Donor Umbilical Cord Blood Transplant By Injection Into the Bone Marrow in Treating Patients With Hematologic Cancer

• ClinicalTrials.gov Identifier: NCT00295880
• Recruitment Status: Terminated
• First Posted: February 24, 2006
• Results First Posted: September 3, 2009
• Last Update Posted: December 28, 2017
• Sponsor: Masonic Cancer Center, University of Minnesota
• Information provided by (Responsible Party): Masonic Cancer Center, University of Minnesota

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Conditions: Myeloproliferative Disorders; Leukemia; Lymphoma; Myelodysplastic Syndromes
• Intervention: Procedure: umbilical cord blood transplantation
• Enrollment: 12

Participant Flow

Recruitment Details	Patients are recruited from within the Department of Blood and Marrow Transplant Program. (Patients will be co-enrolled on study MT2005-10 (NCT00309842). Eligibility for this study are identical to MT2005-10.)
Pre-assignment Details

Arm/Group Title	Umbilical Cord Blood Transplant Patients
Arm/Group Description	Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
Period Title: Overall Study
Started	12
Completed	10
Not Completed	2
Reason Not Completed
IBMI unit could not be volume reduced	1
Unit randomization did not occur	1

Baseline Characteristics

Arm/Group Title		Umbilical Cord Blood Transplant Patients
Arm/Group Description		Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
Overall Number of Baseline Participants		12
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	12 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	12 100.0%
	>=65 years	0 0.0%
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	12 participants
		34 (8)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	12 participants
	Female	5 41.7%
	Male	7 58.3%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
United States	Number Analyzed	12 participants
		12

Outcome Measures

1. Primary Outcome

Title	Median Number of Days to Neutrophil Engraftment
Description	Number of days to neutrophil recovery observed in recipients of two umbilical cord blood units (UCB)administered i.v. Neutrophil recovery is defined as first of 3 consecutive days with ANC (absolute neutrophil count) greater than or equal to 500/ul.
Time Frame	Daily through Day 60 post transplant

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Umbilical Cord Blood Transplant Patients
Arm/Group Description:	Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
Overall Number of Participants Analyzed	10
Median (Full Range); Unit of Measure: Days
	21; (17 to 49)

2. Secondary Outcome

Title	Number of Patients Achieving Neutrophil Recovery
Description	Number of patients with sustained neutrophil recovery with chimerism (evidence of engraftment of both cord blood transplants) at 6 months.
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

Patients who completed treatment.

Arm/Group Title	Umbilical Cord Blood Transplant Patients
Arm/Group Description:	Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
Overall Number of Participants Analyzed	10
Measure Type: Number; Unit of Measure: Participants
	9

3. Secondary Outcome

Title	Number of Patients With Evidence of Engraftment.
Description	Number of patients who received both cord blood units and achieved sustained donor engraftment
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

1 patient was not evaluable due to graft failure

Arm/Group Title	Umbilical Cord Blood Transplant Patients
Arm/Group Description:	Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
Overall Number of Participants Analyzed	10
Measure Type: Number; Unit of Measure: Participants
	2

4. Secondary Outcome

Title	Number of Patients With Acute Graft-versus-host Disease (GVHD)
Description	Number of patients who exhibited grade II-IV acute GVHD at 100 days post umbilical cord blood transplant.
Time Frame	100 days post transplant

Outcome Measure Data

Analysis Population Description

2 patients were not graded for acute GVHD due to graft failure.

