1

CBMTG 0601 Update

13-Feb-2009 Tampa, Florida

22

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

33

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)

44

Secondary Objectives

• Hematologic recovery

• Acute and chronic GVHD

• Donor and Recipient Quality of Life

• Laboratory analysis of graft characteristics

• Prospective economic analysis

55

Project History

• Grant first submitted July 2005

• Received NIH funding April 2006

• Investigator Meeting June 2006

• First site activated January 2007 (CCMB)

66

Actual versus Projected EnrollmentMarch 2008

7

Vancouver

Winnipeg

LondonHamilton Toronto

OttawaMontreal

Quebec City Halifax

9 Canadian Sites

5 International Sites: Fred Hutch King Faisal Melbourne Auckland Adelaide

88

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

9

Total enrollment = 61

February 2009

10

Cumulative Enrollment

re-projected rate

11

Enrollment by Site (Feb-2009)

12

Re-projected Accrual Timeline

Accrual to be completed by January 2012

13

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

13

14

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

15

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

16

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)

17

• 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

1818

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

19

Number Scheduled for Allo HCT versus Number on Study

9 Activated Sites (48% enrolled on average)

20

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!