Pemphigus VulgarisPhase 4NCT05303272
A Study to Evaluate Efficacy and Safety of Abatacept in Participants of Pemphigus Vulgaris (PV)
A Phase 4 study testing Abatacept Prefilled Syringe and Mycophenolate Mofetil 500Mg Tab in people with Pemphigus Vulgaris, run by Tongji Hospital at 1 site in China.
Trial summary
Pemphigus vulgaris (PV) is a rare, chronic, debilitating, and potentially life-threatening autoimmune disorder that is characterized by mucocutaneous blisters.Abatacept is a biologic drug that belongs to the class of T-cell co-stimulation modulators and is used for the treatment of autoimmune diseases. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Pemphigus Vulgaris
- What is being tested?
- Abatacept Prefilled Syringe and Mycophenolate Mofetil 500Mg Tab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteChina
- Study dates
- 1 Feb 2021to 30 Sep 2022
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults (18 through 80 years of age) with clinically-documented diagnosis of PV for >2 months and <10 years.
- History of biopsy consistent with PV (Hematoxylin and Eosin staining and direct immunofluorescence). Confirmed diagnosis of PV within the previous 24 months, based on the presence of histological features of acantholysis via skin or mucosal biopsy and one of the following: tissue bound immunoglobulin G (IgG) antibodies by direct immunofluorescence on the surface of affected epithelium or serological detection of serum desmoglein-3 (DSg3) autoantibodies against epithelial cell surface either by indirect immunofluorescence microscopy or by enzyme-linked immunosorbent assay.
- At least 1 previous episode of a failed steroid taper (ie, disease flare/relapse) at a prednisone/prednisolone dose >10 mg/day. The following criteria must have been met as evidence of disease severity at the time of the failed steroid taper: a) A Pemphigus Severity of Clinical Disease score of moderate (2) or severe (3) (may be historical/retrospective assessment). b) Required a treatment change at the time of the failed steroid taper of at least one of the following: i) A steroid increase to >=20 mg/day OR ii) The addition of immunosuppressive/immunomodulatory agent/treatment OR iii) A dose increase of immunosuppressive/immunomodulatory agent/treatment
- Screening anti-Dsg antibodies consistent with a diagnosis of PV (ie, elevated antiDsg3 antibodies).
- Has initiated and received a stable dose of prednisone/prednisolone from a minimum of 20 mg/day (example: 0.25 mg/kg/day for an 80 kg person) up to a maximum of 120 mg/day or 1.5 mg/kg/day (whichever is higher) for >=2 weeks prior to randomization.
- Has exhibited PV disease control, defined as no new lesions for >=2 weeks. A female subject is eligible to enter the study if she: Is of non-child bearing potential, who 7. is either surgically sterile (bilateral tubal ligation, bilateral oophorectomy, or post-hysterectomy) or is postmenopausal without menses for >2 years. Women who are <2 years postmenopausal are required to have menopausal status confirmed by follicle-stimulating hormone (FSH) and estradiol levels at the screening evaluation. If FSH and estradiol levels do not provide confirmation of menopause, subject will be considered to be of childbearing potential.
Who cannot join
- Diagnosis of pemphigus foliaceus, paraneoplastic pemphigus, or other autoimmune blistering disease (other than pemphigus vulgaris).
- Past or current history of hypersensitivity to components of the investigational product or medically significant adverse effects (including allergic reactions) from cetirizine (or antihistamine equivalent) or paracetamol/acetaminophen.
- Prior treatment with rituximab without achieving disease control within 6 months of initiating rituximab dosing.
- Prior treatment with immunosuppressant or immunomodulation agents within the protocol specified periods
- Evidence or history of clinically significant infections
- Past or current malignancy, except for cervical carcinoma Stage 1B or less, noninvasive basal cell and squamous cell skin carcinoma and cancer diagnoses with a duration of complete response (remission) >5 years
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- Significant concurrent, uncontrolled medical condition that could affect the subject's safety, impair the subject's reliable participation in the study, impair the evaluation of endpoints, or necessitate the use of medication not allowed by the protocol. This includes subjects who require any systemic steroid treatment for a concurrent medical condition (other than pemphigus vulgaris).
- Use of an investigational drug or other experimental therapy within 4 weeks, 5 pharmacokinetic half-lives, or the duration of biological effect (whichever is longer) prior to Screening.
