Multiple MyelomaPhase 2NCT01923935
Busulfan and Melphalan Conditioning in Multiple Myeloma
A Phase 2 study testing BUSULFEX® and Alkeran® in people with Multiple Myeloma, run by Chonnam National University Hospital at 8 sites in South Korea.
Trial summary
The purpose of this study is to determine whether intravenous busulfan and melphalan as a conditioning regimen is effective in the treatment of multiple myeloma undergoing autologous stem cell transplantation. Read more about this study
- Who is it for?
- Ages 20 to 65 years
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- BUSULFEX® and Alkeran®
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 105 participants
- Where
- 8 sitesSouth Korea
- Study dates
- Jan 2013to Dec 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with a confirmed diagnosis of MM
- Symptomatic MM
- Age 20 - 65 years
- The MM patients who performed the stem cell collection with appropriate stem cell counts, cluster of differentiation 34 positive cells more than 2 x 10^6 /kg
- Eastern Cooperative Oncology Group 0 - 2
- The MM patients who received induction chemotherapy regardless of types of induction
Show all 11Show fewer
- Patient has measurable disease when the patients started the primary induction therapy, defined as follows: Measurable disease is defined as serum M-protein more than 1 g/dL, urine M-protein more than 200 mg/24 hours, or free light chain more than 100 mg/L
- Adequate liver functions before ASCT Transaminase less than 3 x upper normal value Bilirubin less than 2 x upper normal value
- Adequate hematological function - Platelet count more than 50,000 /microliter, hemoglobin more than 8 g / dL but, Prior red blood cell transfusion or recombinant human erythropoietin use is allowed, absolute neutrophil count more than 1,000 / microliter
- Expected survival: 6 months or more
- Informed consent
Who cannot join
- Systemic amyloid light chain amyloidosis, smoldering multiple myeloma or monoclonal gammopathy of undetermined significance
- Patient with plasma cell leukemia
- Patients who received an extensive radiation therapy within 4 weeks
- Patient is known to be Human Immunodeficiency Virus positive
- Patient has known clinically active Hepatitis B or C
- Pregnant or lactating women, women of childbearing potential not employing adequate contraception
- Other serious illness or medical conditions Uncontrolled or severe cardiovascular disease, including myocardial infarction, within 6 months of enrollment, New York Heart Association Class III or IV heart failure, uncontrolled angina, clinically significant pericardial disease, or cardiac amyloidosis History of significant neurological or psychiatric disorders including dementia or seizures Active uncontrolled infection Active ulcers detected at gastroscopy Other serious medical illnesses
- Known hypersensitivity to any of the study drugs or its ingredients concomitant administration of any other experimental drug under investigation, or concomitant chemotherapy, hormonal therapy, or immunotherapy
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
South Korea8 sites, 8 recruiting
- Not yet recruiting
Samsung Medical Center
Gangnam-gu, Seoul, 135-710
Kihyun Kim, M.D., Ph.D., 82-2-3410-3459, kihyunk@skku.edu
Kihyun Kim, M.D., Ph.D.
- Recruiting
National Cancer Center
Goyang-si, Gyeonggi-do, 410-769
Hyeon Seok Eom, M.D., Ph.D., 82-31-920-2402, hseom@ncc.re.kr
Hyeon Seok Eom, M.D., Ph.D.
- Recruiting
Chonnam National University Hwasun Hospital
Hwasun-gun, Jeollanam-do, 519-763
Je-Jung Lee, M.D, Ph.D., 82-61-379-7638, f0115@chonnam.ac.kr
Jae-Sook Ahn, M.D, Ph.D., 82-61-379-7635, ahnjaesook@hanmail.net
- Not yet recruiting
Seoul National University Hospital
Jongno-gu, Seoul, 110-744
Sung Soo Yoon, M.D., Ph.D., 82-2-2072-3079, ssysmc@snu.ac.kr
Sung Soo Yoon, M.D., Ph.D.
- Not yet recruiting
Ewha Womans University Mokdong Hospital
Mokdong, Seoul, 158-710
Yeung-Chul Mun, M.D., Ph.D., 82-2-2650-2777, yeungchul@ewha.ac.kr
Yeung-Chul Mun, M.D., Ph.D.
- Not yet recruiting
Gachon University Gil Hospital
Namdong-gu, Incheon, 405-760
Jae Hoon Lee, M.D., Ph.D., 82-32-460-2186, jhlee@gilhospital.com
Jae Hoon Lee, M.D., Ph.D.
