SCID Bu/Flu/ATG Study With T Cell Depletion

• Sponsor: Neena Kapoor, M.D. (Other)
• Overall Status: Terminated
• CT.gov ID: NCT02127892
• Collaborator: (none)
• Enrollment: 9
• Location: 1
• Arms: 4
• Actual Duration (Months): 115
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with a diagnosis of Severe Combined Immune Deficiency (SCID) who do not have an HLA-matched sibling donor. The stem cells will be derived from a 1) matched unrelated donor (MUD), 2) unrelated cord blood donor, or 3) a haplo-identical (parental) donor (in descending order of preference).Patients will receive a novel conditioning regimen with Busulfan, Fludarabine and Anti-thymocyte globulin (ATG) followed by an unrelated donor hematopoietic stem cell transplant (HSCT) with T-cell depletion using the CliniMACS device.

Condition or Disease	Intervention/Treatment	Phase
Severe Combined Immunodeficiency	Biological: unrelated BM with T cell depletion; Biological: unrelated cord blood; Biological: haplo BM with T cell depletion; Device: unrelated PBSC with T cell depletion	Phase 1/Phase 2

Detailed Description

The study is being conducted to assess the following:

• overall survival

• event-free survival (events are defined as: death,non-engraftment/2nd transplant, immune reconstitution failure)

• acute toxicity of the conditioning regimen

• engraftment frequency immune reconstitution frequency and tempo acute and chronic graft-versus-host disease (GVHD), frequency and severity.

The outcome from this protocol will be compared to the retrospective cohort consisting of all patients who have undergone haplo-identical HSCT for SCID at CHLA from 1984-2006 based on the assessment of the above-listed endpoints.

The CliniMACS device will be used for CD34+ selection in place of the Isolex 300i. The CliniMACS CD34 Reagent System is an investigational medical device that has not yet been approved by the FDA. This device is used in vitro to select and enrich specific cell populations. When using the CliniMACS CD34 Reagent, the system selects CD34+ cells from heterogenous hematological cell populations for transplantation in cases where this is clinically indicated.

Study Design

• Study Type: Interventional
• Actual Enrollment: 9 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Phase I/II Trial of Hematopoietic Stem Cell Transplant (HSCT) for Children With Severe Combined Immune Deficiency (SCID) and Without an HLA-Matched Sibling Donor
• Actual Study Start Date: Jan 2, 2007
• Actual Primary Completion Date: Aug 1, 2016
• Actual Study Completion Date: Aug 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
Other: unrelated BM with T cell depletion Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.	Biological: unrelated BM with T cell depletion; Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).; Other Names: CD34+ selection using CliniMACS
Other: unrelated cord blood Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).	Biological: unrelated cord blood; Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.; Other Names: umbilical cord blood
Other: haplo BM with T cell depletion If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.	Biological: haplo BM with T cell depletion; haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.; Other Names: CD34+ selection with CliniMACS
Other: unrelated PBSC with T cell depletion The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.	Device: unrelated PBSC with T cell depletion; peripheral blood stem cell will be processed for CD34+ cell isolation.; Other Names: CD34+ selection using CliniMACS

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Engraftment [100 day]

   Engraftment is defined as recovery of blood counts (neutrophil and platelet engraftment) with cells of donor origin, documented by either bone marrow or peripheral blood chimerism assays after hematopoietic stem cell transplant.

Secondary Outcome Measures

1. Number of Participants With Donor-derived CD3+ T Lymphocytes >/= 100/mm3 [1 year]

   Absolute number of donor-derived CD3+ T lymphocytes >/= 100/mm3 in participating subjects.

Other Outcome Measures

1. Number of Participants With Veno-occlusive Disease (VOD) - Moderate and Severe [100 days]

   Evaluation of veno-occlusive disease determined by the presence of the following features; fluid retention, weight gain, leaky capillary syndrome, painful liver enlargement, refractoriness to platelet tranfusion and hyperbilirubinemia

2. Number of Participants With Graft Versus Host Disease (GVHD) - Grade III or IV [1 year]

   GVHD disease surveillance done by clinical evaluation, to include history, physical examination, specifically for rash, jaundice, liver dysfunction, nausea and vomiting, diarrhea and failure to thrive.

3. Overall Survival [1 year]

   Overalls survival of patient at 1 year post transplant

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 21 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• All patients with SCID who lack a histocompatible sibling or HLA-matched related donor will be considered as candidates for this study protocol.

• Eligible patients must have adequate physical function to tolerate the chemotherapy conditioning regimen and the HSCT, as measure by:

1. Renal: creatinine clearance or GFR ≥50 ml/min/1.73m2, and not requiring dialysis

2. Pulmonary: Because patients with SCID frequently present with infectious pneumonia causing ventilatory failure, patients will be considered for enrollment in the study even if respiratory failure requiring mechanical ventilatory support is present. In patients recently diagnosed with pneumonia, efforts to stabilize the respiratory status will be made prior to enrollment in the study.