- Electrocardiogram (ECG) showing a clinically significant abnormality or showing a QTc interval ≥450 msec (≥480 msec for subjects with a bundle branch block)
- Woman who is breastfeeding.
- Positive test results for hepatitis B surface antigen (HBsAg), hepatitis B core antibody (HBcAb), or hepatitis C virus (HCV) serology at screening
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Tongji Hospital
Wuhan, Hubei, 430030
AIHUA DU, M.D, +86 2783662886
YIKAI YU, M.D
About this study
Official title: A Crossover, Randomized and Multi-center Study to Evaluate the Efficacy and Safety of Abatacept Versus Mycophenolate Mofetil (MMF) in Treatment of PV
Pemphigus vulgaris (PV) is a rare, chronic, debilitating, and potentially life-threatening autoimmune disorder that is characterized by mucocutaneous blisters.Abatacept is a biologic drug that belongs to the class of T-cell co-stimulation modulators and is used for the treatment of autoimmune diseases.
Read the detailed description
The background therapy is based on prednisolone administration. PV is a rare disorder, therefore this study is designed as a crossover that may require fewer patients than a parallel study. The study enrolled participants with moderate-to-severely active PV requiring ≥ 50 milligrams per day (mg/day) oral prednisone or equivalent. The purpose of this study was to evaluate the efficacy, tolerability, and safety of abatacept injection for subcutaneous use (abatacept SC) 150 mg administered once in a week in subjects with PV. It was anticipated that with sustained immune suppression in the presence of abatacept SC that clinical remission of the disease would be improved.
- Conditions
- Pemphigus Vulgaris
- Being tested
- Abatacept Prefilled Syringe, Mycophenolate Mofetil 500Mg Tab
- Medical subject headings
- Pemphigus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Abatacept Prefilled SyringeDrug
Also called Orencia
Abatacept (Orencia) was provided in prefilled glass syringes initial syringes contained 0.6ml (60mg) of concentration 100 mg/m: drug product subsequently modified to 0.4 mL (20mg) concentration (50mg/ML) drug product. Combined with standard of care prednisone 10-40mg qd
Mycophenolate Mofetil 500Mg TabDrug
Also called MMF,CellCept
MMF will be administered at a starting dose of 1000 milligrams (mg) Q12H and the dose will be tapered to achieve a goal of 0.5-1.0 gram (gm) Q12H. Combined with standard of care prednisone 10-40mg qd through 52 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Abatacept | Experimental | Subject received subcutaneous administration of abatacept 125 mg once every week through the 52 week double blind period. Receives: Drug: Abatacept Prefilled Syringe |
| Mycophenolate mofetil | Active comparator | Subject received subcutaneous administration of matching placebo of abatacept once a weeks through the 24 week double blind period. A washout period of MMF for 4 weeks (24 th-28th week) is used to ensure data integrity. Subsequently,subject were administered subcutaneous administration of abatacept 125 mg once every week through the 28-52 week open label period. Receives: Drug: Mycophenolate Mofetil 500Mg Tab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Achieved Sustained Complete Remission, Evaluated by the Pemphigus Disease Area Index (PDAI) Activity Score
PADAI and ABSIS was proposed by the German Blistering Disease Group in 2007 which was accepted as the most sensitive and reliable systems for evaluation of pemphigus severity. PADAI was developed by the International Pemphigus Definitions Group in 2009
Measured: 24 Weeks
Percentage of Participants Who Achieved Sustained Complete Remission, Evaluated byAutoimmune bullous skin disorder intensity score (ABSIS)
PADAI and ABSIS was proposed by the German Blistering Disease Group in 2007 which was accepted as the most sensitive and reliable systems for evaluation of pemphigus severity. PADAI was developed by the International Pemphigus Definitions Group in 2009
Measured: 24 Weeks
Secondary outcomes (6)
Cumulative Oral Corticosteroid Dose
Calculate the Cumulative Oral Corticosteroid Dose during 52 week
Measured: From 12th, 24th, 36th and 52th Week
Ulcer Severity Score (USS) for the assessment of skin, oral ulcer improvement
The USS incorporates six ulcer characteristics: number, size, duration, ulcer-free period, site, and pain. This scoring template may be of value to future studies assessing treatment efficacy.
Measured: From baseline up to 4th, 8th,12th, 24th, 36th and 52th Week
Physician global assessment (PGA)
Physician global assessment was assessed by an individual researcher
Measured: From baseline up to 4th, 8th,12th, 24th, 36th and 52th Week
Autoimmune bullous disease quality of life (ABQoL)
Total ABQoL scores range from 0 to 30 with higher DLQI scores reflecting greater impairment in a participant's health-related quality of life. The ABQoL score is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. The measure type mean is the estimated mean from adjusted MMRM.
