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Trial record 3 of 5 for:    GATA2 deficiency

Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00923364
Recruitment Status : Completed
First Posted : June 18, 2009
Results First Posted : November 30, 2017
Last Update Posted : November 30, 2017
Sponsor:
Information provided by (Responsible Party):
Dennis Hickstein, M.D., National Institutes of Health Clinical Center (CC)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions MDS
Immunodeficiency
GATA2
Interventions Drug: Cyclophosphamide (CTX, Cytoxan)
Drug: Fludarabine(Fludara,Berlex Laboratories)
Procedure: Total Body Irradiation (TBI)
Procedure: Allogeneic Hematopoietic Stem Cell (HSC)
Drug: Equine Anti-Thymocyte Globulin
Enrollment 19
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Recipients Healthy Related Donors
Hide Arm/Group Description

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2

Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients)

Allogeneic hematopoietic stem cell (HSC): stem cell transplant

[Not Specified]
Period Title: Overall Study
Started 14 5
Completed 10 5
Not Completed 4 0
Reason Not Completed
Death             3             0
Did not go to transplant             1             0
Arm/Group Title Recipients and Healthy Related Donors
Hide Arm/Group Description

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2

Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients)

Allogeneic hematopoietic stem cell (HSC): stem cell transplant Healthy related donors =5; recipients = 14.

Overall Number of Baseline Participants 19
Hide Baseline Analysis Population Description
Data were not collected separately for donors and recipients.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants
<=18 years
1
   5.3%
Between 18 and 65 years
18
  94.7%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 19 participants
30.21  (7.61)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants
Female
11
  57.9%
Male
8
  42.1%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants
Hispanic or Latino
3
  15.8%
Not Hispanic or Latino
16
  84.2%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
  10.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
2
  10.5%
White
15
  78.9%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 19 participants
19
 100.0%
1.Primary Outcome
Title Days to Neutrophil Engraftment
Hide Description Neutrophil engraftment is defined as a neutrophil count of >0.5 x 10(9) cells/L for 3 consecutive days.
Time Frame 30 days
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
10/19 analyzed:5 pts were healthy related donors, 1 pt taken off study prior to transplant & 3 pts had early deaths (before outcome measurements could be evaluated)
Arm/Group Title Recipients Only
Hide Arm/Group Description:

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2 Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients) Allogeneic hematopoietic stem cell (HSC): stem cell transplant

Overall Number of Participants Analyzed 10
Median (Full Range)
Unit of Measure: Days
12
(12 to 80)
2.Primary Outcome
Title Days to Platelet Engraftment
Hide Description Platelet engraftment is defined as a platelet count of >20 x 10(9) cells/L for 7 consecutive days without requiring a platelet transfusion.
Time Frame 30 days
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
10/19 analyzed:5 pts were healthy related donors, 1 pt taken off study prior to transplant & 3 pts had early deaths (before outcome measurements could be evaluated)
Arm/Group Title Recipients Only
Hide Arm/Group Description:

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2 Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients) Allogeneic hematopoietic stem cell (HSC): stem cell transplant

Overall Number of Participants Analyzed 10
Median (Full Range)
Unit of Measure: Days
30
(0 to 30)
3.Secondary Outcome
Title Percentage of Donor Cells at Last Follow Up in Patients With Mutations GATA Binding Protein 2 (GATA2)
Hide Description Engraftment of donor cells was assessed using polymorphisms in regions known to contain short tandem repeats. Peripheral blood cluster of differentiation 14 (CD14+), cluster of differentiation 3 (CD3-)/cluster of differentiation 56 (CD56+), cluster of differentiation 19 (CD19+), and CD3+ subsets were isolated by flow cytometry and chimerism was assessed.
Time Frame 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
10/19 analyzed:5 pts were healthy related donors, 1 pt taken off study prior to transplant & 3 pts had early deaths (before outcome measurements could be evaluated)
Arm/Group Title Recipients Only
Hide Arm/Group Description:

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2 Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients) Allogeneic hematopoietic stem cell (HSC): stem cell transplant

