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Combined T Cell Depleted Haploidentical Peripheral Blood Stem Cell and Unrelated Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies Using a Total Lymphoid Irradiation Based Preparative Regimen

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ClinicalTrials.gov Identifier: NCT02199041
Recruitment Status : Terminated (The study was halted early due to slow accrual.)
First Posted : July 24, 2014
Results First Posted : February 7, 2018
Last Update Posted : February 7, 2018
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hematological Malignancies
Interventions Drug: Cyclophosphamide
Drug: Thiotepa
Drug: Fludarabine
Drug: Melphalan
Drug: Mesna
Biological: G-CSF
Drug: Mycophenolate mofetil
Drug: Tacrolimus
Drug: Methylprednisolone
Radiation: Total lymphoid irradiation
Biological: Lymphocyte infusions
Device: CliniMACS
Enrollment 24
Recruitment Details Twelve participants meeting eligibility criteria and 12 of their blood donors were enrolled at St. Jude Children's Research Hospital between July 2014 and January 2015.
Pre-assignment Details The 12 enrolled blood donors did not undergo transplantation and are therefore not included in the results reported here.
Arm/Group Title Treatment
Hide Arm/Group Description

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, granulocyte colony-stimulating factor (G-CSF), mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Period Title: Overall Study
Started 12
Completed 12
Not Completed 0
Arm/Group Title Treatment
Hide Arm/Group Description

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Overall Number of Baseline Participants 12
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 12 participants
8.7  (7.5)
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 12 participants
5.0
(0.7 to 20.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Female
7
  58.3%
Male
5
  41.7%
Race/Ethnicity, Customized   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Ethnicity Number Analyzed 12 participants
NOS Spanish, Hispanic, Latino
1
   8.3%
Non Spanish Speaking, Non Hispanic
8
  66.7%
Puerto Rican
1
   8.3%
South or Central American
2
  16.7%
[1]
Measure Description: NOS = Not Otherwise Specified
Race/Ethnicity, Customized   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Race Number Analyzed 12 participants
Black
3
  25.0%
Multiple Race (NOS)
1
   8.3%
White
8
  66.7%
[1]
Measure Description: NOS = Not Otherwise Specified
Diagnosis   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Acute Erythroleukemia, FAB M6, BM
1
   8.3%
AL, Lymphoblastic, Pre-B, BM
2
  16.7%
AL, Lymphoblastic, Pre-T Cell, BM
1
   8.3%
AL, Megakaryoblastic, FAB M7, BM
2
  16.7%
AL, Monocytic, FAB M5, BM
1
   8.3%
AL, Myeloid, Minimal Differentiation, FAB M0, BM
1
   8.3%
AL, Myeloid, NOS, BM
2
  16.7%
AL, Myeloid, With Maturation, FAB M2, BM
1
   8.3%
Lymphoma, Non-Hodgkin's, Anaplastic Large Cell
1
   8.3%
[1]
Measure Description: AL = Acute Leukemia; BM = Bone Marrow
Disease Status at HSCT   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
CR1
2
  16.7%
CR2
3
  25.0%
CR3
2
  16.7%
Refractory
4
  33.3%
Relapse 2
1
   8.3%
[1]
Measure Description: Complete remission (CR) defined as less than 5 percent leukemia blasts in a bone marrow with evidence of hematopoietic recovery. Relapse is defined as return of 5 percent leukemia blasts or more in a bone marrow after previous achieving CR. Refractory is defined as never achieving CR despite therapy, also known as induction failure. The disease status worsens going down the list with CR1 indicating the best prognosis and Relapse 2 the worst prognosis.
1.Primary Outcome
Title Number of Participants With Neutrophil Engraftment
Hide Description Neutrophil engraftment is defined as absolute neutrophil count (ANC) recovery of ≥ 0.5 x 10^9/L (500/mm^3) for three consecutive laboratory values obtained on different days (derived from either donor). Date of engraftment is the date of the first of the three consecutive laboratory values. The number of patients engrafted by day +42 post-transplant is provided.
Time Frame Until day 42 post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Overall Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
11
2.Secondary Outcome
Title Number of Participants With Malignant Relapse
Hide Description

Relapse was evaluated using standard World Health Organization (WHO) criteria for each disease. The estimate of cumulative incidence of relapse will be estimated using Kalbfleisch-Prentice method. Relapse defined as the recurrence of original disease. Death is the competing risk event. The analysis will be implemented using Statistical Analysis System (SAS) macro (bmacro252-Excel2007\cin).

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced malignant relapse is provided

Time Frame One year after transplantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Overall Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
7
3.Secondary Outcome
Title Number of Participants With Event-free Survival (EFS)
Hide Description

The Kaplan-Meier estimate of EFS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007\kme) available in the Department of Biostatistics at St. Jude, where EFS = min (date of last follow-up, date of relapse, date of graft failure, date of death due to any cause) – date of transplant, and all participants surviving at the time of analysis without events will be censored. The number of participants who did not experience any of these events through one year post-transplant is given.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not experience any events defined above is provided.

