Trial Outcomes & Findings for CD34+ Cell Enriched and T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Mismatched Related Donors or Borderline Organ Function (NCT NCT02162511)

NCT ID: NCT02162511

Last Updated: 2023-06-26

Results Overview

GVHD is a condition that occurs when donor bone marrow or stem cells attack the recipient.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 3 participants
• Primary outcome timeframe: Day +100
• Results posted on: 2023-06-26

Participant Flow

Enrollment was open to participants with malignant or non-malignant disorders receiving mismatched related donor hematopoietic stem cell transplants who could benefit from augmented CD34+ cells and T cell-depleted products to prevent severe (grade III/IV) acute Graft vs Host Disease (GVHD). Participants were withdrawn from the study at the time of graft failure due to need for exclusionary concurrent treatment (per PI discretion)

Participant milestones

Measure	ARM A Malignant TBI Malignant diseases Conditioning including total body irradiation and chemotherapy CliniMACS CD34+ cell enrichment and T-cell depletion	ARM B Malignant Non-TBI Malignant diseases chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion	ARM C Non-malignant Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Overall Study STARTED	0	0	3
Overall Study Achieved Engraftment	0	0	1
Overall Study COMPLETED	0	0	1
Overall Study NOT COMPLETED	0	0	2

Reasons for withdrawal

Measure	ARM A Malignant TBI Malignant diseases Conditioning including total body irradiation and chemotherapy CliniMACS CD34+ cell enrichment and T-cell depletion	ARM B Malignant Non-TBI Malignant diseases chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion	ARM C Non-malignant Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Overall Study Lack of Efficacy	0	0	2

Baseline Characteristics

CD34+ Cell Enriched and T Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Mismatched Related Donors or Borderline Organ Function

Baseline characteristics by cohort

Measure	ARM A Malignant TBI Malignant diseases Conditioning including total body irradiation and chemotherapy CliniMACS CD34+ cell enrichment and T-cell depletion	ARM B Malignant Non-TBI Malignant diseases chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion	ARM C Non-malignant n=3 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion	Total n=3 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants	3 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	—	—	7.47 years n=5 Participants	7.47 years n=4 Participants
Sex: Female, Male Female	—	—	1 Participants n=5 Participants	1 Participants n=4 Participants
Sex: Female, Male Male	—	—	2 Participants n=5 Participants	2 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	—	—	2 Participants n=5 Participants	2 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	—	—	1 Participants n=5 Participants	1 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	—	—	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	—	—	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	—	—	1 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	—	—	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	—	—	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	—	—	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) More than one race	—	—	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	—	—	2 Participants n=5 Participants	2 Participants n=4 Participants
Region of Enrollment United States	—	—	3 Participants n=5 Participants	3 Participants n=4 Participants

PRIMARY outcome

Timeframe: Day +100

GVHD is a condition that occurs when donor bone marrow or stem cells attack the recipient.

Outcome measures

Measure	ARM C Non-malignant n=3 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Number of Patients With Severe (Grade III/IV) Acute Graft vs Host Disease (GVHD)	0 Participants

SECONDARY outcome

Timeframe: Up to Day +42 after stem cell transplant

Failure of donor stem cells to make neutrophils

Outcome measures

Measure	ARM C Non-malignant n=3 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Number of Participants With Graft Failure	2 Participants

SECONDARY outcome

Timeframe: up to +1 year post-transplant

Population: Patients who had primary graft failure were withdrawn from the study are excluded from the analysis.

Absolute neutrophil count (ANC) >500 for 3 consecutive days and >80% donor cells in blood.

Outcome measures

Measure	ARM C Non-malignant n=1 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Length of Time to Engraftment	10 days Standard Deviation NA Standard Deviation not calculable for one participant

SECONDARY outcome

Timeframe: Day +100 post-transplant

Population: Patients who had primary graft failure were withdrawn from the study are excluded from the analysis.

