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Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease (STRIDE)

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: NCT01565616
Recruitment Status : Completed
First Posted : March 28, 2012
Results First Posted : September 29, 2017
Last Update Posted : November 21, 2017
Sponsor:
Collaborators:
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Lakshmanan Krishnamurti, Emory University

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Sickle Cell Disease
Intervention Drug: Conditioning Regimen with Bone Marrow Transplant
Enrollment 22
Recruitment Details Participants were enrolled from 8 study locations between March, 2012 and June, 2015.
Pre-assignment Details The first component of the study was restricted to 5 patients with a related donor. If no more than 2 of the first 5 patients experienced unacceptable toxicity within six months of transplantation then the safety of the regimen was considered promising and the study could include patients with a related or unrelated HLA matched donor.
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
Period Title: Overall Study
Started 22
First Component: 5 Related Donors [1] 5
Second Component: Related or Unrelated [2] 17
Completed 22
Not Completed 0
[1]
The first component was restricted to patients having an HLA-identical sibling donor.
[2]
The second component included patients having a related or unrelated HLA matched donor.
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description Individuals receiving a bone marrow transplant at one of 10 study locations, between March 2012 and June, 2015.
Overall Number of Baseline Participants 22
Hide Baseline Analysis Population Description
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
16-19 years of age
6
  27.3%
20-24 years of age
8
  36.4%
24-29 years of age
6
  27.3%
30-40 years of age
2
   9.1%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
Female
14
  63.6%
Male
8
  36.4%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
22
 100.0%
White
0
   0.0%
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 22 participants
22
 100.0%
Donor Relation  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
Related
17
  77.3%
Unrelated
5
  22.7%
Indication of Severe Sickle Cell Disease   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
Significant neurological event (stroke)
2
   9.1%
Acute chest syndrome
2
   9.1%
Recurrent pain events
15
  68.2%
Regular RBC transfusion therapy
4
  18.2%
Elevated TRJ Velocity
4
  18.2%
[1]
Measure Description:

Participants were eligible for the study if they had one or more of the following:

  • Significant neurological event, such as a stroke or any neurological deficit lasting longer than 24 hours
  • History of acute chest syndrome (ACS)
  • History of 3 or more pain crises per year
  • Regular red blood cell (RBC) transfusions (8 or more per year)
  • Trans-thoracic echocardiographic evidence of tricuspid valve regurgitant jet (TRJ) velocity 2.7 m/sec
1.Primary Outcome
Title Event -Free Survival Rate
Hide Description Event-free survival is defined as stable donor erythropoiesis with no new clinical evidence of sickle cell disease. Primary or late graft rejection, disease recurrence, and death are considered events for this endpoint.
Time Frame 1 year after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description:
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
Overall Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
19
  86.4%
2.Secondary Outcome
Title Graft Failure
Hide Description Primary graft failure occurs when a transplant recipient does not achieve donor chimerism following a bone marrow transplant. Secondary graft failure occurs when graft fails after donor chimerism had initially occurred.
Time Frame 1 year after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description:
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
Overall Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
Primary graft failure
0
   0.0%
Secondary graft failure
1
   4.5%
3.Secondary Outcome
Title Acute Graft Versus Host Disease (GVHD)
Hide Description

Acute GVHD was graded according to the Center for International Blood and Marrow Transplant Research (CIBMTR) consensus criteria. Clinical manifestations of acute GVHD include skin, liver, and gastrointestinal symptoms. Grading of acute GVHD is determined by size of maculopapular rash, bilirubin and stool output. Acute GVHD grades range from 0 to 4 with 0 indicating no GVHD and 4 representing the most severe grade.

Grade II is defined as a maculopapular rash over 25-50% of body surface area (BSA), bilirubin of 3.1 to 6 mg/dL, and stool output of 1000-1500 mL/d (for adults).

Grade III is defined as a maculopapular rash over more than 50% of BSA, bilirubin of 6.1 to 15 mg/dL, and stool output of greater than 1500 mL/d (for adults).

