A Phase 1/2 interventional study of Abatacept in Wegener's Granulomatosis, sponsored by University of Pennsylvania. Completed at 4 sites in United States. Open to participants aged 15 Years and older. Per ClinicalTrials.gov, last updated 2016-01-18.
Sponsored by University of Pennsylvania · Phase 1/2, Interventional, and Treatment
Wegener's granulomatosis (WG) is a rare disease that causes inflammation of blood vessels, or vasculitis. It may involve many different parts of the body, but typically affects the upper and lower respiratory tract and kidneys. The purpose of this study is to determine the safety and effectiveness of the medication abatacept in treating adults with mild relapsing WG.
Current standard treatment for WG involves various medications and is based on disease severity. Unfortunately, more than 50% of people experience a relapse after remission, placing them at risk for additional organ damage and medication toxicity. To prevent this, safer and more effective treatments for mild relapses are needed. Several studies have shown that activated T cells, a type of white blood cell important in regulating immune responses, play a role in WG. Abatacept, an immunoglobulin-based medication approved by the FDA to treat rheumatoid arthritis, acts by preventing T-cell activation and may be useful in treating mild relapses of WG. The purpose of this study is to determine the safety and effectiveness of abatacept in treating adults with mild relapsing WG.
Participants will receive abatacept intravenously at study visits on Days 1, 15, and 29, and then once a month thereafter. A participant's abatacept dose is based on body weight and will remain the same throughout the study. Participants who are receiving maintenance immunosuppressive medications consisting of methotrexate, azathioprine, or mycophenolate mofetil at the time of enrollment will remain on these medications without dosage increase or reduction. Eligible participants may be on up to prednisone 15mg daily at the time of relapse. Following the development of relapse, participants may be treated with up to prednisone 30mg daily if necessary, but must to be back to the same dose that they had been on prior to relapse by Month 2. All study visits include medication review, physical exam, blood and urine collection, and questionnaires. A chest x-ray, computed tomography (CT) scan of the chest and sinuses, and lung function testing will occur at some study visits. Participants whose symptoms did not improved by Month 2 will be taken off abatacept. Any participants undergoing early termination or, after common closing, will undergo three follow-up study visits at 1, 3, and 6 months after the end of treatment.
104 studies on the registry are indexed under Granulomatosis with Polyangiitis; 19 are open to participants now.
This study's enrollment of 20 is below the median of 70 across 67 interventional studies indexed under Granulomatosis with Polyangiitis.
Browse Granulomatosis with Polyangiitis studies →University of Pennsylvania is the lead sponsor of 1,635 studies on the registry; 239 are open to participants now.
Of its 154 completed or terminated interventional studies of FDA-regulated products, 104 (68%) have results posted.
Counted across the registry records on this site, refreshed daily.
Relapse of WG within the past 28 days where disease activity is confined to one or more of the following sites and where the symptoms/signs are of such a nature that the usual treatment would consist of the reinstitution or increase in GC to no more than prednisone 30mg daily and/or an increase or addition of a second immunosuppressive agent other than CYC (more specific information about this criterion can be found in the protocol):
Exclusion Criteria:
Participants will receive abatacept intravenously at study visits on Days 1, 15, and 29, and then once a month thereafter.
Drug: Abatacept
A participant's abatacept dose depended on body weight and will remain the same throughout the study: * 500 mg of abatacept for body weight less than 60 kg * 750 mg of abatacept for body weight between 60 and 100 kg * 1000 mg of abatacept for body weight greater than 100 kg Abatacept is administered in a 30-minute intravenous infusion.
Safety of Abatacept - Number of Participants With Adverse Events
This study examined the safety profile of this agent when used in Wegener's granulomatosis. Information was gathered on all adverse events with specific events being identified in the protocol for analysis that included the following: * Infection * Infusion reactions * Cytopenias * Transaminase elevation * Skin reactions * GI side effects * Malignancy All adverse events were reportable for this study.
Time frame: Measured continuously from the screening visit through to the 6 month post-treatment study visit, up to 3 years and 4 months.
Disease Remission
Disease remission was measured by a Birmingham Vasculitis Activity Score for Wegener's Granulomatosis (BVAS/WG) of 0. The BVAS/WG is a validated disease activity index. The BVAS/WG is designed to document new or worsening clinically active vasculitis and consists of a set of items divided into nine organ based systems. BVAS/WG scores range from 0 to 63.
Time frame: Measured monthly until common closing or early termination,up to 3 years and 4 months.
Disease Improvement
Disease improvement was measured by a reduction in the Birmingham Vasculitis Activity Score for Wegener's Granulomatosis (BVAS/WG). The BVAS/WG is a validated disease activity index. The BVAS/WG is designed to document new or worsening clinically active vasculitis and consists of a set of items divided into nine organ based systems. BVAS/WG scores range from 0 to 63.
Time frame: Measured monthly until common closing or early termination, up to 3 years and 4 months.
Meeting Common Closing
The number of subjects that reached the common closing date.
Time frame: Number assessed at the time of common closing, up to 3 years and 4 months.
