Trial Outcomes & Findings for Melphalan and Bortezomib Prior to Autologous Stem Cell Transplant in Treating Patients With Multiple Myeloma (NCT NCT02353572)

NCT ID: NCT02353572

Last Updated: 2015-03-09

Results Overview

MTD is defined as one dose below that which 33% of patients within cohort experienced dose limiting toxicity. Unacceptable toxicity is defined as intractable veno-occlusive disorder, new onset of renal failure requiring dialysis, acute heart failure of New York Heart Association class III/IV, or interstitial pneumonia requiring ventilator management for longer than 3 days or grade 4 neuropathy. A 100-day mortality/toxicity rate of 3% or more is considered unacceptable. Will consider as evidence an observed 100-day mortality/toxicity rate whose lower one-sided 90% confidence bound exceeds 3%.

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 3 participants
• Primary outcome timeframe: 100 days
• Results posted on: 2015-03-09

Participant Flow

Between November 2009 and September 2011, 3 patients were enrolled in the study from University of Colorado Cancer Center

Within 35 days of planned therapy, patients were evaluated for eligibility by standard tests: paraprotein assessment, bone marrow biopsy and aspiration, MRI scan, PET/CT scan, echocardiography and pulmonary function test. Patients who were not be able to pursue the tandem autotransplant were eligible for one autotransplant.

Participant milestones

Measure	Melphalan, Bortezomib, Autologous Transplant Patients received melphalan IV continuously on days -5 to -2 and bortezomib IV over 3-5 seconds on days -4 and -1. Patients also received dexamethasone IV on day -1 prior to the second dose of bortezomib. Beginning two days after completion of melphalan infusion, patients underwent autologous hematopoietic stem cell transplant.
Overall Study STARTED	3
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	Melphalan, Bortezomib, Autologous Transplant Patients received melphalan IV continuously on days -5 to -2 and bortezomib IV over 3-5 seconds on days -4 and -1. Patients also received dexamethasone IV on day -1 prior to the second dose of bortezomib. Beginning two days after completion of melphalan infusion, patients underwent autologous hematopoietic stem cell transplant.
Overall Study Withdrawal by Subject	1
Overall Study Study terminated	2

Baseline Characteristics

Melphalan and Bortezomib Prior to Autologous Stem Cell Transplant in Treating Patients With Multiple Myeloma

Baseline characteristics by cohort

Measure	Melphalan, Bortezomib, Autologous Transplant n=3 Participants Patients received melphalan IV continuously on days -5 to -2 and bortezomib IV over 3-5 seconds on days -4 and -1. Patients also received dexamethasone IV on day -1 prior to the second dose of bortezomib. Beginning two days after completion of melphalan infusion, patients underwent autologous hematopoietic stem cell transplant.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	62.3 Years STANDARD_DEVIATION 5.0 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 100 days

Population: The study was terminated, study endpoints were not reached.

MTD is defined as one dose below that which 33% of patients within cohort experienced dose limiting toxicity. Unacceptable toxicity is defined as intractable veno-occlusive disorder, new onset of renal failure requiring dialysis, acute heart failure of New York Heart Association class III/IV, or interstitial pneumonia requiring ventilator management for longer than 3 days or grade 4 neuropathy. A 100-day mortality/toxicity rate of 3% or more is considered unacceptable. Will consider as evidence an observed 100-day mortality/toxicity rate whose lower one-sided 90% confidence bound exceeds 3%.

Outcome measures

Outcome data not reported

Adverse Events

Melphalan, Bortezomib, Autologous Transplant

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Choon-kee Lee, MD

University of Colorado

Phone: 7208489252

Results disclosure agreements

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