Trial Outcomes & Findings for Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease (NCT NCT00578643)

Last Updated: 2018-11-09

Results Overview

To estimate the engraftment rate for patients with CGD using busulfan, cyclophosphamide, fludarabine and alemtuzumab (Campath 1H) as conditioning therapy for SCT from 5/6 or 6/6 HLA-matched unrelated or 5/6 or 6/6 HLA phenotype-matched related donors.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

28 days post transplant

Results posted on

2018-11-09

Participant Flow

Participant milestones

Participant milestones
Measure	Allogeneic Unrelated Transplant Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion. Busulfan: Days -9 through -6 1 mg/kg initially (based on weight) Alemtuzumab: Day -5 through Day -2 Dose is based on weight: Less than 15 kg: 3 mg More than 15 kg to 30 kg: 5 mg More than 30 kg: 15 mg Cyclophosphamide: Days -5 through -2 50 mg/kg Fludarabine: Day -5 through Day -2 30 mg/m\^2 Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours. Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10\^8/kg recipient weight; for cord blood ≥ 3 X 10\^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10\^/kg CD34+ cells.
Overall Study STARTED	15
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Allogeneic Unrelated Transplant Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion. Busulfan: Days -9 through -6 1 mg/kg initially (based on weight) Alemtuzumab: Day -5 through Day -2 Dose is based on weight: Less than 15 kg: 3 mg More than 15 kg to 30 kg: 5 mg More than 30 kg: 15 mg Cyclophosphamide: Days -5 through -2 50 mg/kg Fludarabine: Day -5 through Day -2 30 mg/m\^2 Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours. Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10\^8/kg recipient weight; for cord blood ≥ 3 X 10\^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10\^/kg CD34+ cells.
Overall Study Death	1
Overall Study Lost to Follow-up	1

Baseline Characteristics

Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Allogeneic Unrelated Transplant n=15 Participants Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion. Busulfan: Days -9 through -6 1 mg/kg initially (based on weight) Alemtuzumab: Day -5 through Day -2 Dose is based on weight: Less than 15 kg: 3 mg More than 15 kg to 30 kg: 5 mg More than 30 kg: 15 mg Cyclophosphamide: Days -5 through -2 50 mg/kg Fludarabine: Day -5 through Day -2 30 mg/m\^2 Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours. Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10\^8/kg recipient weight; for cord blood ≥ 3 X 10\^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10\^/kg CD34+ cells.
Age, Continuous	6 years n=93 Participants
Sex: Female, Male Female	1 Participants n=93 Participants
Sex: Female, Male Male	14 Participants n=93 Participants
Race/Ethnicity, Customized Race/Ethnicity · White	7 Participants n=93 Participants
Race/Ethnicity, Customized Race/Ethnicity · Black or African American	1 Participants n=93 Participants
Race/Ethnicity, Customized Race/Ethnicity · Asian	1 Participants n=93 Participants
Race/Ethnicity, Customized Race/Ethnicity · Hispanic or Latino	5 Participants n=93 Participants
Race/Ethnicity, Customized Race/Ethnicity · Unknown	1 Participants n=93 Participants

PRIMARY outcome

Timeframe: 28 days post transplant

Outcome measures

Outcome measures
Measure	Allogeneic Unrelated Transplant n=15 Participants Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion. Busulfan: Days -9 through -6 1 mg/kg initially (based on weight) Alemtuzumab: Day -5 through Day -2 Dose is based on weight: Less than 15 kg: 3 mg More than 15 kg to 30 kg: 5 mg More than 30 kg: 15 mg Cyclophosphamide: Days -5 through -2 50 mg/kg Fludarabine: Day -5 through Day -2 30 mg/m\^2 Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours. Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10\^8/kg recipient weight; for cord blood ≥ 3 X 10\^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10\^/kg CD34+ cells.
Percentage of Participants With Engraftment	100 percentage of participants

SECONDARY outcome

Timeframe: 120 days post transplant

Population: One patient died on day 62 post transplant and was not included in the analysis.

To estimate the likelihood of complete donor chimerism for patients with CGD using busulfan, cyclophosphamide, fludarabine and alemtuzumab (Campath 1H) as conditioning therapy for SCT from 5/6 or 6/6 HLA-matched unrelated or 5/6 or 6/6 HLA phenotype-matched related donors.

