Trial Outcomes & Findings for Exploratory Trial to Estimate Proportion of Patients With Tumor Cell Contaminated Leukapheresis Products With and Without Bortezomib With In-vivo Purging - Multiple Myeloma (MM) (NCT NCT02703779)

Last Updated: 2021-06-18

Results Overview

Estimate the proportion of subjects with plasma cell contamination (defined as \>0.01% and at least 100 cellular events) of harvested stem cell product by multi parametric flow cytometry (MFC) from patients with myeloma undergoing autologous stem cell collection 1) by standard of care mobilization using Granulocyte colony-stimulating factor (G-CSF) with or without Mozobil and 2) after two doses of bortezomib as in vivo purging plus standard of care using G-CSF with or without Mozobil.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

101 participants

Primary outcome timeframe

Day 0 for all subjects (Day 0 is the day of stem cell collection)

Results posted on

2021-06-18

Participant Flow

Participant milestones

Participant milestones
Measure	Stem Cell Collection Without In-vivo Purging With Bortezomib Standard of Care Stem cell collection without in-vivo purging with Bortezomib. Standard of Care drugs Granulocyte colony-stimulating factor (G-CSF) +/- Mozobil (the latter if needed). NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.	Stem Cell Collection With In-vivo Purging With Bortezomib Bortezomib will be given subcutaneously (SQ) at 1.3 mg/m2 on day -11 and day -8 followed by Granulocyte colony-stimulating factor (G-CSF) given SQ on day -4 thru day -1 and continued until the collection is completed. Mozobil will be given if needed. There must be at least 72 hours between each dose of bortezomib. NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Bortezomib: Bortezomib will be given to Group B participants by injection under the skin 11 days and 8 days before stem cell collection. Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.
Overall Study STARTED	51	50
Overall Study COMPLETED	51	50
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Exploratory Trial to Estimate Proportion of Patients With Tumor Cell Contaminated Leukapheresis Products With and Without Bortezomib With In-vivo Purging - Multiple Myeloma (MM)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Stem Cell Collection Without In-vivo Purging With Bortezomib n=51 Participants Standard of Care Stem cell collection without in-vivo purging with Bortezomib. Standard of Care drugs Granulocyte colony-stimulating factor (G-CSF) +/- Mozobil (the latter if needed). NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.	Stem Cell Collection With In-vivo Purging With Bortezomib n=50 Participants Bortezomib will be given subcutaneously (SQ) at 1.3 mg/m2 on day -11 and day -8 followed by Granulocyte colony-stimulating factor (G-CSF) given SQ on day -4 thru day -1 and continued until the collection is completed. Mozobil will be given if needed. There must be at least 72 hours between each dose of bortezomib. NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Bortezomib: Bortezomib will be given to Group B participants by injection under the skin 11 days and 8 days before stem cell collection. Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.	Total n=101 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	37 Participants n=5 Participants	29 Participants n=7 Participants	66 Participants n=5 Participants
Age, Categorical >=65 years	14 Participants n=5 Participants	21 Participants n=7 Participants	35 Participants n=5 Participants
Sex: Female, Male Female	21 Participants n=5 Participants	21 Participants n=7 Participants	42 Participants n=5 Participants
Sex: Female, Male Male	30 Participants n=5 Participants	29 Participants n=7 Participants	59 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	4 Participants n=7 Participants	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	49 Participants n=5 Participants	46 Participants n=7 Participants	95 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 0 for all subjects (Day 0 is the day of stem cell collection)

Outcome measures

Outcome measures
Measure	Stem Cell Collection Without In-vivo Purging With Bortezomib n=51 Participants Standard of Care Stem cell collection without in-vivo purging with Bortezomib. Standard of Care drugs Granulocyte colony-stimulating factor (G-CSF) +/- Mozobil (the latter if needed). NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.	Stem Cell Collection With In-vivo Purging With Bortezomib n=50 Participants Bortezomib will be given subcutaneously (SQ) at 1.3 mg/m2 on day -11 and day -8 followed by Granulocyte colony-stimulating factor (G-CSF) given SQ on day -4 thru day -1 and continued until the collection is completed. Mozobil will be given if needed. There must be at least 72 hours between each dose of bortezomib. NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Bortezomib: Bortezomib will be given to Group B participants by injection under the skin 11 days and 8 days before stem cell collection. Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.
Multiparametric Flow Cytometry - Minimum Residual Disease	11 Participants	13 Participants

SECONDARY outcome

Timeframe: Within the first 30 days after stem cell collection

Estimate the proportion of subjects who have a successful collection of stem cells (\> 2 million Cluster of Differentiation 34 (CD34) cells/Kg of body weight) for autologous transplant in both treatment groups.

