Trial Outcomes & Findings for Tandem Auto Stem Cell Transplant With Melphalan Followed by Melphalan and Bortezomib in Patients With Multiple Myeloma (NCT NCT01241708)

NCT ID: NCT01241708

Last Updated: 2025-09-17

Results Overview

Progression-free survival of participants that received tandem transplants on study

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 148 participants
• Primary outcome timeframe: 3 years
• Results posted on: 2025-09-17

Participant Flow

Participant milestones

Measure	Tandem Transplantation With Melphalan and Bortezomib Tandem autologous hematopoietic stem cell transplantation with melphalan followed by melphalan and bortezomib in patients with multiple myeloma Bortezomib: Bortezomib 1.6mg/m2 on day -4 and day -1
Overall Study STARTED	148
Overall Study COMPLETED	92
Overall Study NOT COMPLETED	56

Reasons for withdrawal

Measure	Tandem Transplantation With Melphalan and Bortezomib Tandem autologous hematopoietic stem cell transplantation with melphalan followed by melphalan and bortezomib in patients with multiple myeloma Bortezomib: Bortezomib 1.6mg/m2 on day -4 and day -1
Overall Study Withdrawal by Subject	6
Overall Study Screen Failure	6
Overall Study Did not move forward with 2nd Transplant	39
Overall Study Lost to Follow-up	5

Baseline Characteristics

Tandem Auto Stem Cell Transplant With Melphalan Followed by Melphalan and Bortezomib in Patients With Multiple Myeloma

Baseline characteristics by cohort

Measure	Tandem Transplantation With Melphalan and Bortezomib n=142 Participants Tandem autologous hematopoietic stem cell transplantation with melphalan followed by melphalan and bortezomib in patients with multiple myeloma Bortezomib: Bortezomib 1.6mg/m2 on day -4 and day -1
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	126 Participants n=93 Participants
Age, Categorical >=65 years	16 Participants n=93 Participants
Age, Continuous	56.4 years n=93 Participants
Sex: Female, Male Female	52 Participants n=93 Participants
Sex: Female, Male Male	90 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	15 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	123 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	4 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=93 Participants
Race (NIH/OMB) Asian	7 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=93 Participants
Race (NIH/OMB) Black or African American	13 Participants n=93 Participants
Race (NIH/OMB) White	108 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	12 Participants n=93 Participants
Region of Enrollment United States	142 participants n=93 Participants

PRIMARY outcome

Timeframe: 3 years

Progression-free survival of participants that received tandem transplants on study

Outcome measures

Measure	Tandem Transplantation With Melphalan and Bortezomib n=94 Participants Tandem autologous hematopoietic stem cell transplantation with melphalan followed by melphalan and bortezomib in patients with multiple myeloma Bortezomib: Bortezomib 1.6mg/m2 on day -4 and day -1
To Determine the Progression-free Survival of Patients With Multiple Myeloma Treated With Tandem Cycles of High-dose Melphalan Followed by High-dose Melphalan in Combination With Bortezomib With Autologous HSC Transplantation.	73.8 Percent Interval 64.3 to 82.2

SECONDARY outcome

Timeframe: 3 years

Population: 94 participant response assessed after completion of tandem transplant

Overall response rate of participants after completing tandem transplant. Overall Response rate defined as the composite endpoint of response to treatment which includes Complete Response (CR), Partial Response (PR), stable disease (SD) as defined in International Response Criteria.

Outcome measures

Measure	Tandem Transplantation With Melphalan and Bortezomib n=94 Participants Tandem autologous hematopoietic stem cell transplantation with melphalan followed by melphalan and bortezomib in patients with multiple myeloma Bortezomib: Bortezomib 1.6mg/m2 on day -4 and day -1
To Determine the Response Rate of Patients With Multiple Myeloma Treated With High-dose Melphalan or High-dose Melphalan in Combination With Bortezomib Given in Tandem Transplants.	86 Participants

SECONDARY outcome

Timeframe: 3 years

Population: 94 participant survival assessed after completion of tandem transplant

Survival rate of participants after completion of tandem transplant

Outcome measures

Measure	Tandem Transplantation With Melphalan and Bortezomib n=94 Participants Tandem autologous hematopoietic stem cell transplantation with melphalan followed by melphalan and bortezomib in patients with multiple myeloma Bortezomib: Bortezomib 1.6mg/m2 on day -4 and day -1
To Determine the Overall Survival of Patients With Multiple Myeloma Treated With High-dose Melphalan or High-dose Melphalan in Combination With Bortezomib Given in Tandem Transplants.	76 Participants

Adverse Events

Tandem Transplantation With Melphalan and Bortezomib

Serious events: 67 serious events

Other events: 0 other events

Deaths: 46 deaths

Serious adverse events

Measure	Tandem Transplantation With Melphalan and Bortezomib n=142 participants at risk Tandem autologous hematopoietic stem cell transplantation with melphalan followed by melphalan and bortezomib in patients with multiple myeloma Bortezomib: Bortezomib 1.6mg/m2 on day -4 and day -1
Blood and lymphatic system disorders Febrile Neutropenia	2.1% 3/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Infections and infestations Infection	10.6% 15/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Investigations Disease Progression	18.3% 26/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Investigations Fever	2.1% 3/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Immune system disorders Engraftment Syndrome	2.8% 4/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Investigations Failure to Thrive	1.4% 2/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Infections and infestations RSV	0.70% 1/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Gastrointestinal disorders Nausea	0.70% 1/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Gastrointestinal disorders Diarrhea	1.4% 2/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Infections and infestations Perrirectal Abcess	0.70% 1/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Vascular disorders Subdermal Hematoma	0.70% 1/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Renal and urinary disorders Acute Renal Insufficiency	0.70% 1/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Metabolism and nutrition disorders Tumor Lysis Syndrome	0.70% 1/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Metabolism and nutrition disorders Acidosis	0.70% 1/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Infections and infestations CMV Viremia	0.70% 1/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed
Skin and subcutaneous tissue disorders Rash	2.8% 4/142 • All-Cause Mortality was assessed for up to 3 years. Serious Adverse Events were assessed up to 100 days after bone marrow transplant SAEs are reported through 100 days after bone marrow transplant. Other (Not Including Serious) Adverse Events were not analyzed

Other adverse events

Adverse event data not reported

Additional Information

Joshua Zenreich

Hackensack Meridian Health

Phone: 5519964248

Email: joshua.zenreich@hmhn.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place