Trial Outcomes & Findings for T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts (NCT NCT00368355)

Last Updated: 2020-01-21

Results Overview

Percentage of participants with hematopoietic engraftment post-transplant. Engraftment is defined as the first day absolute neutrophil counts exceeded 0.5 X 10\^9/ml.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

46 participants

Primary outcome timeframe

28 days

Results posted on

2020-01-21

Participant Flow

Participant milestones

Participant milestones
Measure	CLINIMACS Device Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the CLINIMACS Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	ISOLEX Device Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the ISOLEX Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0
Overall Study STARTED	18	28
Overall Study COMPLETED	7	11
Overall Study NOT COMPLETED	11	17

Reasons for withdrawal

Reasons for withdrawal
Measure	CLINIMACS Device Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the CLINIMACS Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	ISOLEX Device Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the ISOLEX Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0
Overall Study Adverse Event	1	0
Overall Study Death	2	4
Overall Study Relapse	6	10
Overall Study Subsequent transplant	0	3
Overall Study Treatment - hematopoietic cell product	2	0

Baseline Characteristics

T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CLINIMACS Device n=18 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the CLINIMACS Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	ISOLEX Device n=28 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the ISOLEX Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	Total n=46 Participants Total of all reporting groups
Age, Continuous	7 years n=5 Participants	7 years n=7 Participants	7 years n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	9 Participants n=7 Participants	16 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants	19 Participants n=7 Participants	30 Participants n=5 Participants
Race/Ethnicity, Customized White	2 Participants n=5 Participants	5 Participants n=7 Participants	7 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	3 Participants n=5 Participants	8 Participants n=7 Participants	11 Participants n=5 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	12 Participants n=5 Participants	14 Participants n=7 Participants	26 Participants n=5 Participants

PRIMARY outcome

Timeframe: 28 days

Population: A participant is evaluable for engraftment if the participant underwent transplant and either completed 28 days observation or engrafted. Of the 46 participants at baseline, one in CLINIMACS group did not undergo transplant and one in ISOLEX group died 5 days after transplant.Thus, 2 participants were not included in the analysis.

Percentage of participants with hematopoietic engraftment post-transplant. Engraftment is defined as the first day absolute neutrophil counts exceeded 0.5 X 10\^9/ml.

Outcome measures

Outcome measures
Measure	CLINIMACS Device n=17 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the CLINIMACS Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	ISOLEX Device n=27 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the ISOLEX Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0
Engraftment Rate After Transplant	100 percentage of participants Interval 80.5 to 100.0	100 percentage of participants Interval 87.2 to 100.0

SECONDARY outcome

Timeframe: 100 Days

Population: Of the 46 participants at baseline, one participant in CLINIMACS Device group did not undergo haploidentical stem cell transplant and was not included in this analysis.

Number of participants who experience organ failure or severe infections (defined as Grade III/IV by NCI CTC for Adverse Events (CTCAE), version 2.0)

Outcome measures

Outcome measures
Measure	CLINIMACS Device n=17 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the CLINIMACS Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	ISOLEX Device n=28 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the ISOLEX Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0
Early Post BMT Toxicities	14 Participants	14 Participants

SECONDARY outcome

Timeframe: 100 Days

Population: A participant is evaluable for acute GVHD if the participant engrafted and either completed 100 days observation after transplant or experienced acute GVHD. Of the 46 participants at baseline, eight participants in CLINIMACS and five in ISOLEX were not evaluable for acute GVHD and were not included in this analysis.

Percentage of participants with Grade III/IV acute GVHD. Severe GVHD is defined as Grade III/IV acute GVHD.

