Trial Outcomes & Findings for Trial of High Dose Melphalan/Stem Cell Transplant With or Without Bortezomib (NCT NCT02489500)

Last Updated: 2018-09-10

Results Overview

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

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

3 participants

Primary outcome timeframe

6 months

Results posted on

2018-09-10

Participant Flow

Participant milestones

Participant milestones
Measure	Melphalan 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	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 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
Overall Study STARTED	2	1
Overall Study COMPLETED	1	0
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Melphalan 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	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 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
Overall Study Death	1	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Melphalan n=2 Participants 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	Melphalan + Bortezomib n=1 Participants 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 n=3 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=99 Participants	1 Participants n=107 Participants	2 Participants n=206 Participants
Age, Categorical >=65 years	1 Participants n=99 Participants	0 Participants n=107 Participants	1 Participants n=206 Participants
Sex: Female, Male Female	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Sex: Female, Male Male	2 Participants n=99 Participants	1 Participants n=107 Participants	3 Participants n=206 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=99 Participants	1 Participants n=107 Participants	3 Participants n=206 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants	1 Participants n=107 Participants	1 Participants n=206 Participants
Race (NIH/OMB) White	1 Participants n=99 Participants	0 Participants n=107 Participants	1 Participants n=206 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=99 Participants	0 Participants n=107 Participants	1 Participants n=206 Participants

PRIMARY outcome

Timeframe: 6 months

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

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

Outcome measures

Outcome measures
Measure	Melphalan n=1 Participants 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	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 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
Number of Participants With Hematologic Response	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 100 days

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

Outcome measures

Outcome measures
Measure	Melphalan n=2 Participants 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	Melphalan + Bortezomib n=1 Participants 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
Toxicities	8.5 events per participant Interval 4.0 to 13.0	15 events per participant Interval 15.0 to 15.0

SECONDARY outcome

Timeframe: 5 years

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

duration of overall survival measured in days

Outcome measures

Outcome measures
Measure	Melphalan n=1 Participants 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	Melphalan + Bortezomib n=1 Participants 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
Overall Survival	43 days	29 days

SECONDARY outcome

Timeframe: 5 years

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

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

Outcome measures

Outcome data not reported

Adverse Events

Melphalan

Serious events: 2 serious events

Other events: 2 other events

Deaths: 1 deaths

Melphalan + Bortezomib

Serious events: 1 serious events

Other events: 1 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Additional Information

Vaishali Sanchorawala, MD

Boston Medical Center

Phone: 6176388261

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Melphalan n=2 participants at risk 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	Melphalan + Bortezomib n=1 participants at risk 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
Blood and lymphatic system disorders neutropenia	100.0% 2/2 • Number of events 2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Blood and lymphatic system disorders leukopenia	100.0% 2/2 • Number of events 2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Blood and lymphatic system disorders thrombocytopenia	100.0% 2/2 • Number of events 2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Blood and lymphatic system disorders pancytopenia	100.0% 2/2 • Number of events 2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Blood and lymphatic system disorders Febrile Neutropenia	0.00% 0/2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
General disorders fatigue	50.0% 1/2 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
General disorders weakness	50.0% 1/2 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Infections and infestations sepsis	0.00% 0/2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Infections and infestations parainfluenza 4	0.00% 0/2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Infections and infestations bacteroids bacteremia	0.00% 0/2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
General disorders edema	50.0% 1/2 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	0.00% 0/1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Respiratory, thoracic and mediastinal disorders hypoxia	50.0% 1/2 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Renal and urinary disorders anuria	50.0% 1/2 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	0.00% 0/1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Respiratory, thoracic and mediastinal disorders oliguria	50.0% 1/2 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	0.00% 0/1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Vascular disorders hypotension	50.0% 1/2 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Vascular disorders thromboembolic event	0.00% 0/2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Nervous system disorders somnolence	50.0% 1/2 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	0.00% 0/1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Respiratory, thoracic and mediastinal disorders pleural effusion	50.0% 1/2 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	0.00% 0/1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.
Respiratory, thoracic and mediastinal disorders shortness of breath	0.00% 0/2 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.	100.0% 1/1 • Number of events 1 • 3 months Adverse event data were collected from the time of study entry through the date of program discharge, once blood cell counts returned to normal.