Time Frame One year after transplantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Overall Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
4
4.Secondary Outcome
Title Number of Participants With Overall Survival (OS)
Hide Description

The Kaplan-Meier estimate of OS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007\kme) available in the Department of Biostatistics at St. Jude, where OS = min (date of last follow-up, date of death) – date of hematopoietic cell transplantation (HCT) and all participants surviving after 1 year post-transplant will be considered as censored.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not die at 1 year post-transplant is provided.

Time Frame One year after transplantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Overall Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
6
5.Secondary Outcome
Title Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
Hide Description

Cumulative incidence of acute GVHD was estimated using Kalbfleisch-Prentice method. Death is the competing risk event. SAS macro (bmacro252-Excel2007\cin) available at St. Jude was used for analysis. Severity of GVHD and stage were determined using the Clinical Oncology Group (COG) Stem Cell Committee Consensus Guidelines for establishing organ stage and overall grade of acute GVHD. Participants are graded on a scale from I to IV, with I being mild and IV being severe.

Overall Clinical Grade (based on the highest stage obtained):

Grade 0: No stage 1-4 of any organ. Grade I: Stage 1-2 skin and no liver or gut involvement. Grade II: Stage 3 skin, or Stage I liver involvement, or Stage 1 gastrointestinal (GI).

Grade III: Stage 0-3 skin, with Stage 2-3 liver, or Stage 2-3 GI. Grade IV: Stage 4 skin, liver or GI involvement.

Due to early close of study, a small number of patients were enrolled. The number of patients who experienced acute GVHD is provided.

Time Frame 100 days after transplantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Overall Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
No Acute GVHD 8
Grade I 0
Grade II 1
Grade III 2
Grade IV 1
6.Secondary Outcome
Title Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
Hide Description

Cumulative incidence of acute and chronic GVHD was estimated using Kalbfleisch-Prentice method. Death is competing risk event. SAS macro (bmacro252-Excel2007\cin) available St. Jude was used for such analysis. Severity of chronic GVHD was evaluated using National Institutes of Health (NIH) Consensus Global Severity Scoring. Mild is considered a better outcome with severe being the worst.

Criteria for grading chronic GVHD:

Mild: 1-2 organs/sites, maximum organ score of 1, and lung score of 1. Moderate: 3 or more organs/sites and maximum organ score of 1 and lung score of 1, OR at least 1 organ/site and maximum organ score of 2 and lung score of 1. Severe: At least 1 organ/site and maximum organ score of 3 and lung score of 2-3.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced chronic GVHD is provided.

Time Frame 100 days after transplantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Overall Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
No Chronic GVHD 11
Mild 0
Moderate 0
Severe 1
7.Secondary Outcome
Title Number of Participants With Secondary Graft Failure
Hide Description

The cumulative incidence of secondary graft failure will be estimated using the Kalbfleisch-Prentice method. Deaths due to toxicity and relapse before day 100 are the competing events.

Secondary graft failure or graft rejection will be defined as no evidence of donor chimerism by umbilical cord blood (UCB) and/or haploidentical donor (<10%), or too few cells to perform adequate chimerism analysis, in research participants with prior neutrophil engraftment.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced secondary graft failure is provided.

Time Frame 100 days after transplantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Overall Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
0
8.Secondary Outcome
Title Number of Participants With Transplant-related Mortality (TRM)
Hide Description

TRM is any death in remission and related to protocol therapy. The cumulative incidence of TRM was estimated using the Kalbfleisch-Prentice method. Deaths before day 100 because of other reasons are the completing events.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced TRM is provided.

Time Frame 100 days after transplantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Overall Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
0
9.Secondary Outcome
Title Number of Participants With Transplant-related Morbidity
Hide Description

Any patient who had adverse events listed either as probable or definite in the first 100 days post-transplant are counted as transplant-related morbidity. The cumulative incidence of transplant-related morbidity will be estimated using the Kalbfleisch-Prentice method. Deaths before day 100 are the competing risk events.

Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced at least one-transplant-related morbidity is provided.

Time Frame 100 days after transplantation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment
Hide Arm/Group Description:

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Overall Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
12
Time Frame Adverse events were collected from participants beginning with the on-study date through one year post-transplantation.
Adverse Event Reporting Description Adverse events were monitored for donors beginning with the on-study date through one year post-transplantation.
 
Arm/Group Title Treatment Blood Donors
Hide Arm/Group Description

Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, G-CSF, mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.

Cells for infusion are prepared using the CliniMACS System.

Prior to stem cell infusion, participants receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.