Outcome measures

Outcome measures
Measure	Stem Cell Collection Without In-vivo Purging With Bortezomib n=51 Participants Standard of Care Stem cell collection without in-vivo purging with Bortezomib. Standard of Care drugs Granulocyte colony-stimulating factor (G-CSF) +/- Mozobil (the latter if needed). NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.	Stem Cell Collection With In-vivo Purging With Bortezomib n=50 Participants Bortezomib will be given subcutaneously (SQ) at 1.3 mg/m2 on day -11 and day -8 followed by Granulocyte colony-stimulating factor (G-CSF) given SQ on day -4 thru day -1 and continued until the collection is completed. Mozobil will be given if needed. There must be at least 72 hours between each dose of bortezomib. NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Bortezomib: Bortezomib will be given to Group B participants by injection under the skin 11 days and 8 days before stem cell collection. Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.
Multiparametric Flow Cytometry - Cluster of Differentiation 34 (CD34)Assessment	51 Participants	50 Participants

SECONDARY outcome

Timeframe: Within the first 30 days after stem cell collection

Estimate the percentage of Cluster of Differentiation 34 (CD34) positive cells in circulating peripheral blood as a measure of mobilization on the days of collection.

Outcome measures

Outcome measures
Measure	Stem Cell Collection Without In-vivo Purging With Bortezomib n=51 Participants Standard of Care Stem cell collection without in-vivo purging with Bortezomib. Standard of Care drugs Granulocyte colony-stimulating factor (G-CSF) +/- Mozobil (the latter if needed). NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.	Stem Cell Collection With In-vivo Purging With Bortezomib n=50 Participants Bortezomib will be given subcutaneously (SQ) at 1.3 mg/m2 on day -11 and day -8 followed by Granulocyte colony-stimulating factor (G-CSF) given SQ on day -4 thru day -1 and continued until the collection is completed. Mozobil will be given if needed. There must be at least 72 hours between each dose of bortezomib. NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Bortezomib: Bortezomib will be given to Group B participants by injection under the skin 11 days and 8 days before stem cell collection. Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.
Cluster of Differentiation 34 (CD34) Enumeration	8.9 percentage of CD34 positive cells Standard Deviation 0.1	7.8 percentage of CD34 positive cells Standard Deviation 0.1

