

    Measured: 52 weeks

  3. 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

    Measured: 52 weeks

  4. 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

    Measured: 52 weeks

  5. 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

    Measured: 52 weeks

  6. Rescue therapy rate

    Proportion of subjects who received rescue therapy

    Measured: 52 weeks

  7. Pemphigus autoantibody titer

    Anti-desmoglein 1 antibodies, anti-desmoglein 3 antibodies

    Measured: 52 weeks

  8. Oral corticosteroid dose

    At the equivalent dose of prednisolone

    Measured: 52 weeks

  9. QOL evaluation

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

    Measured: 52 weeks

  10. Adverse events and adverse drug reactions

    Measured: 52 weeks

  11. Clinical laboratory tests

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

    Measured: 52 weeks

  12. 12-lead ECG

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

    Measured: 52 weeks

  13. Vital signs

    Systolic/diastolic blood pressure

    Measured: 52 weeks

  14. Vital signs

    Body temperature

    Measured: 52 weeks

  15. 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

    Measured: 52 weeks

  16. Plasma ONO-4059 concentration

    Measured: 52 weeks

  17. Immunophenotyping

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

    Measured: 52 weeks

  18. Cytokines and chemokines

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

    Measured: 52 weeks

  19. RNA sequencing

    Measured: 52 weeks

  20. Anti-virus antibody

    Measured: 52 weeks

  21. 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

    Measured: 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Nov 2024
  2. Study start15 Aug 2022
  3. Primary completion30 Sep 2027 (estimated)
  4. Study completion31 Mar 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
34 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06696716
Sponsor's protocol ID
ONO-4059-10
Other IDs
jRCT2031220043
Status last verified
Jul 2026
First submitted
7 Nov 2024
Last updated
20 Jul 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing ONO-4059 in people with Pemphigus and Steroid-resistant Pemphigus, run by Ono Pharmaceutical Co., Ltd. at 25 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 20 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 25 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06696716.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2026.

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