Overall Number of Participants Analyzed 10
Mean (Full Range)
Unit of Measure: percentage of donor cells
Donor CD14+ cells, %
99.8
(99 to 100)
Donor CD19+ cells, %
100
(100 to 100)
Donor CD3-/CD56+ cells, %
99.8
(99 to 100)
Donor CD3+ cells, %
97.6
(91 to 100)
4.Secondary Outcome
Title Incidence of Acute and Chronic Graft-versus-host Disease (GVHD)
Hide Description Acute GVHD is assessed according to the 1994 Consensus Conference Grading Criteria. Chronic GVHD is assessed by the 2005 Chronic GVHD Consensus Project. GVHD can affect performance status and attack multiple organ systems such as the skin, liver, gut, mouth, eyes, joints, lung, etc. that can lead to a rash, diarrhea, metabolic changes, infection and/or death. The clinical grading of acute GVHD is grade 0 (none) to 4 (severe). Chronic GVHD is a delayed form of GVHD that may occur after day 100 post transplant.
Time Frame 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
10/19 analyzed:5 pts were healthy related donors, 1 pt taken off study prior to transplant & 3 pts had early deaths (before outcome measurements could be evaluated)
Arm/Group Title Recipients Only
Hide Arm/Group Description:

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2 Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients) Allogeneic hematopoietic stem cell (HSC): stem cell transplant

Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: participants
Acute 2
Chronic 3
5.Secondary Outcome
Title Overall Survival
Hide Description Overall survival is defined as date of on-study to date of death from any cause or last follow up.
Time Frame 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
10/19 analyzed:5 pts were healthy related donors, 1 pt taken off study prior to transplant & 3 pts had early deaths (before outcome measurements could be evaluated)
Arm/Group Title Recipients Only
Hide Arm/Group Description:

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2 Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients) Allogeneic hematopoietic stem cell (HSC): stem cell transplant

Overall Number of Participants Analyzed 10
Median (Full Range)
Unit of Measure: months
76
(18 to 95)
6.Secondary Outcome
Title Number of Participants With Disease Free Survival
Hide Description Number of participants with documented evidence of disease progression following start of treatment.
Time Frame 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
10/19 analyzed:5pts were healthy related donors, 1 pt taken off study prior to transplant & 3 pts had early deaths (before outcome measurements could be evaluated)
Arm/Group Title Recipients Only
Hide Arm/Group Description:

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2 Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients) Allogeneic hematopoietic stem cell (HSC): stem cell transplant

Overall Number of Participants Analyzed 10
Measure Type: Count of Participants
Unit of Measure: Participants
8
  80.0%
7.Secondary Outcome
Title Number of Participants With Serious and Non-serious Adverse Events
Hide Description Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events v3.0. A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.
Time Frame 91 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data were not collected separately for donors and recipients.
Arm/Group Title Recipients and Healthy Related Donors
Hide Arm/Group Description:

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2 Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients) Allogeneic hematopoietic stem cell (HSC): stem cell transplant Healthy related donors =5; recipients = 14.

Overall Number of Participants Analyzed 19
Measure Type: Count of Participants
Unit of Measure: Participants
16
  84.2%
Time Frame 91 months
Adverse Event Reporting Description Data were not collected separately for donors and recipients.
 
Arm/Group Title Recipients and Healthy Related Donors
Hide Arm/Group Description

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2 Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients) Allogeneic hematopoietic stem cell (HSC): stem cell transplant Healthy related donors =5; recipients = 14.

