Trial Outcomes & Findings for Haploidentical Donor Hematopoietic Progenitor Cell and NK Cell Transplantation for Hematologic Malignancy (NCT NCT01807611)

Last Updated: 2022-10-31

Results Overview

Neutrophil engraftment will be determined using the parameters put forth by the Center for International Blood and Marrow Registry. Assessments will be made upon review of daily complete blood count and serial chimerism studies. Successful engraftment for the purposes of this objective will be patients who do not experience graft failure.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

82 participants

Primary outcome timeframe

42 days post engraftment

Results posted on

2022-10-31

Participant Flow

There are in total 82 transplant recipients enrolled.

Participant milestones

Participant milestones
Measure	Transplant Recipients Participants undergo a preparative regimen of total lymphoid irradiation, fludarabine, cyclophosphamide, fludarabine, thiotepa, melphalan, and mycophenolate mofetil, followed by HPC,A infusion and TC-NK infusion. They also receive G-CSF and mesna. Cells for infusion are prepared using the CliniMACS System. Total Lymphoid Irradiation: Participants receive total lymphoid irradiation over four doses. Fludarabine: Given IV. Cyclophosphamide: Given IV. Thiotepa: Given IV. Melphalan: Given IV. HPC,A Infusion: Participants received infusions of HPC,A (CD34+ selected) and HPC,A (CD45RA depleted). TC-NK Infusion: Participants receive infusions of TC-NK. G-CSF: Participants receive G-CSF subcutaneously or intravenously. Donors receive G-CSF subcutaneously during cell mobilization. Mesna: Mesna is generally dosed at approximately 25% of the cyclophosphamide dose. It is generally given intravenously prior to and again at 3, 6 and 9 hours following each dose of cyclophosphamide. CliniMACS: The mechanism of action of the CliniMACS Cell Selection System is based on magnetic-activated cell sorting (MACS). The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures, (e.g. apheresis products). These can then be separated in a magnetic field using an immunomagnetic label specific for the cell type of interest, such as CD3+ human T cells. Mycophenolate mofetil: Given intravenously or orally.
Overall Study STARTED	82
Overall Study COMPLETED	78
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Haploidentical Donor Hematopoietic Progenitor Cell and NK Cell Transplantation for Hematologic Malignancy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Experimental: Transplant Recipients n=72 Participants Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.
Age, Categorical <=18 years	63 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	9.5 years STANDARD_DEVIATION 6.23 • n=5 Participants
Sex: Female, Male Female	30 Participants n=5 Participants
Sex: Female, Male Male	42 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	19 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	53 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	14 Participants n=5 Participants
Race (NIH/OMB) White	52 Participants n=5 Participants
Race (NIH/OMB) More than one race	3 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Donor relationship to patient: Father, Mother, Sibling, Other. Father	35 Participants n=5 Participants
Donor relationship to patient: Father, Mother, Sibling, Other. Mother	31 Participants n=5 Participants
Donor relationship to patient: Father, Mother, Sibling, Other. Sibling	4 Participants n=5 Participants
Donor relationship to patient: Father, Mother, Sibling, Other. Other	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: 42 days post engraftment

Population: Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.

Outcome measures

Outcome measures
Measure	Experimental: Transplant Recipients n=72 Participants Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.	Number of Transplant Recipients With Chronic Graft Versus Host Disease (cGVHD) Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.
Number of Transplant Recipients With Successful Engraftment	70 Participants	—

SECONDARY outcome

Timeframe: One-year post-transplantation

Bone marrow studies for disease status evaluation will be performed at 1-year post-transplant. Testing will include a research evaluation for minimal residual disease.

Outcome measures

Outcome measures
Measure	Experimental: Transplant Recipients n=72 Participants Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.	Number of Transplant Recipients With Chronic Graft Versus Host Disease (cGVHD) Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.
Number of Transplant Recipients With Malignant Relapse	18 Participants	—

SECONDARY outcome

Timeframe: One year post-transplantation

The one-year event free survival is defined by the patient who has neither experienced relapse nor death within one year after post transplantation. And the rate is calculated by computing the ratio between total number of one year event free survival patients and the total number of patients.