- Not yet recruiting
Seoul St. Mary's hospital
Seocho-gu, Seoul, 137-701
Chang Ki Min, M.D., Ph.D., 82-2-2258-6053, ckmin@catholic.ac.kr
Chang Ki Min, M.D., Ph.D.
- Not yet recruiting
Severance Hospital
Seodaemun-gu, Seoul, 120-752
Soo Jung Kim, M.D., 82-2-2228-5487, ALVIN97@yuhs.ac
Soo Jung Kim, M.D., Ph.D.
About this study
Official title: A Phase 2, Open-label, Prospective, Multicenter Study to Evaluate the Efficacy of Intravenous Busulfan and Melphalan as a Conditioning Regimen in Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplantation
The purpose of this study is to determine whether intravenous busulfan and melphalan as a conditioning regimen is effective in the treatment of multiple myeloma undergoing autologous stem cell transplantation.
Read the detailed description
Title
- A phase 2, open-label, prospective, multicenter study to evaluate the efficacy of intravenous busulfan and melphalan as a conditioning regimen in patients with multiple myeloma (MM) undergoing autologous stem cell transplantation (ASCT)
Principal Investigator
- Je-Jung Lee (Chonnam National University Hwasun Hospital)
Co-investigators
- Hyeon Seok Eom (National Cancer Center)
- Kihyun Kim (Samsung Medical Center)
- Chang Ki Min (Seoul St. Mary's Hospital)
- Soo Jung Kim (Severance Hospital)
- Sung Soo Yoon (Seoul National University Hospital)
- Jae Hoon Lee (Gachon University Gil Hospital)
- Yeung-Chul Mun (Ewha Womans University Mokdong Hospital)
Duration
- 2 years
Study phase
- Phase II
Patients
- Patients with multiple myeloma who undergo autologous stem cell transplantation
Objectives(end points)
- Primary objective:
- Treatment response up to 2 months after ASCT
- Safety and toxicity (frequency of grade 3 or worse toxicities) of the conditioning regimen
- Secondary objective:
- Progression free survival (PFS)
- Overall survival (OS)
Total patients
- Initial 105 evaluable patients
- Complete Response (CR) rate of 200mg/m2 melphalan conditioning chemotherapy followed by ASCT in patients with newly diagnosed MM was about 26% and CR rate of busulfan and melphalan conditioning chemotherapy followed by ASCT in patients with newly diagnosed MM was about 40%. If the CR rate of busulfan and melphalan conditioning chemotherapy followed by ASCT is more than 40%, this combination will be accepted as active conditioning regimen that may be worth for investigating in phase III trial. But, if the CR rate of this regimen is lower than 26%, this has not a merit than 200mg/m2 melphalan conditioning chemotherapy. Based upon the above assumption, this trial was designed by using Simon's optimal two-stage testing procedure. Assuming a target level of interest, p1=0.4, and a lower activity level, p0=0.26 and drop rate 0.1. Initially 44 patients will be accrued. If 13 or more CR rate were observed, the trial will be continued. Accrual will be planned to a total of 105 patients, If total 35 or more patients were assessed as CR, busulfan and melphalan conditioning regimen will be accepted as active regimen, This design provides probability ≤ 0.05 of accepting drugs worse than p0 and probability ≤ 0.20 of rejecting drugs better than p1.if we assume that drop-out rate is 10%, total accrual patient will be 105
Treatment Schedule
- Busulfan 3.2 mg/kg/day iv once daily over 3 hours(day-6 ~ day-4)
- Melphalan 70 mg/m2/day iv once daily over 30 minutes(day-3 ~ day-1). If Creatinine Clearance (mL/min) < 50, reduce to 50 mg/m2. If Creatinine Clearance (mL/min) < 30, excluded from the this trial
Informed consent
- Written informed consent must be obtained before any study-specific screening procedures are performed
Screening
- Baseline assessments should be made within 28 days before treatment start:
- Demographic data (date of birth and sex)
- Eastern Cooperative Oncology Group performance status
- Vital signs and physical examination (including height, and weight)
- Medical history (including previous diseases/treatments and concomitant diseases/ treatments)
- Hematology - complete blood counts with differential count examination
- Serum electrolytes (Na, K, Cl, Ca, phosphorus)
- Serum lactate dehydrogenase
- Hepatitis B virus/hepatitis C virus/HIV serology (If serologic tests were conducted within 6 months prior to screening, retests are not required)
- Serum Beta2-microglobulin
- Quantitative serum M-protein (Serum protein electrophoresis), including immunofixation or immune electrophoresis
- Quantitative urine M-protein in 24 hrs urine (Urine protein electrophoresis), including immunofixation or immune electrophoresis