Outcome measures

Outcome measures
Measure	CLINIMACS Device n=10 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the CLINIMACS Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	ISOLEX Device n=23 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the ISOLEX Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0
Severe GVHD Rate	0 percentage of participants Interval 0.0 to 30.8	4.3 percentage of participants Interval 0.1 to 21.9

SECONDARY outcome

Timeframe: First 100 Days

Number of participants with acute GVHD graded by the method of Przepiorka et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD

Outcome measures

Outcome measures
Measure	CLINIMACS Device n=10 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the CLINIMACS Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	ISOLEX Device n=23 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the ISOLEX Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0
Patients With Acute GVHD Grade 0	7 Participants	16 Participants
Patients With Acute GVHD Grade I	3 Participants	6 Participants
Patients With Acute GVHD Grade II	0 Participants	0 Participants
Patients With Acute GVHD Grade III	0 Participants	0 Participants
Patients With Acute GVHD Grade IV	0 Participants	1 Participants

SECONDARY outcome

Timeframe: Up to 1 Year

Population: Patients surviving more than 100 days were evaluable for chronic GvHD. Of the 46 participants at baseline, eight participants in CLINIMACS and six in ISOLEX were not evaluable for chronic GVHD and were not included in this analysis.

Number of participants with chronic GVHD graded by the method of Przepiorka et al, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3.

Outcome measures

Outcome measures
Measure	CLINIMACS Device n=10 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the CLINIMACS Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	ISOLEX Device n=22 Participants Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the ISOLEX Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0
Patients With Chronic GVHD No	10 Participants	20 Participants
Patients With Chronic GVHD Yes	0 Participants	2 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 Year

To evaluate the effect of T-cell depletion by positive selection for CD34 on immune reconstitution, which will be determined from total lymphocyte count, from T and B cell numbers and from T cell subset analyses.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 1 Year

Median length of remission in patients with high risk leukemia treated with myeloablative chemotherapy, radiotherapy and CD34 selected peripheral blood stem cells from haploidentical related donors using Kaplan-Meier method

Outcome measures

Outcome data not reported

Adverse Events

CLINIMACS Device

Serious events: 14 serious events

Other events: 13 other events

Deaths: 2 deaths

ISOLEX Device

Serious events: 15 serious events

Other events: 23 other events

Deaths: 4 deaths

Serious adverse events

Other adverse events

Additional Information

Dr. Robert A. Krance

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

Measure	CLINIMACS Device n=18 participants at risk Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the CLINIMACS Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	ISOLEX Device n=28 participants at risk Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the ISOLEX Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0
Cardiac disorders Hypotension	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Cardiac disorders Sinus tachycardia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Cardiac disorders Ventricular Arrhythmia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Gastrointestinal disorders Colitis	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Gastrointestinal disorders Gastrointestinal-Other: Appendicitis	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Gastrointestinal disorders Stomatitis/pharyngitis (oral/pharyngeal mucositis)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Gastrointestinal disorders pharyngeal ulcers	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Renal and urinary disorders Hematuria (in the absence of vaginal bleeding)	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Blood and lymphatic system disorders Hemorrhage/bleeding with grade 3 or 4 thrombocytopenia	11.1% 2/18 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Hepatobiliary disorders Liver Dysfunction/Failure (clinical)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Hepatobiliary disorders SGOT (AST) (serum glutamic oxaloacetic transaminase)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	7.1% 2/28 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Hepatobiliary disorders SGPT (ALT) (serum glutamic pyruvic transaminase)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Infections and infestations Catheter-Related Infection	11.1% 2/18 • Number of events 3 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	7.1% 2/28 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Infections and infestations Infection/Febrile Neutropenia-Other: HHV6 viremia	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Infections and infestations Infection(documented clinically or microbiologically)with grade 3 or 4 neutropenia(ANC <1.0 x 10e9/L	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Infections and infestations Infection without neutropenia	55.6% 10/18 • Number of events 14 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	21.4% 6/28 • Number of events 8 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Hyperglycemia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Hypokalemia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	7.1% 2/28 • Number of events 4 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Nervous system disorders Ataxia (incoordination)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Nervous system disorders Neuropathy - sensory	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Respiratory, thoracic and mediastinal disorders Adult Respiratory Distress Syndrome(ARDS)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Respiratory, thoracic and mediastinal disorders Hypoxia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Respiratory, thoracic and mediastinal disorders Pneumothorax	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Respiratory, thoracic and mediastinal disorders Pulmonary-Other: Acute respiratory failure	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Respiratory, thoracic and mediastinal disorders Pulmonary-Other: Mediastinal hematoma after replacing central line.	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Respiratory, thoracic and mediastinal disorders Pulmonary-Other: Respiratory failure	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Renal and urinary disorders Renal/Genitourinary-Other: Hemorrhagic Cystitis	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	7.1% 2/28 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Renal and urinary disorders Renal/Genitourinary-Other: Hydronephrosis	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Renal and urinary disorders Renal/Genitourinary-Other: Nephrolithiasis	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Renal and urinary disorders Renal Failure	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.