Outcome measures

Outcome measures
Measure	Allogeneic Unrelated Transplant n=14 Participants Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion. Busulfan: Days -9 through -6 1 mg/kg initially (based on weight) Alemtuzumab: Day -5 through Day -2 Dose is based on weight: Less than 15 kg: 3 mg More than 15 kg to 30 kg: 5 mg More than 30 kg: 15 mg Cyclophosphamide: Days -5 through -2 50 mg/kg Fludarabine: Day -5 through Day -2 30 mg/m\^2 Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours. Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10\^8/kg recipient weight; for cord blood ≥ 3 X 10\^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10\^/kg CD34+ cells.
Number of Patients That Have Complete Donor Chimerism After Transplant.	13 participants

SECONDARY outcome

Timeframe: Assessed between day 0 and day 100 post transplant

Population: One patient who died on day 62 post transplant without developing acute GVHD was not assessed for this Outcome Measure.

To estimate the risk for acute GVHD and regimen related morbidity/mortality for patients with CGD following stem cell transplant from 5/6 or 6/6 HLA matched unrelated or 5/6 or 6/6 HLA phenotype matched related donors.

Outcome measures

Outcome measures
Measure	Allogeneic Unrelated Transplant n=14 Participants Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion. Busulfan: Days -9 through -6 1 mg/kg initially (based on weight) Alemtuzumab: Day -5 through Day -2 Dose is based on weight: Less than 15 kg: 3 mg More than 15 kg to 30 kg: 5 mg More than 30 kg: 15 mg Cyclophosphamide: Days -5 through -2 50 mg/kg Fludarabine: Day -5 through Day -2 30 mg/m\^2 Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours. Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10\^8/kg recipient weight; for cord blood ≥ 3 X 10\^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10\^/kg CD34+ cells.
Number of Patients That Have Acute GVHD and Regimen Related Morbidity/Mortality Post Transplant. None	8 Participants
Number of Patients That Have Acute GVHD and Regimen Related Morbidity/Mortality Post Transplant. Grade I	6 Participants
Number of Patients That Have Acute GVHD and Regimen Related Morbidity/Mortality Post Transplant. Grade II	0 Participants
Number of Patients That Have Acute GVHD and Regimen Related Morbidity/Mortality Post Transplant. Grade III-IV	0 Participants

SECONDARY outcome

Timeframe: Assessed between day 100 and day 365 post transplant

Population: One patient died on day 62 post transplant, and one patient was lost to F/U on day 163 post transplant. They were not assessed for this Outcome Measure.

To estimate the risk for chronic GVHD and regimen related morbidity/mortality for patients with CGD following stem cell transplant from 5/6 or 6/6 HLA matched unrelated or 5/6 or 6/6 HLA phenotype matched related donors.

Outcome measures

Outcome measures
Measure	Allogeneic Unrelated Transplant n=13 Participants Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion. Busulfan: Days -9 through -6 1 mg/kg initially (based on weight) Alemtuzumab: Day -5 through Day -2 Dose is based on weight: Less than 15 kg: 3 mg More than 15 kg to 30 kg: 5 mg More than 30 kg: 15 mg Cyclophosphamide: Days -5 through -2 50 mg/kg Fludarabine: Day -5 through Day -2 30 mg/m\^2 Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours. Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10\^8/kg recipient weight; for cord blood ≥ 3 X 10\^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10\^/kg CD34+ cells.
Number of Patients That Have Chronic GVHD and Regimen Related Morbidity/Mortality Post Transplant. Yes	0 Participants
Number of Patients That Have Chronic GVHD and Regimen Related Morbidity/Mortality Post Transplant. No	13 Participants

Adverse Events

Allogeneic Unrelated Transplant

Serious events: 9 serious events

Other events: 14 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Allogeneic Unrelated Transplant n=15 participants at risk Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion. Busulfan: Days -9 through -6 1 mg/kg initially (based on weight) Alemtuzumab: Day -5 through Day -2 Dose is based on weight: Less than 15 kg: 3 mg More than 15 kg to 30 kg: 5 mg More than 30 kg: 15 mg Cyclophosphamide: Days -5 through -2 50 mg/kg Fludarabine: Day -5 through Day -2 30 mg/m\^2 Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours. Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10\^8/kg recipient weight; for cord blood ≥ 3 X 10\^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10\^/kg CD34+ cells.
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Cardiac disorders Hypertension	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
General disorders Constitutional Symptoms - Other: Multiorgan failure	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Gastrointestinal disorders Diarrhea patients without colostomy	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Gastrointestinal disorders Gastrointestinal-Other: Eosinofilic enteritis	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Gastrointestinal disorders Pancreatitis	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Hematuria (in the absence of vaginal bleeding)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Infection (documented clinically or microbiologically) with grade 3 or 4 neutropenia (ANC <1.0 x 10e	6.7% 1/15 • Number of events 2 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Infection without neutropenia	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Infection - Other: Adenovirus viremia, BK viruria, CMV viremia	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Infection - Other: Hemorrhagic cystitis with BK virus, adenovirus, and CMV	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils - Blood	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Metabolism and nutrition disorders SGOT (AST) (serum glutamic oxaloacetic transaminase)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Nervous system disorders Seizure	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Respiratory, thoracic and mediastinal disorders Pleural effusion (non-malignant) 1	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Respiratory, thoracic and mediastinal disorders Pneumonitis/pulmonary infiltrates	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Respiratory, thoracic and mediastinal disorders Pulmonary/Upper Respiratory - Other: Respiratory distress	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Renal and urinary disorders Renal failure	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.