- Serum free light chain assay
- Creatinine clearance if increased serum creatinine
- ECG
- multigated radionuclide angiography or 2D ECHO
- Chest X-ray, Radiographic skeletal survey including skull, pelvis, vertebral column and long bones
- Bone marrow aspiration and biopsy with chromosome study, and flow cytometry or immunohistochemistry
Assessment
- Primary outcome measure
- To evaluate the objective responses after ASCT, the guidelines from the International Myeloma Working (IMW) Group uniform response criteria will be used
- Response Criteria for Multiple Myeloma the guidelines from the IMW Group uniform response criteria + Add for criteria of near CR (Immunofixation positive CR)
- Serum free light chain study will be added at the every evaluation of response
- To confirm the stringent complete response (sCR) after ASCT, flow cytometry or immunohistochemistry will be used
- NCI Common Toxicity Criteria for Adverse Effects version 4.0 will be used for the examination of toxicities
- Secondary outcome measure
- PFS will be defined from the time of ASCT to the time of first sign of disease progression or death
- OS will be defined as the period from the time of ASCT to the date of the last follow-up or death from any cause
- Conditions
- Multiple Myeloma
- Being tested
- BUSULFEX®, Alkeran®
- Keywords
- Multiple myeloma, busulfan, melphalan,transplantation
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BUSULFEX®Drug
BUSULFEX® 3.2 mg/kg/day iv once daily over 3 hours (day-6~day-4)
Alkeran®Drug
Alkeran® 70 mg/m2/day iv once daily over 30 minutes (day-3~day-2)
| Study group | Role | What they receive |
|---|---|---|
| BUSULFEX®, Alkeran® | Experimental | BUSULFEX® 3.2 mg/kg/day iv once daily over 3 hours (day-6~day-4)
Alkeran® 70 mg/m2/day iv once daily over 30 minutes (day-3~day-2) Receives: Drug: BUSULFEX®; Drug: Alkeran® |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment response up to 2 months after ASCT
- To evaluate the objective responses after ASCT, the guidelines from the International Myeloma Working (IMW) Group uniform response criteria will be used.
- Serum free light chain study will be added at the every evaluation of response.
- To confirm the stringent complete response (sCR) after ASCT, flow cytometry or immunohistochemistry will be used
Response Criteria for Multiple Myeloma:
the guidelines from the IMW Group uniform response criteria + Add for criteria of near CR (Immunofixation positive CR)
Measured: 2 months later after last patent received ASCT
Safety and toxicity (frequency of grade 3 or worse toxicities) of the conditioning regimen
NCI Common Toxicity Criteria for Adverse Effects version 4.0 will be used for the examination of toxicities.
Measured: 2 months later after last patent received ASCT
Secondary outcomes (2)
Progression free survival(PFS)
PFS will be defined from the time of ASCT to the time of first sign of disease progression or death.
Measured: 2 months later after last patient received ASCT
Overall survival (OS)
OS will be defined as the period from the time of ASCT to the date of the last follow-up or death from any cause.
Measured: 2 months later after last patient received ASCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2013 (estimated)
- Study startJan 2013
- Primary completionJan 2014 (estimated)
- Study completionDec 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (32)Papers the sponsor has linked to this study.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 105 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chonnam National University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Korea Otsuka Pharmaceutical Co., Ltd.
- Responsible party
- Principal investigator, Je-Jung Lee, Chonnam National University Hospital
- Investigators
- Je-Jung Lee, M.D., Ph.D., Principal investigator, Chonnam National University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01923935
- Sponsor's protocol ID
- KFDA20120110139
- Status last verified
- Aug 2013
- First submitted
- 9 Aug 2013
- Last updated
- 19 Aug 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
McElwain TJ, Powles RL. High-dose intravenous melphalan for plasma-cell leukaemia and myeloma. Lancet. 1983 Oct 8;2(8354):822-4. doi: 10.1016/s0140-6736(83)90739-0.
PubMed 6137651Selby PJ, McElwain TJ, Nandi AC, Perren TJ, Powles RL, Tillyer CR, Osborne RJ, Slevin ML, Malpas JS. Multiple myeloma treated with high dose intravenous melphalan. Br J Haematol. 1987 May;66(1):55-62. doi: 10.1111/j.1365-2141.1987.tb06890.x.
