A Phase 2 interventional study of Autologous Hematopoietic Stem Cell Transplantation and Busulfan in Plasma Cell Leukemia, Plasmacytoma and Recurrent Plasma Cell Myeloma, sponsored by M.D. Anderson Cancer Center. Completed at 1 site in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2025-03-18.
Sponsored by M.D. Anderson Cancer Center · Phase 2, Interventional, and Treatment
This phase II trial studies how well panobinostat, gemcitabine hydrochloride, busulfan, and melphalan before stem cell transplant work in treating patients with multiple myeloma that does not respond to treatment (refractory) or has returned (relapsed). Panobinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving high-dose chemotherapy, such as gemcitabine hydrochloride, busulfan, and melphalan, before a peripheral blood stem cell transplant helps kill any cancer cells that are in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. Previously collected stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
PRIMARY OBJECTIVES:
I. To determine the progression-free survival (PFS) in patients with refractory or relapsed myeloma receiving panobinostat/gemcitabine hydrochloride (gemcitabine)/busulfan/melphalan (panobinostat/Gem/Bu/Mel) with autologous stem-cell transplant, either as a first or a salvage stem-cell transplant.
SECONDARY OBJECTIVES:
I. To evaluate the complete response (CR) rate. II. To determine the overall survival (OS). III. To determine the CR + very good partial remission (VGPR) rate. IV. To determine the overall response rate (ORR). V. To determine minimal residual disease posttransplant, measured by multiparametric flow cytometry (MFC).
VI. To describe the toxicity profile of panobinostat/Gem/Bu/Mel. VII. To analyze the predictive value of pretransplant levels in myeloma cells of X-box binding protein 1 (XBP1), inositol-requiring enzyme 1 (IRE1), unspliced XBP1 (XBP1u), sliced XBP1 (XPB1s), XBP1u/XPBs ratio and v-myc myelocytomatosis viral oncogene homolog (avian) (Myc), by analyzing their correlation with CR, VGPR+CR and response rate (RR).
VIII. To study the prognostic effect of pretransplant levels in myeloma cells of XBP1, IRE1, XBP1u, XPB1s, XBP1u/XPBs ratio and Myc, by analyzing their correlation with PFS and OS.
OUTLINE:
Patients receive panobinostat orally (PO) once daily (QD) on days -9 to -2, gemcitabine hydrochloride intravenously (IV) over 4 hours on days -8 and -3, busulfan IV over 3 hours on days -8 to -5, and melphalan IV over 30 minutes on days -3 and -2. Patients then undergo autologous peripheral blood stem cell transplant on day 0.
After completion of study treatment, patients are followed up at 1 month, 100 days, 6 months, 1 year, and then every 3-6 months for at least 2 years.
60 studies on the registry are indexed under Leukemia, Plasma Cell; 33 are open to participants now.
This study's enrollment of 83 is above the median of 27 across 53 interventional studies indexed under Leukemia, Plasma Cell.
Browse Leukemia, Plasma Cell studies →M.D. Anderson Cancer Center is the lead sponsor of 3,047 studies on the registry; 578 are open to participants now.
Of its 612 completed or terminated interventional studies of FDA-regulated products, 409 (67%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
or relapsed myeloma, defined as one or more of the following:
Treated with therapy including at least 2 cycles of lenalidomide, bortezomib or thalidomide, and one or more of the following:
Exclusion Criteria:
Impaired cardiac function or cardiac diseases, including any one of the following:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
FEV1
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients receive panobinostat PO QD on days -9 to -2, gemcitabine hydrochloride IV over 4 hours on days -8 and -3, busulfan IV over 3 hours on days -8 to -5, and melphalan IV over 30 minutes on days -3 and -2. Patients then undergo autologous peripheral blood stem cell transplant on day 0.
Procedure: Autologous Hematopoietic Stem Cell Transplantation · Drug: Busulfan · Drug: Gemcitabine Hydrochloride · Drug: Melphalan · Drug: Panobinostat · Procedure: Peripheral Blood Stem Cell Transplantation · Other: Pharmacological Study
Patients receive panobinostat PO QD on days -9 to -2, gemcitabine hydrochloride IV over 4 hours on days -8 and -3, busulfan IV over 3 hours on days -8 to -5, and melphalan IV over 30 minutes on days -3 and -2. Patients then undergo autologous peripheral blood stem cell transplant on day 0.
