A Phase 1/2 interventional study of Autologous Hematopoietic Stem Cell Transplantation and Filgrastim-sndz in Plasma Cell Myeloma, sponsored by M.D. Anderson Cancer Center. Completed at 1 site in United States. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2025-05-01.
Sponsored by M.D. Anderson Cancer Center · Phase 1/2, Interventional, and Treatment
This phase I/II trial studies the side effects and best dose of melphalan hydrochloride in treating participants with newly-diagnosed multiple myeloma who are undergoing a donor stem cell transplantation. Giving chemotherapy before a donor stem cell transplantation helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the participant, they may help the participant's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Giving melphalan hydrochloride before a donor stem cell transplantation may work better than standard chemotherapy in helping to prevent multiple myeloma from coming back.
PRIMARY OBJECTIVES:
I. To determine the optimal dose and schedule of melphalan for injection (melphalan hydrochloride [Evomela]) prior to autologous hematopoietic stem cell transplantation (auto-HCT) for multiple myeloma (MM).
II. To collect the pharmacokinetic data and compare the exposure-response evaluations between the 2 infusion schedules.
SECONDARY OBJECTIVES:
I. To determine the incidence of treatment related mortality (TRM) at day 90 after auto-HCT in newly diagnosed myeloma patients treated on different schedules and doses of Evomela.
II. To determine the rate of minimal residual disease (MRD) negative complete response (CR) rate at day 90 after auto-HCT in newly diagnosed myeloma patients treated on different schedules and doses of Evomela.
III. To determine the progression-free survival (PFS) after auto-HCT in newly diagnosed myeloma patients treated on different schedules and doses of Evomela.
OUTLINE: This is a phase I, dose escalation study of melphalan hydrochloride followed by a phase II study.
PREPARATIVE REGIMEN: Participants are randomized to 1 of 2 groups.
GROUP 1: Participants receive melphalan hydrochloride intravenously (IV) over 30-60 minutes on day -2.
GROUP 2: Participants receive melphalan hydrochloride IV over 8-9 hours on day -2.
TRANSPLANT: Participants in both groups undergo donor stem cell transplantation IV on day 0 over 30-60 minutes.
POST-TRANSPLANT: Participants in both groups receive filgrastim-sndz subcutaneously (SC) once daily (QD) starting on day 5 and continuing in the absence of disease progression, unacceptable toxicity, or until evidence of an absolute neutrophil count (ANC) of 0.5 x 10\^9/L.
After completion of study treatment, participants are followed up at 3 months, every 3 months for 1 year, and then annually for 1 year.
3,760 studies on the registry are indexed under Multiple Myeloma; 769 are open to participants now.
This study's enrollment of 62 is above the median of 41 across 3,005 interventional studies indexed under Multiple Myeloma.
Browse Multiple Myeloma 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.
Exclusion Criteria:
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.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
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.
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.
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.
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.
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.
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).
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
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.
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.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
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.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
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.
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.
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.
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.
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.
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.
Reproductive potential
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask 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.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
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.
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.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
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.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
PREPARATIVE REGIMEN: Participants receive melphalan hydrochloride IV over 30-60 minutes on day -2. TRANSPLANT: Participants in both groups undergo donor stem cell transplantation IV on day 0 over 30-60 minutes. POST-TRANSPLANT: Participants in both groups receive filgrastim-sndz SC QD starting on day 5 and continuing in the absence of disease progression, unacceptable toxicity, or until evidence of an ANC of 0.5 x 10\^9/L.
Procedure: Autologous Hematopoietic Stem Cell Transplantation · Biological: Filgrastim-sndz · Drug: Melphalan Hydrochloride
PREPARATIVE REGIMEN: Participants receive melphalan hydrochloride IV over 8-9 hours on day -2. TRANSPLANT: Participants in both groups undergo donor stem cell transplantation IV on day 0 over 30-60 minutes. POST-TRANSPLANT: Participants in both groups receive filgrastim-sndz SC QD starting on day 5 and continuing in the absence of disease progression, unacceptable toxicity, or until evidence of an ANC of 0.5 x 10\^9/L.
Procedure: Autologous Hematopoietic Stem Cell Transplantation · Biological: Filgrastim-sndz · Drug: Melphalan Hydrochloride
Undergo HSCT
Also known as: AHSCT, Autologous Hematopoietic Cell Transplantation, Autologous Stem Cell Transplantation, Stem Cell Transplantation, Autologous
Given SC
Also known as: Filgrastim Biosimilar Filgrastim-sndz, Zarxio
Given IV
Also known as: Alkeran, Alkerana, Evomela
Optimal Dose and Schedule of Evomela Prior to Autologous Transplant.
Dose limiting toxicity will be defined as grade 4 mucositis or any grade 4 or 5 non-hematologic or non-infectious toxicity occurring within 30 days from the start of infusion.
Time frame: Within 30 days after the start of infusion
Progression-free Survival (PFS)
Number of participants that are still alive and disease free one year post autologous transplant.
Time frame: From the date of Evomela injection up to 1 year
Treatment Related Mortality (TRM).
Number of participants expired from non-disease relapse within 90 days post transplant
Time frame: At day 90 post-transplant
Number of Participants That Have Achieved Complete Response (CR).
Number of participants that have achieved Complete response (CR). Complete response definition is negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates.
