Trial Outcomes & Findings for Stem Cell Transplantation for Patients With Cancers of the Blood (NCT NCT00467961)

Last Updated: 2018-07-13

Results Overview

Patients will receive a selectively photodepleted lymphocyte product which will be delivered together with the T cell depleted stem cell product on the day of transplantation. Subjects will receive a conditioning regimen of cyclophosphamide, fludarabine and total body irradiation followed by an infusion of a stem cell product prepared using the Miltenyi CliniMacs system for CD34-selection and a lymphocyte product that has been selectively depleted using the photodepletion approach. Older subjects will receive a lower dose of irradiation to reduce the regimen intensity. To determine appropriate level of post transplant immunosuppression, a three sequential de-escalation stage design for timing of cyclosporine will be utilized. To determine if selective T cell depletion using the photodepletion procedure can substantially reduce the number of severe acute GVHD (grade III/IV) after transplantation followed by low-dose or no immunosuppression.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

61 participants

Primary outcome timeframe

Day 90

Results posted on

2018-07-13

Participant Flow

Participant milestones

Participant milestones
Measure	Selective T Cell Depletion Transplant Recipients Subjects will receive a myeloablative conditioning regimen of cyclophosphamide, fludarabine and total body irradiation, followed by an infusion of a stem cell product prepared using the Miltenyi CliniMacs system for CD34 selection and a lymphocyte product that has been selectively depleted using the photodepletion approach. Older subjects will receive a lower dose of irradiation to reduce the regimen intensity. To determine appropriate level of post transplant immunosuppression, a three sequential de-escalation stage design for timing of cyclosporine will be utilized.
Overall Study STARTED	31
Overall Study COMPLETED	24
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Reasons for withdrawal
Measure	Selective T Cell Depletion Transplant Recipients Subjects will receive a myeloablative conditioning regimen of cyclophosphamide, fludarabine and total body irradiation, followed by an infusion of a stem cell product prepared using the Miltenyi CliniMacs system for CD34 selection and a lymphocyte product that has been selectively depleted using the photodepletion approach. Older subjects will receive a lower dose of irradiation to reduce the regimen intensity. To determine appropriate level of post transplant immunosuppression, a three sequential de-escalation stage design for timing of cyclosporine will be utilized.
Overall Study Technical Failures	5
Overall Study Disease Progression	2

Baseline Characteristics

Stem Cell Transplantation for Patients With Cancers of the Blood

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Selective T Cell Depletion Transplant Recipients n=31 Participants Subjects will receive a myeloablative conditioning regimen of cyclophosphamide, fludarabine and total body irradiation, followed by an infusion of a stem cell product prepared using the Miltenyi CliniMacs system for CD34 selection and a lymphocyte product that has been selectively depleted using the photodepletion approach. Older subjects will receive a lower dose of irradiation to reduce the regimen intensity. To determine appropriate level of post transplant immunosuppression, a three sequential de-escalation stage design for timing of cyclosporine will be utilized.
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	15 Participants n=5 Participants
Region of Enrollment United States	31 participants n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	28 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 90

Population: The study accrued 31 transplant recipents and 30 donors. Of the 31 transplant recipients, there were 24 evaluable recipients. Seven of the 24 recipients did not receive transplantation.

Outcome measures

Outcome measures
Measure	Selective T Cell Depletion Transplant Recipients n=24 Participants Subjects will receive a myeloablative conditioning regimen of cyclophosphamide, fludarabine and total body irradiation, followed by an infusion of a stem cell product prepared using the Miltenyi CliniMacs system for CD34 selection and a lymphocyte product that has been selectively depleted using the photodepletion approach (Kiadis Pharma). Older subjects will receive a lower dose of irradiation to reduce the regimen intensity. To determine appropriate level of post transplant immunosuppression.
To Determine if Selective T Cell Depletion Using the Photodepletion Procedure Can Substantially Reduce the Rate of Severe Acute GVHD (Grade III/IV) After Matched Sibling Transplantation Followed by Low-dose or no Immunosuppression. Subjects affected by Acute Grade III/ IV GVHD	3 participants
To Determine if Selective T Cell Depletion Using the Photodepletion Procedure Can Substantially Reduce the Rate of Severe Acute GVHD (Grade III/IV) After Matched Sibling Transplantation Followed by Low-dose or no Immunosuppression. Subjects unaffected by Acute Grade III/ IV GVHD	21 participants

Adverse Events

Selective T Cell Depletion Transplant Recipients

Serious events: 24 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Selective T Cell Depletion Transplant Recipients n=31 participants at risk Subjects will receive a myeloablative conditioning regimen of cyclophosphamide, fludarabine and total body irradiation, followed by an infusion of a stem cell product prepared using the Miltenyi CliniMacs system for CD34 selection and a lymphocyte product that has been selectively depleted using the photodepletion approach. Older subjects will receive a lower dose of irradiation to reduce the regimen intensity. To determine appropriate level of post transplant immunosuppression, a three sequential de-escalation stage design for timing of cyclosporine will be utilized.
Infections and infestations Infection with normal absolute nuetrophil count	9.7% 3/31 • Number of events 3
Infections and infestations Infection	38.7% 12/31 • Number of events 12
Infections and infestations Infection with cytomegalovirus reactivation	9.7% 3/31 • Number of events 3
Infections and infestations Febrile neutropenia	3.2% 1/31 • Number of events 1
Infections and infestations Infection with neutropenic fever	22.6% 7/31 • Number of events 7
Infections and infestations Infection cytomegalovirus gastritis	3.2% 1/31 • Number of events 1
Infections and infestations pulmonary infection	3.2% 1/31 • Number of events 1
Infections and infestations renal infection	3.2% 1/31 • Number of events 1
Infections and infestations multiple opportunistic infection	3.2% 1/31 • Number of events 1
Immune system disorders GVHD	19.4% 6/31 • Number of events 6
Immune system disorders GVHD/ infection	3.2% 1/31 • Number of events 1
Immune system disorders GVHD/ gastrointestinal	9.7% 3/31 • Number of events 3
Nervous system disorders GVHD/ neurology	3.2% 1/31 • Number of events 1
Immune system disorders GVHD/ pulmonary	3.2% 1/31 • Number of events 1
Immune system disorders GVHD/ cytomegalovirus reactivation	3.2% 1/31 • Number of events 1
Blood and lymphatic system disorders Disease progression/ Infection	3.2% 1/31 • Number of events 1
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Adbdominal mantle cell lymphoma	3.2% 1/31 • Number of events 1
Blood and lymphatic system disorders Disease relapse	19.4% 6/31 • Number of events 6

Other adverse events

Adverse event data not reported

Additional Information

Minocher Battiwalla, MD

NIH NHLBI

Phone: 301-827-0939

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place