Prospective Cohort for the Evaluation of Biomarkers Following HCT (BMT CTN 1202)

• Sponsor: Medical College of Wisconsin (Other)
• Overall Status: Completed
• CT.gov ID: NCT01879072
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI) (NIH), National Cancer Institute (NCI) (NIH), Blood and Marrow Transplant Clinical Trials Network (Other), National Marrow Donor Program (Other)
• Enrollment: 1,860
• Locations: 42
• Actual Duration (Months): 39
• Patients Per Site: 44.3
• Patients Per Site Per Month: 1.1

Study Details

Study Description

Brief Summary

The goal of this protocol is to establish a cohort of at least 1500 biologic samples collected prospectively from patients treated in BMT CTN centers that will be a shared bio specimen resource for conducting future allogeneic hematopoietic stem cell transplantation (HCT) correlative studies.

Condition or Disease	Intervention/Treatment	Phase
Allogeneic Hematopoietic Stem Cell Transplantation

Detailed Description

The goal of this protocol is to establish a cohort of biologic samples collected prospectively from patients treated in BMT CTN centers that will be a shared bio specimen resource for conducting future allogeneic HCT correlative studies. This resource is designed to allow genomic, proteomic and transcriptional data to be integrated with high quality clinical phenotype and outcomes data to identify risk factors for development and severity of acute GVHD, chronic GVHD, organ toxicity, relapse, mortality, infection and other clinically significant complications occurring after allogeneic HCT.

To achieve this goal, patients and donors will be recruited and consent obtained at the time that they enroll on BMT CTN protocols where enrollment occurs at or before transplantation or prior to start of conditioning for patients enrolled on non-BMT CTN studies or treated as standard of care. Samples will be collected: (1) from patients and donors pre-transplant; and, (2) from patients post-transplant on a calendar schedule through the first 3 months post-HCT. For patients co-enrolled on BMT CTN studies, clinical data will be collected in the context of the primary transplant protocols. For patients not enrolled on BMT CTN protocols, clinical data on early post-transplant events will be collected using the same data collection forms and systems that are used on BMT CTN trials. Additional clinical data for both BMT CTN and non-BMT CTN patients will be available from data submitted to the Center for International Blood and Marrow Transplant Research (CIBMTR) using the CIBMTR Comprehensive Report Forms. This protocol also leverages ongoing pre-transplant donor-recipient sample collection performed by the CIBMTR and National Marrow Donor Program (NMDP). Success in establishing this shared resource will inspire future investigator initiated research proposals and will allow investigators to take advantage of National Institutes of Health (NIH) funding initiatives.

Study Design

• Study Type: Observational [Patient Registry]
• Actual Enrollment: 1860 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Prospective Multi-Center Cohort for the Evaluation of Biomarkers Predicting Risk of Complications and Mortality Following Allogeneic HCT (BMT CTN #1202)
• Actual Study Start Date: Jun 1, 2013
• Actual Primary Completion Date: Sep 1, 2016
• Actual Study Completion Date: Sep 1, 2016

Outcome Measures

Primary Outcome Measures

1. Number of Participants Providing Biologic Samples [Two years from hematopoietic stem cell transplant]

   The primary outcome will be measured by the number of participants who supply biologic samples. The prospectively collected samples will be a shared bio specimen resource for conducting future correlative studies.

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Recipients of first allogeneic hematopoietic cell transplants that are transplanted in U.S. centers that participate in the NMDP/CIBMTR's "Protocol for a Research Sample Repository for Allogeneic Hematopoietic Stem Cell Transplantation and Marrow Toxic Injuries" and receive a cord blood graft or receive a bone marrow or peripheral blood graft from a related donor or from an unrelated donor in an NMDP-affiliated Donor Center or Registry participating in that same protocol.

This transplant and donor center restriction is to allow linkage with pretransplant donor specimens collected under the NMDP/CIBMTR protocol. Current data indicate that

90% of donors approached under this protocol agree to provide samples

1. Patients with any malignant or non-malignant hematologic disorder will be eligible for enrollment on this protocol. A subset of 240 sequential patients with acute leukemia in first or second remission will also provide research samples for gene expression studies.

2. Children may participate in this study but must weigh at least 20 kilograms given the volume (100ml) and number of blood draws during this study. Subjects must weigh at least 30 kg to provide research samples for gene expression studies (additional 40 ml).

3. All participants or parent/legal guardian must sign an informed consent for this study.

Because studies using this resource will require linking with clinical data collected by CIBMTR, all participants or parent/legal guardian must also consent to participate in "Protocol for a Research Database for Hematopoietic Cell Transplantation and Marrow Toxic Injuries".

Exclusion Criteria:

• N/A

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Phoenix Children's Hospital	Phoenix	Arizona	United States	85016
2	City of Hope National Medical Center	Duarte	California	United States	91010
3	Children's Hospital at Oakland	Oakland	California	United States	94609
4	University of CA, SF	San Francisco	California	United States	94143
5	Stanford Hospitals and Clinics	Stanford	California	United States	94305
6	Nemours/Alfred I. duPont Hospital for Children	Wilmington	Delaware	United States	19803
7	Children's National Medical Center	Washington	District of Columbia	United States	20010
8	University of Florida College of Medicine (Shands)	Gainesville	Florida	United States	32610
9	All Children's Hospital	Saint Petersburg	Florida	United States	33701
10	H. Lee Moffitt Cancer Center	Tampa	Florida	United States	33624
11	Children's Healthcare of Atlanta	Atlanta	Georgia	United States	30322
12	Emory University	Atlanta	Georgia	United States	30322
13	BMT at Northside Hospital	Atlanta	Georgia	United States	30342
14	Ann and Robert H. Lurie Children's Hospital	Chicago	Illinois	United States	60611
15	University of Kansas Hospital	Kansas City	Kansas	United States	66160
16	Johns Hopkins	Baltimore	Maryland	United States	21231
17	DFCI/Brigham & Women's	Boston	Massachusetts	United States	02115
18	University of Michigan Medical Center	Ann Arbor	Michigan	United States	48105-2967
19	University of Minnesota	Minneapolis	Minnesota	United States	55455
20	Mayo Clinic - Rochester	Rochester	Minnesota	United States	55905
21	Washington University/Barnes Jewish Hospital	Saint Louis	Missouri	United States	63110
22	Washington University/St. Louis Children's Hospital	Saint Louis	Missouri	United States	63110
23	University of Nebraska Medical Center	Omaha	Nebraska	United States	68198
24	Hackensack University Medical Center	Hackensack	New Jersey	United States	07601
25	Roswell Park	Buffalo	New York	United States	14263
26	Mount Sinai Medical Center	New York	New York	United States	10029
27	Memorial Sloan-Kettering Cancer Center	New York	New York	United States	10065
28	University of North Carolina Hospital at Chapel Hill	Chapel Hill	North Carolina	United States	27599
29	Duke University	Durham	North Carolina	United States	27705
30	University Hospitals of Cleveland/Case Western	Cleveland	Ohio	United States	44106
31	Cleveland Clinic Foundation	Cleveland	Ohio	United States	44195
32	Ohio State/Arthur G. James Cancer Hospital	Columbus	Ohio	United States	43210
33	Oregon Health & Science University	Portland	Oregon	United States	97239
34	University of Pennsylvania Cancer Center	Philadelphia	Pennsylvania	United States	19104
35	Children's Medical Center of Dallas	Dallas	Texas	United States	75235
36	Cook Children's Hospital	Fort Worth	Texas	United States	76104
37	Baylor College of Medicine/The Methodist Hospital	Houston	Texas	United States	77030
38	University of Texas/MD Anderson CRC	Houston	Texas	United States	77030
39	Texas Transplant Institute	San Antonio	Texas	United States	78229
40	Utah BMT/Primary Children's Medical Center	Salt Lake City	Utah	United States	84132
41	Virginia Commonwealth University	Richmond	Virginia	United States	23298
42	Fred Hutchinson Cancer Research Center	Seattle	Washington	United States	98109

Sponsors and Collaborators

• Medical College of Wisconsin
• National Heart, Lung, and Blood Institute (NHLBI)
• National Cancer Institute (NCI)
• Blood and Marrow Transplant Clinical Trials Network
• National Marrow Donor Program

Investigators

• Study Director: Mary Horowitz, MD, MS, Center for International Blood and Marrow Transplant Research (CIBMTR), Medical College of Wisconsin

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Blood and Marrow Transplant Clinical Trials Network Website
• National Marrow Donor Program

Publications

None provided.

• Responsible Party: Medical College of Wisconsin
• ClinicalTrials.gov Identifier: NCT01879072
• Other Study ID Numbers: BMTCTN1202; U10HL069294-11; 5U24CA076518
• First Posted: Jun 17, 2013
• Last Update Posted: Dec 31, 2018
• Last Verified: Dec 1, 2018

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Keywords provided by Medical College of Wisconsin: Biomarkers; Hematopoietic stem cell transplant

Study Results

Participant Flow

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Participants received allogeneic hematopoietic stem cell transplantation
Period Title: Overall Study
STARTED	1860
COMPLETED	1709
NOT COMPLETED	151

Baseline Characteristics

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Participants received allogeneic hematopoietic stem cell transplantation
Overall Participants	1709
Age (years) [Median (Full Range) ]
Median (Full Range) [years]	52.7
Sex: Female, Male (Count of Participants)
Female	701 41%
Male	1008 59%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	128 7.5%
Not Hispanic or Latino	1552 90.8%
Unknown or Not Reported	29 1.7%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	9 0.5%
Asian	59 3.5%
Native Hawaiian or Other Pacific Islander	5 0.3%
Black or African American	203 11.9%
White	1370 80.2%
More than one race	13 0.8%
Unknown or Not Reported	50 2.9%

Outcome Measures

1. Primary Outcome

Title	Number of Participants Providing Biologic Samples
Description	The primary outcome will be measured by the number of participants who supply biologic samples. The prospectively collected samples will be a shared bio specimen resource for conducting future correlative studies.
Time Frame	Two years from hematopoietic stem cell transplant

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Allogeneic Hematopoietic Stem Cell Transplant
Arm/Group Description	Participants received allogeneic hematopoietic stem cell transplantation
Measure Participants	1709
Count of Participants [Participants]	1709 100%

Adverse Events

Time Frame	Two years post-transplant
Arm/Group Title	Hematopoietic Stem Cell Transplant
Arm/Group Description
All Cause Mortality
	Hematopoietic Stem Cell Transplant
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Hematopoietic Stem Cell Transplant
	Affected / at Risk (%)	# Events
Total	0/1709 (0%)
Other (Not Including Serious) Adverse Events
	Hematopoietic Stem Cell Transplant
	Affected / at Risk (%)	# Events
Total	0/1709 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.