The percentage of donor cells for all evaluable (without disease progression) patients

Outcome measures

Measure	ARM C Non-malignant n=1 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Chimerism of Donor Cells Whole Blood	95 Percentage of cells from donor Standard Deviation NA Standard Deviation not calculable for one participant
Chimerism of Donor Cells CD3 Cells	70 Percentage of cells from donor Standard Deviation NA Standard Deviation not calculable for one participant
Chimerism of Donor Cells CD15 Cells	100 Percentage of cells from donor Standard Deviation NA Standard Deviation not calculable for one participant
Chimerism of Donor Cells CD19	99 Percentage of cells from donor Standard Deviation NA Standard Deviation not calculable for one participant
Chimerism of Donor Cells CD34	98 Percentage of cells from donor Standard Deviation NA Standard Deviation not calculable for one participant
Chimerism of Donor Cells CD56	99 Percentage of cells from donor Standard Deviation NA Standard Deviation not calculable for one participant

SECONDARY outcome

Timeframe: up to +1 year post-transplant

Population: Patients who had primary graft failure and were withdrawn from the study are excluded from the analysis.

The time to CD4 count >100

Outcome measures

Measure	ARM C Non-malignant n=1 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Immune Recovery (CD4)	148 days Standard Deviation NA Standard Deviation not calculable for one participant

SECONDARY outcome

Timeframe: up to +1 year post-transplant

Population: Patients who had primary graft failure and were withdrawn from the study are excluded from the analysis.

Outcome measures

Measure	ARM C Non-malignant n=1 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Number of Participants With Immune Recovery (CD4 >200) by Year 1	0 Participants

SECONDARY outcome

Timeframe: 6 months and 1 year post-transplant

Population: Participants who had primary graft failure were withdrawn from the study and unable to be evaluated for this outcome measure.

Immune recovery defined as achieving normal levels of PHA (53,000-200,000 CPM)

Outcome measures

Measure	ARM C Non-malignant n=1 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Immune Recovery Shown as Phytohemagglutin (PHA) 6 months	33736 Net CPM Standard Deviation NA Standard Deviation not calculable for one participant
Immune Recovery Shown as Phytohemagglutin (PHA) 1 year	110809 Net CPM Standard Deviation NA Standard Deviation not calculable for one participant

SECONDARY outcome

Timeframe: up to +1 year post-transplant

Post-transplant lymphoproliferative disorder (PTLD) is a well-known, life-threatening complication of organ transplantation, predominantly occurring after solid organ transplantation (SOT) and hematopoietic stem cell transplantation (HSCT).

Outcome measures

Measure	ARM C Non-malignant n=3 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Number of Patients With Post-transplant Lymphoproliferative Disease (PTLD)	0 Participants

SECONDARY outcome

Timeframe: up to +1 year post-transplant

Incidence of transplant-related toxicities

Outcome measures

Measure	ARM C Non-malignant n=3 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Number of Patients With Severe Toxicities	2 Participants

SECONDARY outcome

Timeframe: up to +1 year post-transplant

Post-transplant infections will be described by incidence and type. Participants may have had more than one type of infection.

Outcome measures

Measure	ARM C Non-malignant n=3 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Number of Participants Experiencing Post-transplant Infections BK viremia	1 Participants
Number of Participants Experiencing Post-transplant Infections BK viruria	1 Participants
Number of Participants Experiencing Post-transplant Infections Norovirus	1 Participants
Number of Participants Experiencing Post-transplant Infections HHV6 viremia	2 Participants
Number of Participants Experiencing Post-transplant Infections EBV viremia	1 Participants
Number of Participants Experiencing Post-transplant Infections Rhinovirus/Enterovirus	1 Participants
Number of Participants Experiencing Post-transplant Infections Parainfluenza 3 upper respiratory infection	1 Participants
Number of Participants Experiencing Post-transplant Infections Adenovirus (low level reactivation)	1 Participants
Number of Participants Experiencing Post-transplant Infections Cytomegalovirus (low level reactivation)	1 Participants

SECONDARY outcome

Timeframe: at Day +100 and +1 year post-transplant

Death related to transplant

Outcome measures

Measure	ARM C Non-malignant n=3 Participants Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion
Transplant-related Mortality (TRM) Day 100	1 Participants
Transplant-related Mortality (TRM) Between Day 100 and 1 Year	1 Participants

Adverse Events

ARM C Non-malignant

Serious events: 0 serious events

Other events: 0 other events

Deaths: 2 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Erica Goodwin

Stanford University

Phone: 253-508-3556

Email: egood55@stanford.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place