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CBMTG 0601 Update
13-Feb-2009 Tampa, Florida
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Key Information• Pivotal Phase III trial comparing G-PB with G-BM
(experimental arm)
• 230 recipient-donor pairs
• Sponsor: CBMTG
• Grant PI: Dr. Kirk Schultz
• Clinical Chair: Dr. Stephen Couban; Co-Chair: Dr. Jeff Lipton
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Primary Objective
• Will we see less chronic GvHD with G-BM?
• Treatment Failure (Composite Endpoint): - extensive chronic GVHD - relapse - death
(Time to the earliest of 3 events after HSCT)
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Secondary Objectives
• Hematologic recovery
• Acute and chronic GVHD
• Donor and Recipient Quality of Life
• Laboratory analysis of graft characteristics
• Prospective economic analysis
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Project History
• Grant first submitted July 2005
• Received NIH funding April 2006
• Investigator Meeting June 2006
• First site activated January 2007 (CCMB)
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Actual versus Projected EnrollmentMarch 2008
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Vancouver
Winnipeg
LondonHamilton Toronto
OttawaMontreal
Quebec City Halifax
9 Canadian Sites
5 International Sites: Fred Hutch King Faisal Melbourne Auckland Adelaide
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Site State Depart
Approval
IRB
Approval Contract
Site
Activation
Fred Hutch Jul-2008
King Faisal Nov-2008
Auckland City Hospital
Royal Melbourne
IMVS – Adelaide
International Sites
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Total enrollment = 61
February 2009
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Cumulative Enrollment
re-projected rate
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Enrollment by Site (Feb-2009)
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Re-projected Accrual Timeline
Accrual to be completed by January 2012
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Data Safety Monitoring Committee
• SAE’s are submitted to DSMC within 48 hours
• Formal review every 6 months (most recent Dec-08)
• Monitors accrual
• Requires updates on data submission compliance every 6 months
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Data Submission ComplianceClinical Data
Time since
Randomization(in months)
Number
enrolled
0-5 6-11 12-17 24 +
0-5 19 19 - - -
6-11 13 8 4 - -
12-17 17 15 2 0 -
18-23 8 7 0 1 -
24+ 4 4 0 0 0
• 7 pairs are more than 6 months overdue • Overall, data submission compliance is excellent
Data current within number of months
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Timelines for Data Submission
Data Form Submission Deadlines
Questionnaires Same day
Section 3 to 6
Pre-HCT and graft product
1 month post HCT
Section 7 and 8
Day 30 and Day 100
1 month from day +100
Section 9
Follow-up
1 month from date of follow-up
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What’s new?
• No more invoicing for per patient payments
• Electronic data capture on the horizon
• Collection of more GvHD data at Year 1 and Year 3
(NIH Consensus Data Collection Form)
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• All deaths to be reported as SAEs up until last follow-up (required by the DSMC)
• Data on immunosuppressive therapy
(Section 9: Follow-Up)
• Revised Health Care Questionnaire
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Consent and Enrollment
• Approach recipients first
• Quality of Life can be done
prior to randomization if consent obtained
• Product can be cryopreserved if scheduling difficulties arise
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Number Scheduled for Allo HCT versus Number on Study
9 Activated Sites (48% enrolled on average)
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Summary
• Continued commitment necessary in order to complete accrual by 2012
• Let’s try to submit data on schedule!
• Changes to SAE reporting are coming
• Sites are making a great contribution!