Trial Outcomes & Findings for T-Cell Depletion, Donor Hematopoietic Stem Cell Transplant (HSCT), and T-Cell Infusions in Treating Patients With Hematologic Cancer or Other Diseases (NCT NCT00589602)

Last Updated: 2017-05-30

Results Overview

The complication rate in matched unrelated donor (MUD) allogeneic bone marrow transplant (allo BMT) is known to be high. Graft failure and severe graft versus host disease (GvHD) are the most significant contributors to treatment related mortality (TRM). This treatment regimen will be considered unacceptable if the number of patients that experience TRM is 55% or greater, and effective if TRM is 33% or less.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

13 participants

Primary outcome timeframe

180 days after transplant

Results posted on

2017-05-30

Participant Flow

Patients were recruited from local hospital from January, 2006 through January, 2009.

Participant milestones

Participant milestones
Measure	T-Cell Depletion Transplant Our protocol is designed to attempt to improve the current results of MUD allo BMT and will be a major step towards the introduction and refinement of graft engineering. Our approach will address in a rational fashion all major technical and clinical aspects of MUD allo BMT. Peripheral blood lymphocyte therapy; cyclophosphamide, tacrolimus, peripheral blood stem cell transplantation; total-body irradiation; 'allogeneic hematopoietic stem cell transplantation' peripheral blood lymphocyte therapy: T-cell depletion cyclophosphamide: T-cell depletion tacrolimus: T-cell depletion allogeneic hematopoietic stem cell transplantation: T-cell depletion peripheral blood stem cell transplantation: T-cell depletion total-body irradiation: T-cell depletion
Overall Study STARTED	13
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	T-Cell Depletion Transplant Our protocol is designed to attempt to improve the current results of MUD allo BMT and will be a major step towards the introduction and refinement of graft engineering. Our approach will address in a rational fashion all major technical and clinical aspects of MUD allo BMT. Peripheral blood lymphocyte therapy; cyclophosphamide, tacrolimus, peripheral blood stem cell transplantation; total-body irradiation; 'allogeneic hematopoietic stem cell transplantation' peripheral blood lymphocyte therapy: T-cell depletion cyclophosphamide: T-cell depletion tacrolimus: T-cell depletion allogeneic hematopoietic stem cell transplantation: T-cell depletion peripheral blood stem cell transplantation: T-cell depletion total-body irradiation: T-cell depletion
Overall Study Did not collect enough CD34+ cells	1

Baseline Characteristics

T-Cell Depletion, Donor Hematopoietic Stem Cell Transplant (HSCT), and T-Cell Infusions in Treating Patients With Hematologic Cancer or Other Diseases

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	T-Cell Depletion Transplant n=13 Participants Our protocol is designed to attempt to improve the current results of MUD allo BMT and will be a major step towards the introduction and refinement of graft engineering. Our approach will address in a rational fashion all major technical and clinical aspects of MUD allo BMT. Peripheral blood lymphocyte therapy; cyclophosphamide, tacrolimus, peripheral blood stem cell transplantation; total-body irradiation; 'allogeneic hematopoietic stem cell transplantation' peripheral blood lymphocyte therapy: T-cell depletion cyclophosphamide: T-cell depletion tacrolimus: T-cell depletion allogeneic hematopoietic stem cell transplantation: T-cell depletion peripheral blood stem cell transplantation: T-cell depletion total-body irradiation: T-cell depletion
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	12 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	46 years n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Region of Enrollment United States	13 participants n=5 Participants

PRIMARY outcome

Timeframe: 180 days after transplant

Population: Patients that received treatment

Outcome measures

Outcome measures
Measure	T-Cell Depletion Transplant n=12 Participants Our protocol is designed to attempt to improve the current results of MUD allo BMT and will be a major step towards the introduction and refinement of graft engineering. Our approach will address in a rational fashion all major technical and clinical aspects of MUD allo BMT. Peripheral blood lymphocyte therapy; cyclophosphamide, tacrolimus, peripheral blood stem cell transplantation; total-body irradiation; 'allogeneic hematopoietic stem cell transplantation' peripheral blood lymphocyte therapy: T-cell depletion cyclophosphamide: T-cell depletion tacrolimus: T-cell depletion allogeneic hematopoietic stem cell transplantation: T-cell depletion peripheral blood stem cell transplantation: T-cell depletion total-body irradiation: T-cell depletion
Treatment-related Mortality (TRM)	8 participants