All-Cause Mortality
Recipients and Healthy Related Donors
Affected / at Risk (%)
Total   4/19 (21.05%)    
Show Serious Adverse Events Hide Serious Adverse Events
Recipients and Healthy Related Donors
Affected / at Risk (%) # Events
Total   12/19 (63.16%)    
Blood and lymphatic system disorders   
Leukocytes (total WBC)  1  1/19 (5.26%)  3
Lymphopenia  1  2/19 (10.53%)  2
Neutrophils/granulocytes (ANC/AGC)  1  1/19 (5.26%)  1
Platelets  1  2/19 (10.53%)  8
Cardiac disorders   
Cardiac troponin I (cTnI)  1  1/19 (5.26%)  1
Cardiopulmonary arrest, cause unknown (non-fatal)  1  1/19 (5.26%)  1
Hypotension  1  2/19 (10.53%)  2
Left ventricular systolic dysfunction  1  1/19 (5.26%)  2
Pericardial effusion (non-malignant)  1  1/19 (5.26%)  1
Gastrointestinal disorders   
Diarrhea  1  1/19 (5.26%)  1
Distension/bloating, abdominal  1  1/19 (5.26%)  1
Dysphagia (difficulty swallowing)  1  1/19 (5.26%)  1
Hemorrhage, GI::Upper GI NOS  1  1/19 (5.26%)  2
Mucositis/stomatitis (clinical exam)::Stomach  1  1/19 (5.26%)  1
Mucositis/stomatitis (functional/symptomatic)::Esophagus  1  1/19 (5.26%)  1
Mucositis/stomatitis (functional/symptomatic)::Oral cavity  1  1/19 (5.26%)  1
Nausea  1  1/19 (5.26%)  1
Pain::Abdomen NOS  1  1/19 (5.26%)  1
Vomiting  1  1/19 (5.26%)  1
General disorders   
Death not associated with CTCAE term::Death NOS  1  4/19 (21.05%)  4
Hepatobiliary disorders   
Cholecystitis  1  1/19 (5.26%)  1
Hepatobiliary/Pancreas - Other (Specify, __)  1  1/19 (5.26%)  1
Infections and infestations   
Febrile neutropenia  1 [1]  2/19 (10.53%)  4
Infection  1 [2]  1/19 (5.26%)  1
Infection (documented clinically or microbiologically)  1 [3]  1/19 (5.26%)  1
Infection (documented clinically or microbiologically)  1 [4]  1/19 (5.26%)  1
Infection  1 [5]  1/19 (5.26%)  1
Infection - Other (Specify, __)  1  1/19 (5.26%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Blood  1  1/19 (5.26%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Lung (pneumonia)  1  1/19 (5.26%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Upper aerodigestive NOS  1  1/19 (5.26%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Upper airway NOS  1  1/19 (5.26%)  1
Infection with unknown ANC::Blood  1  1/19 (5.26%)  1
Infection with unknown ANC::Colon  1  1/19 (5.26%)  1
Metabolism and nutrition disorders   
ALT, SGPT (serum glutamic pyruvic transaminase)  1  2/19 (10.53%)  3
AST, SGOT(serum glutamic oxaloacetic transaminase)  1  1/19 (5.26%)  1
Proteinuria  1  1/19 (5.26%)  1
Nervous system disorders   
Leak (including anastomotic), GI::Leak NOS  1  1/19 (5.26%)  1
Neuropathy: motor  1  1/19 (5.26%)  1
Renal and urinary disorders   
Cystitis  1  1/19 (5.26%)  1
Hemorrhage, GU::Vagina  1  1/19 (5.26%)  1
Renal failure  1  1/19 (5.26%)  1
Respiratory, thoracic and mediastinal disorders   
Adult Respiratory Distress Syndrome (ARDS)  1  1/19 (5.26%)  1
Dyspnea (shortness of breath)  1  2/19 (10.53%)  2
Hemorrhage, pulmonary/upper respiratory::Lung  1  1/19 (5.26%)  1
Hypoxia  1  2/19 (10.53%)  3
Pleural effusion (non-malignant)  1  2/19 (10.53%)  2
Pneumonitis/pulmonary infiltrates  1  2/19 (10.53%)  3
Vascular disorders   
DIC (disseminated intravascular coagulation)  1  1/19 (5.26%)  1
Thrombosis/thrombus/embolism  1  2/19 (10.53%)  2
Vessel injury-artery::Other NOS  1  1/19 (5.26%)  1
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
[1]
(fever of unknown origin without clinically or microbiologically documented infection)(ANC <1.0 x 10e9/L, fever >=38.5 degrees C)
