Pemphigus VulgarisEarly Phase 1NCT07769333
Rituximab With Corticosteroids Versus Corticosteroids Plus Azathioprine for Moderate-to-Severe Pemphigus Vulgaris
A Early Phase 1 study testing Rituxan (rituximab) and Azathioprine (AZA)' plus corticosteroids in people with Pemphigus Vulgaris and Rituximab (RTx), run by Khyber Teaching Hospital.
Trial summary
This quasi-experimental study aims to compare the effectiveness and safety of rituximab combined with corticosteroids versus the standard regimen of oral corticosteroids plus azathioprine in patients with moderate to severe pemphigus vulgaris. Pemphigus vulgaris is a rare autoimmune blistering disease. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Pemphigus Vulgaris, Rituximab (RTx)
- What is being tested?
- Rituxan (rituximab) and Azathioprine (AZA)' plus corticosteroids
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 92 participants
- Where
- No sites listed yet
- Study dates
- 2 Dec 2026to 6 Nov 2027
- Phase
- Early Phase 1
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults aged 18-65 years.
- Active moderate-to-severe pemphigus vulgaris.
- Newly diagnosed disease or disease flare.
- Diagnosis confirmed by biopsy and direct immunofluorescence
Who cannot join
- Mild pemphigus vulgaris.
- Pregnancy or lactation.
- Active malignancy.
- Serious infection.
- Severe cardiac disease.
- Severe renal disease.
- Severe hepatic disease.
- Prior exposure to rituximab within the last 12 months.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Effectiveness of Rituximab With Corticosteroids Versus Standard Oral Corticosteroids Plus Azathioprine in Participants With Moderate-to-Severe Pemphigus Vulgaris: A Quasi-Experimental Study
This quasi-experimental study aims to compare the effectiveness and safety of rituximab combined with corticosteroids versus the standard regimen of oral corticosteroids plus azathioprine in patients with moderate to severe pemphigus vulgaris.
Pemphigus vulgaris is a rare autoimmune blistering disease. Conventional treatment with high-dose corticosteroids and azathioprine, though effective, is associated with significant long-term side effects. Rituximab, a B-cell depleting monoclonal antibody, has emerged as a promising alternative that may achieve higher rates of complete remission, faster disease control, fewer relapses, and reduced cumulative steroid exposure.
Read more
Patients will be allocated non-randomly into two groups based on clinical suitability, affordability, and preference. Outcomes will be assessed over 12 months, focusing primarily on complete remission rates, with secondary evaluation of time to disease control, relapse rates, cumulative corticosteroid dose, and adverse events.
The study seeks to generate local evidence from Pakistan to guide more effective and safer treatment protocols for moderate to severe pemphigus vulgaris.
Read the detailed description
Pemphigus vulgaris is a rare but serious autoimmune disease in which the body's immune system produces antibodies that attack proteins (desmogleins) responsible for holding skin and mucosal cells together. This leads to painful blisters and erosions on the skin and mucous membranes, particularly in the mouth. If not treated effectively, the condition can cause significant morbidity and, in severe cases, life-threatening complications.
For many years the standard approach has involved high-dose oral corticosteroids combined with an immunosuppressive agent such as azathioprine. While this combination can control the disease, prolonged use of corticosteroids frequently results in serious adverse effects, including diabetes, osteoporosis, increased susceptibility to infections, and Cushingoid features. These complications often limit the long-term safety of conventional therapy.
Rituximab is a monoclonal antibody that specifically targets CD20-positive B-cells, the cells responsible for producing the harmful autoantibodies. By depleting these cells, rituximab addresses the underlying immunological mechanism of the disease more selectively than broad immunosuppression. Growing evidence from international studies suggests that rituximab, when used with a short course of corticosteroids, can achieve higher rates of sustained remission, reduce the frequency of relapses, and substantially lower the total amount of corticosteroids patients need to take.
Despite these promising findings, comparative data from Pakistan and similar resource-constrained settings remain limited. Cost, availability, and local clinical experience still influence treatment choices. A quasi-experimental design is therefore appropriate: patients with moderate to severe disease will be assigned to one of two treatment arms according to clinical suitability, affordability, and informed preference rather than by randomisation. One group will receive rituximab plus corticosteroids; the other will receive the conventional regimen of oral corticosteroids plus azathioprine.
