Trial Outcomes & Findings for High-Dose Gemcitabine, Busulfan and Melphalan With Hematopoietic-Cell Support for Patients With Poor-Risk Myeloma (NCT NCT01237951)

Last Updated: 2020-05-05

Results Overview

Stringent complete remission was defined as negative immunofixation on the serum and urine, disappearance of any soft tissue plasmacytomas, 5% or fewer plasma cells in bone marrow, normal free light chain ratio, and the absence of clonal cells in bone marrow by immunohistochemistry or immunofluorescence.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

75 participants

Primary outcome timeframe

100 days post-transplant

Results posted on

2020-05-05

Participant Flow

Participants were enrolled at the University of Texas MD Anderson Cancer Center. Out of 75, 1 participant died before starting the treatment.

Participant milestones

Participant milestones
Measure	Gemcitabine/Busulfan/Melphalan Patients ages 18-70 with primary refractory or relapsed myeloma, candidates for an autologous SCT (ASCT) who had previously received treatment with bortezomib, an immunomodulatory drug, or both, or who were receiving a salvage ASCT.
Overall Study STARTED	75
Overall Study COMPLETED	74
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Gemcitabine/Busulfan/Melphalan Patients ages 18-70 with primary refractory or relapsed myeloma, candidates for an autologous SCT (ASCT) who had previously received treatment with bortezomib, an immunomodulatory drug, or both, or who were receiving a salvage ASCT.
Overall Study Death	1

Baseline Characteristics

High-Dose Gemcitabine, Busulfan and Melphalan With Hematopoietic-Cell Support for Patients With Poor-Risk Myeloma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Gemcitabine/Busulfan/Melphalan n=74 Participants Patients ages 18-70 with primary refractory or relapsed myeloma, candidates for an autologous SCT (ASCT) who had previously received treatment with bortezomib, an immunomodulatory drug, or both, or who were receiving a salvage ASCT.
Age, Continuous	58.5 years STANDARD_DEVIATION 5.5 • n=93 Participants
Sex: Female, Male Female	17 Participants n=93 Participants
Sex: Female, Male Male	57 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	3 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	16 Participants n=93 Participants
Race (NIH/OMB) White	55 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	74 Participants n=93 Participants
Overall study group 1st SCT/salvage SCT	13 participants n=93 Participants
Overall study group Poor-risk cytogenetics	6 participants n=93 Participants
Overall study group Extramedullary disease	1 participants n=93 Participants
Overall study group Double refractory (IMiDs + proteasome inhibitors)	11 participants n=93 Participants
Overall study group Disease Status: Primary refractory	4 participants n=93 Participants
Overall study group Disease Status: Refractory relapse	11 participants n=93 Participants
Overall study group Disease Status: Sensitive relapse	4 participants n=93 Participants
Overall study group Response at ASCT: Response	8 participants n=93 Participants
Overall study group Response at ASCT: Refractory	11 participants n=93 Participants
Overall study group No. prior lines of treatment	10 participants n=93 Participants
Overall study group No. prior lines of treatment > 2	9 participants n=93 Participants
Overall study group Post-ASCT maintenance	16 participants n=93 Participants

PRIMARY outcome

Timeframe: 100 days post-transplant

Population: Only 65 participants had measurable disease at the time of ASCT.

Outcome measures

Outcome measures
Measure	Gemcitabine/Busulfan/Melphalan n=65 Participants Patients with refractory or relapsed myeloma received high-dose GemBuMel with ASCT
Participants Who Had Measurable Disease at Time of Transplant and Achieved a Stringent Complete Remission	16 Participants

SECONDARY outcome

Timeframe: From date of transplant until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 2 years.

Number of participants remain free of progression or death after ASCT

Outcome measures

Outcome measures
Measure	Gemcitabine/Busulfan/Melphalan n=74 Participants Patients with refractory or relapsed myeloma received high-dose GemBuMel with ASCT
Progression-free Survival (PFS)	31 Participants

SECONDARY outcome

Timeframe: From date of transplant until the date of death from any cause, assessed up to 2 years

Number of participants from ASCT to death or last contact

Outcome measures

Outcome measures
Measure	Gemcitabine/Busulfan/Melphalan n=74 Participants Patients with refractory or relapsed myeloma received high-dose GemBuMel with ASCT
Overall Survival	49 Participants

SECONDARY outcome

Timeframe: From date of transplant until the date of death from treatment-related complications, assessed up to 2 years

Participants who died from treatment-related complications from the time of ASCT.

Outcome measures

Outcome measures
Measure	Gemcitabine/Busulfan/Melphalan n=74 Participants Patients with refractory or relapsed myeloma received high-dose GemBuMel with ASCT
Percent of Participants Dying From Treatment-Related Complications	4 Participants

Adverse Events

Gemcitabine/Busulfan/Melphalan

Serious events: 31 serious events

Other events: 71 other events

Deaths: 5 deaths

Serious adverse events

Serious adverse events
Measure	Gemcitabine/Busulfan/Melphalan n=74 participants at risk Participants with refractory or relapsed myeloma received high-dose GemBuMel with ASCT.
Gastrointestinal disorders Diarrhea	2.7% 2/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Infections and infestations Infection	6.8% 5/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Cardiac disorders Cardiac	2.7% 2/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Gastrointestinal disorders Mucositis	17.6% 13/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Skin and subcutaneous tissue disorders Dermatitis	6.8% 5/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Hepatobiliary disorders Hyperbilirubinemia	12.2% 9/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.

Other adverse events

Other adverse events
Measure	Gemcitabine/Busulfan/Melphalan n=74 participants at risk Participants with refractory or relapsed myeloma received high-dose GemBuMel with ASCT.
Gastrointestinal disorders Mucositis	74.3% 55/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Skin and subcutaneous tissue disorders Dermatitis	45.9% 34/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Gastrointestinal disorders Diarrhea	17.6% 13/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Immune system disorders Infection	95.9% 71/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Cardiac disorders Cardiac	73.0% 54/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Hepatobiliary disorders Hyperbilirubinemia	12.2% 9/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.
Skin and subcutaneous tissue disorders Hand-foot syndrome	1.4% 1/74 • Up to 2 years All-Cause Mortality was monitored in for all enrolled 75 participants ( 1 participant died before receiving treatment and 4 while on study) and 74 participants with Serious and Other Adverse Events.

Additional Information

Dr. Nieto, Yago, M.D./Stem Cell Transplantation

UT MD Anderson Cancer Center

Phone: 713-792-2466

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place