Trial Outcomes & Findings for Stem Cell Transplantation and T-Cell Add-Back to Treat Bone Marrow Malignancies (NCT NCT00079391)

Results Overview

The proportion of patients who develop full donor CD3+ lymphocyte chimerism by day 30. Full chimerism is defined as \>95% donor alleles by molecular profiling (Short Tandem Repeat analysis).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

50 participants

Primary outcome timeframe

Day 30

Results posted on

2015-10-26

Participant Flow

Participant milestones

Participant milestones
Measure	Bone Marrow Transplantation Using "Nexell Isolex 300i" Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. cluster of differentiation 34 (CD34) selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
Overall Study STARTED	50
Overall Study COMPLETED	49
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Bone Marrow Transplantation Using "Nexell Isolex 300i" Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. cluster of differentiation 34 (CD34) selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Stem Cell Transplantation and T-Cell Add-Back to Treat Bone Marrow Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bone Marrow Transplantation Using Nexell Isolex 300i n=50 Participants Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. CD34 selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
Age, Categorical <=18 years	4 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	46 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	33 years STANDARD_DEVIATION 10 • n=5 Participants
Sex: Female, Male Female	28 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants
Region of Enrollment United States	50 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 30

Population: Per protocol; only patients who survived to day 30 and were evaluable.

The proportion of patients who develop full donor CD3+ lymphocyte chimerism by day 30. Full chimerism is defined as \>95% donor alleles by molecular profiling (Short Tandem Repeat analysis).

Outcome measures

Outcome measures
Measure	Bone Marrow Transplantation Using Nexell Isolex 300i n=49 Participants Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. CD34 selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
The Proportion of Patients Who Develop Full Donor T Cell Chimerism at Day 30	44.9 percentage of participants

SECONDARY outcome

Timeframe: at 5 years post transplant

Kaplan Meier estimate of survival

Outcome measures

Outcome measures
Measure	Bone Marrow Transplantation Using Nexell Isolex 300i n=49 Participants Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. CD34 selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
Overall Survival	47.9 percentage of participants

SECONDARY outcome

Timeframe: at 5 years post transplant

Non relapse mortality: death without relapse Kaplan Meier estimate

Outcome measures

Outcome measures
Measure	Bone Marrow Transplantation Using Nexell Isolex 300i n=49 Participants Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. CD34 selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
Non Relapse Mortality.	21.1 percentage of participants

SECONDARY outcome

Timeframe: at 5 years post transplant

Kaplan Meier-estimate of relapse incidence

Outcome measures

Outcome measures
Measure	Bone Marrow Transplantation Using Nexell Isolex 300i n=49 Participants Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. CD34 selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
Cumulative Incidence of Relapse	39.8 percentage of participants

SECONDARY outcome

Timeframe: First 60 days

Population: Per protocol

Incidence of acute Graft versus host disease (GVHD) grades II-IV (before day 60 T cell add back) Modified "Glucksberg" grading

Outcome measures

Outcome measures
Measure	Bone Marrow Transplantation Using Nexell Isolex 300i n=49 Participants Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. CD34 selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
Acute Graft Versus Host Disease (Before Day 60 T Cell Add Back)	20 participants

SECONDARY outcome

Timeframe: First 100 days

Incidence of acute GVHD grades II-IV (before and after T cell add back) Modified Glucksberg grading

Outcome measures

Outcome measures
Measure	Bone Marrow Transplantation Using Nexell Isolex 300i n=49 Participants Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. CD34 selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
Acute GVHD Overall	39 participants

Adverse Events

Bone Marrow Transplantation Using Nexell Isolex 300i

Serious events: 25 serious events

Other events: 49 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Bone Marrow Transplantation Using Nexell Isolex 300i n=49 participants at risk Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. CD34 selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
Immune system disorders Transplant Related Death	18.4% 9/49 • Number of events 9 • Overall study
Gastrointestinal disorders Hospitalization	2.0% 1/49 • Number of events 1 • Overall study
Immune system disorders Hospitalization	4.1% 2/49 • Number of events 2 • Overall study
Blood and lymphatic system disorders Death	24.5% 12/49 • Number of events 12 • Overall study
Infections and infestations Life threatening	2.0% 1/49 • Number of events 1 • Overall study

Other adverse events

Other adverse events
Measure	Bone Marrow Transplantation Using Nexell Isolex 300i n=49 participants at risk Bone marrow stem cell transplant program to improve the outcome of allogeneic Bone Marrow Transplant for hematologic malignancies. Immunosuppression including cyclosporine will be given six days prior to transplant up to 21 days post transplant. CD34 selection and T cell depletion using Isolex 300i immuno-magnetic cell selection and immunosuppression during peri- transplant.
Blood and lymphatic system disorders Acute GVHD before day 60	20.4% 10/49 • Number of events 10 • Overall study
Blood and lymphatic system disorders Chronic GVHD	44.9% 22/49 • Number of events 22 • Overall study
Immune system disorders Hemorrhagic cystitis	8.2% 4/49 • Number of events 4 • Overall study
Immune system disorders Cytomegalovirus disease	75.5% 37/49 • Number of events 37 • Overall study
Blood and lymphatic system disorders Disease Relapse	36.7% 18/49 • Number of events 18 • Overall study
Blood and lymphatic system disorders Acute GVHD after day 60	38.8% 19/49 • Number of events 19 • Overall study

Additional Information

Minocher Battiwalla

NIH National Heart, Lung and Blood Institute

Phone: 301-402-4170

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place