[2]
(documented clinically or microbiologically) with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Blood
[3]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Gallbladder (cholecystitis)
[4]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Sinus
[5]
(documented clinically or microbiologically) with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Urinary tract NOS
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Recipients and Healthy Related Donors
Affected / at Risk (%) # Events
Total   16/19 (84.21%)    
Blood and lymphatic system disorders   
CD4 count  1  1/19 (5.26%)  3
Edema: limb  1  2/19 (10.53%)  2
Hemoglobin  1  9/19 (47.37%)  72
Leukocytes (total WBC)  1  7/19 (36.84%)  90
Lymphedema-related fibrosis  1  1/19 (5.26%)  1
Lymphopenia  1  9/19 (47.37%)  126
Neutrophils/granulocytes (ANC/AGC)  1  6/19 (31.58%)  20
PTT (Partial Thromboplastin Time)  1  4/19 (21.05%)  6
Pain::Lymph node  1  1/19 (5.26%)  1
Platelets  1  6/19 (31.58%)  37
Cardiac disorders   
Cardiac Arrhythmia - Other (Specify, persistent tachycardia)  1  1/19 (5.26%)  1
Left ventricular systolic dysfunction  1  1/19 (5.26%)  1
Pericarditis  1  1/19 (5.26%)  1
Prolonged QTc interval  1  1/19 (5.26%)  1
Pulmonary hypertension  1  1/19 (5.26%)  1
Supraventricular and nodal arrhythmia::Sinus bradycardia  1  1/19 (5.26%)  1
Supraventricular and nodal arrhythmia::Sinus tachycardia  1  3/19 (15.79%)  4
Ear and labyrinth disorders   
Hearing: patients without baseline audiogram and not enrolled in a monitoring program  1  1/19 (5.26%)  1
Eye disorders   
Dry eye syndrome  1  4/19 (21.05%)  4
Ocular surface disease  1  1/19 (5.26%)  1
Ocular/Visual - Other (Specify, decreased vision)  1  1/19 (5.26%)  1
Gastrointestinal disorders   
Ascites (non-malignant)  1  2/19 (10.53%)  2
Diarrhea  1  6/19 (31.58%)  13
Distension/bloating, abdominal  1  1/19 (5.26%)  1
Dry mouth/salivary gland (xerostomia)  1  1/19 (5.26%)  1
Enteritis (inflammation of the small bowel)  1  1/19 (5.26%)  1
Esophagitis  1  3/19 (15.79%)  3
Gastrointestinal - Other (Specify, v)  1  1/19 (5.26%)  1
Hemorrhage, GI::Lower GI NOS  1  1/19 (5.26%)  1
Ileus, GI (functional obstruction of bowel, i.e., neuroconstipation)  1  1/19 (5.26%)  1
Mucositis/stomatitis (clinical exam)::Oral cavity  1  1/19 (5.26%)  1
Mucositis/stomatitis (functional/symptomatic)::Oral cavity  1  2/19 (10.53%)  3
Nausea  1  4/19 (21.05%)  7
Pain::Abdomen NOS  1  1/19 (5.26%)  1
Pain::Anus  1  1/19 (5.26%)  1
Pain::Dental/teeth/peridontal  1  1/19 (5.26%)  2
Vomiting  1  5/19 (26.32%)  5
General disorders   
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)  1  4/19 (21.05%)  8
Hemorrhage/Bleeding - Other (Specify, diathesis)  1  1/19 (5.26%)  1
Pain::Pain NOS  1  1/19 (5.26%)  1
Weight loss  1  2/19 (10.53%)  2
Hepatobiliary disorders   
Liver dysfunction/failure (clinical)  1  1/19 (5.26%)  1
Infections and infestations   
Febrile neutropenia  1 [1]  3/19 (15.79%)  11
Infection (documented clinically or microbiologically)  1 [2]  4/19 (21.05%)  9
Infection (documented clinically or microbiologically)  1 [3]  1/19 (5.26%)  1
Infection (documented clinically or microbiologically)  1 [4]  1/19 (5.26%)  1
Infection (documented clinically or microbiologically)  1 [5]  1/19 (5.26%)  1
Infection (documented clinically or microbiologically)  1 [6]  1/19 (5.26%)  1
Infection (documented clinically or microbiologically)  1 [7]  1/19 (5.26%)  1
Infection (documented clinically or microbiologically)  1 [8]  3/19 (15.79%)  4
