Trial Outcomes & Findings for Autologous or Syngeneic Stem Cell Transplant Followed by Donor Stem Cell Transplant and Bortezomib in Treating Patients With Newly Diagnosed High-Risk, Relapsed, or Refractory Multiple Myeloma (NCT NCT00793572)

Last Updated: 2020-01-22

Results Overview

Number of subjects surviving without progressive disease post-transplant. Progressive disease criteria: 1. Greater than 25% increase in serum (absolute increase must be ≥0.5 g/dL) or urine (absolute increase must be ≥200 mg/24h) M proteins compared to best response status after autologous transplant. 2. Appearance of new lytic bone lesions or plasmacytomas.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

32 participants

Primary outcome timeframe

At 2 years after the autograft

Results posted on

2020-01-22

Participant Flow

Participant milestones

Participant milestones
Measure	Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy See Detailed Description Autologous Hematopoietic Stem Cell Transplantation: Undergo transplantation Bortezomib: Given SC Cyclosporine: Given IV Cyclosporine: Given PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given PO Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo transplantation Peripheral Blood Stem Cell Transplantation: Undergo transplantation Syngeneic Bone Marrow Transplantation: Undergo transplantation Total-Body Irradiation: Undergo radiotherapy
Overall Study STARTED	32
Overall Study COMPLETED	32
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Autologous or Syngeneic Stem Cell Transplant Followed by Donor Stem Cell Transplant and Bortezomib in Treating Patients With Newly Diagnosed High-Risk, Relapsed, or Refractory Multiple Myeloma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy n=32 Participants See Detailed Description Autologous Hematopoietic Stem Cell Transplantation: Undergo transplantation Bortezomib: Given SC Cyclosporine: Given IV Cyclosporine: Given PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given PO Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo transplantation Peripheral Blood Stem Cell Transplantation: Undergo transplantation Syngeneic Bone Marrow Transplantation: Undergo transplantation Total-Body Irradiation: Undergo radiotherapy
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	30 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age, Continuous	49.2 years n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants
Region of Enrollment United States	32 participants n=5 Participants

PRIMARY outcome

Timeframe: At 2 years after the autograft

Outcome measures

Outcome measures
Measure	Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy n=32 Participants See Detailed Description Autologous Hematopoietic Stem Cell Transplantation: Undergo transplantation Bortezomib: Given SC Cyclosporine: Given IV Cyclosporine: Given PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given PO Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo transplantation Peripheral Blood Stem Cell Transplantation: Undergo transplantation Syngeneic Bone Marrow Transplantation: Undergo transplantation Total-Body Irradiation: Undergo radiotherapy
Number of Patients Surviving Progression-free	14 Participants

SECONDARY outcome

Timeframe: 100 days post allo transplant

Number of patients who developed acute GVHD post allogeneic transplant. aGVHD Stages Skin: 1. \- a maculopapular eruption involving \< 25% BSA 2. \- a maculopapular eruption involving 25 - 50% BSA 3. \- generalized erythroderma 4. \- generalized erythroderma w/ bullous formation and often w/ desquamation Liver: 1. \- bilirubin 2.0 - 3.0 mg/100 mL 2. \- bilirubin 3 - 5.9 mg/100 mL 3. \- bilirubin 6 - 14.9 mg/100 mL 4. \- bilirubin \> 15 mg/100 mL Gut: Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall. aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death

Outcome measures

Outcome measures
Measure	Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy n=32 Participants See Detailed Description Autologous Hematopoietic Stem Cell Transplantation: Undergo transplantation Bortezomib: Given SC Cyclosporine: Given IV Cyclosporine: Given PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given PO Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo transplantation Peripheral Blood Stem Cell Transplantation: Undergo transplantation Syngeneic Bone Marrow Transplantation: Undergo transplantation Total-Body Irradiation: Undergo radiotherapy
Number of Patients With Grade II-IV Acute GVHD	14 Participants

SECONDARY outcome

Timeframe: 1 year post allo

Number of subjects with classic chronic or chronic extensive GVHD post allogeneic transplant.

Outcome measures

Outcome measures
Measure	Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy n=32 Participants See Detailed Description Autologous Hematopoietic Stem Cell Transplantation: Undergo transplantation Bortezomib: Given SC Cyclosporine: Given IV Cyclosporine: Given PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given PO Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo transplantation Peripheral Blood Stem Cell Transplantation: Undergo transplantation Syngeneic Bone Marrow Transplantation: Undergo transplantation Total-Body Irradiation: Undergo radiotherapy
Number of Patients With Chronic GVHD	10 Participants

SECONDARY outcome

Timeframe: Up to 100 days after the autograft or allograft

Number of subjects with toxicities related to bortezomib maintenance therapy post-transplant.

Outcome measures

Outcome measures
Measure	Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy n=32 Participants See Detailed Description Autologous Hematopoietic Stem Cell Transplantation: Undergo transplantation Bortezomib: Given SC Cyclosporine: Given IV Cyclosporine: Given PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given PO Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo transplantation Peripheral Blood Stem Cell Transplantation: Undergo transplantation Syngeneic Bone Marrow Transplantation: Undergo transplantation Total-Body Irradiation: Undergo radiotherapy
Number of Patients With Toxicities Related to Bortezomib Maintenance Therapy	1 Participants

SECONDARY outcome

Timeframe: 200 and 365 days after allo

Number of subjects with non-relapse mortalities post allogeneic transplant.