Blood donors were enrolled on the study to provide cells for transplantation used in the Treatment Arm.
All-Cause Mortality
Treatment Blood Donors
Affected / at Risk (%) Affected / at Risk (%)
Total   6/12 (50.00%)      0/12 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Treatment Blood Donors
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/12 (41.67%)      0/12 (0.00%)    
Blood and lymphatic system disorders     
Thrombotic Thrombocytopenic Purpura * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Cardiac disorders     
Hypotension * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Pericardial Effusion * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Gastrointestinal disorders     
Diarrhea * 1  1/12 (8.33%)  2 0/12 (0.00%)  0
General disorders     
Fever without Neutropenia * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infections and infestations     
Infection, Enterobacter Cloacae, Blood * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Fungal and Viral, Pneumonia * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Staphylococcus Aureus, Blood * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Pneumonia * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Pneumonia, Staph Aureus * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Investigations     
Weight Loss * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Nervous system disorders     
Seizure * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
1
Term from vocabulary, CTCAE (3.0)
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Treatment Blood Donors
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   12/12 (100.00%)      0/12 (0.00%)    
Blood and lymphatic system disorders     
Disseminated Intravascular Coagulation * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Cardiac disorders     
Hypertension * 1  6/12 (50.00%)  8 0/12 (0.00%)  0
Left Ventricular Cardiac Dysfunction (Disorder) * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Pericardial Effusion * 1  3/12 (25.00%)  4 0/12 (0.00%)  0
Gastrointestinal disorders     
Colitis (Disorder) * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Dehydration * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Diarrhea * 1  2/12 (16.67%)  2 0/12 (0.00%)  0
Esophagitis * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Hypernatremic Dehydration (Disorder) * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Mucositis * 1  2/12 (16.67%)  2 0/12 (0.00%)  0
Nausea * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Vomiting (Disorder) * 1  2/12 (16.67%)  2 0/12 (0.00%)  0
General disorders     
Pain, Abdomen * 1  2/12 (16.67%)  2 0/12 (0.00%)  0
Pain, Neuropathic, Legs and Feet, Bilateral * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Hepatobiliary disorders     
Veno-Occlusive Disease, Hepatic * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Immune system disorders     
Acute Infusion Reaction, Stem Cells * 1  2/12 (16.67%)  2 0/12 (0.00%)  0
Allergic Reaction, Platelet Transfusion * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Allergy to Diary Product (Disorder) * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Engraftment Syndrome * 1  2/12 (16.67%)  2 0/12 (0.00%)  0
Infections and infestations     
Cellulitis, Gastrostomy Site * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Fever without Neutropenia * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Febrile Neutropenia * 1  12/12 (100.00%)  13 0/12 (0.00%)  0
Hepatitis * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Adenovirus, Respiratory Tract * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Adenovirus, Stool * 1  4/12 (33.33%)  5 0/12 (0.00%)  0
Infection, BK Virus, Blood * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, BK Virus, Urine * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, CMV, Respiratory/Nasopharynx * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Candida Albicans, Oral Mucosa * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Candida Lipolytica, Oral Mucosa * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Clostridium Difficile Colitis * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Clostridium Difficile, Stool * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Cytomegalovirus, Blood * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Due to Klebsiella, Bone Marrow Site * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Enterobacter Asburiae, Blood * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Enterobacter Cloacae, Blood * 1  1/12 (8.33%)  2 0/12 (0.00%)  0
Infection, Enterobacter asburiae and Coagulase Negative Staphylococcus, Gastrostomy Wound Site * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Fungal, Liver * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Herpes Simplex Virus, Oral Mucosa * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Human Herpes Simplex Virus, Oral * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Human Herpes Virus 6, Blood * 1  8/12 (66.67%)  8 0/12 (0.00%)  0
Infection, Human Herpes Virus 6, Cerebrospinal Fluid * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Klebsiella pneumoniae, Colon * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Malassezia Furfur, Blood * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Norovirus, Stool * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Pediococcus Pentosaceua * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Presumed Viral, Oral Mucosa * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Infection, Rotavirus, Stool * 1  3/12 (25.00%)  6 0/12 (0.00%)  0
Infection, Staphylococcus Epidermidis, Blood * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Reactivation, Human Herpes Virus 6, Blood * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Hypoxia * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Ulceration, Larynx * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Investigations     
Weight Loss * 1  2/12 (16.67%)  2 0/12 (0.00%)  0
Metabolism and nutrition disorders     
Hyperglycemia * 1  2/12 (16.67%)  3 0/12 (0.00%)  0
Psychiatric disorders     
Anxiety * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Renal and urinary disorders     
Acute renal failure * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Renal Insufficiency * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Renal, Acute Kidney Injury * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Adult Respiratory Distress Syndrome * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Skin and subcutaneous tissue disorders     
Rash * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
Rash, Skin * 1  1/12 (8.33%)  1 0/12 (0.00%)  0
1
Term from vocabulary, CTCAE (3.0)
*
Indicates events were collected by non-systematic assessment
The study was halted early due to slow accrual.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Brandon Triplett, MD
Organization: St. Jude Children's Research Hospital
Phone: 901-595-2766
Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT02199041     History of Changes
Other Study ID Numbers: HAPCORD
NCI-2014-00526 ( Registry Identifier: NCI Clinical Trial Registration Program )
First Submitted: July 9, 2014
First Posted: July 24, 2014
Results First Submitted: August 1, 2017
Results First Posted: February 7, 2018
Last Update Posted: February 7, 2018