Time frame: At 90 days post-transplant
All participants were registered in MD Anderson Cancer Center.
| Milestone | Group1_Dose 1 200mgm2 | Group 1_Dose 2 225mgm2 | Group 2_Dose 1 200mgm2 | Group 2_Dose 225mgm2 |
|---|---|---|---|---|
| Started | 4 | 27 | 4 | 27 |
| Completed | 3 | 27 | 3 | 27 |
| Not completed | 1 | 0 | 1 | 0 |
| Withdrew: Inadequate supply on study drug | 1 | 0 | 0 | 0 |
| Withdrew: Disease progression | 0 | 0 | 1 | 0 |
Dose limiting toxicity will be defined as grade 4 mucositis or any grade 4 or 5 non-hematologic or non-infectious toxicity occurring within 30 days from the start of infusion.
| Participants | Group1_Dose 1 200mgm2 | Group 1_Dose 2 225mgm2 | Group 2_Dose 1 200mgm2 | Group 2_Dose 225mgm2 |
|---|---|---|---|---|
| Grade 4 Mucositis | 0 | 0 | 0 | 0 |
| Grade 4 non-hematologic or non-infectious toxicity | 0 | 0 | 0 | 0 |
Number of participants that are still alive and disease free one year post autologous transplant.
| Participants | Group1_Dose 1 200mgm2 | Group 1_Dose 2 225mgm2 | Group 2_Dose 1 200mgm2 | Group 2_Dose 225mgm2 |
|---|---|---|---|---|
| Progression-free Survival (PFS) | 2 | 21 | 2 | 21 |
Number of participants expired from non-disease relapse within 90 days post transplant
| Participants | Group1_Dose 1 200mgm2 | Group 1_Dose 2 225mgm2 | Group 2_Dose 1 200mgm2 | Group 2_Dose 225mgm2 |
|---|---|---|---|---|
| Treatment Related Mortality (TRM). | 0 | 0 | 0 | 0 |
Number of participants that have achieved Complete response (CR). Complete response definition is negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow aspirates.
| Participants | Group1_Dose 1 200mgm2 | Group 1_Dose 2 225mgm2 | Group 2_Dose 1 200mgm2 | Group 2_Dose 225mgm2 |
|---|---|---|---|---|
| Number of Participants That Have Achieved Complete Response (CR). | 1 | 13 | 2 | 13 |
Collected over Up to one year. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Group1_Dose 1 200mgm2 | 1/4 (25%) | 0/4 (0%) | 3/4 (75%) |
| Group 1_Dose 2 225mgm2 | 0/27 (0%) | 0/27 (0%) | 27/27 (100%) |
| Group 2_Dose 1 200mgm2 | 0/4 (0%) | 0/4 (0%) | 4/4 (100%) |
| Group 2_Dose 225mgm2 | 0/27 (0%) | 0/27 (0%) | 27/27 (100%) |
| Event | Group1_Dose 1 200mgm2 | Group 1_Dose 2 225mgm2 | Group 2_Dose 1 200mgm2 | Group 2_Dose 225mgm2 |
|---|---|---|---|---|
| NauseaGastrointestinal disorders | 3/4 | 27/27 | 4/4 | 27/27 |
| DiarrheaGastrointestinal disorders | 3/4 | 25/27 | 3/4 | 25/27 |
| Oral mucositisGastrointestinal disorders | 2/4 | 17/27 | 3/4 | 13/27 |
| RashSkin and subcutaneous tissue disorders | 3/4 | 0/27 | 1/4 | 0/27 |
| Febrile neutropeniaBlood and lymphatic system disorders | 1/4 | 9/27 | 1/4 | 13/27 |
| Abdominal painGastrointestinal disorders | 0/4 | 8/27 | 1/4 | 8/27 |
| BacterialInfections and infestations | 1/4 | 1/27 | 1/4 | 3/27 |
| DyspepsiaGastrointestinal disorders | 0/4 | 2/27 | 1/4 | 3/27 |
| HypotensionVascular disorders | 0/4 | 1/27 | 1/4 | 4/27 |
| T bilirubin increasedInvestigations | 0/4 | 1/27 | 1/4 | 1/27 |
| Age, Categorical(Participants) | Group1_Dose 1 200mgm2 | Group 1_Dose 2 225mgm2 | Group 2_Dose 1 200mgm2 | Group 2_Dose 225mgm2 | Total |
|---|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 2 | 19 | 4 | 22 | 47 |
| >=65 years | 2 | 8 | 0 | 5 | 15 |
| Sex: Female, Male(Participants) | Group1_Dose 1 200mgm2 | Group 1_Dose 2 225mgm2 | Group 2_Dose 1 200mgm2 | Group 2_Dose 225mgm2 | Total |
|---|---|---|---|---|---|
| Female | 1 | 12 | 2 | 5 | 20 |
| Male | 3 | 15 | 2 | 22 | 42 |
| Ethnicity (NIH/OMB)(Participants) | Group1_Dose 1 200mgm2 | Group 1_Dose 2 225mgm2 | Group 2_Dose 1 200mgm2 | Group 2_Dose 225mgm2 | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 3 | 1 | 4 | 9 |
| Not Hispanic or Latino | 3 | 24 | 3 | 23 | 53 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 |
| Region of Enrollment(participants) | Group1_Dose 1 200mgm2 | Group 1_Dose 2 225mgm2 | Group 2_Dose 1 200mgm2 | Group 2_Dose 225mgm2 | Total |
|---|---|---|---|---|---|
| United States | 4 | 27 | 4 | 27 | 62 |
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Studies of the drug tested here: Melphalan hydrochloride