Procedure: Autologous Hematopoietic Stem Cell Transplantation · Drug: Busulfan · Drug: Gemcitabine Hydrochloride · Drug: Melphalan · Drug: Panobinostat · Procedure: Peripheral Blood Stem Cell Transplantation · Other: Pharmacological Study
Undergo autologous peripheral blood stem cell transplant
Also known as: Autologous Hematopoietic Cell Transplantation
Given IV
Also known as: 1, 4-Bis[methanesulfonoxy]butane, BUS, Bussulfam, Busulfanum, Busulfex, Busulphan, CB 2041, CB-2041, Glyzophrol, GT 41, GT-41, Joacamine, Methanesulfonic Acid Tetramethylene Ester, Methanesulfonic acid, tetramethylene ester, Mielucin, Misulban, Misulfan, Mitosan, Myeleukon, Myeloleukon, Myelosan, Mylecytan, Myleran, Sulfabutin, Tetramethylene Bis(methanesulfonate), Tetramethylene bis[methanesulfonate], WR-19508
Given IV
Also known as: dFdCyd, Difluorodeoxycytidine Hydrochloride, FF 10832, FF-10832, FF10832, Gemcitabine HCI, Gemzar, LY-188011, LY188011
Given IV
Also known as: Alanine Nitrogen Mustard, CB-3025, L-PAM, L-Phenylalanine Mustard, L-Sarcolysin, L-Sarcolysin Phenylalanine mustard, L-Sarcolysine, Melphalanum, Phenylalanine Mustard, Phenylalanine Nitrogen Mustard, Sarcoclorin, Sarkolysin, WR-19813
Given PO
Also known as: Faridak, LBH589
Undergo autologous peripheral blood stem cell transplant
Also known as: PBPC transplantation, PBSCT, Peripheral Blood, Peripheral Blood Progenitor Cell Transplantation, PERIPHERAL BLOOD STEM CELL TRANSPLANT, Peripheral Stem Cell Support, Peripheral Stem Cell Transplant, Peripheral Stem Cell Transplantation
Correlative studies
Progression Free Survival (PFS)
Number of participants alive and disease free one year post auto transplant in patients with refractory or relapsed myeloma receiving panobinostat/gemcitabine/busulfan/melphalan (panobinostat/Gem/Bu/Mel) with autologous stem-cell transplant, either as a first or a salvage stem-cell transplant.
Time frame: 1 year
Overall Survival (OS)
Number of participants with refractory or relapse Myeloma who still remain alive two years post transplant
Time frame: Up to 2 years
Participants Who Experienced Grade 3 or Higher Adverse Events
Number of Participants who experienced grade 3 or higher Adverse Events during the first 100 days of the transplant.
Time frame: Up to day 100
| Milestone | Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel |
|---|---|---|
| Started | 50 | 33 |
| Completed | 48 | 32 |
| Not completed | 2 | 1 |
| Withdrew: Physician decision | 2 | 1 |
Number of participants alive and disease free one year post auto transplant in patients with refractory or relapsed myeloma receiving panobinostat/gemcitabine/busulfan/melphalan (panobinostat/Gem/Bu/Mel) with autologous stem-cell transplant, either as a first or a salvage stem-cell transplant.
| Participants | Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel |
|---|---|---|
| Progression Free Survival (PFS) | 38 | 24 |
Number of participants with refractory or relapse Myeloma who still remain alive two years post transplant
| Participants | Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel |
|---|---|---|
| Overall Survival (OS) | 27 | 16 |
Number of Participants who experienced grade 3 or higher Adverse Events during the first 100 days of the transplant.
| Participants | Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel |
|---|---|---|
| Participants Who Experienced Grade 3 or Higher Adverse Events | 47 | 29 |
Collected over Up to 2 years. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | 23/50 (46%) | 2/50 (4%) | 47/50 (94%) |
| Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | 17/33 (51.5%) | 4/33 (12.1%) | 30/33 (90.9%) |
| Event | Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel |
|---|---|---|
| ViralInfections and infestations | 1/50 | 4/33 |
| FungalInfections and infestations | 1/50 | 2/33 |
| Pulmonary edemaRespiratory, thoracic and mediastinal disorders | 2/50 | 0/33 |
| AST increasedInvestigations | 0/50 | 1/33 |
| Creatinine increasedInvestigations | 0/50 | 1/33 |
| DiarrheaGastrointestinal disorders | 0/50 | 1/33 |
| EncephalopathyNervous system disorders | 0/50 | 1/33 |
| Oral mucositisGastrointestinal disorders | 0/50 | 1/33 |
| T bilirubin increasedInvestigations | 0/50 | 1/33 |
| Bleeding (no GI no PUL)Blood and lymphatic system disorders | 1/50 | 0/33 |
| Event | Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel |
|---|---|---|
| Oral mucositisGastrointestinal disorders | 47/50 | 26/33 |
| NauseaGastrointestinal disorders | 46/50 | 30/33 |
| DiarrheaGeneral disorders | 43/50 | 28/33 |
| Febrile neutropeniaBlood and lymphatic system disorders | 43/50 | 28/33 |
| Fluid overloadGeneral disorders | 36/50 | 25/33 |
| RashSkin and subcutaneous tissue disorders | 27/50 | 23/33 |
| ViralInfections and infestations | 18/50 | 23/33 |
| ALT increasedInvestigations | 25/50 | 20/33 |
| BacterialInfections and infestations | 18/50 | 14/33 |
| T bilirubin increasedInvestigations | 15/50 | 12/33 |
| Age, Categorical(Participants) | Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 48 | 33 | 81 |
| >=65 years | 2 | 0 | 2 |
| Sex: Female, Male(Participants) | Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Total |
|---|---|---|---|
| Female | 15 | 15 | 30 |
| Male | 35 | 18 | 53 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Total |
|---|---|---|---|
| Hispanic or Latino | 9 | 2 | 11 |
| Not Hispanic or Latino | 41 | 31 | 72 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(Participants) | Cohort 1_1st Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Cohort 2_2nd Auto TP Conditioned With Panobinostat and Gem/Bu/Mel | Total |
|---|---|---|---|
| United States | 50 | 33 | 83 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Sep 2015 to primary completion in Jun 2024, 104 months.
Results were first posted in Mar 2025, 9 months after primary completion.
How these figures are worked out, and which studies are left out of them.
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Studies of the drugs tested here: Busulfan · Gemcitabine · Melphalan · Panobinostat