PubMed 3593657Barlogie B, Tricot G, Anaissie E, Shaughnessy J, Rasmussen E, van Rhee F, Fassas A, Zangari M, Hollmig K, Pineda-Roman M, Lee C, Talamo G, Thertulien R, Kiwan E, Krishna S, Fox M, Crowley J. Thalidomide and hematopoietic-cell transplantation for multiple myeloma. N Engl J Med. 2006 Mar 9;354(10):1021-30. doi: 10.1056/NEJMoa053583.
PubMed 16525139Attal M, Harousseau JL, Stoppa AM, Sotto JJ, Fuzibet JG, Rossi JF, Casassus P, Maisonneuve H, Facon T, Ifrah N, Payen C, Bataille R. A prospective, randomized trial of autologous bone marrow transplantation and chemotherapy in multiple myeloma. Intergroupe Francais du Myelome. N Engl J Med. 1996 Jul 11;335(2):91-7. doi: 10.1056/NEJM199607113350204.
PubMed 8649495Fermand JP, Ravaud P, Chevret S, Divine M, Leblond V, Belanger C, Macro M, Pertuiset E, Dreyfus F, Mariette X, Boccacio C, Brouet JC. High-dose therapy and autologous peripheral blood stem cell transplantation in multiple myeloma: up-front or rescue treatment? Results of a multicenter sequential randomized clinical trial. Blood. 1998 Nov 1;92(9):3131-6.
PubMed 9787148Child JA, Morgan GJ, Davies FE, Owen RG, Bell SE, Hawkins K, Brown J, Drayson MT, Selby PJ; Medical Research Council Adult Leukaemia Working Party. High-dose chemotherapy with hematopoietic stem-cell rescue for multiple myeloma. N Engl J Med. 2003 May 8;348(19):1875-83. doi: 10.1056/NEJMoa022340.
PubMed 12736280Blade J, Rosinol L, Sureda A, Ribera JM, Diaz-Mediavilla J, Garcia-Larana J, Mateos MV, Palomera L, Fernandez-Calvo J, Marti JM, Giraldo P, Carbonell F, Callis M, Trujillo J, Gardella S, Moro MJ, Barez A, Soler A, Font L, Fontanillas M, San Miguel J; Programa para el Estudio de la Terapeutica en Hemopatia Maligna (PETHEMA). High-dose therapy intensification compared with continued standard chemotherapy in multiple myeloma patients responding to the initial chemotherapy: long-term results from a prospective randomized trial from the Spanish cooperative group PETHEMA. Blood. 2005 Dec 1;106(12):3755-9. doi: 10.1182/blood-2005-03-1301. Epub 2005 Aug 16.
PubMed 16105975Fermand JP, Katsahian S, Divine M, Leblond V, Dreyfus F, Macro M, Arnulf B, Royer B, Mariette X, Pertuiset E, Belanger C, Janvier M, Chevret S, Brouet JC, Ravaud P; Group Myelome-Autogreffe. High-dose therapy and autologous blood stem-cell transplantation compared with conventional treatment in myeloma patients aged 55 to 65 years: long-term results of a randomized control trial from the Group Myelome-Autogreffe. J Clin Oncol. 2005 Dec 20;23(36):9227-33. doi: 10.1200/JCO.2005.03.0551. Epub 2005 Nov 7.
PubMed 16275936Barlogie B, Kyle RA, Anderson KC, Greipp PR, Lazarus HM, Hurd DD, McCoy J, Moore DF Jr, Dakhil SR, Lanier KS, Chapman RA, Cromer JN, Salmon SE, Durie B, Crowley JC. Standard chemotherapy compared with high-dose chemoradiotherapy for multiple myeloma: final results of phase III US Intergroup Trial S9321. J Clin Oncol. 2006 Feb 20;24(6):929-36. doi: 10.1200/JCO.2005.04.5807. Epub 2006 Jan 23.
PubMed 16432076Alexanian R, Barlogie B, Tucker S. VAD-based regimens as primary treatment for multiple myeloma. Am J Hematol. 1990 Feb;33(2):86-9. doi: 10.1002/ajh.2830330203.