Patients will be followed for twelve months. The primary focus will be the proportion of patients who achieve complete clinical remission. Secondary measures will include the time required to achieve disease control, the number and timing of relapses, the cumulative dose of corticosteroids administered, and the frequency and severity of treatment-related adverse events. Disease activity will be monitored using the Pemphigus Disease Area Index (PDAI) at regular intervals.
By generating local comparative data, this study aims to clarify whether rituximab-based therapy offers meaningful advantages in effectiveness and safety over the conventional regimen in the Pakistani population, thereby supporting more informed treatment decisions for patients with moderate to severe pemphigus vulgaris.
- Conditions
- Pemphigus Vulgaris, Rituximab (RTx)
- Being tested
- Rituxan (rituximab), Azathioprine (AZA)' plus corticosteroids
- Medical subject headings
- Pemphigus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rituxan (rituximab)Drug
a biological drug plus corticosteroids in the treatment of P.V
Azathioprine (AZA)' plus corticosteroidsDrug
A CONVRNTIONAL TREATMENT FOR P.V
| Study group | Role | What they receive |
|---|---|---|
| RITUXIMAB | Experimental | Not described Receives: Drug: Rituxan (rituximab) |
| CORTICOSTEROIDS PLUS AZATHIOPRINE | Active comparator | Not described Receives: Drug: Azathioprine (AZA)' plus corticosteroids |
What is being measured
The primary outcome is the main question the study is designed to answer.
PDAI SCORE
PDAI score will be assessed to measure disease activity in patients with pemphigus vulgaris. The PDAI score ranges from 0 to 250. Higher scores indicate more severe disease activity and a worse outcome.
Measured: Baseline, 2 weeks, 1 month, 3 months, and 6 months after baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2026
- Study start2 Dec 2026 (estimated)
- Primary completion5 May 2027 (estimated)
- Study completion6 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Pemphigus VulgarisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 92 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Khyber Teaching Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Noor Ayaz, Khyber Teaching Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07769333
- Sponsor's protocol ID
- 343/IREB/KTH
- Status last verified
- Aug 2026
- First submitted
- 12 Aug 2026
- Last updated
- 18 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Szymanski K, Kowalewski C, Walecka I, Wozniak K. Eight Years of Follow-Up of Rituximab in Pemphigus Vulgaris and Foliaceus at a Single Center: Assessing Efficacy and Safety in Light of Several Factors. J Clin Med. 2025 Oct 16;14(20):7318. doi: 10.3390/jcm14207318.
PubMed 41156189Cao S, Yang B, Wang Z, Liu T, Sun Y, Zhang Z, Pan F, Yang Q, Geng H, Zhao Q, Liu H, Zhang F. Efficacy, safety, and B-cell depletion capacity of 3 rituximab dosing regimens in the treatment of moderate-to-severe pemphigus vulgaris and pemphigus foliaceus: A 52-week clinical trial. J Am Acad Dermatol. 2025 Sep;93(3):634-643. doi: 10.1016/j.jaad.2025.05.1374. Epub 2025 May 13.
PubMed 40374120Liu SH, Wang SH, Zuo YG. Efficacy and safety of low dose rituximab in pemphigus: an updated systematic review and meta-analysis. Front Immunol. 2025 Jul 25;16:1605243. doi: 10.3389/fimmu.2025.1605243. eCollection 2025.
PubMed 40787442Branisteanu DE, Lotti T, Colac Botoc C, Hutanu AE, Munteanu CA, Ciobanu RP, Branisteanu DC, Colac AG, Branisteanu CIO, Branisteanu G, Toader SV, Toader MP. Real-world outcomes with rituximab vs. conventional therapy in pemphigus vulgaris: a single-center Romanian cohort. Front Med (Lausanne). 2025 Nov 12;12:1691897. doi: 10.3389/fmed.2025.1691897. eCollection 2025.