SECONDARY outcome

Timeframe: D+100 from transplant

Outcome measures

Outcome measures
Measure	T-Cell Depletion Transplant n=12 Participants Our protocol is designed to attempt to improve the current results of MUD allo BMT and will be a major step towards the introduction and refinement of graft engineering. Our approach will address in a rational fashion all major technical and clinical aspects of MUD allo BMT. Peripheral blood lymphocyte therapy; cyclophosphamide, tacrolimus, peripheral blood stem cell transplantation; total-body irradiation; 'allogeneic hematopoietic stem cell transplantation' peripheral blood lymphocyte therapy: T-cell depletion cyclophosphamide: T-cell depletion tacrolimus: T-cell depletion allogeneic hematopoietic stem cell transplantation: T-cell depletion peripheral blood stem cell transplantation: T-cell depletion total-body irradiation: T-cell depletion
The Rate of Acute Graft Versus Host Disease (GVHD)	11 participants

SECONDARY outcome

Timeframe: D+100 from transplant

Outcome measures

Outcome measures
Measure	T-Cell Depletion Transplant n=12 Participants Our protocol is designed to attempt to improve the current results of MUD allo BMT and will be a major step towards the introduction and refinement of graft engineering. Our approach will address in a rational fashion all major technical and clinical aspects of MUD allo BMT. Peripheral blood lymphocyte therapy; cyclophosphamide, tacrolimus, peripheral blood stem cell transplantation; total-body irradiation; 'allogeneic hematopoietic stem cell transplantation' peripheral blood lymphocyte therapy: T-cell depletion cyclophosphamide: T-cell depletion tacrolimus: T-cell depletion allogeneic hematopoietic stem cell transplantation: T-cell depletion peripheral blood stem cell transplantation: T-cell depletion total-body irradiation: T-cell depletion
Number of Participants With Duration of Absolute Neutropenia	10 participants

SECONDARY outcome

Timeframe: through D+100

Patients receive defined doses of donor T cells by IV infusion on days 45 and 100, in absence of active graft-versus-host disease (GVHD) requiring steroids.

Outcome measures

Outcome measures
Measure	T-Cell Depletion Transplant n=12 Participants Our protocol is designed to attempt to improve the current results of MUD allo BMT and will be a major step towards the introduction and refinement of graft engineering. Our approach will address in a rational fashion all major technical and clinical aspects of MUD allo BMT. Peripheral blood lymphocyte therapy; cyclophosphamide, tacrolimus, peripheral blood stem cell transplantation; total-body irradiation; 'allogeneic hematopoietic stem cell transplantation' peripheral blood lymphocyte therapy: T-cell depletion cyclophosphamide: T-cell depletion tacrolimus: T-cell depletion allogeneic hematopoietic stem cell transplantation: T-cell depletion peripheral blood stem cell transplantation: T-cell depletion total-body irradiation: T-cell depletion
Number of Participants Able to Receive T-cell Add Backs	3 participants

SECONDARY outcome

Timeframe: after 7 years of follow up

Population: All patients that received treatment

number of patients that were still alive and relapse free

Outcome measures

Outcome measures
Measure	T-Cell Depletion Transplant n=12 Participants Our protocol is designed to attempt to improve the current results of MUD allo BMT and will be a major step towards the introduction and refinement of graft engineering. Our approach will address in a rational fashion all major technical and clinical aspects of MUD allo BMT. Peripheral blood lymphocyte therapy; cyclophosphamide, tacrolimus, peripheral blood stem cell transplantation; total-body irradiation; 'allogeneic hematopoietic stem cell transplantation' peripheral blood lymphocyte therapy: T-cell depletion cyclophosphamide: T-cell depletion tacrolimus: T-cell depletion allogeneic hematopoietic stem cell transplantation: T-cell depletion peripheral blood stem cell transplantation: T-cell depletion total-body irradiation: T-cell depletion
Number of Participants With Relapse-free Survival	2 participants