Adverse Events

Stem Cell Collection Without In-vivo Purging With Bortezomib

Serious events: 12 serious events

Other events: 39 other events

Deaths: 3 deaths

Stem Cell Collection With In-vivo Purging With Bortezomib

Serious events: 24 serious events

Other events: 39 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Stem Cell Collection Without In-vivo Purging With Bortezomib n=51 participants at risk Standard of Care Stem cell collection without in-vivo purging with Bortezomib. Standard of Care drugs Granulocyte colony-stimulating factor (G-CSF) +/- Mozobil (the latter if needed). NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.	Stem Cell Collection With In-vivo Purging With Bortezomib n=50 participants at risk Bortezomib will be given subcutaneously (SQ) at 1.3 mg/m2 on day -11 and day -8 followed by Granulocyte colony-stimulating factor (G-CSF) given SQ on day -4 thru day -1 and continued until the collection is completed. Mozobil will be given if needed. There must be at least 72 hours between each dose of bortezomib. NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Bortezomib: Bortezomib will be given to Group B participants by injection under the skin 11 days and 8 days before stem cell collection. Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.
Blood and lymphatic system disorders Febrile neutropenia	15.7% 8/51 • Number of events 8 • From the time of signing the consent until 30 days after collection or up to 2 months.	14.0% 7/50 • Number of events 7 • From the time of signing the consent until 30 days after collection or up to 2 months.
Cardiac disorders Atrial fibrillation	2.0% 1/51 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.	4.0% 2/50 • Number of events 2 • From the time of signing the consent until 30 days after collection or up to 2 months.
Gastrointestinal disorders Diarrhea	2.0% 1/51 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.	2.0% 1/50 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.
Gastrointestinal disorders Enterocolitis	2.0% 1/51 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.	0.00% 0/50 • From the time of signing the consent until 30 days after collection or up to 2 months.
General disorders Fatigue	2.0% 1/51 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.	0.00% 0/50 • From the time of signing the consent until 30 days after collection or up to 2 months.
Gastrointestinal disorders Abdominal pain	0.00% 0/51 • From the time of signing the consent until 30 days after collection or up to 2 months.	2.0% 1/50 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.
Renal and urinary disorders Acute kidney injury	0.00% 0/51 • From the time of signing the consent until 30 days after collection or up to 2 months.	2.0% 1/50 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.
General disorders Fever	0.00% 0/51 • From the time of signing the consent until 30 days after collection or up to 2 months.	2.0% 1/50 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.
Vascular disorders Hypotension	0.00% 0/51 • From the time of signing the consent until 30 days after collection or up to 2 months.	4.0% 2/50 • Number of events 2 • From the time of signing the consent until 30 days after collection or up to 2 months.
Infections and infestations Infection	0.00% 0/51 • From the time of signing the consent until 30 days after collection or up to 2 months.	2.0% 1/50 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.
Gastrointestinal disorders Mucositis oral	0.00% 0/51 • From the time of signing the consent until 30 days after collection or up to 2 months.	8.0% 4/50 • Number of events 4 • From the time of signing the consent until 30 days after collection or up to 2 months.
Nervous system disorders Peripheral sensory neuropathy	0.00% 0/51 • From the time of signing the consent until 30 days after collection or up to 2 months.	2.0% 1/50 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.
Infections and infestations Sepsis	0.00% 0/51 • From the time of signing the consent until 30 days after collection or up to 2 months.	2.0% 1/50 • Number of events 1 • From the time of signing the consent until 30 days after collection or up to 2 months.
Nervous system disorders Syncope	0.00% 0/51 • From the time of signing the consent until 30 days after collection or up to 2 months.	4.0% 2/50 • Number of events 2 • From the time of signing the consent until 30 days after collection or up to 2 months.