Measured: From baseline up to 4th, 8th,12th, 24th, 36th and 52th Week
Anti-desmoglein 1 (anti-Dsg1) and anti-Dsg3 autoantibody titers
anti-Dsg1 and anti-Dsg3 will be performed using ELISA
Measured: From Baseline up to 12th, 24th and 52th Week
Change From Baseline for CD19+ B Cell Count
CD19+ B cell count will be performed using Flow Cytometry
Measured: From Baseline up to 12th, 24th and 52th Week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2022
- Study start1 Feb 2021
- Primary completion1 Sep 2022 (estimated)
- Study completion30 Sep 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pemphigus VulgarisStudies of the same condition, open and under way.
- More trials run by Tongji HospitalThe sponsor's other registered studies.
- 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
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Care provider, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tongji Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Wuhan Central Hospital, Wuhan Hospital of Traditional Chinese Medicine
- Responsible party
- Principal investigator, YIKAI YU, Tongji Hospital
- Investigators
- AIHUA DU, M.D, Study chair, Tongji Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05303272
- Sponsor's protocol ID
- TJRH2021109
- Status last verified
- Mar 2022
- First submitted
- 25 Feb 2022
- Last updated
- 31 Mar 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Crossover, Randomized and Multi-center Study to Evaluate the Efficacy and Safety of Abatacept Versus Mycophenolate Mofetil (MMF) in Treatment of PV
- ClinicalTrials.gov ID
- NCT05303272
- Acronym / sponsor ID
- TJRH2021109
- Conditions
- Pemphigus Vulgaris
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Tongji HospitalUniversity, hospital or other
- Collaborators
- Wuhan Central Hospital, Wuhan Hospital of Traditional Chinese Medicine
- Study start
- 1 Feb 2021 (actual)
- Primary completion
- 1 Sep 2022 (estimated)
- Study completion
- 30 Sep 2022 (estimated)
- First posted
- 31 Mar 2022 (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.
Pemphigus vulgaris (PV) is a rare, chronic, debilitating, and potentially life-threatening autoimmune disorder that is characterized by mucocutaneous blisters.Abatacept is a biologic drug that belongs to the class of T-cell co-stimulation modulators and is used for the treatment of autoimmune diseases.
Detailed description
The background therapy is based on prednisolone administration. PV is a rare disorder, therefore this study is designed as a crossover that may require fewer patients than a parallel study. The study enrolled participants with moderate-to-severely active PV requiring ≥ 50 milligrams per day (mg/day) oral prednisone or equivalent. The purpose of this study was to evaluate the efficacy, tolerability, and safety of abatacept injection for subcutaneous use (abatacept SC) 150 mg administered once in a week in subjects with PV. It was anticipated that with sustained immune suppression in the presence of abatacept SC that clinical remission of the disease would be improved.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (care provider, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Abatacept | Experimental | Drug: Abatacept Prefilled SyringeSubject received subcutaneous administration of abatacept 125 mg once every week through the 52 week double blind period. |
| Mycophenolate mofetil | Active comparator | Drug: Mycophenolate Mofetil 500Mg TabSubject received subcutaneous administration of matching placebo of abatacept once a weeks through the 24 week double blind period. A washout period of MMF for 4 weeks (24 th-28th week) is used to ensure data integrity. Subsequently,subject were administered subcutaneous administration of abatacept 125 mg once every week through the 28-52 week open label period. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Adults (18 through 80 years of age) with clinically-documented diagnosis of PV for >2 months and <10 years.
- History of biopsy consistent with PV (Hematoxylin and Eosin staining and direct immunofluorescence). Confirmed diagnosis of PV within the previous 24 months, based on the presence of histological features of acantholysis via skin or mucosal biopsy and one of the following: tissue bound immunoglobulin G (IgG) antibodies by direct immunofluorescence on the surface of affected epithelium or serological detection of serum desmoglein-3 (DSg3) autoantibodies against epithelial cell surface either by indirect immunofluorescence microscopy or by enzyme-linked immunosorbent assay.