Outcome measures

Outcome measures
Measure	Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy n=32 Participants See Detailed Description Autologous Hematopoietic Stem Cell Transplantation: Undergo transplantation Bortezomib: Given SC Cyclosporine: Given IV Cyclosporine: Given PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given PO Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo transplantation Peripheral Blood Stem Cell Transplantation: Undergo transplantation Syngeneic Bone Marrow Transplantation: Undergo transplantation Total-Body Irradiation: Undergo radiotherapy
Number of Patients With Non-relapse Mortality 200 days post allo	1 Participants
Number of Patients With Non-relapse Mortality 1 Year post allo	1 Participants

SECONDARY outcome

Timeframe: At 2 years after the autograft

Number of subjects surviving two years post autologous transplant.

Outcome measures

Outcome measures
Measure	Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy n=32 Participants See Detailed Description Autologous Hematopoietic Stem Cell Transplantation: Undergo transplantation Bortezomib: Given SC Cyclosporine: Given IV Cyclosporine: Given PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given PO Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo transplantation Peripheral Blood Stem Cell Transplantation: Undergo transplantation Syngeneic Bone Marrow Transplantation: Undergo transplantation Total-Body Irradiation: Undergo radiotherapy
Number of Patients Surviving Overall	21 Participants

Adverse Events

Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy

Serious events: 16 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy n=32 participants at risk See Detailed Description Autologous Hematopoietic Stem Cell Transplantation: Undergo transplantation Bortezomib: Given SC Cyclosporine: Given IV Cyclosporine: Given PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given PO Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo transplantation Peripheral Blood Stem Cell Transplantation: Undergo transplantation Syngeneic Bone Marrow Transplantation: Undergo transplantation Total-Body Irradiation: Undergo radiotherapy
Renal and urinary disorders Acute kidney injury	6.2% 2/32 • Number of events 2 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Infections and infestations Bronchial infection	6.2% 2/32 • Number of events 2 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Cardiac disorders Chest pain - cardiac	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Gastrointestinal disorders Enterocolitis	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
General disorders Fatigue	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Blood and lymphatic system disorders Febrile neutropenia	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
General disorders Fever	9.4% 3/32 • Number of events 3 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Gastrointestinal disorders Gastric hemorrhage	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Immune system disorders GVHD	9.4% 3/32 • Number of events 3 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Nervous system disorders Headache	6.2% 2/32 • Number of events 2 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Respiratory, thoracic and mediastinal disorders Hypoxia	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Gastrointestinal disorders Lower gastrointestinal hemorrhage	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Infections and infestations Mucosal infection	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
General disorders Multi-organ failure	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Gastrointestinal disorders Nausea	12.5% 4/32 • Number of events 4 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
General disorders Non-cardiac chest pain	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Gastrointestinal disorders Obstruction gastric	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Infections and infestations Sepsis	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Infections and infestations Sinusitis	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Vascular disorders Thromboembolic event	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Infections and infestations Upper respiratory infection	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Blood and lymphatic system disorders Blood and lymphatic system disorders, Other (Microangiopathy)	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Infections and infestations Infections and infestations, Other (Serum)	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto

Other adverse events

Other adverse events
Measure	Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy n=32 participants at risk See Detailed Description Autologous Hematopoietic Stem Cell Transplantation: Undergo transplantation Bortezomib: Given SC Cyclosporine: Given IV Cyclosporine: Given PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Melphalan: Given IV Mycophenolate Mofetil: Given PO Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation: Undergo transplantation Peripheral Blood Stem Cell Transplantation: Undergo transplantation Syngeneic Bone Marrow Transplantation: Undergo transplantation Total-Body Irradiation: Undergo radiotherapy
Cardiac disorders Atrial fibrillation	6.2% 2/32 • Number of events 2 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Investigations Blood bilirubin increased	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Gastrointestinal disorders Colitis	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Gastrointestinal disorders Diarrhea	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Respiratory, thoracic and mediastinal disorders Dyspnea	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Blood and lymphatic system disorders Febrile neutropenia	9.4% 3/32 • Number of events 3 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Vascular disorders Hypotension	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Respiratory, thoracic and mediastinal disorders Hypoxia	6.2% 2/32 • Number of events 3 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Cardiac disorders Left ventricular dysfunction	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Gastrointestinal disorders Mucositis oral	12.5% 4/32 • Number of events 5 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Cardiac disorders Pericardial effusion	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Cardiac disorders Pericardial tamponade	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto
Cardiac disorders Supraventricular tachycardia	3.1% 1/32 • Number of events 1 • AEs: Conditioning through Day 100 post Auto; SAEs: Conditioning through Day 200 post Auto

Additional Information

Dr. Marco Mielcarek

Fred Hutchinson Cancer Research Center

Phone: (206) 667-2827

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place