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Information Request Email, March 30, 2012 - ALLOCORD

 

From:        Tull, Lori
Sent:          Friday, March 30, 2012 12:55 PM
To:             'Donna Regan'
Subject:     RE: Clinical/Statistical information request (revision)
 
 
Hi Donna,
 
Drs. Xu and Rees revised their list and asked me to forward to you. I left the original request email at the bottom for your reference.
 
Please provide the following information.
1.     One spreadsheet that contains all the data (not multiple sheets within one workbook). Please include one variable in the spreadsheet that uniquely identifies each patient.
2.     A data dictionary (i.e., a definition of each variable in the spreadsheet).
3.     In Patient Outcomes and Product Safety, section III (vii) of your application, you analyze data that is not available in the Excel spreadsheet containing the raw safety data set sent to the FDA on December 7, 2011 (e.g., HLA match). Furthermore, Table 31 of this section lists the number of patients analyzed for single and dual cord transplants as 1173 and 589, respectively, for a total of 1763 patients, but the Excel spreadsheet contains only 1211 units (rows). Please clarify which dataset was used in your analyses.
4.     Data on HLA match.
5.     Storage time or collection date information.
6.     Manufacturing procedure used for each unit.
7.     Last contact date for patients who are still alive.
8.     Are there any AE (adverse events) reported directly from physicians to the applicant?
9.     Are all the units in the spreadsheets (including all the units for multiple unit recipients) manufactured by the St. Louis Cord Blood Bank?
10. Please clarify if the applicant has the SOPP regarding SAE report to the FDA. If yes, please submit for review.
 
Please clarify the following information in the dataset.
11. Patient with CIBMTR ID 176950 has a death date of (b)(6), one day earlier than the last contact date of (b)(6).
12. Patient with CIBMTR ID 2980581 has a death date of (b)(6), two days earlier than the infusion date of (b)(6).
13. “Infused Nucleated Cell Count” ≤ “Total Infused Nucleated Cell Count” for many single unit recipients. Please clarify the difference between these variables.
14. One patient (CIBMTR ID 64552) achieved neutrophil recovery (“ANC 500” is ‘Y’), but is missing the number of days to recovery (DAYS to ANC 500).
15. Please clarify the distinction between a response of ‘N’ and ‘Never’ for the variable “ANC 500”.
16. One patient (CIBMTR ID 190555) has a response of 1122 for “Days to ANC 500” and another (CIBMTR ID 205890) has 181 days; please confirm these large number of days.
17. 15 patients have a ‘Y’ response for “Platelets 50000”, but are missing the number of days to recovery (“Days to Platelet 50,000”).
18. 16 patients have a ‘Y’ response for “Platelets 20000”, but are missing the number of days to recovery (“Days to Platelet 20,000”).
19. What does a response of “U” represent for the variable “Platelet 50,000”? 
 
_____________________________________________
From:                    Tull, Lori 
Sent:                      Wednesday, March 28, 2012 10:29 AM
To:                         'Donna Regan'
Subject:                 Clinical/Statistical information request
 
Information/Clarifications for St. Louis BLA 125413 (Cord Blood) Dataset
Hi Donna,
 
Drs. Le Xu (clinical) and Renee Rees (statistical) have the following information requests. They also asked me to set up a telecon to discuss these requests. We have these times (in EDT) available: tomorrow, 3/29 at 1pm; Friday, 3/30 at 1 or 3pm, Wednesday, 4/4 at 1 or 2pm, or Friday, 4/6 anytime between 1 and 3pm. Would any of those times work for your group? Just let me know.
 
Please provide the following information.
1.     One spreadsheet that contains all the data (not multiple sheets within one workbook). Please include one variable in the spreadsheet that uniquely identifies each patient.
2.     A data dictionary (i.e., a definition of each variable in the spreadsheet).
3.     In Patient Outcomes and Product Safety, section III (vii) of your application, you analyze data that is not available in the Excel spreadsheet containing the raw safety data set sent to the FDA on December 7, 2011 (e.g., HLA match). Furthermore, Table 31 of this section lists the number of patients analyzed for single and dual cord transplants as 1173 and 589, respectively, for a total of 1763 patients, but the Excel spreadsheet contains only 1211 units (rows). Please clarify which dataset was used in your analyses.
4.     Data on HLA match.
5.     Storage or collection data information.
6.     Are all the units in the spreadsheets (including all the units for multiple unit recipients) manufactured by the St. Louis Cord Blood Bank?
 
Please clarify the following information in the dataset.
7.   Patient with CIBMTR ID 176950 has a death date of (b)(6), one day earlier than the last contact date of (b)(6).
8.   Patient with CIBMTR ID 2980581 has a death date of (b)(6), two days earlier than the infusion date of (b)(6).
9.   “Infused Nucleated Cell Count” ≤ “Total Infused Nucleated Cell Count” for many single unit recipients. Please clarify the difference between these variables.
10. One patient (CIBMTR ID 64552) achieved neutrophil recovery (“ANC 500” is ‘Y’), but is missing the number of days to recovery (DAYS to ANC 500).
11. Please clarify the distinction between a response of ‘N’ and ‘Never’ for the variable “ANC 500”.
12. One patient (CIBMTR ID 190555) has a response of 1122 for “Days to ANC 500” and another (CIBMTR ID 205890) has 181 days; please confirm these large number of days.
13. 15 patients have a ‘Y’ response for “Platelets 50000”, but are missing the number of days to recovery (“Days to Platelet 50,000”).
14. 16 patients have a ‘Y’ response for “Platelets 20000”, but are missing the number of days to recovery (“Days to Platelet 20,000”).
15. What does a response of “U” represent for the variable “Platelet 50,000”? 
 
Best Regards,
Lori
 
Lori A. Tull, RAC
Regulatory Project Manager
Office of Cellular, Tissue, and Gene Therapies
Center for Biologics Evaluation and Research
(301) 827-5359
 
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