PubMed 41312452
Study at a glance
From the registry record, last updated by the sponsor on 18 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness of Rituximab With Corticosteroids Versus Standard Oral Corticosteroids Plus Azathioprine in Participants With Moderate-to-Severe Pemphigus Vulgaris: A Quasi-Experimental Study
- ClinicalTrials.gov ID
- NCT07769333
- Acronym / sponsor ID
- 343/IREB/KTH
- Conditions
- Pemphigus Vulgaris, Rituximab (RTx)
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 92 participants (estimated)
- Lead sponsor
- Khyber Teaching HospitalUniversity, hospital or other
- Study start
- 2 Dec 2026 (estimated)
- Primary completion
- 5 May 2027 (estimated)
- Study completion
- 6 Nov 2027 (estimated)
- First posted
- 17 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This quasi-experimental study aims to compare the effectiveness and safety of rituximab combined with corticosteroids versus the standard regimen of oral corticosteroids plus azathioprine in patients with moderate to severe pemphigus vulgaris.
Pemphigus vulgaris is a rare autoimmune blistering disease. Conventional treatment with high-dose corticosteroids and azathioprine, though effective, is associated with significant long-term side effects. Rituximab, a B-cell depleting monoclonal antibody, has emerged as a promising alternative that may achieve higher rates of complete remission, faster disease control, fewer relapses, and reduced cumulative steroid exposure.
Patients will be allocated non-randomly into two groups based on clinical suitability, affordability, and preference. Outcomes will be assessed over 12 months, focusing primarily on complete remission rates, with secondary evaluation of time to disease control, relapse rates, cumulative corticosteroid dose, and adverse events.
The study seeks to generate local evidence from Pakistan to guide more effective and safer treatment protocols for moderate to severe pemphigus vulgaris.
Detailed description
Pemphigus vulgaris is a rare but serious autoimmune disease in which the body's immune system produces antibodies that attack proteins (desmogleins) responsible for holding skin and mucosal cells together. This leads to painful blisters and erosions on the skin and mucous membranes, particularly in the mouth. If not treated effectively, the condition can cause significant morbidity and, in severe cases, life-threatening complications.
For many years the standard approach has involved high-dose oral corticosteroids combined with an immunosuppressive agent such as azathioprine. While this combination can control the disease, prolonged use of corticosteroids frequently results in serious adverse effects, including diabetes, osteoporosis, increased susceptibility to infections, and Cushingoid features. These complications often limit the long-term safety of conventional therapy.
Rituximab is a monoclonal antibody that specifically targets CD20-positive B-cells, the cells responsible for producing the harmful autoantibodies. By depleting these cells, rituximab addresses the underlying immunological mechanism of the disease more selectively than broad immunosuppression. Growing evidence from international studies suggests that rituximab, when used with a short course of corticosteroids, can achieve higher rates of sustained remission, reduce the frequency of relapses, and substantially lower the total amount of corticosteroids patients need to take.
Despite these promising findings, comparative data from Pakistan and similar resource-constrained settings remain limited. Cost, availability, and local clinical experience still influence treatment choices. A quasi-experimental design is therefore appropriate: patients with moderate to severe disease will be assigned to one of two treatment arms according to clinical suitability, affordability, and informed preference rather than by randomisation. One group will receive rituximab plus corticosteroids; the other will receive the conventional regimen of oral corticosteroids plus azathioprine.
Patients will be followed for twelve months. The primary focus will be the proportion of patients who achieve complete clinical remission. Secondary measures will include the time required to achieve disease control, the number and timing of relapses, the cumulative dose of corticosteroids administered, and the frequency and severity of treatment-related adverse events. Disease activity will be monitored using the Pemphigus Disease Area Index (PDAI) at regular intervals.
By generating local comparative data, this study aims to clarify whether rituximab-based therapy offers meaningful advantages in effectiveness and safety over the conventional regimen in the Pakistani population, thereby supporting more informed treatment decisions for patients with moderate to severe pemphigus vulgaris.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RITUXIMAB | Experimental | Drug: Rituxan (rituximab) |
| CORTICOSTEROIDS PLUS AZATHIOPRINE | Active comparator | Drug: Azathioprine (AZA)' plus corticosteroids |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Adults aged 18-65 years.
- Active moderate-to-severe pemphigus vulgaris.
- Newly diagnosed disease or disease flare.
- Diagnosis confirmed by biopsy and direct immunofluorescence
Key exclusion criteria (8 listed)
- Mild pemphigus vulgaris.
- Pregnancy or lactation.
