Trial Outcomes & Findings for Ex Vivo TCR αβ T Cell Depletion for Graft-Versus-Host Disease Prophylaxis in Mismatched Donor Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies (NCT NCT03717480)

NCT ID: NCT03717480

Last Updated: 2022-05-10

Results Overview

Number of participants with severe acute GVHD-free survival will be assessed at 100 days post-SCT

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

100 Days

Results posted on

2022-05-10

Participant Flow

Participant milestones

Participant milestones
Measure	TCR α/β Reagent System The stem cell apheresis product was depleted of TCRαβ T cells by negative selection using the automated CliniMACS® Plus device. CD34+ stem cell counts were obtained before and after processing with the Miltenyi ClinicMACs device. ClinicMACs: The Reagent System removed certain cells (called T-Cell Receptor (TCR) α/β positive T-cells) that are thought to cause GVHD from donor product before it was given to participants.
Overall Study STARTED	2
Overall Study Receipt of Investigational Product/Study Treatment	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	TCR α/β Reagent System The stem cell apheresis product was depleted of TCRαβ T cells by negative selection using the automated CliniMACS® Plus device. CD34+ stem cell counts were obtained before and after processing with the Miltenyi ClinicMACs device. ClinicMACs: The Reagent System removed certain cells (called T-Cell Receptor (TCR) α/β positive T-cells) that are thought to cause GVHD from donor product before it was given to participants.
Overall Study Never received investigational product due to product viability limitations.	1

Baseline Characteristics

Ex Vivo TCR αβ T Cell Depletion for Graft-Versus-Host Disease Prophylaxis in Mismatched Donor Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	TCR α/β Reagent System n=1 Participants The stem cell apheresis product was depleted of TCRαβ T cells by negative selection using the automated CliniMACS® Plus device. CD34+ stem cell counts were obtained before and after processing with the Miltenyi ClinicMACs device. ClinicMACs: The Reagent System removed certain cells (called T-Cell Receptor (TCR) α/β positive T-cells) that are thought to cause GVHD from donor product before it was given to participants.
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Sex: Female, Male Female	1 Participants n=99 Participants
Sex: Female, Male Male	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	1 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants
Race (NIH/OMB) White	0 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Region of Enrollment United States	1 participants n=99 Participants

PRIMARY outcome

Timeframe: 100 Days

Number of participants with severe acute GVHD-free survival will be assessed at 100 days post-SCT

Outcome measures

Outcome measures
Measure	TCR α/β Reagent System n=1 Participants The stem cell apheresis product was depleted of TCRαβ T cells by negative selection using the automated CliniMACS® Plus device. CD34+ stem cell counts were obtained before and after processing with the Miltenyi ClinicMACs device. ClinicMACs: The Reagent System removed certain cells (called T-Cell Receptor (TCR) α/β positive T-cells) that are thought to cause GVHD from donor product before it was given to participants.
Number of Participants With Severe Acute GVHD-free Survival	1 Participants

SECONDARY outcome

Timeframe: 2 years

Population: No participants were analyzed for this secondary outcome measure, because the only participant who would have been evaluable for analysis did not survive to the analysis timepoint of 2 years post-transplant.

Grades II-IV acute GVHD will be assessed at 2 years post-SCT. Grade I GVHD is characterized as mild disease, grade II as moderate, grade III as severe, and grade IV as life-threatening. Higher grades of acute GVHD are associated with worse outcomes. Acute GVHD will be staged by assessment of clinical manifestations in the skin, gastrointestinal tract, and liver.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Number of participants with chronic GVHD will be assessed at 2 years post-SCT.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

GRFS will be defined as alive without having experienced grade III-IV acute GVHD, moderate/severe chronic GVHD, or relapse of underlying malignancy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Number of participants with immunosuppression-free survival will be assessed at 2 years post-SCT.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Hematologic recovery will be assessed in participants at 2 years post-SCT.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Immune reconstitution will be assessed in participants at 2 years post-SCT.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Disease relapse will be assessed in participants at 2 years post-SCT.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Participant transplant-related mortality will be assessed at 2 years post-SCT.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Participant organ toxicity will be assessed at 2 years post-SCT.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Participant rate of infections will be assessed at 2 years post-SCT.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Number of participants with relapse-free and overall survival will be assessed at 2 years post-SCT.

Outcome measures

Outcome data not reported

Adverse Events

TCR α/β Reagent System

Serious events: 1 serious events

Other events: 1 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	TCR α/β Reagent System n=1 participants at risk The stem cell apheresis product was depleted of TCRαβ T cells by negative selection using the automated CliniMACS® Plus device. CD34+ stem cell counts were obtained before and after processing with the Miltenyi ClinicMACs device. ClinicMACs: The Reagent System removed certain cells (called T-Cell Receptor (TCR) α/β positive T-cells) that are thought to cause GVHD from donor product before it was given to participants.
Hepatobiliary disorders Hepatic Hemorrhage	100.0% 1/1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Gastrointestinal disorders Colonic Perforation	100.0% 1/1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Investigations Platelet Count Decreased	100.0% 1/1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Respiratory, thoracic and mediastinal disorders Respiratory Failure	100.0% 1/1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Renal and urinary disorders Acute Kidney Injury	100.0% 1/1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Nervous system disorders Stroke	100.0% 1/1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Gastrointestinal disorders Colitis	100.0% 1/1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.

Other adverse events

Other adverse events
Measure	TCR α/β Reagent System n=1 participants at risk The stem cell apheresis product was depleted of TCRαβ T cells by negative selection using the automated CliniMACS® Plus device. CD34+ stem cell counts were obtained before and after processing with the Miltenyi ClinicMACs device. ClinicMACs: The Reagent System removed certain cells (called T-Cell Receptor (TCR) α/β positive T-cells) that are thought to cause GVHD from donor product before it was given to participants.
Gastrointestinal disorders Abdominal Pain	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Investigations Alanine Aminotransferase Increased	100.0% 1/1 • Number of events 3 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Blood and lymphatic system disorders Anemia	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Metabolism and nutrition disorders Anorexia	100.0% 1/1 • Number of events 2 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Investigations Aspartate Aminotransferase Increased	100.0% 1/1 • Number of events 3 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Infections and infestations Bacteremia	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Respiratory, thoracic and mediastinal disorders Cough	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Investigations Creatinine Increased	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Gastrointestinal disorders Diarrhea	100.0% 1/1 • Number of events 2 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Nervous system disorders Dizziness	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Respiratory, thoracic and mediastinal disorders Epistaxis	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
General disorders Fatigue	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Renal and urinary disorders Hematuria	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Psychiatric disorders Insomnia	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Infections and infestations Infections and Infestations - Other, specify	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Gastrointestinal disorders Mucositis Oral	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Gastrointestinal disorders Nausea	100.0% 1/1 • Number of events 3 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Investigations Platelet Count Decreased	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Skin and subcutaneous tissue disorders Pruritus	100.0% 1/1 • Number of events 2 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Skin and subcutaneous tissue disorders Rash Maculo-papular	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Reproductive system and breast disorders Vaginal Pain	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Investigations Weight Loss	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.
Investigations White Blood Cell Decreased	100.0% 1/1 • Number of events 1 • Adverse event data was collected for each participant up until 2 years post-transplant or until the participant's death, whichever came first. The only participant who received study treatment did not survive to the analysis timepoint of 2 years post-transplant, so all documented adverse events were collected from the date of their transplant up until their death approximately 9 months post-transplant. Adverse events were collected through regular investigator assessment and laboratory testing.

Additional Information

Dr. Vincent Ho

Dana-Farber Cancer Institute

Phone: 617-632-6256

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place