Infection (documented clinically or microbiologically)  1 [9]  1/19 (5.26%)  1
Infection (documented clinically or microbiologically)  1 [10]  2/19 (10.53%)  2
Infection - Other (Specify, skin:zoster; thrush)  1  2/19 (10.53%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils::Anal/perianal  1  1/19 (5.26%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Bladder (urinary)  1  2/19 (10.53%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils::Blood  1  9/19 (47.37%)  21
Infection with normal ANC or Grade 1 or 2 neutrophils::Catheter-related  1  2/19 (10.53%)  5
Infection with normal ANC or Grade 1 or 2 neutrophils::Ileum  1  1/19 (5.26%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Larynx  1  1/19 (5.26%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Lung (pneumonia)  1  3/19 (15.79%)  3
Infection with normal ANC or Grade 1 or 2 neutrophils::Nose  1  1/19 (5.26%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils::Oral cavity-gums (gingivitis)  1  1/19 (5.26%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Peritoneal cavity  1  2/19 (10.53%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils::Pharynx  1  1/19 (5.26%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils::Sinus  1  3/19 (15.79%)  6
Infection with normal ANC or Grade 1 or 2 neutrophils::Skin (cellulitis)  1  2/19 (10.53%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils::Upper aerodigestive NOS  1  1/19 (5.26%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Upper airway NOS  1  3/19 (15.79%)  4
Infection with normal ANC or Grade 1 or 2 neutrophils::Urinary tract NOS  1  3/19 (15.79%)  6
Infection with normal ANC or Grade 1 or 2 neutrophils::Vulva  1  1/19 (5.26%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils::Wound  1  1/19 (5.26%)  3
Infection with unknown ANC::Blood  1  2/19 (10.53%)  5
Infection with unknown ANC::Brain + Spinal cord (encephalomyelitis)  1  1/19 (5.26%)  1
Infection with unknown ANC::Bronchus  1  1/19 (5.26%)  1
Infection with unknown ANC::Catheter-related  1  1/19 (5.26%)  1
Infection with unknown ANC::Lung (pneumonia)  1  1/19 (5.26%)  1
Infection with unknown ANC::Upper airway NOS  1  1/19 (5.26%)  1
Metabolism and nutrition disorders   
ALT, SGPT (serum glutamic pyruvic transaminase)  1  11/19 (57.89%)  37
AST, SGOT(serum glutamic oxaloacetic transaminase)  1  10/19 (52.63%)  38
Acidosis (metabolic or respiratory)  1  1/19 (5.26%)  3
Albumin, serum-low (hypoalbuminemia)  1  5/19 (26.32%)  20
Alkaline phosphatase  1  6/19 (31.58%)  23
Bilirubin (hyperbilirubinemia)  1  5/19 (26.32%)  12
CPK (creatine phosphokinase)  1  1/19 (5.26%)  1
Cholesterol, serum-high (hypercholesteremia)  1  1/19 (5.26%)  1
Creatinine  1  2/19 (10.53%)  7
GGT (gamma-Glutamyl transpeptidase)  1  4/19 (21.05%)  4
Glucose, serum-high (hyperglycemia)  1  3/19 (15.79%)  48
Glucose, serum-low (hypoglycemia)  1  1/19 (5.26%)  1
Hemolysis (e.g., immune hemolytic anemia, drug-related hemolysis)  1  1/19 (5.26%)  1
Magnesium, serum-high (hypermagnesemia)  1  3/19 (15.79%)  9
Magnesium, serum-low (hypomagnesemia)  1  1/19 (5.26%)  1
Phosphate, serum-low (hypophosphatemia)  1  6/19 (31.58%)  23
Potassium, serum-high (hyperkalemia)  1  1/19 (5.26%)  1
Potassium, serum-low (hypokalemia)  1  2/19 (10.53%)  11
Proteinuria  1  1/19 (5.26%)  1
Sodium, serum-low (hyponatremia)  1  1/19 (5.26%)  2
Triglyceride, serum-high (hypertriglyceridemia)  1  4/19 (21.05%)  9
Uric acid, serum-high (hyperuricemia)  1  2/19 (10.53%)  2