3. Infectious disease status. The presence of infection per se will not be a reason for exclusion from the study. Patients with SCID are frequently infected with both routine pathogens as well as opportunistic infections. Antibiotic, antifungal and antiviral prophylaxis and therapy will be instituted as clinically indicated. Despite the use of antimicrobial therapy, the ability to control infections will not be achieved unless HSCT is performed. Therefore, subjects may be enrolled in the study, even though infection is present, because control of infection may depend on engraftment of a donor immune system.

4. Patients will be 0-21 years of age.

Exclusion Criteria:

• Patient with histocompatible sibling or other related donor

• End-organ failure that precludes the ability to tolerate the transplant procedure, including conditioning.

• Renal failure requiring dialysis

• Congenital heart disease resulting in congestive heart failure

• Severe CNS disease, e.g., coma or intractable seizures

• Ventilatory failure due to non-infectious etiology

• Major congenital anomalies that adversely affect survival, eg CNS malformations

• Metabolic diseases that would affect transplant survival, eg urea cycle disorders

• HIV infection

Since the only chance of survival for patients with SCID is successful transplantation, all patients with SCID will be considered to be potential subjects for the study, regardless of end-organ dysfunction.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital Los Angeles	Los Angeles	California	United States	90027

Sponsors and Collaborators

• Neena Kapoor, M.D.

Investigators

• Principal Investigator: Neena Kapoor, M.D., Children's Hospital Los Angeles, University of Southern California

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Neena Kapoor, M.D., Professor of Pediatrics, Keck School of Medicine; Division Head, Division of Research Immunology/BMT, Children's Hospital Los Angeles
• ClinicalTrials.gov Identifier: NCT02127892
• Other Study ID Numbers: CCI-06-00243
• First Posted: May 1, 2014
• Last Update Posted: Sep 18, 2017
• Last Verified: Aug 1, 2017

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: Yes
• Keywords provided by Neena Kapoor, M.D., Professor of Pediatrics, Keck School of Medicine; Division Head, Division of Research Immunology/BMT, Children's Hospital Los Angeles: Severe Combined Immune Deficiency; SCID; HLA-matched unrelated donor; unrelated; MUD; bone marrow; cord blood; haplo-identical; peripheral blood; CD34+ selection; T cell depletion; HSCT; BMT; CliniMACS
• Additional relevant MeSH terms: Severe Combined Immunodeficiency

Study Results

Participant Flow

Arm/Group Title	Unrelated BM With T Cell Depletion	Unrelated Cord Blood	Haplo BM With T Cell Depletion	Unrelated PBSC With T Cell Depletion
Arm/Group Description	Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles. unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).	Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles). unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.	If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors. haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.	The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed. unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
Period Title: Overall Study
STARTED	7	2	0	0
COMPLETED	6	1	0	0
NOT COMPLETED	1	1	0	0

Baseline Characteristics

Arm/Group Title	Unrelated BM With T Cell Depletion	Unrelated Cord Blood	Haplo BM With T Cell Depletion	Unrelated PBSC With T Cell Depletion	Total
Arm/Group Description	Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles. unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).	Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles). unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.	If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors. haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.	The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed. unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.	Total of all reporting groups
Overall Participants	7	2	0	0	9
Age (Count of Participants)
<=18 years	7 100%	2 100%	0 NaN	0 NaN	9 100%
Between 18 and 65 years	0 0%	0 0%	0 NaN	0 NaN	0 0%
>=65 years	0 0%	0 0%	0 NaN	0 NaN	0 0%
Sex: Female, Male (Count of Participants)
Female	1 14.3%	0 0%	1 Infinity
Male	6 85.7%	2 100%	8 Infinity
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	3 42.9%	2 100%	0 NaN	0 NaN	5 55.6%
Not Hispanic or Latino	3 42.9%	0 0%	0 NaN	0 NaN	3 33.3%
Unknown or Not Reported	1 14.3%	0 0%	0 NaN	0 NaN	1 11.1%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 NaN
Asian	1 14.3%	0 0%	1 Infinity
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 NaN
Black or African American	0 0%	0 0%	0 NaN
White	2 28.6%	0 0%	2 Infinity
More than one race	0 0%	0 0%	0 NaN
Unknown or Not Reported	4 57.1%	2 100%	6 Infinity
Region of Enrollment (participants) [Number]
United States	7 100%	2 100%	9 Infinity

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Engraftment
Description	Engraftment is defined as recovery of blood counts (neutrophil and platelet engraftment) with cells of donor origin, documented by either bone marrow or peripheral blood chimerism assays after hematopoietic stem cell transplant.
Time Frame	100 day

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Unrelated BM With T Cell Depletion	Unrelated Cord Blood	Haplo BM With T Cell Depletion	Unrelated PBSC With T Cell Depletion
Arm/Group Description	Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles. unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).	Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles). unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.	If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors. haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.	The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed. unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
Measure Participants	7	2	0	0
Count of Participants [Participants]	7 100%	2 100%

