Carfilzomib, Rituximab and Dexamethasone in Waldenstrom's Macroglobulinemia (CaRD)
Primary Purpose
Waldenstrom's Macroglobulinemia
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Dexamethasone
Carfilzomib
Rituximab
Sponsored by
About this trial
This is an interventional treatment trial for Waldenstrom's Macroglobulinemia focused on measuring WM, untreated, symptomatic
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of Waldenstrom's Macroglobulinemia
- Symptomatic disease
- Measurable disease
- Life expectancy of greater than 12 weeks
- Adequate organ and marrow function
- CD20 positive based on any previous performed bone marrow immunohistochemistry or flow cytometric analysis
- Disease free of prior malignancies for >/= 5 years with the exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast
Exclusion Criteria:
- More than one prior therapy
- Previous therapy with a proteasome inhibitor or rituximab
- Chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or not recovered from adverse events due to agents administered more than 4 weeks earlier
- Currently receiving treatment for any malignancy
- Major surgery within 21 days prior to study entry
- Acute active infection requiring treatment (systemic antibiotics, antivirals, or antifungals) within 14 days prior to study entry
- Uncontrolled hypertension or uncontrolled diabetes
- Significant neuropathy (Grades 3-4, or Grade 2 with pain) within 14 days prior to study entry
- Known history of allergy to Captisol
- Receiving any other study agents
- Known brain metastases
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to carfilzomib, rituximab, and/or dexamethasone
- Contraindication to any of the required concomitant drugs or supportive treatments, including hypersensitivity to all anticoagulation and antiplatelet options, antiviral drugs, or intolerance to hydration due to preexisting pulmonary or cardiac impairment
- Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Pregnant or lactating
- HIV-positive on combination antiretroviral therapy
Sites / Locations
- Dana-Farber Cancer Institute
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treatment Arm
Arm Description
Carfilzomib, dexamethasone, rituximab
Outcomes
Primary Outcome Measures
Overall Response Rate
Overall Response Rate= Minor response (>25%-50% reduction in serum IgM from baseline + Partial Response (>50-90% reduction in serum IgM from baseline) + Very Good Partial Response (>90% reduction in serum IgM from baseline) + Complete Response (resolution of all symptoms, normalization of serum IgM with disappearance of IgM paraprotein, resolution of any adenopathy or splenomegaly).
Neuropathy Incidence Rate
Number and percentage of participants who experienced neuropathy attributable to CaRD therapy
Time to Progression
Progression-free survival is the defined as the time from study entry to disease progression (PD) or death. Patients without PD are censored at the date of last disease evaluation. PD is defined as a greater than 25% increase in serum IgM and 500mg/dL absolute increase from the lowest attained response value as determined by serum electrophoresis, confirmed by at least one other investigation, or progression of clinically significant disease related symptom(s).
Major Response Rate
Major Response Rate= Partial Response (>50-90% reduction in serum IgM from baseline) + Very Good Partial Response (>90% reduction in serum IgM from baseline) + Complete Response (resolution of all symptoms, normalization of serum IgM with disappearance of IgM paraprotein, resolution of any adenopathy or splenomegaly).
Very Good Partial Response and Complete Response Rate
This is the rate of VGPR and CR in patients on CaRD therapy. Very good partial responses are >90% reduction in serum IgM from baseline. Complete response is defined as having resolution of all symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly.
Secondary Outcome Measures
Full Information
NCT ID
NCT01470196
First Posted
September 22, 2011
Last Updated
October 26, 2018
Sponsor
Dana-Farber Cancer Institute
Collaborators
Amgen
1. Study Identification
Unique Protocol Identification Number
NCT01470196
Brief Title
Carfilzomib, Rituximab and Dexamethasone in Waldenstrom's Macroglobulinemia
Acronym
CaRD
Official Title
Carfilzomib, Rituximab, and Dexamethasone (CaRD) in Waldenstrom's Macroglobulinemia
Study Type
Interventional
2. Study Status
Record Verification Date
October 2018
Overall Recruitment Status
Completed
Study Start Date
October 2011 (undefined)
Primary Completion Date
September 2016 (Actual)
Study Completion Date
September 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Dana-Farber Cancer Institute
Collaborators
Amgen
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Carfilzomib is a drug that has shown anti-tumor activity by inhibiting the proteasome within the cell, which is responsible for degrading or breaking down a wide variety of proteins. Carfilzomib has not been approved by the FDA.
Rituximab and dexamethasone are often used to treat Waldenstrom's Macroglobulinemia (WM), alone or in combination with other drugs. Combinations with rituximab, dexamethasone and proteasome inhibitors, like carfilzomib, show high levels of activity in WM patients.
In this research study, the investigators are testing the safety and efficacy of Carfilzomib when used along with Rituximab and Dexamethasone as a possible treatment for Waldenstrom's Macroglobulinemia.
Detailed Description
If you take part in this research study, you will receive Carfilzomib and dexamethasone as an infusion on Days 1, 2, 8, and 9 for Cycles 1-6. You will then have a Rituximab infusion on Days 2 and 9. Each cycles lasts 21 days. After completing Cycle 6 and if you are eligible, there will be a 2 month break before the maintenance phase is started. During this break, you will have a study visit with a physical exam, blood tests, and a bone marrow biopsy. If you continue to the maintenance phase, you will receive Carfilzomib and Dexamethasone on Days 1 and 2 and Rituximab on Day 2 of Cycles 1-8. Each cycle will continue to last 21 days, but will take place every 2 months. Infusions will last between 2-6 hours.