- At least 1 previous episode of a failed steroid taper (ie, disease flare/relapse) at a prednisone/prednisolone dose >10 mg/day. The following criteria must have been met as evidence of disease severity at the time of the failed steroid taper: a) A Pemphigus Severity of Clinical Disease score of moderate (2) or severe (3) (may be historical/retrospective assessment). b) Required a treatment change at the time of the failed steroid taper of at least one of the following: i) A steroid increase to >=20 mg/day OR ii) The addition of immunosuppressive/immunomodulatory agent/treatment OR iii) A dose increase of immunosuppressive/immunomodulatory agent/treatment
- Screening anti-Dsg antibodies consistent with a diagnosis of PV (ie, elevated antiDsg3 antibodies).
- Has initiated and received a stable dose of prednisone/prednisolone from a minimum of 20 mg/day (example: 0.25 mg/kg/day for an 80 kg person) up to a maximum of 120 mg/day or 1.5 mg/kg/day (whichever is higher) for >=2 weeks prior to randomization.
Key exclusion criteria (11 listed)
- Diagnosis of pemphigus foliaceus, paraneoplastic pemphigus, or other autoimmune blistering disease (other than pemphigus vulgaris).
- Past or current history of hypersensitivity to components of the investigational product or medically significant adverse effects (including allergic reactions) from cetirizine (or antihistamine equivalent) or paracetamol/acetaminophen.
- Prior treatment with rituximab without achieving disease control within 6 months of initiating rituximab dosing.
- Prior treatment with immunosuppressant or immunomodulation agents within the protocol specified periods
- Evidence or history of clinically significant infections
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 |
|---|---|---|
| Abatacept Prefilled SyringeAlso: OrenciaAbatacept (Orencia) was provided in prefilled glass syringes initial syringes contained 0.6ml (60mg) of concentration 100 mg/m: drug product subsequently modified to 0.4 mL (20mg) concentration (50mg/ML) drug product. Combined with standard of care prednisone 10-40mg qd | Drug | Abatacept |
| Mycophenolate Mofetil 500Mg TabAlso: MMF,CellCeptMMF will be administered at a starting dose of 1000 milligrams (mg) Q12H and the dose will be tapered to achieve a goal of 0.5-1.0 gram (gm) Q12H. Combined with standard of care prednisone 10-40mg qd through 52 weeks. | Drug | Mycophenolate mofetil |
Primary outcome
Percentage of Participants Who Achieved Sustained Complete Remission, Evaluated by the Pemphigus Disease Area Index (PDAI) Activity Score
Time frame: 24 Weeks
PADAI and ABSIS was proposed by the German Blistering Disease Group in 2007 which was accepted as the most sensitive and reliable systems for evaluation of pemphigus severity. PADAI was developed by the International Pemphigus Definitions Group in 2009
Percentage of Participants Who Achieved Sustained Complete Remission, Evaluated byAutoimmune bullous skin disorder intensity score (ABSIS)
Time frame: 24 Weeks
PADAI and ABSIS was proposed by the German Blistering Disease Group in 2007 which was accepted as the most sensitive and reliable systems for evaluation of pemphigus severity. PADAI was developed by the International Pemphigus Definitions Group in 2009
6 secondary outcomes
Cumulative Oral Corticosteroid Dose
Time frame: From 12th, 24th, 36th and 52th Week
Ulcer Severity Score (USS) for the assessment of skin, oral ulcer improvement
Time frame: From baseline up to 4th, 8th,12th, 24th, 36th and 52th Week
Physician global assessment (PGA)
Time frame: From baseline up to 4th, 8th,12th, 24th, 36th and 52th Week
Autoimmune bullous disease quality of life (ABQoL)
Time frame: From baseline up to 4th, 8th,12th, 24th, 36th and 52th Week
Anti-desmoglein 1 (anti-Dsg1) and anti-Dsg3 autoantibody titers
Time frame: From Baseline up to 12th, 24th and 52th Week
Change From Baseline for CD19+ B Cell Count
Time frame: From Baseline up to 12th, 24th and 52th Week
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2022.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
AIHUA DU, M.D study chair
Tongji Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Abatacept Prefilled Syringe and Mycophenolate Mofetil 500Mg Tab in people with Pemphigus Vulgaris, run by Tongji Hospital at 1 site in China.
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 6 inclusion and 11 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: status not confirmed, as of the sponsor's last update on 31 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact YIKAI YU (+1 484-995-5917, yuyikai@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants Who Achieved Sustained Complete Remission, Evaluated by the Pemphigus Disease Area Index (PDAI) Activity Score (time frame: 24 Weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 31 Mar 2022.