Cord Blood Transplantation for Patients With Cancer

• Sponsor: University of Chicago (Other)
• Overall Status: Terminated
• CT.gov ID: NCT01359254
• Collaborator: (none)
• Enrollment: 1
• Location: 1
• Arms: 2
• Duration (Months): 26
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

The primary objective of this study is to assess the rate of engraftment with combined haploidentical-cord blood transplantation in patients with pre-existing donor specific antibodies and in those with active disease.

Condition or Disease	Intervention/Treatment	Phase
Hematological Disease	Drug: Melphalan; Drug: Fludarabine; Drug: Antithymocyte Globulin (ATG); Drug: Busulfan; Drug: Total Body Irradiation (TBI)	Phase 2

Detailed Description

Primary Objective

• To assess the rate of engraftment with combined haploidentical-cord blood transplantation in patients with pre-existing donor specific anti-HLA antibodies and in those with active disease

Secondary Objectives

• To determine the long term fate of the haplo transplant and the cord blood transplant in this setting.

• To describe the incidence and severity of acute and chronic GVHD.

Study Design

• Study Type: Interventional
• Actual Enrollment: 1 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Combined Transplantation of Unmanipulated Haploidentical and a SingleCord Blood Unit for Patients With Hematologic Malignancies
• Study Start Date: Apr 1, 2010
• Actual Primary Completion Date: Sep 1, 2011
• Actual Study Completion Date: Jun 1, 2012

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Conditioning Regimen I Arm I contains fludarabine, melphalan, and antithymocyte globulin (ATG)	Drug: Melphalan; Melphalan is given daily for 2 days, overlapping with the completion of fludarabine.; Other Names: Alkeran Sarcolysin ; Drug: Fludarabine; Fludarabine is given through the vein daily for 5 days.; Other Names: fludarabine phosphate Fludara ; Drug: Antithymocyte Globulin (ATG); ATG is given every other day for 4 days.
Experimental: Conditioning Regimen II Arm II contains fludarabine, busulfan, antithymocyte globulin (ATG), and total body irradiation (TBI).	Drug: Fludarabine; Fludarabine is given through the vein daily for 5 days.; Other Names: fludarabine phosphate Fludara ; Drug: Antithymocyte Globulin (ATG); ATG is given every other day for 4 days.; Drug: Busulfan; Busulfan is given daily for 4 days.; Other Names: Myleran Busulfex IV ; Drug: Total Body Irradiation (TBI); TBI is given twice on the last day.

Outcome Measures

Primary Outcome Measures

1. Cord Blood Engraftment by Day 100 [100 days]

   Percent of subjects with cord blood engraftment on or before day 100. Detectable cord blood engraftment should be present by day 100 in at least 50% of patients. As of 44 days post-transplant, only haploidentical donor chimerism achieved in the one patient enrolled in the Fludarabine, melphalan, and ATG arm. There were no cord cells detected for this patient.

Secondary Outcome Measures

1. Survival at Day 100 [100 days]

   Percent of subjects who are alive 100 days after the stem cell infusion

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Relapsed or refractory acute leukemia (myeloid or lymphoid)

• Acute leukemia in first remission at high-risk for recurrence

• Chronic myelogenous leukemia in accelerated phase or blast-crisis

• Chronic myelogenous leukemia in chronic phase

• Recurrent or refractory malignant lymphoma or Hodgkin lymphoma

• Chronic lymphocytic leukemia, relapsed or with poor prognostic features

• Multiple myeloma

• Myelodysplastic syndrome

• Chronic myeloproliferative disease

• Hemoglobinopathies

• Aplastic anemia

Exclusion Criteria:

• Zubrod performance status > 2

• Life expectancy is severely limited by concomitant illness

• Patients with severely decreased LVEF or impaired pulmonary function tests

• Estimated Creatinine Clearance <50 ml/min

• Serum bilirubin> 2.0 mg/dl or SGPT >3 x upper limit of normal

• Evidence of chronic active hepatitis or cirrhosis

• HIV-positive

• Patient is pregnant

• Patient or guardian not able to sign informed consent

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Chicago	Chicago	Illinois	United States	60637

Sponsors and Collaborators

• University of Chicago

Investigators

• Principal Investigator: Andrew Artz, MD, University of Chicago

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: University of Chicago
• ClinicalTrials.gov Identifier: NCT01359254
• Other Study ID Numbers: 10-020-B
• First Posted: May 24, 2011
• Last Update Posted: Apr 30, 2015
• Last Verified: Apr 1, 2015

• Additional relevant MeSH terms: Hematologic Diseases; Vidarabine; Fludarabine; Fludarabine phosphate; Melphalan; Busulfan; Antilymphocyte Serum

Study Results

Participant Flow

Arm/Group Title	Fludarabine, Melphalan, and ATG	Fludarabine, Busulfan, ATG, and TBI
Arm/Group Description	Arm I contains fludarabine, melphalan, and antithymocyte globulin (ATG) Melphalan: Melphalan is given daily for 2 days, overlapping with the completion of fludarabine. Fludarabine: Fludarabine is given through the vein daily for 5 days. Antithymocyte Globulin (ATG): ATG is given every other day for 4 days.	Arm II contains fludarabine, busulfan, antithymocyte globulin (ATG), and total body irradiation (TBI). Fludarabine: Fludarabine is given through the vein daily for 5 days. Antithymocyte Globulin (ATG): ATG is given every other day for 4 days. Busulfan: Busulfan is given daily for 4 days. Total Body Irradiation (TBI): TBI is given twice on the last day.
Period Title: Overall Study
STARTED	1	0
COMPLETED	0	0
NOT COMPLETED	1	0