During all cycles you will have a physical exam and you will be asked questions about your general health and specific questions about any problems that you might be having and any medications you may be taking. Blood tests will also be done at each Cycle visit, and you will complete a questionnaire. Bone marrow and CT scan will only be repeated at physician discretion when appropriate and in order to ensure your response to treatment.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Waldenstrom's Macroglobulinemia
Keywords
WM, untreated, symptomatic
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
31 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Treatment Arm
Arm Type
Experimental
Arm Description
Carfilzomib, dexamethasone, rituximab
Intervention Type
Drug
Intervention Name(s)
Dexamethasone
Other Intervention Name(s)
Decadron
Intervention Description
20 mg IV on Days 1, 2, 8, 9, of 21 day cycle for cycles 1-6 20 mg IV on Days 1, 2 of 21 day cycles q 2 months for cycles 1-8
Intervention Type
Drug
Intervention Name(s)
Carfilzomib
Other Intervention Name(s)
PR-171
Intervention Description
20 mg/m2 IV on Days 1, 2, 8, 9 of 21 day cycles for Cycle 1 36 mg/m2 IV on Days 1, 2, 8, 9 of 21 day cycles for Cycles 2-6 36 mg/m2 IV on Days 1, 2 of 21 day cycles q 2 months for Cycles 1-8
Intervention Type
Drug
Intervention Name(s)
Rituximab
Other Intervention Name(s)
Rituxan
Intervention Description
375 mg/m2 IV on Days 2, 9 of 21 day cycles for Cycles 1-6 375 mg/m2 IV on Day 2 of 21 day cycles q 2 months for Cycles 1-8
Primary Outcome Measure Information:
Title
Overall Response Rate
Description
Overall Response Rate= Minor response (>25%-50% reduction in serum IgM from baseline + Partial Response (>50-90% reduction in serum IgM from baseline) + Very Good Partial Response (>90% reduction in serum IgM from baseline) + Complete Response (resolution of all symptoms, normalization of serum IgM with disappearance of IgM paraprotein, resolution of any adenopathy or splenomegaly).
Time Frame
4 years
Title
Neuropathy Incidence Rate
Description
Number and percentage of participants who experienced neuropathy attributable to CaRD therapy
Time Frame
3 years
Title
Time to Progression
Description
Progression-free survival is the defined as the time from study entry to disease progression (PD) or death. Patients without PD are censored at the date of last disease evaluation. PD is defined as a greater than 25% increase in serum IgM and 500mg/dL absolute increase from the lowest attained response value as determined by serum electrophoresis, confirmed by at least one other investigation, or progression of clinically significant disease related symptom(s).
Time Frame
4 years
Title
Major Response Rate
Description
Major Response Rate= Partial Response (>50-90% reduction in serum IgM from baseline) + Very Good Partial Response (>90% reduction in serum IgM from baseline) + Complete Response (resolution of all symptoms, normalization of serum IgM with disappearance of IgM paraprotein, resolution of any adenopathy or splenomegaly).
Time Frame
4 years
Title
Very Good Partial Response and Complete Response Rate
Description
This is the rate of VGPR and CR in patients on CaRD therapy. Very good partial responses are >90% reduction in serum IgM from baseline. Complete response is defined as having resolution of all symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly.
Time Frame
4 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of Waldenstrom's Macroglobulinemia
Symptomatic disease
Measurable disease
Life expectancy of greater than 12 weeks
Adequate organ and marrow function
CD20 positive based on any previous performed bone marrow immunohistochemistry or flow cytometric analysis
Disease free of prior malignancies for >/= 5 years with the exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast
Exclusion Criteria:
More than one prior therapy
Previous therapy with a proteasome inhibitor or rituximab
Chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or not recovered from adverse events due to agents administered more than 4 weeks earlier
Currently receiving treatment for any malignancy
Major surgery within 21 days prior to study entry
Acute active infection requiring treatment (systemic antibiotics, antivirals, or antifungals) within 14 days prior to study entry
Uncontrolled hypertension or uncontrolled diabetes
Significant neuropathy (Grades 3-4, or Grade 2 with pain) within 14 days prior to study entry
Known history of allergy to Captisol
Receiving any other study agents
Known brain metastases
History of allergic reactions attributed to compounds of similar chemical or biologic composition to carfilzomib, rituximab, and/or dexamethasone
Contraindication to any of the required concomitant drugs or supportive treatments, including hypersensitivity to all anticoagulation and antiplatelet options, antiviral drugs, or intolerance to hydration due to preexisting pulmonary or cardiac impairment
Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
Pregnant or lactating
HIV-positive on combination antiretroviral therapy
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Steven P Treon, MD, PhD
Organizational Affiliation
Dana-Farber Cancer Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
Dana-Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Citations:
PubMed Identifier
24859363
Citation
Treon SP, Tripsas CK, Meid K, Kanan S, Sheehy P, Chuma S, Xu L, Cao Y, Yang G, Liu X, Patterson CJ, Warren D, Hunter ZR, Turnbull B, Ghobrial IM, Castillo JJ. Carfilzomib, rituximab, and dexamethasone (CaRD) treatment offers a neuropathy-sparing approach for treating Waldenstrom's macroglobulinemia. Blood. 2014 Jul 24;124(4):503-10. doi: 10.1182/blood-2014-03-566273. Epub 2014 May 23.
Results Reference
derived
Learn more about this trial
Carfilzomib, Rituximab and Dexamethasone in Waldenstrom's Macroglobulinemia
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