Grade IV is defined as generalized erythroderma with bullous formation, bilirubin greater than 15 mg/dL, and severe abdominal pain with or without ileus.

Time Frame 1 year after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description:
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
Overall Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
Grade II Acute GVHD
3
  13.6%
Grade III Acute GVHD
1
   4.5%
Grade IV Acute GVHD
0
   0.0%
4.Secondary Outcome
Title Chronic Graft Versus Host Disease (GVHD)
Hide Description Chronic GVHD was graded according to the National Institutes of Health (NIH) 2014 Consensus Criteria Diagnosis and scoring the severity of chronic GVHD is determined by evaluating symptoms of the skin, nails, hair, mouth, eyes, genitalia, gastrointestinal tract, liver, lungs, muscles, fascia and joints, immune function as well as other symptoms such as ascites and neuropathy. Chronic GVHD is graded as mild, moderate or severe based on the number of organ sites impacted and the severity of symptoms.
Time Frame 1 year after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description:
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
Overall Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
Mild Chronic GVHD
3
  13.6%
Moderate Chronic GVHD
2
   9.1%
Severe Chronic GVHD
1
   4.5%
5.Secondary Outcome
Title Overall Survival
Hide Description Overall survival is defined as survival with or without sickle cell disease after hematopoietic cell transplantation (HCT).
Time Frame 1 year after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description:
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
Overall Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
20
  90.9%
6.Secondary Outcome
Title Time to Neutrophil and Platelet Engraftment
Hide Description Time to neutrophil engraftment is defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count of at least 500/µL after conditioning. Time to Platelet engraftment is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count > 50,000/µL, without receiving a platelet transfusion in the previous 7 days.
Time Frame 1 year after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description:
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
Overall Number of Participants Analyzed 22
Median (Full Range)
Unit of Measure: Days
Neutrophil engraftment
17
(12 to 26)
Platelet
21
(13 to 26)
7.Secondary Outcome
Title Transplant Related Outcomes
Hide Description Common transplant related complications were monitored as a secondary outcome measure of this study. These transplant related complications include hepatic veno-occlusive disease (VOD), idiopathic pneumonia syndrome (IPS), central nervous system (CNS) toxicity complications of posterior reversible encephalopathy syndrome (PRES), hemorrhage, and seizures, cytomegalovirus (CMV) infection, adenovirus infection, Epstein-Barr virus (EBV) infection, post-transplant lymphoproliferative disease (PTLD), and invasive fungal infection.
Time Frame 1 year after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description:
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
Overall Number of Participants Analyzed 22
Measure Type: Count of Participants
Unit of Measure: Participants
Hepatic Veno-occlusive Disease (VOD)
0
   0.0%
Idiopathic Pneumonia Syndrome (IPS)
1
   4.5%
PRES
1
   4.5%
Central Nervous System Toxicity: Hemorrhage
1
   4.5%
Central Nervous System Toxicity: Seizure
1
   4.5%
Cytomegalovirus (CMV) Infection
11
  50.0%
Adenovirus Infection
0
   0.0%
Epstein-Barr Virus (EBV) Infection
4
  18.2%
Post-transplant Lymphoproliferative Disease
1
   4.5%
Invasive Fungal Infection
0
   0.0%
8.Post-Hoc Outcome
Title PROMIS-57 Scores Health Related Quality of Life
Hide Description

Health related quality of life was measured with the 57 item Patient-Reported Outcomes Measurement Information System (PROMIS-57). The PROMIS-57 includes 8 domains of health. The domains of Anxiety, Depression, Fatigue, Pain Interference, Physical Function, Satisfaction with Social Role, and Sleep Disturbances each include 8 items. Respondents indicate the degree to which statements about specific health issues are problematic on a scale of 1 to 5 and responses are converted to a t-score metric. A score of 50 is the mean score for the general population in the United States, with a standard deviation of 10. Scores above 50 indicate the topic of the domain is being experienced more than average while scores below 50 mean that it is less than average.

The domain of Pain Intensity is measured with a single item asking participants to rate their average pain on a scale from 0 (no pain) to 10 (worst imaginable pain). The raw mean score is used.

Time Frame Baseline, 1 year after transplant
Hide Outcome Measure Data
Hide Analysis Population Description
This outcome measure compared quality of life scores one year post-HCT to baseline in the 17 participants who completed the surveys.
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description:
Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
Overall Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: units on a scale
Anxiety domain score: baseline Number Analyzed 17 participants
50.6  (10.4)
Anxiety domain score: 1 year Number Analyzed 16 participants
48.1  (10.5)
Depression domain score: baseline Number Analyzed 17 participants
44.7  (6.7)
Depresssion domain score: 1 year Number Analyzed 16 participants
46.9  (10.3)
Fatigue domain score: baseline Number Analyzed 17 participants
48.0  (10.5)
Fatigue domain score: 1 year Number Analyzed 16 participants
46.1  (13.4)
Pain interference domain score: baseline Number Analyzed 17 participants
58.5  (10.3)
Pain interference domain score: 1 year Number Analyzed 16 participants
50.1  (11.6)
Physical function domain score: baseline Number Analyzed 17 participants
44.1  (8.0)
Physical function domain score: 1 year Number Analyzed 16 participants
50.1  (11.0)
Social role satisfaction domain score: baseline Number Analyzed 17 participants
51.7  (11.6)
Social role satisfaction domain score: 1 year Number Analyzed 16 participants
52.4  (12.3)
Sleep disturbance domain score: baseline Number Analyzed 17 participants
47.7  (11.9)
Sleep disturbance domain score: 1 year Number Analyzed 16 participants
46.9  (9.6)
Pain intensity domain score: baseline Number Analyzed 17 participants
3.2  (3.4)
Pain intensity domain score: 1 year Number Analyzed 16 participants
2.4  (3.0)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Bone Marrow Transplant Recipients
Comments This statistical analysis is the paired difference in t-scores of the anxiety domain of the PROMIS-57 quality of life scale. 16 participants with paired measurements of the PROMIS-57 survey (baseline and 1 year) are included in this analysis.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.52
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.7
Parameter Dispersion
Type: Standard Deviation
Value: 10.4
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Bone Marrow Transplant Recipients
Comments This statistical analysis is the paired difference in t-scores of the depression domain of the PROMIS-57 quality of life scale. 16 participants with paired measurements of the PROMIS-57 survey (baseline and 1 year) are included in this analysis.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.12
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 2.7
Parameter Dispersion
Type: Standard Deviation
Value: 6.4
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Bone Marrow Transplant Recipients
Comments This statistical analysis is the paired difference in t-scores of the fatigue domain of the PROMIS-57 quality of life scale. 16 participants with paired measurements of the PROMIS-57 survey (baseline and 1 year) are included in this analysis.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.54
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.3
Parameter Dispersion
Type: Standard Deviation
Value: 8.5
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Bone Marrow Transplant Recipients
Comments This statistical analysis is the paired difference in t-scores of the pain interference domain of the PROMIS-57 quality of life scale. 16 participants with paired measurements of the PROMIS-57 survey (baseline and 1 year) are included in this analysis.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.006
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -7.5
Parameter Dispersion
Type: Standard Deviation
Value: 9.3
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Bone Marrow Transplant Recipients
Comments This statistical analysis is the paired difference in t-scores of the physical function domain of the PROMIS-57 quality of life scale. 16 participants with paired measurements of the PROMIS-57 survey (baseline and 1 year) are included in this analysis.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.044
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 5.8
Parameter Dispersion
Type: Standard Deviation
Value: 10.5
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Bone Marrow Transplant Recipients
Comments This statistical analysis is the paired difference in t-scores of the satisfaction with social role domain of the PROMIS-57 quality of life scale. 16 participants with paired measurements of the PROMIS-57 survey (baseline and 1 year) are included in this analysis.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.85
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.6
Parameter Dispersion
Type: Standard Deviation
Value: 11.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Bone Marrow Transplant Recipients
Comments This statistical analysis is the paired difference in t-scores of the sleep disturbances domain of the PROMIS-57 quality of life scale. 16 participants with paired measurements of the PROMIS-57 survey (baseline and 1 year) are included in this analysis.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.88
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.5
Parameter Dispersion
Type: Standard Deviation
Value: 13.5
Estimation Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Bone Marrow Transplant Recipients
Comments This statistical analysis is the paired difference in raw scores of the pain intensity domain of the PROMIS-57 quality of life scale. 16 participants with paired measurements of the PROMIS-57 survey (baseline and 1 year) are included in this analysis.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.53
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.5
Parameter Dispersion
Type: Standard Deviation
Value: 3.1
Estimation Comments [Not Specified]
Time Frame Data on adverse events were collected from when the patient consented to participate in the study through 1 year after hematopoietic cell transplantation (HCT).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Bone Marrow Transplant Recipients
Hide Arm/Group Description Individuals with severe sickle cell disease receiving hematopoietic cell transplantation (HCT) from a human leukocyte antigen (HLA)-identical sibling donor or an unrelated HLA-matched donor
All-Cause Mortality
Bone Marrow Transplant Recipients
Affected / at Risk (%)
Total   2/22 (9.09%)    
Hide Serious Adverse Events
Bone Marrow Transplant Recipients
Affected / at Risk (%) # Events
Total   14/22 (63.64%)    
Blood and lymphatic system disorders   
Anemia secondary to hemolysis *  1/22 (4.55%)  1
Hemolysis with rising creatinine *  1/22 (4.55%)  1
Hypercalcemia *  1/22 (4.55%)  1
Hospitalization due to pain crisis *  1/22 (4.55%)  1
Hospitalization due to progressive respiratory distress with hypoxemia *  1/22 (4.55%)  1
Removal of central venous catheter due to possible thrombus *  1/22 (4.55%)  1
Respiratory distress secondary to large pharyngeal blood clot *  1/22 (4.55%)  1
Cardiac disorders   
Hospitalization due to pericardial effusion *  2/22 (9.09%)  2
Pericarditis *  1/22 (4.55%)  1
Gastrointestinal disorders   
Hospitalization due to abdominal pain, diarrhea, and bloody stools *  1/22 (4.55%)  1
Hospitalization due to abdominal pain, nausea, vomiting, and elevated liver enzymes *  1/22 (4.55%)  1
Gastritis *  1/22 (4.55%)  2
Mucositis *  2/22 (9.09%)  2
Hospitalization due to severe nausea, vomiting and diarrhea *  1/22 (4.55%)  1
General disorders   
Hospitalization due to acute on chronic pain *  1/22 (4.55%)  1
Hospitalization due to exacerbation of pain syndrome *  1/22 (4.55%)  1
Facial swelling with sore throat *  1/22 (4.55%)  1
Generalized pain *  1/22 (4.55%)  1
Headache requiring Norco *  1/22 (4.55%)  1
Mucosistis, esophageal pain, and low back pain *  1/22 (4.55%)  1
Hospitalization due to persistent fever *  1/22 (4.55%)  1
Hepatobiliary disorders   
Cholelithiasis *  1/22 (4.55%)  1
Elevated liver enzymes *  2/22 (9.09%)  3
Gallbladder obstruction *  1/22 (4.55%)  1
Gallstones with hyperbilirubinemia *  1/22 (4.55%)  1
Infections and infestations   
Hospitalization due to fever, malaise, and chills *  1/22 (4.55%)  1
Hospitalization due to seizure and infection *  1/22 (4.55%)  1
Injury, poisoning and procedural complications   
Opioid-induced over-sedation *  1/22 (4.55%)  1
Nervous system disorders   
Neurological changes *  2/22 (9.09%)  2
Renal and urinary disorders   
Urinary retention, dysuria, and hesitancy *  1/22 (4.55%)  1
Respiratory, thoracic and mediastinal disorders   
Hospitalization due to acute inflammatory bronchitis *  1/22 (4.55%)  1
Hospitalization due to chest pain, secondary to pneumonia *  1/22 (4.55%)  1
Hospitalization due to pneumonia with hypoxia *  1/22 (4.55%)  1
Surgical and medical procedures   
Hospitalization due to elevated prograf level *  1/22 (4.55%)  1
Prograf toxicity *  1/22 (4.55%)  1
Vascular disorders   
PRES with intercerebral hemorrhage associated with severe hypertension *  1/22 (4.55%)  1
Vascular disorder with facial swelling *  1/22 (4.55%)  1
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Bone Marrow Transplant Recipients
Affected / at Risk (%) # Events
Total   12/22 (54.55%)    
Blood and lymphatic system disorders   
Reduced hemoglobin *  1/22 (4.55%)  1
Central nervous system hemorrhage *  1/22 (4.55%)  1
Endocrine disorders   
Diabetes mellitus *  1/22 (4.55%)  1
Hyperglycemia *  2/22 (9.09%)  2
Gastrointestinal disorders   
Diarrhea *  1/22 (4.55%)  1
Stomatitis *  7/22 (31.82%)  10
General disorders   
Anorexia *  1/22 (4.55%)  1
Deep bone pain *  1/22 (4.55%)  1
Extremity pain *  1/22 (4.55%)  1
Fever *  1/22 (4.55%)  1
Headache *  1/22 (4.55%)  1
Hyperkalemia *  1/22 (4.55%)  1
Hypocalcemia *  1/22 (4.55%)  1
Hypokalemia *  1/22 (4.55%)  1
Hypomagnesemia *  1/22 (4.55%)  1
Lethargy and confusion *  1/22 (4.55%)  1
Rectal pain *  1/22 (4.55%)  1
Severe itching *  1/22 (4.55%)  1
Severe pain *  1/22 (4.55%)  1
Somnolence, depressed level of consciousness *  1/22 (4.55%)  1
Tremors *  1/22 (4.55%)  1
Hepatobiliary disorders   
Elevated alkaline phosphatase *  1/22 (4.55%)  1
Elevated alanine transaminase *  2/22 (9.09%)  2
Increased liver function tests *  1/22 (4.55%)  2
Immune system disorders   
Alopecia *  1/22 (4.55%)  1
Infections and infestations   
Catheter related infection *  1/22 (4.55%)  1
Infection *  1/22 (4.55%)  1
Increased white blood cell count *  1/22 (4.55%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia *  1/22 (4.55%)  1
Nervous system disorders   
Cranial Bell's Palsy *  1/22 (4.55%)  1
Neuropathic extremity pain *  1/22 (4.55%)  1
Psychiatric disorders   
Anxiety *  1/22 (4.55%)  1
Renal and urinary disorders   
Renal failure *  1/22 (4.55%)  1
Renal insufficiency *  2/22 (9.09%)  4
Urinary disorder *  1/22 (4.55%)  1
Reproductive system and breast disorders   
Vaginal pain *  1/22 (4.55%)  1
Respiratory, thoracic and mediastinal disorders   
Bronchiolitis obliterans *  1/22 (4.55%)  1
Dyspnea *  1/22 (4.55%)  1
Idiopathic pneumonia syndrome *  1/22 (4.55%)  1
Pulmonary edema *  1/22 (4.55%)  1
Surgical and medical procedures   
Post transplant lymphoproliferative disorder *  1/22 (4.55%)  1
Prograf toxicity *  1/22 (4.55%)  1
Vascular disorders   
Hypertension *  3/22 (13.64%)  4
Pulmonary hypertension *  1/22 (4.55%)  1
*
Indicates events were collected by non-systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Lakshmanan Krishnamurti, MD
Organization: Emory University
EMail: lakshmanan.krishnamurti@emory.edu
Layout table for additonal information
Responsible Party: Lakshmanan Krishnamurti, Emory University
ClinicalTrials.gov Identifier: NCT01565616    
Other Study ID Numbers: IRB00068287
1R34HL108761-01 ( U.S. NIH Grant/Contract )
First Submitted: March 26, 2012
First Posted: March 28, 2012
Results First Submitted: August 29, 2017
Results First Posted: September 29, 2017
Last Update Posted: November 21, 2017