Other adverse events

Additional Information

Dr. Sid Ganguly

University of Kansas Cancer Center

Phone: 913-588-6030

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Stem Cell Collection Without In-vivo Purging With Bortezomib n=51 participants at risk Standard of Care Stem cell collection without in-vivo purging with Bortezomib. Standard of Care drugs Granulocyte colony-stimulating factor (G-CSF) +/- Mozobil (the latter if needed). NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.	Stem Cell Collection With In-vivo Purging With Bortezomib n=50 participants at risk Bortezomib will be given subcutaneously (SQ) at 1.3 mg/m2 on day -11 and day -8 followed by Granulocyte colony-stimulating factor (G-CSF) given SQ on day -4 thru day -1 and continued until the collection is completed. Mozobil will be given if needed. There must be at least 72 hours between each dose of bortezomib. NOTE: Multiparametric Flow Cytometry to be performed on samples for BOTH groups A and B Bortezomib: Bortezomib will be given to Group B participants by injection under the skin 11 days and 8 days before stem cell collection. Granulocyte colony-stimulating factor (G-CSF): Granulocyte colony-stimulating factor (G-CSF) will be given to all participants by injection under the skin 4 days and 1 day before stem cell collection, and then continued until the stem cell collection is completed. Mozobil: Mozobil will be given to all participants by injection under the skin only if needed per Investigator.
Blood and lymphatic system disorders Anemia	17.6% 9/51 • Number of events 26 • From the time of signing the consent until 30 days after collection or up to 2 months.	14.0% 7/50 • Number of events 8 • From the time of signing the consent until 30 days after collection or up to 2 months.
Metabolism and nutrition disorders Anorexia	51.0% 26/51 • Number of events 34 • From the time of signing the consent until 30 days after collection or up to 2 months.	54.0% 27/50 • Number of events 33 • From the time of signing the consent until 30 days after collection or up to 2 months.
Psychiatric disorders Anxiety	21.6% 11/51 • Number of events 11 • From the time of signing the consent until 30 days after collection or up to 2 months.	18.0% 9/50 • Number of events 10 • From the time of signing the consent until 30 days after collection or up to 2 months.
Musculoskeletal and connective tissue disorders Back pain	17.6% 9/51 • Number of events 11 • From the time of signing the consent until 30 days after collection or up to 2 months.	16.0% 8/50 • Number of events 10 • From the time of signing the consent until 30 days after collection or up to 2 months.
Musculoskeletal and connective tissue disorders Bone pain	19.6% 10/51 • Number of events 10 • From the time of signing the consent until 30 days after collection or up to 2 months.	12.0% 6/50 • Number of events 7 • From the time of signing the consent until 30 days after collection or up to 2 months.
Gastrointestinal disorders Constipation	29.4% 15/51 • Number of events 16 • From the time of signing the consent until 30 days after collection or up to 2 months.	28.0% 14/50 • Number of events 14 • From the time of signing the consent until 30 days after collection or up to 2 months.
Gastrointestinal disorders Diarrhea	56.9% 29/51 • Number of events 55 • From the time of signing the consent until 30 days after collection or up to 2 months.	58.0% 29/50 • Number of events 45 • From the time of signing the consent until 30 days after collection or up to 2 months.
Respiratory, thoracic and mediastinal disorders Dyspnea	23.5% 12/51 • Number of events 12 • From the time of signing the consent until 30 days after collection or up to 2 months.	12.0% 6/50 • Number of events 7 • From the time of signing the consent until 30 days after collection or up to 2 months.
General disorders Edema limbs	23.5% 12/51 • Number of events 14 • From the time of signing the consent until 30 days after collection or up to 2 months.	20.0% 10/50 • Number of events 11 • From the time of signing the consent until 30 days after collection or up to 2 months.
General disorders Fatigue	41.2% 21/51 • Number of events 32 • From the time of signing the consent until 30 days after collection or up to 2 months.	42.0% 21/50 • Number of events 30 • From the time of signing the consent until 30 days after collection or up to 2 months.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	21.6% 11/51 • Number of events 12 • From the time of signing the consent until 30 days after collection or up to 2 months.	30.0% 15/50 • Number of events 16 • From the time of signing the consent until 30 days after collection or up to 2 months.
Vascular disorders Hypertension	17.6% 9/51 • Number of events 14 • From the time of signing the consent until 30 days after collection or up to 2 months.	8.0% 4/50 • Number of events 4 • From the time of signing the consent until 30 days after collection or up to 2 months.
Metabolism and nutrition disorders Hypokalemia	52.9% 27/51 • Number of events 53 • From the time of signing the consent until 30 days after collection or up to 2 months.	38.0% 19/50 • Number of events 43 • From the time of signing the consent until 30 days after collection or up to 2 months.
Metabolism and nutrition disorders Hypomagnesemia	27.5% 14/51 • Number of events 16 • From the time of signing the consent until 30 days after collection or up to 2 months.	14.0% 7/50 • Number of events 13 • From the time of signing the consent until 30 days after collection or up to 2 months.
Metabolism and nutrition disorders Hyponatremia	17.6% 9/51 • Number of events 16 • From the time of signing the consent until 30 days after collection or up to 2 months.	14.0% 7/50 • Number of events 9 • From the time of signing the consent until 30 days after collection or up to 2 months.
Psychiatric disorders Insomnia	25.5% 13/51 • Number of events 15 • From the time of signing the consent until 30 days after collection or up to 2 months.	8.0% 4/50 • Number of events 4 • From the time of signing the consent until 30 days after collection or up to 2 months.
Gastrointestinal disorders Mucositis oral	39.2% 20/51 • Number of events 27 • From the time of signing the consent until 30 days after collection or up to 2 months.	36.0% 18/50 • Number of events 28 • From the time of signing the consent until 30 days after collection or up to 2 months.
Gastrointestinal disorders Nausea	64.7% 33/51 • Number of events 59 • From the time of signing the consent until 30 days after collection or up to 2 months.	58.0% 29/50 • Number of events 45 • From the time of signing the consent until 30 days after collection or up to 2 months.
Investigations Neutrophil count decreased	41.2% 21/51 • Number of events 37 • From the time of signing the consent until 30 days after collection or up to 2 months.	32.0% 16/50 • Number of events 24 • From the time of signing the consent until 30 days after collection or up to 2 months.
Investigations Platelet count decreased	49.0% 25/51 • Number of events 68 • From the time of signing the consent until 30 days after collection or up to 2 months.	36.0% 18/50 • Number of events 24 • From the time of signing the consent until 30 days after collection or up to 2 months.
Gastrointestinal disorders Vomiting	47.1% 24/51 • Number of events 37 • From the time of signing the consent until 30 days after collection or up to 2 months.	42.0% 21/50 • Number of events 27 • From the time of signing the consent until 30 days after collection or up to 2 months.
Investigations White blood cell decreased	52.9% 27/51 • Number of events 46 • From the time of signing the consent until 30 days after collection or up to 2 months.	46.0% 23/50 • Number of events 46 • From the time of signing the consent until 30 days after collection or up to 2 months.