Trial Outcomes & Findings for Non-Myeloablative Allogeneic HSCT From HLA Matched Related or Unrelated Donors for the Treatment of Low Grade B Cell Malignancies (NCT NCT00714259)

Results Overview

subjects surviving without disease progression at 100 days after transplant as evidenced by decreased disease and no new disease showing on radiologic scans and / or bone marrow pathology.

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

5 participants

Primary outcome timeframe

100 days post transplant

Results posted on

2017-07-24

Participant Flow

Participant milestones

Participant milestones
Measure	Single Arm Trial Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Overall Study STARTED	5
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Single Arm Trial Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Overall Study Death	2

Baseline Characteristics

Non-Myeloablative Allogeneic HSCT From HLA Matched Related or Unrelated Donors for the Treatment of Low Grade B Cell Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Single Arm Trial n=5 Participants Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: 100 days post transplant

subjects surviving without disease progression at 100 days after transplant as evidenced by decreased disease and no new disease showing on radiologic scans and / or bone marrow pathology.

Outcome measures

Outcome measures
Measure	Single Arm Trial n=5 Participants Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Progressive Free Survival Post Transplant	4 participants

PRIMARY outcome

Timeframe: 180 days post transplant

Subjects surviving without disease progression 180 days after transplant as evidenced by decreased disease and no new disease showing on radiologic scans and / or bone marrow pathology.

Outcome measures

Outcome measures
Measure	Single Arm Trial n=4 Participants Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Progressive Free Survival Post Transplant	4 participants

PRIMARY outcome

Timeframe: 365 days post transplant

Subjects surviving without disease progression 365 days after transplant as evidenced by decreased disease and no new disease showing on radiologic scans and / or bone marrow pathology.

Outcome measures

Outcome measures
Measure	Single Arm Trial n=4 Participants Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Progressive Free Survival Post Transplant	4 participants

PRIMARY outcome

Timeframe: 2 years post transplant

Subjects surviving without disease progression 2 years after transplant as evidenced by no new disease showing on radiologic scans and / or bone marrow pathology.

Outcome measures

Outcome measures
Measure	Single Arm Trial n=4 Participants Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Progression Free Survival Post Transplant	2 participants

SECONDARY outcome

Timeframe: Within 100 days post transplant

Death related to treatment without relapse within 100 days after transplant

Outcome measures

Outcome measures
Measure	Single Arm Trial n=5 Participants Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Non-relapse Treatment Related Mortality	0 participants

SECONDARY outcome

Timeframe: Post transplant up to 100 days post transplant

Measured by number of participants that have chimerism study results that show the number of donor cells and the number of recipient cells present in the blood after post-transplant lymphocyte infusions continue to be predominately either donor or recipient.

Outcome measures

Outcome measures
Measure	Single Arm Trial n=5 Participants Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Number of Participants With Detectable Donor Chimerism at up to 100 Days Post Transplant	1 participant

SECONDARY outcome

Timeframe: Up to 100 days post transplant.

Outcome measures

Outcome measures
Measure	Single Arm Trial n=5 Participants Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Composite Incidence of Acute and Chronic Graft Versus Host Disease	2 participants

Adverse Events

Single Arm Trial

Serious events: 3 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Single Arm Trial n=5 participants at risk Single Arm Intervention trial as noted in interventions section Fludarabine: Fludarabine 30 mg/m2/day x 3 days Total Body Irradiation: TBI 200cGy x1 dose on transplant day Infusion of Stem Cells: On Day 0 pts will received an infusion of HLA matched sibling donor stem cells. Dose is determined by the volume of cells obtained from donor. Minimum dose is 2x10\*6 CD34+ cells per kilogram of recipient weight.
Respiratory, thoracic and mediastinal disorders RSV pneumonia Infection	20.0% 1/5 • Number of events 1
Respiratory, thoracic and mediastinal disorders Klebsiella pneumonia Infection	20.0% 1/5 • Number of events 1
Respiratory, thoracic and mediastinal disorders Pneumonia	20.0% 1/5 • Number of events 1

Other adverse events

Adverse event data not reported

Additional Information

William Vaughan, MD

UAB

Phone: 20-934-0066

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place