Arm/Group Title	Umbilical Cord Blood Transplant Patients
Arm/Group Description:	Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
Overall Number of Participants Analyzed	8
Measure Type: Number; Unit of Measure: Participants
	7

5. Secondary Outcome

Title	Number of Patients With Transplant-related Mortality (TRM)
Description	Number of patients who were deceased at days 100 and 180 from any cause other than relapse.
Time Frame	Day 100 and Day 180

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Umbilical Cord Blood Transplant Patients
Arm/Group Description:	Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
Overall Number of Participants Analyzed	10
Measure Type: Number; Unit of Measure: Participants
	5

6. Secondary Outcome

Title	Number of Patients Surviving at Day 100 and 1 Year.
Description	Overall survival of patients-Number of patients who were alive at Day 100 and 1 year post transplant.
Time Frame	Day 100 and 1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Umbilical Cord Blood Transplant Patients
Arm/Group Description:	Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
Overall Number of Participants Analyzed	10
Measure Type: Number; Unit of Measure: Participants
	5

7. Secondary Outcome

Title	Number of Patients With Grade III-IV Acute Graft-versus-host Disease (GVHD)
Description	Number of umbilical cord blood transplant patients developing severe GVHD at 100 days post transplant.
Time Frame	100 days post transplant

Outcome Measure Data

Analysis Population Description

2 patients were not yet graded for acute gvhd

Arm/Group Title	Umbilical Cord Blood Transplant Patients
Arm/Group Description:	Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
Overall Number of Participants Analyzed	8
Measure Type: Number; Unit of Measure: Participants
	2

8. Secondary Outcome

Title	Number of Patients With Chronic Graft-versus-host Disease (GVHD).
Description	Number of umbilical cord blood transplant patients with limited and extensive chronic GVHD.
Time Frame	1 year post transplant

Outcome Measure Data

Analysis Population Description

Only 7 patients were at risk for chronic GVHD

Arm/Group Title	Umbilical Cord Blood Transplant Patients
Arm/Group Description:	Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
Overall Number of Participants Analyzed	7
Measure Type: Number; Unit of Measure: Participants
	2

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Umbilical Cord Blood Transplant Patients
Arm/Group Description	Patients with high-risk hematologic malignancy transplanted with umbilical cord blood via direct marrow injection.
All-Cause Mortality
	Umbilical Cord Blood Transplant Patients
	Affected / at Risk (%)
Total	--/--
Serious Adverse Events
	Umbilical Cord Blood Transplant Patients
	Affected / at Risk (%)	# Events
Total	5/12 (41.67%)
Blood and lymphatic system disorders
graft failure †	1/12 (8.33%)	1
Acute GVHD Grade 3 †	2/12 (16.67%)	2
General disorders
Death *	5/12 (41.67%)	5
† Indicates events were collected by systematic assessment; * Indicates events were collected by non-systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Umbilical Cord Blood Transplant Patients
	Affected / at Risk (%)	# Events
Total	0/12 (0.00%)

Limitations and Caveats

This study was terminated early based on interim statistical analysis that indicated time to engraftment will not be improved compared to historical controls. While safe, lack of faster marrow recovery supports early discontinuation.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

• Name/Title: John E. Wagner, M.D.
• Organization: Masonic Cancer Center, University of Minnesota Blood and Marrow Transplant Program
• Phone: 612-626-2961
• EMail: wagne002@umn.edu

Publications of Results:

Brunstein CG, Barker JN, Weisdorf DJ, Defor TE, McKenna D, Chong SY, Miller JS, McGlave PB, Wagner JE. Intra-BM injection to enhance engraftment after myeloablative umbilical cord blood transplantation with two partially HLA-matched units. Bone Marrow Transplant. 2009 Jun;43(12):935-40. doi: 10.1038/bmt.2008.417. Epub 2009 Jan 12.

• Responsible Party: Masonic Cancer Center, University of Minnesota
• ClinicalTrials.gov Identifier: NCT00295880
• Other Study ID Numbers: 2004LS072; UMN-MT2004-26 ( Other Identifier: Blood and Bone Marrow Transplant Program ); UMN-0412M65789 ( Other Identifier: IRB at University of Minnesota )
• First Submitted: February 23, 2006
• First Posted: February 24, 2006
• Results First Submitted: March 25, 2009
• Results First Posted: September 3, 2009
• Last Update Posted: December 28, 2017