Measure	CLINIMACS Device n=18 participants at risk Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the CLINIMACS Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0	ISOLEX Device n=28 participants at risk Subjects will receive transplant conditioning with Ara-C, Cyclophosphamide, Campath-1H, Total Body Irradiation and will then receive T cell depleted stem cell infusion processed by the ISOLEX Device Ara-C: day-8 through day-5 3 g/m2 q 12 hours Cyclophosphamide: day-7 and day-6 45 mg/kg Campath-1H: day-3 through day-1 Dosing for children: 5 - 15kg : 3mg IV in 30ml NS 15.1 - 30kg : 5mg IV in 50ml NS \>30 kg : 10mg IV in 100ml NS Adults will receive 10mg IV in 100ml NS Total Body Irradiation: day-4 through day-1 175 cGy x 2 at 24 cGy/min Stem Cell Infusion: Stem cells are infused on day 0
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	11.1% 2/18 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
General disorders Fatigue (lethargy, malaise, asthenia)	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Skin and subcutaneous tissue disorders Rash/Desquamation	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Anorexia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	7.1% 2/28 • Number of events 3 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Gastrointestinal disorders Diarrhea patients without colostomy	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	7.1% 2/28 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Gastrointestinal disorders Stomatitis/pharyngitis (oral/pharyngeal mucositis)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Gastrointestinal disorders Vomiting	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Reproductive system and breast disorders Vaginal bleeding	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Investigations GGT (Gamma-Glutamyl transpeptidase)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	25.0% 7/28 • Number of events 10 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Hepatobiliary disorders Hepatic enlargement	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	7.1% 2/28 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Hepatobiliary disorders Portal vein flow	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Investigations SGOT (AST) (serum glutamic oxaloacetic transaminase)	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	10.7% 3/28 • Number of events 4 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Investigations SGPT (ALT) (serum glutamic pyruvic transaminase)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	10.7% 3/28 • Number of events 7 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Infections and infestations Catheter-Related Infection	11.1% 2/18 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	21.4% 6/28 • Number of events 11 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Infections and infestations Infection/Febrile Neutropenia -Other: Parainfluenza	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Infections and infestations Infection(documented clinically or microbiologically)with grade 3 or 4 neutropenia(ANC <1.0 x 10e9/L	50.0% 9/18 • Number of events 10 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	21.4% 6/28 • Number of events 8 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Infections and infestations Infection without neutropenia	11.1% 2/18 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Acidosis (metabolic or respiratory)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Alkalosis (metabolic or respiratory)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Hypercalcemia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Hyperglycemia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	10.7% 3/28 • Number of events 3 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Hypernatremia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Hypocalcemia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Hypokalemia	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	39.3% 11/28 • Number of events 18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Hyponatremia	16.7% 3/18 • Number of events 4 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	10.7% 3/28 • Number of events 3 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Metabolism and nutrition disorders Hypophosphatemia	16.7% 3/18 • Number of events 5 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	10.7% 3/28 • Number of events 4 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Investigations Lipase	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	7.1% 2/28 • Number of events 3 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Musculoskeletal and connective tissue disorders Muscle Weakness	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Psychiatric disorders Confusion	5.6% 1/18 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	0.00% 0/28 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Gastrointestinal disorders Abdominal Pain or Cramping	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Musculoskeletal and connective tissue disorders Bone Pain	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Musculoskeletal and connective tissue disorders Myalgia (muscle pain)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Respiratory, thoracic and mediastinal disorders Pleural Effusion (non-malignant)	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Respiratory, thoracic and mediastinal disorders Pneumonitis/Pulmonary Infiltrates	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Respiratory, thoracic and mediastinal disorders Pulmonary-Other: Sinusitis	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 2 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Investigations BUN	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.
Investigations Creatinine	0.00% 0/18 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.	3.6% 1/28 • Number of events 1 • Adverse events were collected from the time participants started preparative regimen to 100 days post transplant.