Disease Relapse
Disease relapse was measured by a rise in the Birmingham Vasculitis Activity Score for Wegener's Granulomatosis (BVAS/WG) of greater than or equal to 1 after achieving remission. The BVAS/WG is a validated disease activity index. The BVAS/WG is designed to document new or worsening clinically active vasculitis and consists of a set of items divided into nine organ based systems. BVAS/WG scores range from 0 to 63.
Time frame: Measured monthly until common closing or early termination, up to 3 years and 4 months.
Recruitment began in February 2008 and the final subject was enrolled in June 2010. Subjects were recruited through the clinical practices of each site investigator.
| Milestone | Open-label Abatacept |
|---|---|
| Started | 20 |
| Completed | 20 |
| Not completed | 0 |
This study examined the safety profile of this agent when used in Wegener's granulomatosis. Information was gathered on all adverse events with specific events being identified in the protocol for analysis that included the following: * Infection * Infusion reactions * Cytopenias * Transaminase elevation * Skin reactions * GI side effects * Malignancy All adverse events were reportable for this study.
| participants | Open-label Abatacept |
|---|---|
| Serious adverse events | 7 |
| Non-serious adverse events | 16 |
| Infection | 14 |
| Infusion related (systemic) | 1 |
| Infusion related( intravenous site reaction) | 1 |
| Cytopenia | 4 |
Disease remission was measured by a Birmingham Vasculitis Activity Score for Wegener's Granulomatosis (BVAS/WG) of 0. The BVAS/WG is a validated disease activity index. The BVAS/WG is designed to document new or worsening clinically active vasculitis and consists of a set of items divided into nine organ based systems. BVAS/WG scores range from 0 to 63.
| participants | Open-label Abatacept |
|---|---|
| Disease Remission | 16 |
Disease improvement was measured by a reduction in the Birmingham Vasculitis Activity Score for Wegener's Granulomatosis (BVAS/WG). The BVAS/WG is a validated disease activity index. The BVAS/WG is designed to document new or worsening clinically active vasculitis and consists of a set of items divided into nine organ based systems. BVAS/WG scores range from 0 to 63.
| participants | Open-label Abatacept |
|---|---|
| Disease Improvement | 18 |
The number of subjects that reached the common closing date.
| participants | Open-label Abatacept |
|---|---|
| Meeting Common Closing | 14 |
Disease relapse was measured by a rise in the Birmingham Vasculitis Activity Score for Wegener's Granulomatosis (BVAS/WG) of greater than or equal to 1 after achieving remission. The BVAS/WG is a validated disease activity index. The BVAS/WG is designed to document new or worsening clinically active vasculitis and consists of a set of items divided into nine organ based systems. BVAS/WG scores range from 0 to 63.
| participants | Open-label Abatacept |
|---|---|
| Disease Relapse | 3 |
Collected over Adverse event data was collected for subjects from time of signed consent through common close out of the study. The total timeframe of adverse event collection for the study was 3 years, 3 months.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Open-label Abatacept | — | 7/20 (35%) | 16/20 (80%) |
| Event | Open-label Abatacept |
|---|---|
| Infection - OcularInfections and infestations | 2/20 |
| Infection - DentalInfections and infestations | 1/20 |
| Infection - LungInfections and infestations | 1/20 |
| Infection - Upper airwayInfections and infestations | 1/20 |
| Infection - GastrointestinalInfections and infestations | 1/20 |
| Obstruction/stenosis of airwayRespiratory, thoracic and mediastinal disorders | 1/20 |
| Event | Open-label Abatacept |
|---|---|
| Dermatology/Skin - OtherSkin and subcutaneous tissue disorders | 5/20 |
| Infection - OtherInfections and infestations | 4/20 |
| Musculoskeletal/Soft TissueMusculoskeletal and connective tissue disorders | 4/20 |
| Pulmonary/Upper respiratoryRespiratory, thoracic and mediastinal disorders | 4/20 |
| Infection - Upper airwayInfections and infestations | 3/20 |
| Blood/Bone marrow - HemoglobinBlood and lymphatic system disorders | 2/20 |
| Blood/Bone marrow - LeukocytesBlood and lymphatic system disorders | 2/20 |
| Gastrointestinal - OtherGastrointestinal disorders | 2/20 |
| Infection - BronchusInfections and infestations | 2/20 |
| Infection - StomachInfections and infestations | 2/20 |
| Age, Categorical(Participants) | Open-label Abatacept |
|---|---|
| <=18 years | 1 |
| Between 18 and 65 years | 17 |
| >=65 years | 2 |
| Age, Continuous(years) | Open-label Abatacept |
|---|---|
| Mean | 45.1 ± 16.8 |
| Sex: Female, Male(Participants) | Open-label Abatacept |
|---|---|
| Female | 9 |
| Male | 11 |
| Ethnicity (NIH/OMB)(Participants) | Open-label Abatacept |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 17 |
| Unknown or Not Reported | 2 |
| Race (NIH/OMB)(Participants) | Open-label Abatacept |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 20 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Open-label Abatacept |
|---|---|
| United States | 20 |
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Granulomatosis with Polyangiitis→
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