Outcome measures

Outcome measures
Measure	Experimental: Transplant Recipients n=72 Participants Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.	Number of Transplant Recipients With Chronic Graft Versus Host Disease (cGVHD) Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.
Event-free Survival	49 Participants	—

SECONDARY outcome

Timeframe: one year post-transplantation

The one-year survival is defined by the patient who has not died within one year after post transplantation. And the rate is calculated by computing the ratio between total number of one year survival patients and the total number of patients.

Outcome measures

Outcome measures
Measure	Experimental: Transplant Recipients n=72 Participants Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.	Number of Transplant Recipients With Chronic Graft Versus Host Disease (cGVHD) Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.
Overall Survival	59 Participants	—

SECONDARY outcome

Timeframe: 100 days post-transplant for acute GVHD; one year post-transplant for chronic GVHD .

Acute and chronic graft-vs.-host disease will be evaluated using the standard grading criteria. The estimate will be the number of recipients who experienced GVHD divided by the total number of patients considered in this group.

Outcome measures

Outcome measures
Measure	Experimental: Transplant Recipients n=72 Participants Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.	Number of Transplant Recipients With Chronic Graft Versus Host Disease (cGVHD) n=72 Participants Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.
Number of Transplant Recipients With Acute and/or Chronic Graft Versus Host Disease (GVHD)	24 Participants	16 Participants

SECONDARY outcome

Timeframe: In the first 100 days after transplantation

Estimate the proportion of patients died within 100 days after the transplantation who has not experienced a relapse. The estimate will be the number of TRM divides the total number of patients considered in this group.

Outcome measures

Outcome measures
Measure	Experimental: Transplant Recipients n=72 Participants Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.	Number of Transplant Recipients With Chronic Graft Versus Host Disease (cGVHD) Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.
Number of Transplant Recipients With Transplant-related Mortality (TRM)	4 Participants	—

SECONDARY outcome

Timeframe: 100 days post-transplant for acute GVHD.

Ongoing assessment of toxicity will be done using the NCI CTCAE version 3.0. Acute and chronic graft-vs.-host disease will be evaluated using the standard grading criteria. The severity of acute GvHD and chronic GvHD will be described. The analysis for this objective will be performed when the last evaluable participant has been followed for 100 days post transplant. Acute GvHD is graded from 1-4 with 4 being the worst outcome.

Outcome measures

Outcome measures
Measure	Experimental: Transplant Recipients n=72 Participants Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.	Number of Transplant Recipients With Chronic Graft Versus Host Disease (cGVHD) Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.
Severity of Acute Graft Versus Host Disease (aGVHD) No aGVHD within 100 days	48 Participants	—
Severity of Acute Graft Versus Host Disease (aGVHD) Grade 1 aGVHD within 100 days	2 Participants	—
Severity of Acute Graft Versus Host Disease (aGVHD) Grade 2 aGVHD within 100 days	2 Participants	—
Severity of Acute Graft Versus Host Disease (aGVHD) Grade 3 aGVHD within 100 days	16 Participants	—
Severity of Acute Graft Versus Host Disease (aGVHD) Grade 4 aGVHD within 100 days	4 Participants	—

SECONDARY outcome

Timeframe: 1 year post-transplant for chronic GVHD .

Ongoing assessment of toxicity will be done using the NCI CTCAE version 3.0. Acute and chronic graft-vs.-host disease will be evaluated using the standard grading criteria. The severity of acute GvHD and chronic GvHD will be described. The analysis for this objective will be performed when the last evaluable participant has been followed for 1 year post-transplant. Chronic GvHD is graded as "mild", "moderate" or "severe" with "severe" being the worst outcome.