Musculoskeletal and connective tissue disorders   
Pain::Bone  1  1/19 (5.26%)  1
Muscle weakness, generalized or specific area (not due to neuropathy)::Facial  1  1/19 (5.26%)  2
Osteoporosis  1  1/19 (5.26%)  1
Pain::Back  1  1/19 (5.26%)  1
Pain::Bone  1  2/19 (10.53%)  4
Pain::Extremity-limb  1  1/19 (5.26%)  4
Pain::Neck  1  1/19 (5.26%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Secondary Malignancy - possibly related to cancer treatment (Specify, donor cell leukemia)  1  1/19 (5.26%)  1
Nervous system disorders   
Pain::Head/headache  1  2/19 (10.53%)  2
Leak, cerebrospinal fluid (CSF)  1  1/19 (5.26%)  1
Mood alteration::Agitation  1  1/19 (5.26%)  1
Neuropathy: sensory  1  1/19 (5.26%)  1
Pain::Head/headache  1  3/19 (15.79%)  4
Renal and urinary disorders   
Bladder spasms  1  1/19 (5.26%)  1
Cystitis  1  1/19 (5.26%)  1
Renal failure  1  2/19 (10.53%)  2
Reproductive system and breast disorders   
Pain::Testicle  1  1/19 (5.26%)  1
Vaginal dryness  1  1/19 (5.26%)  1
Vaginal stenosis/length  1  1/19 (5.26%)  1
Respiratory, thoracic and mediastinal disorders   
Apnea  1  1/19 (5.26%)  1
Carbon monoxide diffusion capacity (DL(co))  1  3/19 (15.79%)  4
Dyspnea (shortness of breath)  1  2/19 (10.53%)  3
FEV(1)  1  1/19 (5.26%)  1
Hemorrhage, pulmonary/upper respiratory::Nose  1  2/19 (10.53%)  3
Hypoxia  1  5/19 (26.32%)  9
Nasal cavity/paranasal sinus reactions  1  1/19 (5.26%)  1
Pleural effusion (non-malignant)  1  1/19 (5.26%)  1
Pneumonitis/pulmonary infiltrates  1  1/19 (5.26%)  1
Skin and subcutaneous tissue disorders   
Dermal change lymphedema, phlebolymphedema  1  1/19 (5.26%)  1
Dermatology/Skin - Other (Specify, v dermatitis per biopsy)  1  1/19 (5.26%)  1
Pain::Skin  1  1/19 (5.26%)  1
Rash/desquamation  1  9/19 (47.37%)  13
Rash: acne/acneiform  1  1/19 (5.26%)  1
Urticaria (hives, welts, wheals)  1  1/19 (5.26%)  1
Vascular disorders   
Hypertension  1  4/19 (21.05%)  5
Hypotension  1  4/19 (21.05%)  4
Thrombotic microangiopathy (e.g., thrombotic thrombocytopenic purpura [TTP]  1 [11]  1/19 (5.26%)  1
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
[1]
(fever of unknown origin without clinically or microbiologically documented infection)(ANC <1.0 x 10e9/L, fever >=38.5 degrees C)
[2]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Blood
[3]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Catheter-related
[4]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Pharynx
[5]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Pleura (empyema)
[6]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Rectum
[7]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Spinal cord (myelitis)
[8]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Urinary tract NOS
[9]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Vagina
[10]
with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L)::Wound
[11]
or hemolytic uremic syndrome [HUS])
Certain Agreements
Principal Investigators are NOT 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.
Results Point of Contact
Name/Title: Dr. Dennis Hickstein
Organization: National Cancer Institute
Phone: 301-594-1718
Responsible Party: Dennis Hickstein, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00923364     History of Changes
Obsolete Identifiers: NCT00884923
Other Study ID Numbers: 090096
09-C-0096
First Submitted: June 17, 2009
First Posted: June 18, 2009
Results First Submitted: May 3, 2017
Results First Posted: November 30, 2017
Last Update Posted: November 30, 2017