Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Genentech, Inc. | INDUSTRY |
Not provided
Not provided
Not provided
The purpose of this pilot study is to test whether an 8-week course of glucocorticoids, combined with rituximab, is effective in treating ANCA-associated vasculitis.
The primary aim of this pilot study is to examine whether an 8 week course of glucocorticoids, in combination with rituximab, is effective in inducing and maintaining disease remission for up to 6 months in a subset of patients with ANCA-associated vasculitis (AAV) who have a more favorable prognosis.
This pilot study will enroll 20 patients with active AAV. Close patient follow-up will insure that any patients who require courses of glucocorticoids longer than two months will receive longer therapy, if appropriate for their well-being.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Glucocorticoids and Rituximab | Experimental | This is a single-arm trial. All patients receive both rituximab and glucocorticoids. The protocol calls for the discontinuation of prednisone within two months of the baseline visit. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Glucocorticoids | Drug | Patients will begin prednisone therapy at a dose selected by the investigator or the treating physician with oral prednisone 60mg or 1mg/kg (if weight less than 60kg) or intravenous methylprednisolone, up to 1g/day for three days. Prednisone will be tapered over 8 weeks as follows:
|
| Measure | Description | Time Frame |
|---|---|---|
| Complete Remission | We examined whether an 8-week glucocorticoid course in combination with rituximab (RTX) would induce disease remission in patients with AAV. The primary outcome was disease remission off steroids at 6 months. | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Disease Response | Number of patients achieving disease response defined as, no new disease manifestations; no worsening of existing disease; stable or improved BVAS/WG score at 4 weeks. | 4 weeks |
| Partial Remission |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| John H Stone, MD | Massachusetts General Hospital and Harvard Medical School | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
Individual participant data will be shared upon request - without personal health information - following completion of the analysis. Inquiries should be directed to the Principal Investigator.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Glucocorticoids and Rituximab | All patients receive both rituximab and glucocorticoids. The protocol calls for the discontinuation of prednisone within two months of the baseline visit. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Glucocorticoids and Rituximab | This is a single-arm trial. All patients receive both rituximab and glucocorticoids. The protocol calls for the discontinuation of prednisone within two months of the baseline visit. Glucocorticoids: Patients will begin prednisone therapy at a dose selected by the investigator or the treating physician with oral prednisone 60mg or 1mg/kg (if weight less than 60kg) or intravenous methylprednisolone, up to 1g/day for three days. Prednisone will be tapered over 8 weeks as follows:
Rituximab: Rituximab will be administered in four weekly doses at 375mg/m2 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Complete Remission | We examined whether an 8-week glucocorticoid course in combination with rituximab (RTX) would induce disease remission in patients with AAV. The primary outcome was disease remission off steroids at 6 months. | Posted | Count of Participants | Participants | No | 6 months |
|
Reports of unanticipated problems involving risks to subjects or others are to be submitted to the IRB within 5 working days/7 calendar days of the date the investigator first becomes aware of the problem. Adverse events were collected through the 6 month study duration.
Serious adverse event means any event temporally associated with the subject's participation in research that meets any of the following criteria:
1) hospitalization or prolonged hospitalization 2.)One severe flare - defined as BVAS/WG ≥ 3 or experiencing one of the major BVAS/WG items) 3.) unexpected and related to the investigation drug
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Glucocorticoids and Rituximab | Patients will begin prednisone therapy at a dose selected by the investigator or the treating physician with oral prednisone 60mg or 1mg/kg (if weight less than 60kg) or intravenous methylprednisolone, up to 1g/day for three days. Prednisone will be tapered over 8 weeks as follows:
Rituximab: Rituximab will be administered in four weekly doses at 375mg/m2 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Severe flares | Immune system disorders | Systematic Assessment |
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. John Stone | Massachusetts General Hospital, Division of Rheumatology | 617-643-2140 | adfernandes@mgh.harvard.edu |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jun 28, 2015 | May 15, 2018 | Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D014890 | Granulomatosis with Polyangiitis |
| D055953 | Microscopic Polyangiitis |
| ID | Term |
|---|---|
| D017563 | Lung Diseases, Interstitial |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D056648 | Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis |
Not provided
Not provided
| ID | Term |
|---|---|
| D005938 | Glucocorticoids |
| D011241 | Prednisone |
| D000069283 | Rituximab |
| ID | Term |
|---|---|
| D000305 | Adrenal Cortex Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D045505 | Physiological Effects of Drugs |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
|
| Rituximab | Drug | Rituximab will be administered in four weekly doses at 375mg/m2 |
|
|
Number of patients entering partial remission, defined as no new disease manifestations, no worsening of existing disease and BVAS/WG < 3.