Outcome measures

Outcome measures
Measure	Experimental: Transplant Recipients n=72 Participants Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.	Number of Transplant Recipients With Chronic Graft Versus Host Disease (cGVHD) Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are excluded.
Severity of Chronic Graft Versus Host Disease (cGVHD) No cGVHD within 1st year	56 Participants	—
Severity of Chronic Graft Versus Host Disease (cGVHD) Mild	6 Participants	—
Severity of Chronic Graft Versus Host Disease (cGVHD) Moderate	5 Participants	—
Severity of Chronic Graft Versus Host Disease (cGVHD) Severe	5 Participants	—

Adverse Events

Experimental: Transplant Recipients

Serious events: 56 serious events

Other events: 78 other events

Deaths: 14 deaths

Serious adverse events

Serious adverse events
Measure	Experimental: Transplant Recipients n=78 participants at risk Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are included.
Infections and infestations Febrile Without Neutropenia	21.8% 17/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Febrile Neutropenia	12.8% 10/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Cardiac disorders Pericardial Effusion	9.0% 7/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Respiratory, thoracic and mediastinal disorders Respiratory Distress	9.0% 7/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Cardiac disorders Hypotension	7.7% 6/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Renal and urinary disorders Acute Kidney Injury	6.4% 5/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Staphylococcus Epidermidis, Blood	6.4% 5/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Nervous system disorders Seizure	6.4% 5/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
General disorders Fever	5.1% 4/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Blood and lymphatic system disorders Hemorrhage, Pulmonary	5.1% 4/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.

Other adverse events

Other adverse events
Measure	Experimental: Transplant Recipients n=78 participants at risk Participants meeting eligibility criteria and who completed study therapy: haploidentical stem cell transplantation with additional natural killer (NK) cells and total lymphoid irradiation (TLI) based conditioning regimen. Participants who received a therapeutic variance (maraviroc) are included.
Infections and infestations Febrile Neutropenia	85.9% 67/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Gastrointestinal disorders Mucositis	64.1% 50/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Clostridium Difficile, Stool	42.3% 33/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Human Herpesvirus 6, Blood	41.0% 32/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Febrile Without Neutropenia	35.9% 28/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Adenovirus, Stool	33.3% 26/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Immune system disorders Engraftment Syndrome	30.8% 24/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Candida, Oral	28.2% 22/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Cardiac disorders Hypertension	25.6% 20/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Cytomegalovirus, Blood	23.1% 18/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Cardiac disorders Tachycardia (finding)	20.5% 16/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Gastrointestinal disorders Nausea	19.2% 15/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, BK Virus, Urine	15.4% 12/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Gastrointestinal disorders Diarrhea	14.1% 11/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Renal and urinary disorders Hemorrhagic Cystitis	14.1% 11/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Cardiac disorders Hypotension	12.8% 10/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Rotavirus, Stool	12.8% 10/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Immune system disorders Cytokine Release Syndrome	10.3% 8/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Respiratory, thoracic and mediastinal disorders Hypoxia	10.3% 8/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Renal and urinary disorders Acute Kidney Injury	9.0% 7/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Staphylococcus Epidermidis, Blood	9.0% 7/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Metabolism and nutrition disorders Alanine aminotransferase (ALT) (serum glutamic-pyruvic transaminase) (SGPT) level abnormal (finding)	7.7% 6/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Cardiac disorders Diastolic dysfunction (finding)	7.7% 6/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Adenovirus, Blood	7.7% 6/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Epstein Barr Virus, Blood	7.7% 6/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Adenovirus, Respiratory Tract	6.4% 5/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Norovirus, Stool	6.4% 5/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Metabolism and nutrition disorders Transaminitis	6.4% 5/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Nervous system disorders Confusion	5.1% 4/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Metabolism and nutrition disorders Dehydration	5.1% 4/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Blood and lymphatic system disorders Epistaxis	5.1% 4/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, BK Virus, Blood	5.1% 4/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Infections and infestations Infection, Streptococcus Viridans, Blood	5.1% 4/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Gastrointestinal disorders Pneumatosis Intestinalis	5.1% 4/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.
Metabolism and nutrition disorders Weight Loss	5.1% 4/78 • Transplant recipients will be followed for adverse events from the start of conditioning and throughout the first-year post-transplant Recipient participants were followed for all NCI Grade III-V adverse events from the start of conditioning through the first-year post HCT, regardless of their relationship to the treatment given. Clinically significant NCI Grade I-II adverse events that are judged to be related/possibly related may be collected per the discretion and judgment of the PI. Adverse events that affected more than 5 participants are included in this description.

Additional Information

Brandon M. Triplett, MD

St. Jude Children's Research Hospital

Phone: 866-278-5833

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place