Other adverse events

Other adverse events
Measure	Allogeneic Unrelated Transplant n=15 participants at risk Conditioning from Day -9 to Day -1. Stem cells given on Day 0. Busulfan, alemtuzumab, cyclophosphamide, fludarabine, cyclosporine, stem cell infusion. Busulfan: Days -9 through -6 1 mg/kg initially (based on weight) Alemtuzumab: Day -5 through Day -2 Dose is based on weight: Less than 15 kg: 3 mg More than 15 kg to 30 kg: 5 mg More than 30 kg: 15 mg Cyclophosphamide: Days -5 through -2 50 mg/kg Fludarabine: Day -5 through Day -2 30 mg/m\^2 Cyclosporine: Cyclosporine will be administered beginning Day -2. Initial dose will 5 mg/kg infused over 24 hours. Stem Cell Infusion: Stem Cell: Either bone marrow, cord blood, or peripheral blood stem cells may be used for stem cell transplantation. It is desired to infuse: for bone marrow, nucleated cells ≥ 4 X 10\^8/kg recipient weight; for cord blood ≥ 3 X 10\^7/kg nucleated cells; for peripheral blood stem cells ≥ 1 X 10\^/kg CD34+ cells.
Cardiac disorders Hypertension	20.0% 3/15 • Number of events 3 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Gastrointestinal disorders Dehydration	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Gastrointestinal disorders Diarrhea	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Gastrointestinal disorders Diarrhea patients without colostomy	6.7% 1/15 • Number of events 3 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Gastrointestinal disorders Stomatitis/pharyngitis (oral/pharyngeal mucositis)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Hematuria (in the absence of vaginal bleeding)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Hepatobiliary disorders GGT (Gamma-Glutamyl transpeptidase)	13.3% 2/15 • Number of events 3 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Hepatobiliary disorders SGOT (AST) (serum glutamic oxaloacetic transaminase)	13.3% 2/15 • Number of events 2 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Hepatobiliary disorders SGPT (ALT) (serum glutamic pyruvic transaminase)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Catheter-related infection	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Infection (documented clinically or microbiologically) with grade 3 or 4 neutropenia(ANC<1.0x10e9/L)	20.0% 3/15 • Number of events 3 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Infection without neutropenia	13.3% 2/15 • Number of events 5 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils - Bladder (urinary)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Metabolism and nutrition disorders ALT, SGPT (serum glutamic pyruvic transaminase)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Metabolism and nutrition disorders AST, SGOT(serum glutamic oxaloacetic transaminase)	33.3% 5/15 • Number of events 6 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Metabolism and nutrition disorders Acidosis (metabolic or respiratory)	6.7% 1/15 • Number of events 2 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Metabolism and nutrition disorders Amylase	6.7% 1/15 • Number of events 2 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Metabolism and nutrition disorders Calcium, serum-low (hypocalcemia)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Metabolism and nutrition disorders GGT (gamma-Glutamyl transpeptidase)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Metabolism and nutrition disorders Hypocalcemia	13.3% 2/15 • Number of events 2 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Metabolism and nutrition disorders Hyponatremia	13.3% 2/15 • Number of events 2 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Metabolism and nutrition disorders Lipase	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.
Nervous system disorders Seizure(s)	6.7% 1/15 • Number of events 1 • Adverse events excluding fevers and hematological toxicities were collected up to 30 days after transplant. SAEs excluding fevers and hematological toxicities were collected up to 100 days after transplant. An adverse event was defined as grade III or grade IV toxicity by the NCI Common Toxicity Criteria Version 3.0. A SAE is any adverse event that was fatal, life threatening , required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the investigational product(s) or considered a significant medical event by the investigator.

Additional Information

Robert Krance, MD

Baylor College of Medicine

Phone: (832) 824-4661

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place