2. Secondary Outcome

Title	Number of Participants With Donor-derived CD3+ T Lymphocytes >/= 100/mm3
Description	Absolute number of donor-derived CD3+ T lymphocytes >/= 100/mm3 in participating subjects.
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Unrelated BM With T Cell Depletion	Unrelated Cord Blood	Haplo BM With T Cell Depletion	Unrelated PBSC With T Cell Depletion
Arm/Group Description	Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles. unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).	Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles). unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.	If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors. haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.	The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed. unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
Measure Participants	7	2	0	0
Count of Participants [Participants]	7 100%	1 50%	0 NaN	0 NaN

3. Other Pre-specified Outcome

Title	Number of Participants With Veno-occlusive Disease (VOD) - Moderate and Severe
Description	Evaluation of veno-occlusive disease determined by the presence of the following features; fluid retention, weight gain, leaky capillary syndrome, painful liver enlargement, refractoriness to platelet tranfusion and hyperbilirubinemia
Time Frame	100 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Unrelated BM With T Cell Depletion	Unrelated Cord Blood	Haplo BM With T Cell Depletion	Unrelated PBSC With T Cell Depletion
Arm/Group Description	Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles. unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).	Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles). unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.	If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors. haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.	The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed. unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
Measure Participants	7	2	0	0
Count of Participants [Participants]	2 28.6%	1 50%	0 NaN	0 NaN

4. Other Pre-specified Outcome

Title	Number of Participants With Graft Versus Host Disease (GVHD) - Grade III or IV
Description	GVHD disease surveillance done by clinical evaluation, to include history, physical examination, specifically for rash, jaundice, liver dysfunction, nausea and vomiting, diarrhea and failure to thrive.
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Unrelated BM With T Cell Depletion	Unrelated Cord Blood	Haplo BM With T Cell Depletion	Unrelated PBSC With T Cell Depletion
Arm/Group Description	Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles. unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).	Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles). unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.	If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors. haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.	The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed. unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
Measure Participants	7	2	0	0
Count of Participants [Participants]	1 14.3%	0 0%	0 NaN	0 NaN

5. Other Pre-specified Outcome

Title	Overall Survival
Description	Overalls survival of patient at 1 year post transplant
Time Frame	1 year

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Unrelated BM With T Cell Depletion	Unrelated Cord Blood	Haplo BM With T Cell Depletion	Unrelated PBSC With T Cell Depletion
Arm/Group Description	Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles. unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).	Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles). unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.	If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors. haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.	The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed. unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
Measure Participants	7	2	0	0
Count of Participants [Participants]	6 85.7%	1 50%	0 NaN	0 NaN

Adverse Events

Time Frame	Adverse event data was collected over a period of 1 year post transplant.
Arm/Group Title	Unrelated BM With T Cell Depletion		Unrelated Cord Blood		Haplo BM With T Cell Depletion		Unrelated PBSC With T Cell Depletion
Arm/Group Description	Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles. unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).		Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles). unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.		If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors. haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.		The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed. unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.
All Cause Mortality
	Unrelated BM With T Cell Depletion		Unrelated Cord Blood		Haplo BM With T Cell Depletion		Unrelated PBSC With T Cell Depletion
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/7 (14.3%)		1/2 (50%)		0/0 (NaN)		0/0 (NaN)
Serious Adverse Events
	Unrelated BM With T Cell Depletion		Unrelated Cord Blood		Haplo BM With T Cell Depletion		Unrelated PBSC With T Cell Depletion
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/7 (14.3%)		1/2 (50%)		0/0 (NaN)		0/0 (NaN)
Hepatobiliary disorders
Sinusoidal obstruction syndrome	1/7 (14.3%)	1	1/2 (50%)	1	0/0 (NaN)	0	0/0 (NaN)	0
Other (Not Including Serious) Adverse Events
	Unrelated BM With T Cell Depletion		Unrelated Cord Blood		Haplo BM With T Cell Depletion		Unrelated PBSC With T Cell Depletion
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	5/7 (71.4%)		1/2 (50%)		0/0 (NaN)		0/0 (NaN)
Infections and infestations
Infection - Aspergillis	1/7 (14.3%)	1	1/2 (50%)	1	0/0 (NaN)	0	0/0 (NaN)	0
Infection - Fungal	1/7 (14.3%)	1	0/2 (0%)	0	0/0 (NaN)	0	0/0 (NaN)	0
Nervous system disorders
Seizure	1/7 (14.3%)	1	0/2 (0%)	0	0/0 (NaN)	0	0/0 (NaN)	0
Respiratory, thoracic and mediastinal disorders
Pulmonary Hemorrhage	0/7 (0%)	0	1/2 (50%)	1	0/0 (NaN)	0	0/0 (NaN)	0
Skin and subcutaneous tissue disorders
Graft Versus Host Disease	5/7 (71.4%)	5	1/2 (50%)	1	0/0 (NaN)	0	0/0 (NaN)	0

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.