- Active malignancy.
- Serious infection.
- Severe cardiac disease.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Rituxan (rituximab)a biological drug plus corticosteroids in the treatment of P.V | Drug | RITUXIMAB |
| Azathioprine (AZA)' plus corticosteroidsA CONVRNTIONAL TREATMENT FOR P.V | Drug | CORTICOSTEROIDS PLUS AZATHIOPRINE |
Primary outcome
PDAI SCORE
Time frame: Baseline, 2 weeks, 1 month, 3 months, and 6 months after baseline
PDAI score will be assessed to measure disease activity in patients with pemphigus vulgaris. The PDAI score ranges from 0 to 250. Higher scores indicate more severe disease activity and a worse outcome.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2026.
- Results publication: PubMed 41156189Szymanski K, Kowalewski C, Walecka I, Wozniak K. Eight Years of Follow-Up of Rituximab in Pemphigus Vulgaris and Foliaceus at a Single Center: Assessing Efficacy and Safety in Light of Several Factors. J Clin Med. 2025 Oct 16;14(20):7318. doi: 10.3390/jcm14207318.
- Results publication: PubMed 40374120Cao S, Yang B, Wang Z, Liu T, Sun Y, Zhang Z, Pan F, Yang Q, Geng H, Zhao Q, Liu H, Zhang F. Efficacy, safety, and B-cell depletion capacity of 3 rituximab dosing regimens in the treatment of moderate-to-severe pemphigus vulgaris and pemphigus foliaceus: A 52-week clinical trial. J Am Acad Dermatol. 2025 Sep;93(3):634-643. doi: 10.1016/j.jaad.2025.05.1374. Epub 2025 May 13.
- Results publication: PubMed 40787442Liu SH, Wang SH, Zuo YG. Efficacy and safety of low dose rituximab in pemphigus: an updated systematic review and meta-analysis. Front Immunol. 2025 Jul 25;16:1605243. doi: 10.3389/fimmu.2025.1605243. eCollection 2025.
- Results publication: PubMed 41312452Branisteanu DE, Lotti T, Colac Botoc C, Hutanu AE, Munteanu CA, Ciobanu RP, Branisteanu DC, Colac AG, Branisteanu CIO, Branisteanu G, Toader SV, Toader MP. Real-world outcomes with rituximab vs. conventional therapy in pemphigus vulgaris: a single-center Romanian cohort. Front Med (Lausanne). 2025 Nov 12;12:1691897. doi: 10.3389/fmed.2025.1691897. eCollection 2025.
- Individual participant dataSharing planned: No
Related trials for Pemphigus Vulgaris
RecruitingPhase 1/2
A Phase 1/2, Open-label, Safety and Dosing Study of Autologous CART Cells (Desmoglein 3 Chimeric Autoantibody Receptor T Cells [DSG3-CAART] or CD19-specific Chimeric Antigen Receptor T Cells [CABA-201]) in Subjects With Active, Pemphigus Vulgaris (RESET-PV)
Pemphigus Vulgaris
Recruiting
CD19 CAR-T Therapy for Refractory Pemphigus Vulgaris
Pemphigus Vulgaris
Recruiting
Comparison of the Efficacy of Clobetasol Propionate 0.05% Mouthwash, Photobiomodulation, and Their Combination in Managing of Oral Lesions in Patient With Pemphigus Vulgaris.
Oral Lesions in Patients With Pemphigus Vulgaris
RecruitingObservational study
Autoimmune Blistering Diseases Study
Pemphigus Vulgaris, Pemphigus Foliaceus
Not yet recruiting
Low-level Laser Therapy on Oral Pemphigus Vulgaris Patients.
Pemphigus Vulgaris
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Rituxan (rituximab) and Azathioprine (AZA)' plus corticosteroids in people with Pemphigus Vulgaris and Rituximab (RTx), run by Khyber Teaching Hospital.
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 4 inclusion and 8 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: opening soon, as of the sponsor's last update on 18 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Contact NOOR AYAZ, MBBS (+923345536315, drnoorayaz@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PDAI SCORE (time frame: Baseline, 2 weeks, 1 month, 3 months, and 6 months after baseline). 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 NCT07769333, which also lists 4 linked publications.
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 18 Aug 2026.