Baseline Characteristics

Arm/Group Title	Fludarabine, Melphalan, and ATG	Fludarabine, Busulfan, ATG, and TBI	Total
Arm/Group Description	Arm I contains fludarabine, melphalan, and antithymocyte globulin (ATG) Melphalan: Melphalan is given daily for 2 days, overlapping with the completion of fludarabine. Fludarabine: Fludarabine is given through the vein daily for 5 days. Antithymocyte Globulin (ATG): ATG is given every other day for 4 days.	Arm II contains fludarabine, busulfan, antithymocyte globulin (ATG), and total body irradiation (TBI). Fludarabine: Fludarabine is given through the vein daily for 5 days. Antithymocyte Globulin (ATG): ATG is given every other day for 4 days. Busulfan: Busulfan is given daily for 4 days. Total Body Irradiation (TBI): TBI is given twice on the last day.	Total of all reporting groups
Overall Participants	1	0	1
Age (participants) [Number]
<=18 years	0 0%	0 NaN
Between 18 and 65 years	1 100%	1 Infinity
>=65 years	0 0%	0 NaN
Gender (participants) [Number]
Female	0 0%	0 NaN
Male	1 100%	1 Infinity

Outcome Measures

1. Primary Outcome

Title	Cord Blood Engraftment by Day 100
Description	Percent of subjects with cord blood engraftment on or before day 100. Detectable cord blood engraftment should be present by day 100 in at least 50% of patients. As of 44 days post-transplant, only haploidentical donor chimerism achieved in the one patient enrolled in the Fludarabine, melphalan, and ATG arm. There were no cord cells detected for this patient.
Time Frame	100 days

Outcome Measure Data

Analysis Population Description

The only enrolled patient failed to complete the study due to death.

Arm/Group Title	Fludarabine, Melphalan, and ATG	Fludarabine, Busulfan, ATG, and TBI
Arm/Group Description	Arm I contains fludarabine, melphalan, and antithymocyte globulin (ATG) Melphalan: Melphalan is given daily for 2 days, overlapping with the completion of fludarabine. Fludarabine: Fludarabine is given through the vein daily for 5 days. Antithymocyte Globulin (ATG): ATG is given every other day for 4 days.	Arm II contains fludarabine, busulfan, antithymocyte globulin (ATG), and total body irradiation (TBI). Fludarabine: Fludarabine is given through the vein daily for 5 days. Antithymocyte Globulin (ATG): ATG is given every other day for 4 days. Busulfan: Busulfan is given daily for 4 days. Total Body Irradiation (TBI): TBI is given twice on the last day.
Measure Participants	0	0

2. Secondary Outcome

Title	Survival at Day 100
Description	Percent of subjects who are alive 100 days after the stem cell infusion
Time Frame	100 days

Outcome Measure Data

Analysis Population Description

The only enrolled patient failed to complete the study due to death.

Arm/Group Title	Fludarabine, Melphalan, and ATG	Fludarabine, Busulfan, ATG, and TBI
Arm/Group Description	Arm I contains fludarabine, melphalan, and antithymocyte globulin (ATG) Melphalan: Melphalan is given daily for 2 days, overlapping with the completion of fludarabine. Fludarabine: Fludarabine is given through the vein daily for 5 days. Antithymocyte Globulin (ATG): ATG is given every other day for 4 days.	Arm II contains fludarabine, busulfan, antithymocyte globulin (ATG), and total body irradiation (TBI). Fludarabine: Fludarabine is given through the vein daily for 5 days. Antithymocyte Globulin (ATG): ATG is given every other day for 4 days. Busulfan: Busulfan is given daily for 4 days. Total Body Irradiation (TBI): TBI is given twice on the last day.
Measure Participants	0	0

Adverse Events

Time Frame	100 days
Arm/Group Title	Fludarabine, Melphalan, and ATG		Fludarabine, Busulfan, ATG, and TBI
Arm/Group Description	Arm I contains fludarabine, melphalan, and antithymocyte globulin (ATG) Melphalan: Melphalan is given daily for 2 days, overlapping with the completion of fludarabine. Fludarabine: Fludarabine is given through the vein daily for 5 days. Antithymocyte Globulin (ATG): ATG is given every other day for 4 days.		Arm II contains fludarabine, busulfan, antithymocyte globulin (ATG), and total body irradiation (TBI). Fludarabine: Fludarabine is given through the vein daily for 5 days. Antithymocyte Globulin (ATG): ATG is given every other day for 4 days. Busulfan: Busulfan is given daily for 4 days. Total Body Irradiation (TBI): TBI is given twice on the last day.
All Cause Mortality
	Fludarabine, Melphalan, and ATG		Fludarabine, Busulfan, ATG, and TBI
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	Fludarabine, Melphalan, and ATG		Fludarabine, Busulfan, ATG, and TBI
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/1 (100%)		0/0 (NaN)
General disorders
Septic shock	1/1 (100%)		0/0 (NaN)
Renal and urinary disorders
Renal failure	1/1 (100%)		0/0 (NaN)
Other (Not Including Serious) Adverse Events
	Fludarabine, Melphalan, and ATG		Fludarabine, Busulfan, ATG, and TBI
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/1 (100%)		0/0 (NaN)
General disorders
Acute graft versus host disease	1/1 (100%)		0/0 (NaN)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE 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.