Safety and Efficacy of BL-8040 for the Mobilization of Donor Hematopoietic Stem Cells and Allogeneic Transplantation in Patients With Advanced Hematological Malignancies
Study Details
Study Description
Brief Summary
Current protocols use G-CSF to mobilize hematopoietic progenitor cells from matched sibling and volunteer unrelated donors. Unfortunately, this process requires four to six days of G-CSF injection and can be associated with side effects, most notably bone pain and rarely splenic rupture. BL-8040 is given as a single SC injection, and collection of cells occurs on the same day as BL-8040 administration. This study will evaluate the safety and efficacy of this novel agent for hematopoietic progenitor cell mobilization and allogeneic transplantation based on the following hypotheses:
-
Healthy HLA-matched donors receiving one injection of BL-8040 will mobilize sufficient CD34+ cells (at least 2.0 x 10^6 CD34+ cells/kg recipient weight) following no more than two leukapheresis collections to support a hematopoietic cell transplant.
-
The hematopoietic cells mobilized by SC BL-8040 will be functional and will result in prompt and durable hematopoietic engraftment following transplantation into HLA-identical siblings with advanced hematological malignancies using various non-myeloablative and myeloablative conditioning regimens and regimens for routine GVHD prophylaxis.
-
If these hypotheses 1 and 2 are confirmed after an interim safety analysis of the data, then the study will continue and include recruitment of haploidentical donors.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. |
Drug: BL-8040
Procedure: Leukapheresis
|
| Experimental: Arm 2: Recipients -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
Procedure: Hematopoietic cell transplant
|
Outcome Measures
Primary Outcome Measures
- Number of Donors That Mobilize ≥ 2 x 10^6 CD34+ Cells/kg of Recipients Weight After a Single Injection of BL-8040 After no More Than Two Leukapheresis Collections (Arm 1 - Donors Only) [Up to Day 2]
Secondary Outcome Measures
- Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) [Up to 5 years]
-Adverse events will be graded according to the NCI CTCAE version 4.03
- Time to Neutrophil Engraftment Post-transplant in Patients Undergoing Allogeneic Stem Cell Transplant (Arm 2 Recipients Only) [Up to Day 28]
-Time to neutrophil engraftment is measured by determining the first of 3 consecutive measurements of neutrophil count ≥ 500/μL following conditioning regimen induced nadir.
- Time to Platelet Engraftment Post-transplant in Patients Undergoing Allogeneic Stem Cell Transplant (Arm 2 Recipients Only) [Through 90 days]
-Time to platelet engraftment is measured by determining the first of 3 consecutive measurements of platelet count ≥ 20,000/μL without platelet transfusion support for 7 days.
- Number of Recipients With Primary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) [Up to 1 year after transplantation]
- Incidence of Secondary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) [Up to 1 year after transplantation]
- Cumulative Incidence of Grade 2-4 Acute Graft Versus Host Disease (GvHD) as Measured by Minnesota Acute GVHD Criteria (Arm 2 Recipients Only) [Day 100]
Acute GVHD rate and worst severity is noted 4 organ categories (skin, liver, lower GI, and upper GI) Skin: Grade I: 1-2 , Grade II: 3, Grade III: N/A, Grade IV: 4 Liver: Grade I: 0, Grade II: 1, Grade III: 2-4, Grade IV: N/A Lower GI: Grade I: 0, Grade II: 1: Grade II: 2-3: Grade IV: 4 Upper GI: Grade I: 0, Grade II: 1, Grade III: N/A, Grade IV: N/A The cumulative incidence of grade 2-4 acute GVHD was determined using competing risk analysis. Competing risks for acute GVHD were death, relapse, and graft failure.
- Cumulative Incidence of Chronic GvHD in Patients Who Have Undergone Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) [From Day 100 through 1 year after transplantation]
Chronic GVHD rate and severity for the first 365 days after PBSC infusion will be assessed based on the NIH criteria The cumulative incidence of chronic GVHD was determined using competing risk analysis. Competing risks for acute GVHD were death, relapse, and graft failure.
- Number of Participants Who Collect 5 x 106 CD34+ Cells/kg of Recipient Weight in a Single Leukapheresis and in 2 Leukapheresis Sessions (Arm 1 Donors Only) [Up to Day 2]
- Incidence of CMV Reactivation After Transplantation of Hematopoietic Cells Mobilized With BL-8040 in CMV Seropositive Recipients [Up to 1 year after transplantation]
-CMV reactivation will be defined as a positive test for CMV viremia as determined by an antigenemia assay or quantitative PCR that results in the administration of antiviral treatment directed against CMV
- Cumulative Incidence of Treatment-related Mortality After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) [Up to 1 year after transplantation]
-Death that results from a transplant procedure related complication (e.g. infection, organ failure, hemorrhage, GVHD) rather than from relapse of the underlying disease or an unrelated cause
- Cumulative Incidence of Disease Relapse/Progression After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) [At 1 year post-tranplantation]
-Disease relapse occurs in recipients who entered transplant in CR. Progression occurs in recipients with existent disease at transplant who meet criteria for progressive disease post-transplant. A recipient will be considered relapsed when there is a recurrence of the original malignant disease after transplantation. Date of relapse/progression is defined as the date at which the first observation of hematologic, radiographic, or cytogenetic changes which signify progression/relapse is made
- Cumulative Incidence of Disease Relapse/Progression After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) [At 2 years post-tranplantation]
-Disease relapse occurs in recipients who entered transplant in CR. Progression occurs in recipients with existent disease at transplant who meet criteria for progressive disease post-transplant. A recipient will be considered relapsed when there is a recurrence of the original malignant disease after transplantation. Date of relapse/progression is defined as the date at which the first observation of hematologic, radiographic, or cytogenetic changes which signify progression/relapse is made
- Kaplan-Meier Estimate of Event Free Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) [At 2 years post-transplantation]
-An event is defined as either graft failure, disease relapse as evidenced by hematologic, radiographic, or cytogenetic changes, or death. The event free survival is the time from Day 0 to occurrence of the first event.
- Kaplan-Meier Estimate of Event Free Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) [At 3 years post-transplantation]
-An event is defined as either graft failure, disease relapse as evidenced by hematologic, radiographic, or cytogenetic changes, or death. The event free survival is the time from Day 0 to occurrence of the first event.
- Kaplan-Meier Estimate of Overall Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) [At 2 years post-transplantation]
-The time from Day 0 to death
- Kaplan-Meier Estimate of Overall Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) [At 3 years post-transplantation]
-The time from Day 0 to death
- Median Peripheral Blood CD34+ Cell Count (Arm 1 Donor Only) [At 3-4 hours after BL-8040]
Eligibility Criteria
Criteria
Inclusion Criteria (DONOR):
-
Age 18 to 70 years of age.
-
ECOG performance status of 0 or 1.
-
PART 1: Donor must be a 5/6 or 6/6 HLA-matched sibling willing to donate PBSC for transplant.
-
PART 2: Donor must be a 5/6 or 6/6 HLA-matched sibling or 3/6 or 4/6 HLA haploidentical donor willing to donate PBSC for transplant. Haploidentical donors will be allowed to participate upon investigator decision and based on the data reached from 5/6 or 6/6 HLA matched transplant done during Part 1 of the study.
-
Adequate organ function defined by:
-
serum creatinine within normal limits or a minimum creatinine clearance (CrCl) value of ≥ 60 ml/min calculated using the Modification of Diet in Renal Disease (MDRD) Study equation
-
AST, ALT and total bilirubin ≤ 2x institutional upper limit of normal.
-
Women of childbearing potential and men must agree to use adequate contraception with two different forms, including one barrier method, during participation in the study and for 2 weeks following dosing with BL-8040. Abstinence is acceptable if this is the established and preferred contraception for the subject.
-
Female subjects must have a negative urine or serum pregnancy test within 10 days prior to taking study medication if of childbearing potential or must be of non-childbearing potential. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. The serum pregnancy test must be negative for the subject to be eligible. Non-childbearing potential is defined as:
-≥ 45 years of age and has not had menses for > 2 years
-
Amenorrheic for > 2 years without a hysterectomy and oophorectomy and a FSH value in the postmenopausal range upon pretrial (screening) evaluation
-
Post-hysterectomy, oophorectomy, or tubal ligation.
-
Able and willing to comply with the requirements of the protocol.
-
Able to understand and willing to sign an IRB-approved written informed consent document.
Inclusion Criteria (RECIPIENT):
-
Age 18 to 75 years
-
ECOG performance status of 0-2 (inclusive)
-
One of the following diagnoses:
-
Acute myelogenous leukemia (AML) in 1st or subsequent remission
-
Acute lymphoblastic leukemia (ALL) in 1st or subsequent remission
-
Chronic myelogenous leukemia (CML) in chronic or accelerated phase
-
Non-Hodgkin lymphoma (NHL) or Hodgkin's disease (HD) in 2nd or greater complete remission, partial remission
-
Chronic lymphocytic leukemia (CLL)
-
Multiple myeloma (MM)
-
Myelodysplastic syndrome (MDS)
-
Myeloproliferative neoplasm (MPN) excluding primary or secondary myelofibrosis
-
Adequate organ function defined by:
-
a creatinine clearance (CrCl) value of ≥ 60 ml/min by MDRD study equation
-
AST, ALT and a total bilirubin ≤ 2x institutional upper limit of normal.
-
Adequate cardiac function with a left ventricular ejection fraction ≥ 40%.
-
Adequate pulmonary function defined as NO severe or symptomatic restrictive or obstructive lung disease, and formal pulmonary function testing showing an FEV1 ≥50% of predicted and a DLCO ≥ 40% of predicted, corrected for hemoglobin.
-
Female subjects must have a negative urine or serum pregnancy test if of childbearing potential or be of non-childbearing potential. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. The serum pregnancy test must be negative for the subject to be eligible. Non-childbearing potential is defined as:
*≥ 45 years of age and has not had menses for > 2 years
-
Amenorrheic for > 2 years without a hysterectomy and oophorectomy and a FSH value in the postmenopausal range upon pretrial (screening) evaluation
-
Post-hysterectomy, oophorectomy, or tubal ligation.
-
Able to understand and willing to sign an IRB-approved written informed consent document.
Exclusion Criteria (DONOR):
-
Received any investigational agent within 30 days and/or 5 half-lives (of the other investigational agent), whichever is longer, of receiving BL-8040.
-
Active HIV or hepatitis B or C infection
-
Pregnant or breastfeeding.
-
Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
-
Known allergy or hypersensitivity to any of the test compounds, materials, or contraindication to test products.
-
Any malignancies in the 2 years prior to baseline, excluding: basal cell carcinoma, in situ malignancy, low-risk prostate cancer, cervix cancer after curative therapy.
-
A comorbid condition which, in the view of the investigators, renders the subject at high risk from treatment complications.
Exclusion Criteria (RECIPIENT):
-
Recipient must not have received any investigational drug within 30 days of starting conditioning treatment.
-
Pregnant or breastfeeding.
-
Active HIV or hepatitis B or C infection.
-
Any medical condition which, in the opinion of the clinical investigator, would interfere with the evaluation of the patient. Subjects with a clinically significant or unstable medical or surgical condition or any other condition that cannot be well-controlled by the allowed medications permitted in the study protocol that would preclude safe and complete study participation, as determined by medical history, physical examinations, ECG, laboratory tests, or chest-X-ray and according to the investigator's judgment.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Northside Hospital Cancer Institute | Atlanta | Georgia | United States | 30342 |
| 2 | Washington University School of Medicine | Saint Louis | Missouri | United States | 63110 |
| 3 | Ohio State University | Columbus | Ohio | United States | 43210 |
Sponsors and Collaborators
- Washington University School of Medicine
- BioLineRx, Ltd.
Investigators
- Principal Investigator: Geoffrey Uy, M.D., Washington University School of Medicine
Study Documents (Full-Text)
More Information
Additional Information:
Publications
None provided.- 201602037
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Period Title: Overall Study | ||
| STARTED | 25 | 25 |
| COMPLETED | 24 | 22 |
| NOT COMPLETED | 1 | 3 |
Baseline Characteristics
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients | Total |
|---|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 | Total of all reporting groups |
| Overall Participants | 25 | 25 | 50 |
| Age (years) [Median (Full Range) ] | |||
| Median (Full Range) [years] |
55
|
58
|
55
|
| Sex: Female, Male (Count of Participants) | |||
| Female |
7
28%
|
10
40%
|
17
34%
|
| Male |
18
72%
|
15
60%
|
33
66%
|
| Ethnicity (NIH/OMB) (Count of Participants) | |||
| Hispanic or Latino |
2
8%
|
2
8%
|
4
8%
|
| Not Hispanic or Latino |
21
84%
|
23
92%
|
44
88%
|
| Unknown or Not Reported |
2
8%
|
0
0%
|
2
4%
|
| Race (NIH/OMB) (Count of Participants) | |||
| American Indian or Alaska Native |
0
0%
|
0
0%
|
0
0%
|
| Asian |
0
0%
|
0
0%
|
0
0%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
0
0%
|
0
0%
|
| Black or African American |
1
4%
|
1
4%
|
2
4%
|
| White |
20
80%
|
22
88%
|
42
84%
|
| More than one race |
0
0%
|
0
0%
|
0
0%
|
| Unknown or Not Reported |
4
16%
|
2
8%
|
6
12%
|
| Region of Enrollment (participants) [Number] | |||
| United States |
25
100%
|
25
100%
|
50
100%
|
Outcome Measures
| Title | Number of Donors That Mobilize ≥ 2 x 10^6 CD34+ Cells/kg of Recipients Weight After a Single Injection of BL-8040 After no More Than Two Leukapheresis Collections (Arm 1 - Donors Only) |
|---|---|
| Description | |
| Time Frame | Up to Day 2 |
Outcome Measure Data
| Analysis Population Description |
|---|
| This outcome measure is for Arm 1 - Donors only. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 24 | 0 |
| Count of Participants [Participants] |
22
88%
|
| Title | Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) |
|---|---|
| Description | -Adverse events will be graded according to the NCI CTCAE version 4.03 |
| Time Frame | Up to 5 years |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title |
|---|
| Arm/Group Description |
| Title | Time to Neutrophil Engraftment Post-transplant in Patients Undergoing Allogeneic Stem Cell Transplant (Arm 2 Recipients Only) |
|---|---|
| Description | -Time to neutrophil engraftment is measured by determining the first of 3 consecutive measurements of neutrophil count ≥ 500/μL following conditioning regimen induced nadir. |
| Time Frame | Up to Day 28 |
Outcome Measure Data
| Analysis Population Description |
|---|
| -Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Median (Full Range) [days] |
13
|
| Title | Time to Platelet Engraftment Post-transplant in Patients Undergoing Allogeneic Stem Cell Transplant (Arm 2 Recipients Only) |
|---|---|
| Description | -Time to platelet engraftment is measured by determining the first of 3 consecutive measurements of platelet count ≥ 20,000/μL without platelet transfusion support for 7 days. |
| Time Frame | Through 90 days |
Outcome Measure Data
| Analysis Population Description |
|---|
| -Donors are not evaluable for this outcome measure. 1 recipient was not evaluable due to now platelet nadir and 1 recipient was not evaluable due to no engraftment. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 20 |
| Median (Full Range) [days] |
20
|
| Title | Number of Recipients With Primary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) |
|---|---|
| Description | |
| Time Frame | Up to 1 year after transplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| -Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Count of Participants [Participants] |
0
0%
|
| Title | Incidence of Secondary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) |
|---|---|
| Description | |
| Time Frame | Up to 1 year after transplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| -Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Count of Participants [Participants] |
0
0%
|
| Title | Cumulative Incidence of Grade 2-4 Acute Graft Versus Host Disease (GvHD) as Measured by Minnesota Acute GVHD Criteria (Arm 2 Recipients Only) |
|---|---|
| Description | Acute GVHD rate and worst severity is noted 4 organ categories (skin, liver, lower GI, and upper GI) Skin: Grade I: 1-2 , Grade II: 3, Grade III: N/A, Grade IV: 4 Liver: Grade I: 0, Grade II: 1, Grade III: 2-4, Grade IV: N/A Lower GI: Grade I: 0, Grade II: 1: Grade II: 2-3: Grade IV: 4 Upper GI: Grade I: 0, Grade II: 1, Grade III: N/A, Grade IV: N/A The cumulative incidence of grade 2-4 acute GVHD was determined using competing risk analysis. Competing risks for acute GVHD were death, relapse, and graft failure. |
| Time Frame | Day 100 |
Outcome Measure Data
| Analysis Population Description |
|---|
| -Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Number (95% Confidence Interval) [proportion of participants] |
0.3636
1.5%
|
| Title | Cumulative Incidence of Chronic GvHD in Patients Who Have Undergone Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) |
|---|---|
| Description | Chronic GVHD rate and severity for the first 365 days after PBSC infusion will be assessed based on the NIH criteria The cumulative incidence of chronic GVHD was determined using competing risk analysis. Competing risks for acute GVHD were death, relapse, and graft failure. |
| Time Frame | From Day 100 through 1 year after transplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| -Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Number (95% Confidence Interval) [proportion of participants] |
0.5000
2%
|
| Title | Number of Participants Who Collect 5 x 106 CD34+ Cells/kg of Recipient Weight in a Single Leukapheresis and in 2 Leukapheresis Sessions (Arm 1 Donors Only) |
|---|---|
| Description | |
| Time Frame | Up to Day 2 |
Outcome Measure Data
| Analysis Population Description |
|---|
| -Recipients are not evaluable for this outcome measure. -Only donors who received 1.25 mg/kg of BL-8040 were evaluable for this outcome measure (the 1st 10 donors received 1.00 mg/kg of BL-8040) |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 9 | 0 |
| Count of Participants [Participants] |
9
36%
|
| Title | Incidence of CMV Reactivation After Transplantation of Hematopoietic Cells Mobilized With BL-8040 in CMV Seropositive Recipients |
|---|---|
| Description | -CMV reactivation will be defined as a positive test for CMV viremia as determined by an antigenemia assay or quantitative PCR that results in the administration of antiviral treatment directed against CMV |
| Time Frame | Up to 1 year after transplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| -Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Count of Participants [Participants] |
8
32%
|
| Title | Cumulative Incidence of Treatment-related Mortality After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) |
|---|---|
| Description | -Death that results from a transplant procedure related complication (e.g. infection, organ failure, hemorrhage, GVHD) rather than from relapse of the underlying disease or an unrelated cause |
| Time Frame | Up to 1 year after transplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| -Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Number (95% Confidence Interval) [proportion of participants] |
0.2273
0.9%
|
| Title | Cumulative Incidence of Disease Relapse/Progression After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) |
|---|---|
| Description | -Disease relapse occurs in recipients who entered transplant in CR. Progression occurs in recipients with existent disease at transplant who meet criteria for progressive disease post-transplant. A recipient will be considered relapsed when there is a recurrence of the original malignant disease after transplantation. Date of relapse/progression is defined as the date at which the first observation of hematologic, radiographic, or cytogenetic changes which signify progression/relapse is made |
| Time Frame | At 1 year post-tranplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Number (95% Confidence Interval) [proportion of participants] |
0.227
0.9%
|
| Title | Cumulative Incidence of Disease Relapse/Progression After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) |
|---|---|
| Description | -Disease relapse occurs in recipients who entered transplant in CR. Progression occurs in recipients with existent disease at transplant who meet criteria for progressive disease post-transplant. A recipient will be considered relapsed when there is a recurrence of the original malignant disease after transplantation. Date of relapse/progression is defined as the date at which the first observation of hematologic, radiographic, or cytogenetic changes which signify progression/relapse is made |
| Time Frame | At 2 years post-tranplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Number (95% Confidence Interval) [proportion of participants] |
0.273
1.1%
|
| Title | Kaplan-Meier Estimate of Event Free Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) |
|---|---|
| Description | -An event is defined as either graft failure, disease relapse as evidenced by hematologic, radiographic, or cytogenetic changes, or death. The event free survival is the time from Day 0 to occurrence of the first event. |
| Time Frame | At 2 years post-transplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Number [percentage of participants] |
50.0
200%
|
| Title | Kaplan-Meier Estimate of Event Free Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) |
|---|---|
| Description | -An event is defined as either graft failure, disease relapse as evidenced by hematologic, radiographic, or cytogenetic changes, or death. The event free survival is the time from Day 0 to occurrence of the first event. |
| Time Frame | At 3 years post-transplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Number [percentage of participants] |
34.1
136.4%
|
| Title | Kaplan-Meier Estimate of Overall Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) |
|---|---|
| Description | -The time from Day 0 to death |
| Time Frame | At 2 years post-transplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Number [percentage of participants] |
63.6
254.4%
|
| Title | Kaplan-Meier Estimate of Overall Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only) |
|---|---|
| Description | -The time from Day 0 to death |
| Time Frame | At 3 years post-transplantation |
Outcome Measure Data
| Analysis Population Description |
|---|
| Donors are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 0 | 22 |
| Number [percentage of participants] |
49.0
196%
|
| Title | Median Peripheral Blood CD34+ Cell Count (Arm 1 Donor Only) |
|---|---|
| Description | |
| Time Frame | At 3-4 hours after BL-8040 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Recipients are not evaluable for this outcome measure. |
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 |
| Measure Participants | 25 | 0 |
| Median (Full Range) [CD34/microliters] |
15
|
Adverse Events
| Time Frame | -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected. | |||
|---|---|---|---|---|
| Adverse Event Reporting Description | ||||
| Arm/Group Title | Arm 1: Donors | Arm 2: Recipients | ||
| Arm/Group Description | -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10^6 CD34+ cells/kg and at least ≥ 2 x 10^6 CD34+ cells/kg from the combined collections. | -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0 | ||
All Cause Mortality |
||||
| Arm 1: Donors | Arm 2: Recipients | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/25 (0%) | 11/22 (50%) | ||
Serious Adverse Events |
||||
| Arm 1: Donors | Arm 2: Recipients | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/25 (0%) | 12/22 (54.5%) | ||
| Blood and lymphatic system disorders | ||||
| Febrile neutropenia | 0/25 (0%) | 2/22 (9.1%) | ||
| Gastrointestinal disorders | ||||
| Diarrhea | 0/25 (0%) | 3/22 (13.6%) | ||
| Mucositis oral | 0/25 (0%) | 1/22 (4.5%) | ||
| Nausea | 0/25 (0%) | 1/22 (4.5%) | ||
| Vomiting | 0/25 (0%) | 1/22 (4.5%) | ||
| General disorders | ||||
| Fever | 0/25 (0%) | 1/22 (4.5%) | ||
| Generalized weakness | 0/25 (0%) | 1/22 (4.5%) | ||
| Infections and infestations | ||||
| Sepsis | 0/25 (0%) | 2/22 (9.1%) | ||
| Upper respiratory infection | 0/25 (0%) | 1/22 (4.5%) | ||
| Injury, poisoning and procedural complications | ||||
| Fall | 0/25 (0%) | 1/22 (4.5%) | ||
| Nervous system disorders | ||||
| Headache | 0/25 (0%) | 1/22 (4.5%) | ||
| Psychiatric disorders | ||||
| Delirium | 0/25 (0%) | 1/22 (4.5%) | ||
| Renal and urinary disorders | ||||
| Acute kidney injury | 0/25 (0%) | 3/22 (13.6%) | ||
| Reproductive system and breast disorders | ||||
| Genital edema | 0/25 (0%) | 1/22 (4.5%) | ||
| Testicular pain | 0/25 (0%) | 1/22 (4.5%) | ||
| Respiratory, thoracic and mediastinal disorders | ||||
| Respiratory failure | 0/25 (0%) | 1/22 (4.5%) | ||
Other (Not Including Serious) Adverse Events |
||||
| Arm 1: Donors | Arm 2: Recipients | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 25/25 (100%) | 22/22 (100%) | ||
| Blood and lymphatic system disorders | ||||
| Anemia | 17/25 (68%) | 0/22 (0%) | ||
| Febrile neutropenia | 0/25 (0%) | 7/22 (31.8%) | ||
| Cardiac disorders | ||||
| Acute systolic heart failure | 0/25 (0%) | 1/22 (4.5%) | ||
| Atrial fibrillation | 1/25 (4%) | 0/22 (0%) | ||
| Chest pain-cardiac | 0/25 (0%) | 1/22 (4.5%) | ||
| Sinus brachycardia | 4/25 (16%) | 0/22 (0%) | ||
| Sinus tachycardia | 2/25 (8%) | 0/22 (0%) | ||
| Eye disorders | ||||
| Eye pain | 1/25 (4%) | 0/22 (0%) | ||
| Watering eyes | 1/25 (4%) | 0/22 (0%) | ||
| Gastrointestinal disorders | ||||
| Abdominal pain | 0/25 (0%) | 1/22 (4.5%) | ||
| Colitis | 0/25 (0%) | 1/22 (4.5%) | ||
| Diarrhea | 0/25 (0%) | 1/22 (4.5%) | ||
| Esophagitis | 0/25 (0%) | 1/22 (4.5%) | ||
| Gastrointestinal reflux disease | 1/25 (4%) | 0/22 (0%) | ||
| Mucositis oral | 0/25 (0%) | 7/22 (31.8%) | ||
| Nausea | 1/25 (4%) | 2/22 (9.1%) | ||
| Oral dysesthesia | 6/25 (24%) | 0/22 (0%) | ||
| Toothache | 1/25 (4%) | 0/22 (0%) | ||
| Vomiting | 2/25 (8%) | 0/22 (0%) | ||
| General disorders | ||||
| Chills | 1/25 (4%) | 0/22 (0%) | ||
| Edema limbs-anasarca | 0/25 (0%) | 1/22 (4.5%) | ||
| Fatigue | 3/25 (12%) | 2/22 (9.1%) | ||
| Fever | 1/25 (4%) | 0/22 (0%) | ||
| Infusion related reaction | 1/25 (4%) | 0/22 (0%) | ||
| Injection site reaction | 20/25 (80%) | 0/22 (0%) | ||
| Localized edema | 1/25 (4%) | 0/22 (0%) | ||
| Pain | 1/25 (4%) | 0/22 (0%) | ||
| Vibration sensation | 1/25 (4%) | 0/22 (0%) | ||
| Hepatobiliary disorders | ||||
| Hepatic failure | 0/25 (0%) | 1/22 (4.5%) | ||
| Immune system disorders | ||||
| Allergic reaction | 20/25 (80%) | 0/22 (0%) | ||
| Infections and infestations | ||||
| Bacteremia-Klebsiella pneumoniae | 0/25 (0%) | 1/22 (4.5%) | ||
| Bacteremia-VRE | 0/25 (0%) | 1/22 (4.5%) | ||
| Catheter related infection | 0/25 (0%) | 1/22 (4.5%) | ||
| Clostridium difficile | 0/25 (0%) | 2/22 (9.1%) | ||
| Hepatitis B Core Ab positive | 2/25 (8%) | 0/22 (0%) | ||
| Lung infection | 0/25 (0%) | 2/22 (9.1%) | ||
| Mild head cold | 1/25 (4%) | 0/22 (0%) | ||
| Oral thrush | 0/25 (0%) | 1/22 (4.5%) | ||
| Papulopustular rash | 1/25 (4%) | 0/22 (0%) | ||
| Skin infection | 0/25 (0%) | 1/22 (4.5%) | ||
| Urinary tract infection | 0/25 (0%) | 2/22 (9.1%) | ||
| Injury, poisoning and procedural complications | ||||
| Bruising | 1/25 (4%) | 0/22 (0%) | ||
| Investigations | ||||
| Activated partial thromboplastin time prolonged | 1/25 (4%) | 0/22 (0%) | ||
| Alanine aminotransferase increased | 1/25 (4%) | 2/22 (9.1%) | ||
| Alkaline phosphatase increased | 1/25 (4%) | 0/22 (0%) | ||
| Aspartate aminotransferase increased | 0/25 (0%) | 1/22 (4.5%) | ||
| Blood bilirubin increased | 0/25 (0%) | 1/22 (4.5%) | ||
| Cholesterol high | 6/25 (24%) | 0/22 (0%) | ||
| GGT increased | 0/25 (0%) | 1/22 (4.5%) | ||
| Hemoglobin increased | 2/25 (8%) | 0/22 (0%) | ||
| Lymphocyte count decreased | 8/25 (32%) | 0/22 (0%) | ||
| Lymphocyte count increased | 22/25 (88%) | 0/22 (0%) | ||
| Neutrophil count decreased | 1/25 (4%) | 0/22 (0%) | ||
| Platelet count decreased | 22/25 (88%) | 0/22 (0%) | ||
| White blood cell decreased | 2/25 (8%) | 0/22 (0%) | ||
| Metabolism and nutrition disorders | ||||
| Anorexia | 0/25 (0%) | 1/22 (4.5%) | ||
| Dehydration | 0/25 (0%) | 1/22 (4.5%) | ||
| Hypercalcemia | 0/25 (0%) | 1/22 (4.5%) | ||
| Hyperglycemia | 3/25 (12%) | 4/22 (18.2%) | ||
| Hyperkalemia | 1/25 (4%) | 2/22 (9.1%) | ||
| Hypermagnesemia | 0/25 (0%) | 1/22 (4.5%) | ||
| Hypertriglyceridemia | 4/25 (16%) | 0/22 (0%) | ||
| Hyperuricemia | 1/25 (4%) | 2/22 (9.1%) | ||
| Hypocalcemia | 10/25 (40%) | 0/22 (0%) | ||
| Hypokalemia | 0/25 (0%) | 4/22 (18.2%) | ||
| Hypomagnesemia | 1/25 (4%) | 0/22 (0%) | ||
| Hyponatremia | 1/25 (4%) | 1/22 (4.5%) | ||
| Hypophosphatemia | 0/25 (0%) | 6/22 (27.3%) | ||
| Type II diabetes | 1/25 (4%) | 0/22 (0%) | ||
| Musculoskeletal and connective tissue disorders | ||||
| Arthralgia | 1/25 (4%) | 0/22 (0%) | ||
| Back pain | 0/25 (0%) | 2/22 (9.1%) | ||
| Bone pain | 2/25 (8%) | 0/22 (0%) | ||
| Foot pain | 1/25 (4%) | 0/22 (0%) | ||
| Leg/knee pain | 1/25 (4%) | 0/22 (0%) | ||
| Pain in extremity | 1/25 (4%) | 0/22 (0%) | ||
| Nervous system disorders | ||||
| Dizziness | 1/25 (4%) | 0/22 (0%) | ||
| Dysesthesia | 1/25 (4%) | 0/22 (0%) | ||
| Dysgeusia | 1/25 (4%) | 0/22 (0%) | ||
| Headache | 3/25 (12%) | 3/22 (13.6%) | ||
| Paresthesia | 3/25 (12%) | 0/22 (0%) | ||
| Syncope | 1/25 (4%) | 1/22 (4.5%) | ||
| Psychiatric disorders | ||||
| Anxiety | 1/25 (4%) | 0/22 (0%) | ||
| Insomnia | 2/25 (8%) | 0/22 (0%) | ||
| Renal and urinary disorders | ||||
| Cystitis noninfective | 0/25 (0%) | 1/22 (4.5%) | ||
| Renal failure | 0/25 (0%) | 1/22 (4.5%) | ||
| Respiratory, thoracic and mediastinal disorders | ||||
| Allergic rhinitis | 2/25 (8%) | 0/22 (0%) | ||
| Dyspnea | 1/25 (4%) | 0/22 (0%) | ||
| Epistaxis | 0/25 (0%) | 1/22 (4.5%) | ||
| Hypoxia | 0/25 (0%) | 4/22 (18.2%) | ||
| Pneumonitis | 0/25 (0%) | 1/22 (4.5%) | ||
| Pulmonary edema | 0/25 (0%) | 1/22 (4.5%) | ||
| Respiratory failure | 0/25 (0%) | 1/22 (4.5%) | ||
| Sore throat | 0/25 (0%) | 1/22 (4.5%) | ||
| Skin and subcutaneous tissue disorders | ||||
| Rash maculo-papular | 1/25 (4%) | 0/22 (0%) | ||
| Scrotal pruritus | 0/25 (0%) | 1/22 (4.5%) | ||
| Vascular disorders | ||||
| Hypertension | 16/25 (64%) | 11/22 (50%) | ||
| Hypotension | 2/25 (8%) | 1/22 (4.5%) | ||
| Veno-occlusive disease | 0/25 (0%) | 1/22 (4.5%) | ||
Limitations/Caveats
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.
Results Point of Contact
| Name/Title | Geoffrey Uy, M.D. |
|---|---|
| Organization | Washington University School of Medicine |
| Phone | 314-454-8304 |
| guy@wustl.edu |
- 201602037