Adverse Events

T-Cell Depletion Transplant

Serious events: 9 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	T-Cell Depletion Transplant n=12 participants at risk peripheral blood lymphocyte therapy: T-cell depletion cyclophosphamide: T-cell depletion tacrolimus: T-cell depletion allogeneic hematopoietic stem cell transplantation: T-cell depletion peripheral blood stem cell transplantation: T-cell depletion total-body irradiation: T-cell depletion
General disorders Death by Relapse	33.3% 4/12 • Number of events 4 • Adverse events data was over the course of the study for approximately 7 years
General disorders Death from Acute GVHD	25.0% 3/12 • Number of events 3 • Adverse events data was over the course of the study for approximately 7 years
Respiratory, thoracic and mediastinal disorders Death due to respiratory failure	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Infections and infestations Death due to Infection	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years

Other adverse events

Other adverse events
Measure	T-Cell Depletion Transplant n=12 participants at risk peripheral blood lymphocyte therapy: T-cell depletion cyclophosphamide: T-cell depletion tacrolimus: T-cell depletion allogeneic hematopoietic stem cell transplantation: T-cell depletion peripheral blood stem cell transplantation: T-cell depletion total-body irradiation: T-cell depletion
General disorders Hospitalization for fever	58.3% 7/12 • Number of events 24 • Adverse events data was over the course of the study for approximately 7 years
Endocrine disorders Hospitalized with hypoglycemia	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
General disorders Hospitalized with GVHD	58.3% 7/12 • Number of events 11 • Adverse events data was over the course of the study for approximately 7 years
Nervous system disorders Hospitalized with mental status changes	16.7% 2/12 • Number of events 2 • Adverse events data was over the course of the study for approximately 7 years
Infections and infestations Hospitalized with Infection	75.0% 9/12 • Number of events 38 • Adverse events data was over the course of the study for approximately 7 years
General disorders Hospitalized for Nausae, Vomiting and Diarrhea	41.7% 5/12 • Number of events 22 • Adverse events data was over the course of the study for approximately 7 years
Respiratory, thoracic and mediastinal disorders Hospitalized for respiratory distress	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
General disorders Hospitalized for broken leg	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Respiratory, thoracic and mediastinal disorders Hospitalized for shortness of breath	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
General disorders Hospitalized for hypertension	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Cardiac disorders Hospitalized for chest pains	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
General disorders Hospitalized for Syncope and dizziness	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Gastrointestinal disorders Hospitalized for abdominal pain	16.7% 2/12 • Number of events 4 • Adverse events data was over the course of the study for approximately 7 years
General disorders Hospitalized for puritus and plolyarthagias	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Respiratory, thoracic and mediastinal disorders Hospitalized for pulmonary embolism	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Infections and infestations Hospitalized for abscess	16.7% 2/12 • Number of events 2 • Adverse events data was over the course of the study for approximately 7 years
Gastrointestinal disorders Hospitalized for blood in stool and	8.3% 1/12 • Number of events 2 • Adverse events data was over the course of the study for approximately 7 years
Blood and lymphatic system disorders Hospitalized for TTP (Thrombotic Thrombocytopenic Purpura)	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Musculoskeletal and connective tissue disorders Hospitalized for muscle spasms	8.3% 1/12 • Number of events 2 • Adverse events data was over the course of the study for approximately 7 years
General disorders Hospitalized for relapse of disease	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Injury, poisoning and procedural complications Hospitalized for a motor vehicle accident	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Musculoskeletal and connective tissue disorders Hospitalized for necrosis of knees	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Surgical and medical procedures Hospitalized for knee replacement	8.3% 1/12 • Number of events 2 • Adverse events data was over the course of the study for approximately 7 years
General disorders Hospitalized for numbness in hands and feet	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Surgical and medical procedures Hospitalized for surgery medial epicondylitis	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Skin and subcutaneous tissue disorders Hospitalized for mucocitus and rash	8.3% 1/12 • Number of events 2 • Adverse events data was over the course of the study for approximately 7 years
Gastrointestinal disorders Constipation	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years
Renal and urinary disorders Hospitalized for dysuria	8.3% 1/12 • Number of events 1 • Adverse events data was over the course of the study for approximately 7 years

Additional Information

Jarek Maciejewski, MD

Cleveland Clinic

Phone: 216-445-5962

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place