| 8 weeks |
| Sustained Complete Remission | Number of patients entering sustained remission defined as BVAS/WG = 0, prednisone dose = 0 and no disease flares during the study period. | 6 months |
| Limited Flares | Number of limited flares defined as a new occurrence or worsening of one or more minor BVAS/WG items and a total BVAS/WG ≤ 3 | 6 months |
| Severe Flares | Number of severe flares defined as flare with BVAS/WG > 3 or experiencing one of the major BVAS/WG items | 6 months |
| Early Treatment Failures | Number of early treatment failures defined as patients who have new or worsening disease manifestations assessed at 4 weeks after study entry | 4 weeks |
| Vasculitis Damage Index (VDI) | The Vasculitis Damage Index (VDI) is a single-page catalog of damage items separated into 11 groupings of items by organ system. There are a total of 60 items. Each item is recorded if it occurred since the onset of vasculitis, has been present for at least 3 months, or occurred at least 3 months ago. Each item of damage is scored as present (1) or absent (0), yielding a maximum score of 60. | 24 months |
| Participants |
| No |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants | No |
|
| Birmingham Vasculitis Activity Score for Wegener's Granulomatosis(BVAS/WG) | Birmingham Vasculitis Activity Score for Wegener's Granulomatosis(BVAS/WG, range 0-64). The total score is composed of 34 predefined items, units on a scale, grouped into 9 organ systems. Each item carries a weight from 1-3, depending on disease severity. Subjects with a score of 3 or higher were invited to participate in the trial. A score of 0 indicates no disease activity; a higher score indicates worsening disease. | Mean | Full Range | units on a scale |
|
| Disease group | Disease type (GPA or MPA) | Count of Participants | Participants | No |
|
| anti-neutrophil cytoplasmic antibody (ANCA) | ANCA type and titer were determined by ELISA. | Count of Participants | Participants | No |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Secondary | Disease Response | Number of patients achieving disease response defined as, no new disease manifestations; no worsening of existing disease; stable or improved BVAS/WG score at 4 weeks. | Number of patients having a disease response | Posted | Count of Participants | Participants | No | 4 weeks |
|
|
|
| Secondary | Partial Remission | Number of patients entering partial remission, defined as no new disease manifestations, no worsening of existing disease and BVAS/WG < 3. | Posted | Count of Participants | Participants | 8 weeks |
|
|
|
| Secondary | Sustained Complete Remission | Number of patients entering sustained remission defined as BVAS/WG = 0, prednisone dose = 0 and no disease flares during the study period. | Posted | Count of Participants | Participants | 6 months |
|
|
|
| Secondary | Limited Flares | Number of limited flares defined as a new occurrence or worsening of one or more minor BVAS/WG items and a total BVAS/WG ≤ 3 | Posted | Count of Participants | Participants | 6 months |
|
|
|
| Secondary | Severe Flares | Number of severe flares defined as flare with BVAS/WG > 3 or experiencing one of the major BVAS/WG items | Posted | Count of Participants | Participants | 6 months |
|
|
|
| Secondary | Early Treatment Failures | Number of early treatment failures defined as patients who have new or worsening disease manifestations assessed at 4 weeks after study entry | Posted | Count of Participants | Participants | No | 4 weeks |
|
|
|
| Secondary | Vasculitis Damage Index (VDI) | The Vasculitis Damage Index (VDI) is a single-page catalog of damage items separated into 11 groupings of items by organ system. There are a total of 60 items. Each item is recorded if it occurred since the onset of vasculitis, has been present for at least 3 months, or occurred at least 3 months ago. Each item of damage is scored as present (1) or absent (0), yielding a maximum score of 60. | Posted | Mean | Inter-Quartile Range | units on a scale | 24 months |
|
|
|
| 0 |
| 20 |
| 11 |
| 20 |
| 0 |
| 20 |
| myocardial infarction | Cardiac disorders | Systematic Assessment |
|
| atrial fibrillation, | Cardiac disorders | Systematic Assessment |
|
| syncope | Immune system disorders | Systematic Assessment |
|
| Thyroid maligancy | Endocrine disorders | Systematic Assessment |
|
| Uterine malignancy | Reproductive system and breast disorders | Systematic Assessment |
|
Not provided
Not provided
| D056647 | Systemic Vasculitis |
| D014657 | Vasculitis |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D017445 | Skin Diseases, Vascular |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D020228 | Pharmacologic Actions |
| D020164 | Chemical Actions and Uses |
| D011244 | Pregnadienediols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D058846 | Antibodies, Monoclonal, Murine-Derived |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |