VelRand: Trial of High Dose Melphalan/Stem Cell Transplant With or Without Bortezomib

• Sponsor: Boston Medical Center (Other)
• Overall Status: Terminated
• CT.gov ID: NCT02489500
• Collaborator: (none)
• Enrollment: 3
• Location: 1
• Arms: 2
• Duration (Months): 22.9
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

Standard treatment for AL Amyloidosis is high-dose melphalan and stem cell transplant.

This study will compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose melphalan and stem cell transplant for AL amyloidosis.

Condition or Disease	Intervention/Treatment	Phase
AL Amyloidosis	Drug: Bortezomib; Drug: Melphalan; Drug: Neupogen; Procedure: Stem Cell Collection; Procedure: Stem cell infusion	Phase 3

Detailed Description

This study seeks to enroll patients with AL amyloidosis who have been recommended for standard treatment with high-dose melphalan and stem cell transplant.

Standard treatment for this disease is high-dose melphalan and stem cell transplant.

The purpose of this study is to compare the safety and effectiveness of standard treatment with high-dose melphalan and stem cell transplant, compared with investigational bortezomib when used in combination with standard treatment with high-dose melphalan and stem cell transplant for AL amyloidosis.

Patients enrolled in this study will receive either standard treatment with high-dose melphalan and stem cell transplant, or investigational bortezomib used in combination with standard treatment with high-dose melphalan and stem cell transplant.

Study Design

• Study Type: Interventional
• Actual Enrollment: 3 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Phase III Trial of High-dose Melphalan and Stem Cell Transplantation Versus High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis
• Study Start Date: Jun 1, 2015
• Actual Primary Completion Date: Apr 28, 2017
• Actual Study Completion Date: Apr 28, 2017

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: melphalan Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion	Drug: Melphalan; Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0; Other Names: Alkeran ; Drug: Neupogen; granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days; Other Names: granulocyte colony-stimulating factor (G-CSF) ; Procedure: Stem Cell Collection; collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells; Other Names: apheresis ; Procedure: Stem cell infusion; infusion of previously collected autologous stem cells; Other Names: infusion
Experimental: melphalan + Bortezomib Neupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion	Drug: Bortezomib; Conditioning Regimen: Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1; Other Names: VELCADE ; Drug: Melphalan; Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0; Other Names: Alkeran ; Drug: Neupogen; granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days; Other Names: granulocyte colony-stimulating factor (G-CSF) ; Procedure: Stem Cell Collection; collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells; Other Names: apheresis ; Procedure: Stem cell infusion; infusion of previously collected autologous stem cells; Other Names: infusion

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Hematologic Response [6 months]

   Hematologic response defined as: at least 50% improvement in the difference between involved and uninvolved free light chains

Secondary Outcome Measures

1. Toxicities [100 days]

   Number of serious adverse events per participant based on Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0

2. Overall Survival [5 years]

   duration of overall survival measured in days

3. Number of Participants With Organ Response [5 years]

   analysis of number of patients with organ response, as defined on page 13 of the detailed protocol for kidney, heart and liver.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Histological diagnosis of primary systemic (AL) amyloidosis based on:

• Deposition of amyloid material by Congo red stain showing characteristic apple green birefringence,AND…

• evidence of a clonal plasma cell dyscrasia with monoclonal protein in the serum or urine by immunofixation electrophoresis studies AND/OR abnormal serum free light chain assay AND/OR clonal plasma cells in the bone marrow exam demonstrated by immunohistochemistry, flow cytometry or in situ hybridization AND…

• evidence of organ involvement other than carpal tunnel syndrome. Patients with senile, secondary, localized, dialysis-related or familial amyloidosis are not eligible. Confirmation of tissue diagnosis at all sites of organ dysfunction is encouraged, but not required.

1. Patients must be > 18 years of age.

2. Patients must have a performance status of 0-2 by Eastern Cooperative Oncology Group (ECOG) criteria

3. Patients must have left ventricular ejection fraction (LVEF) > 45% by echocardiogram within 60 days of enrollment

4. Pulmonary Function Tests must show diffusing capacity of lung for carbon monoxide (DLCO) > 50%.

5. All patients must be informed of the investigational nature of this study and must sign and give written informed consent in accordance with institutional and federal guidelines.

Exclusion Criteria:

1. Patients with recent (< 6 months) myocardial infarction, congestive heart failure, New York Heart Association (NYHA) class III/IV or arrhythmia which are refractory to medical therapy are ineligible.

2. Prior chemotherapy with alkylating agent allowed only if no evidence of Myelodysplastic Dysplastic Syndrome (MDS) morphologically or cytogenetically. Total cumulative dose of oral melphalan must be < 300 mg. Patients should not have received any cytotoxic therapy < 4 weeks prior to registration and should have fully recovered from the effects of such therapy.

3. Patients must not have overt multiple myeloma (>30% bone marrow plasmacytosis and, extensive (>2) lytic lesions and hypercalcemia).

4. No other prior malignancy is allowed except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I or II cancer from which the patient is currently in complete remission, or any other cancer from which the patient has been disease-free for 5 years.

5. Patients must not be HIV positive.

6. Pregnant or nursing women may not participate. Women and men of reproductive potential may not participate unless they have agreed to use an effective contraceptive method.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Boston Medical Center	Boston	Massachusetts	United States	02118

Sponsors and Collaborators

• Boston Medical Center

Investigators

• Principal Investigator: Vaishali Sanchorawala, MD, Boston Medical Center

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Jul 13, 2015

More Information

Publications

None provided.

• Responsible Party: Vaishali Sanchorawala, Principal Investigator, Boston Medical Center
• ClinicalTrials.gov Identifier: NCT02489500
• Other Study ID Numbers: H-33808; VelRand
• First Posted: Jul 3, 2015
• Last Update Posted: Sep 10, 2018
• Last Verified: Aug 1, 2018

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: Yes
• Keywords provided by Vaishali Sanchorawala, Principal Investigator, Boston Medical Center: AL Amyloidosis; Stem Cell Transplantation
• Additional relevant MeSH terms: Immunoglobulin Light-chain Amyloidosis; Amyloidosis; Bortezomib; Melphalan; Lenograstim

Study Results

Participant Flow

Arm/Group Title	Melphalan	Melphalan + Bortezomib
Arm/Group Description	Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells	Neupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion Bortezomib: Conditioning Regimen: Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells
Period Title: Overall Study
STARTED	2	1
COMPLETED	1	0
NOT COMPLETED	1	1

Baseline Characteristics

Arm/Group Title	Melphalan	Melphalan + Bortezomib	Total
Arm/Group Description	Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells	Neupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion Bortezomib: Conditioning Regimen: Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells	Total of all reporting groups
Overall Participants	2	1	3
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%
Between 18 and 65 years	1 50%	1 100%	2 66.7%
>=65 years	1 50%	0 0%	1 33.3%
Sex: Female, Male (Count of Participants)
Female	0 0%	0 0%	0 0%
Male	2 100%	1 100%	3 100%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	0 0%	0 0%	0 0%
Not Hispanic or Latino	2 100%	1 100%	3 100%
Unknown or Not Reported	0 0%	0 0%	0 0%
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	0 0%	1 100%	1 33.3%
White	1 50%	0 0%	1 33.3%
More than one race	0 0%	0 0%	0 0%
Unknown or Not Reported	1 50%	0 0%	1 33.3%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Hematologic Response
Description	Hematologic response defined as: at least 50% improvement in the difference between involved and uninvolved free light chains
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

Two participants were not evaluable as they expired and did not have a post-treatment response evaluation.

Arm/Group Title	Melphalan	Melphalan + Bortezomib
Arm/Group Description	Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells	Neupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion Bortezomib: Conditioning Regimen: Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells
Measure Participants	1	0
Count of Participants [Participants]	0 0%	0 0%

2. Secondary Outcome

Title	Toxicities
Description	Number of serious adverse events per participant based on Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
Time Frame	100 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Melphalan	Melphalan + Bortezomib
Arm/Group Description	Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells	Neupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion Bortezomib: Conditioning Regimen: Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells
Measure Participants	2	1
Mean (Full Range) [events per participant]	8.5	15

3. Secondary Outcome

Title	Overall Survival
Description	duration of overall survival measured in days
Time Frame	5 years

Outcome Measure Data

Analysis Population Description

The protocol was closed prior to the 5-year period of assessment, so 5-year survival assessment of only two participants was assessed.

Arm/Group Title	Melphalan	Melphalan + Bortezomib
Arm/Group Description	Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells	Neupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion Bortezomib: Conditioning Regimen: Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells
Measure Participants	1	1
Number [days]	43	29

4. Secondary Outcome

Title	Number of Participants With Organ Response
Description	analysis of number of patients with organ response, as defined on page 13 of the detailed protocol for kidney, heart and liver.
Time Frame	5 years

Outcome Measure Data

Analysis Population Description

Due to early termination, organ response data was not collected.

Arm/Group Title	Melphalan	Melphalan + Bortezomib
Arm/Group Description	Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells	Neupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion Bortezomib: Conditioning Regimen: Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells
Measure Participants	0	0

Adverse Events

Time Frame	3 months
Adverse Event Reporting Description	Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Arm/Group Title	Melphalan		Melphalan + Bortezomib
Arm/Group Description	Neupogen 16mcg/kg x 4 days Stem cell collection Drug: high dose melphalan 140 or 200 mg/m2 stem cell infusion Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells		Neupogen 16mcg/kg x 4 days Stem Cell collection drug: high-dose melphalan 140 or 200 mg/m2 drug: Bortezomib 1.0 mg/m2/dose x 4 doses stem cell infusion Bortezomib: Conditioning Regimen: Drug: Bortezomib: 1.0 mg/m2/dose D -6, D -3, D +1, D + 4 Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Melphalan: Conditioning Regimen: Drug: Melphalan: 70-100 mg/m2/dose D -2, D -1 Stem Cell Transplant: D 0 Neupogen: granulocyte colony-stimulating factor (G-CSF) mobilization 16mcg/kg x 4 days Stem Cell Collection: collect at least 2.5 million cluster of differentiation 34 (CD34)+ stem cells Stem cell infusion: infusion of previously collected autologous stem cells
All Cause Mortality
	Melphalan		Melphalan + Bortezomib
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/2 (50%)		1/1 (100%)
Serious Adverse Events
	Melphalan		Melphalan + Bortezomib
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	2/2 (100%)		1/1 (100%)
Blood and lymphatic system disorders
neutropenia	2/2 (100%)	2	1/1 (100%)	1
leukopenia	2/2 (100%)	2	1/1 (100%)	1
thrombocytopenia	2/2 (100%)	2	1/1 (100%)	1
pancytopenia	2/2 (100%)	2	1/1 (100%)	1
Febrile Neutropenia	0/2 (0%)	0	1/1 (100%)	1
General disorders
fatigue	1/2 (50%)	1	1/1 (100%)	1
weakness	1/2 (50%)	1	1/1 (100%)	1
edema	1/2 (50%)	1	0/1 (0%)	0
Infections and infestations
sepsis	0/2 (0%)	0	1/1 (100%)	1
parainfluenza 4	0/2 (0%)	0	1/1 (100%)	1
bacteroids bacteremia	0/2 (0%)	0	1/1 (100%)	1
Nervous system disorders
somnolence	1/2 (50%)	1	0/1 (0%)	0
Renal and urinary disorders
anuria	1/2 (50%)	1	0/1 (0%)	0
Respiratory, thoracic and mediastinal disorders
hypoxia	1/2 (50%)	1	1/1 (100%)	2
oliguria	1/2 (50%)	1	0/1 (0%)	0
pleural effusion	1/2 (50%)	1	0/1 (0%)	0
shortness of breath	0/2 (0%)	0	1/1 (100%)	1
Vascular disorders
hypotension	1/2 (50%)	1	1/1 (100%)	1
thromboembolic event	0/2 (0%)	0	1/1 (100%)	1
Other (Not Including Serious) Adverse Events
	Melphalan		Melphalan + Bortezomib
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	2/2 (100%)		1/1 (100%)
Cardiac disorders
tachycardia	2/2 (100%)	2	0/1 (0%)	0
sinus tachycardia	0/2 (0%)	0	1/1 (100%)	1
Ear and labyrinth disorders
photophobia	1/2 (50%)	1	0/1 (0%)	0
Endocrine disorders
syndrome of inappropriate antidiuretic hormone secretion (SIADH)	1/2 (50%)	1	0/1 (0%)	0
adrenal insufficience	1/2 (50%)	1	0/1 (0%)	0
Eye disorders
photosensitivity	1/2 (50%)	1	0/1 (0%)	0
ptosis	0/2 (0%)	0	1/1 (100%)	1
right pupil fixed	0/2 (0%)	0	1/1 (100%)	1
Gastrointestinal disorders
nausea	2/2 (100%)	3	1/1 (100%)	1
constipation	2/2 (100%)	2	1/1 (100%)	1
vomiting	2/2 (100%)	3	0/1 (0%)	0
altered taste	1/2 (50%)	1	0/1 (0%)	0
xerostomia	1/2 (50%)	1	0/1 (0%)	0
abdominal distention	1/2 (50%)	1	1/1 (100%)	1
pain, right upper quadrant	1/2 (50%)	1	0/1 (0%)	0
diarrhea	2/2 (100%)	5	1/1 (100%)	2
enterocolitis	0/2 (0%)	0	1/1 (100%)	1
dysguesia	1/2 (50%)	1	0/1 (0%)	0
throat discomfort	1/2 (50%)	1	0/1 (0%)	0
General disorders
fatigue	1/2 (50%)	1	1/1 (100%)	1
gait disturbance	1/2 (50%)	1	1/1 (100%)	1
bleeding	1/2 (50%)	1	0/1 (0%)	0
Hepatobiliary disorders
hepatic pain, right upper quadrant	1/2 (50%)	1	0/1 (0%)	0
Infections and infestations
clostridium difficile	0/2 (0%)	0	1/1 (100%)	1
Investigations
alkaline phosphatase	2/2 (100%)	2	1/1 (100%)	1
Alanine Aminotransferase increase	2/2 (100%)	2	1/1 (100%)	1
aspartate aminotransferase increase	2/2 (100%)	2	1/1 (100%)	1
hyperbilirubinemia	0/2 (0%)	0	1/1 (100%)	1
Metabolism and nutrition disorders
hyponatremia	1/2 (50%)	1	1/1 (100%)	1
hypernatremia	0/2 (0%)	0	1/1 (100%)	1
hyperglycemia	2/2 (100%)	2	1/1 (100%)	1
hyperphosphatemia	2/2 (100%)	2	0/1 (0%)	0
hypoalbuminemia	2/2 (100%)	2	1/1 (100%)	1
increased creatinine	0/2 (0%)	0	1/1 (100%)	1
alkalosis	1/2 (50%)	1	0/1 (0%)	0
hypermagnesemia	1/2 (50%)	1	1/1 (100%)	1
Musculoskeletal and connective tissue disorders
pain - bone	2/2 (100%)	2	1/1 (100%)	1
pain- apheresis line tenderness	2/2 (100%)	2	1/1 (100%)	1
pain: abdominal	0/2 (0%)	0	1/1 (100%)	1
presyncope	1/2 (50%)	2	0/1 (0%)	0
Nervous system disorders
dizziness	2/2 (100%)	2	0/1 (0%)	0
weakness - bilateral tricep	0/2 (0%)	0	1/1 (100%)	1
neuropathy (unrelated to treatment)	1/2 (50%)	1	0/1 (0%)	0
syncope	1/2 (50%)	1	0/1 (0%)	0
somnolence	1/2 (50%)	1	0/1 (0%)	0
Psychiatric disorders
anxiety	2/2 (100%)	2	1/1 (100%)	1
hyperactive	1/2 (50%)	1	0/1 (0%)	0
insomnia	0/2 (0%)	0	1/1 (100%)	1
Renal and urinary disorders
urinary retention	1/2 (50%)	1	1/1 (100%)	1
creatinine increased	0/2 (0%)	0	1/1 (100%)	1
oliguria	1/2 (50%)	1	0/1 (0%)	0
acute kidney injury	1/2 (50%)	1	0/1 (0%)	0
Respiratory, thoracic and mediastinal disorders
hiccups	1/2 (50%)	1	0/1 (0%)	0
dyspnea	1/2 (50%)	1	1/1 (100%)	1
cough	0/2 (0%)	0	1/1 (100%)	1
pleural effusion	0/2 (0%)	0	1/1 (100%)	1
Skin and subcutaneous tissue disorders
alopecia	2/2 (100%)	2	1/1 (100%)	1
bruising	2/2 (100%)	2	0/1 (0%)	0
purpura	1/2 (50%)	1	0/1 (0%)	0
pruritis	1/2 (50%)	1	0/1 (0%)	0
Vascular disorders
edema	1/2 (50%)	1	1/1 (100%)	1
hypotension	1/2 (50%)	1	1/1 (100%)	3

Limitations/Caveats

Early termination after enrollment held for toxicity evaluation; then closed due to competing trial.

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.