PubMed 2301376Barlogie B, Jagannath S, Desikan KR, Mattox S, Vesole D, Siegel D, Tricot G, Munshi N, Fassas A, Singhal S, Mehta J, Anaissie E, Dhodapkar D, Naucke S, Cromer J, Sawyer J, Epstein J, Spoon D, Ayers D, Cheson B, Crowley J. Total therapy with tandem transplants for newly diagnosed multiple myeloma. Blood. 1999 Jan 1;93(1):55-65.
PubMed 9864146Samson D, Gaminara E, Newland A, Van de Pette J, Kearney J, McCarthy D, Joyner M, Aston L, Mitchell T, Hamon M, et al. Infusion of vincristine and doxorubicin with oral dexamethasone as first-line therapy for multiple myeloma. Lancet. 1989 Oct 14;2(8668):882-5. doi: 10.1016/s0140-6736(89)91549-3.
PubMed 2571813Sirohi B, Powles R, Mehta J, Raje N, Kulkarni S, Ramiah V, Saso R, Horton C, Bhagwati N, Singhal S, Treleaven J. Complete remission rate and outcome after intensive treatment of 177 patients under 75 years of age with IgG myeloma defining a circumscribed disease entity with a new staging system. Br J Haematol. 1999 Dec;107(3):656-66. doi: 10.1046/j.1365-2141.1999.01744.x.
PubMed 10583272Goldschmidt H, Hegenbart U, Wallmeier M, Hohaus S, Haas R. Factors influencing collection of peripheral blood progenitor cells following high-dose cyclophosphamide and granulocyte colony-stimulating factor in patients with multiple myeloma. Br J Haematol. 1997 Sep;98(3):736-44. doi: 10.1046/j.1365-2141.1997.2783095.x.
PubMed 9332333Barlogie B, Alexanian R, Dicke KA, Zagars G, Spitzer G, Jagannath S, Horwitz L. High-dose chemoradiotherapy and autologous bone marrow transplantation for resistant multiple myeloma. Blood. 1987 Sep;70(3):869-72.
PubMed 3304465
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-label, Prospective, Multicenter Study to Evaluate the Efficacy of Intravenous Busulfan and Melphalan as a Conditioning Regimen in Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT01923935
- Acronym / sponsor ID
- KFDA20120110139
- Conditions
- Multiple Myeloma
- Keywords
- Multiple myeloma, busulfan, melphalan,transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 105 participants (estimated)
- Lead sponsor
- Chonnam National University HospitalUniversity, hospital or other
- Collaborators
- Korea Otsuka Pharmaceutical Co., Ltd.
- Study start
- Jan 2013
- Primary completion
- Jan 2014 (estimated)
- Study completion
- Dec 2015 (estimated)
- First posted
- 16 Aug 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether intravenous busulfan and melphalan as a conditioning regimen is effective in the treatment of multiple myeloma undergoing autologous stem cell transplantation.
Detailed description
Title
- A phase 2, open-label, prospective, multicenter study to evaluate the efficacy of intravenous busulfan and melphalan as a conditioning regimen in patients with multiple myeloma (MM) undergoing autologous stem cell transplantation (ASCT)
Principal Investigator
- Je-Jung Lee (Chonnam National University Hwasun Hospital)
Co-investigators
- Hyeon Seok Eom (National Cancer Center)
- Kihyun Kim (Samsung Medical Center)
- Chang Ki Min (Seoul St. Mary's Hospital)
- Soo Jung Kim (Severance Hospital)
- Sung Soo Yoon (Seoul National University Hospital)
- Jae Hoon Lee (Gachon University Gil Hospital)
- Yeung-Chul Mun (Ewha Womans University Mokdong Hospital)
Duration
- 2 years
Study phase
- Phase II
Patients
- Patients with multiple myeloma who undergo autologous stem cell transplantation
Objectives(end points)
- Primary objective:
- Treatment response up to 2 months after ASCT
- Safety and toxicity (frequency of grade 3 or worse toxicities) of the conditioning regimen
- Secondary objective:
- Progression free survival (PFS)
- Overall survival (OS)
Total patients
- Initial 105 evaluable patients
- Complete Response (CR) rate of 200mg/m2 melphalan conditioning chemotherapy followed by ASCT in patients with newly diagnosed MM was about 26% and CR rate of busulfan and melphalan conditioning chemotherapy followed by ASCT in patients with newly diagnosed MM was about 40%. If the CR rate of busulfan and melphalan conditioning chemotherapy followed by ASCT is more than 40%, this combination will be accepted as active conditioning regimen that may be worth for investigating in phase III trial. But, if the CR rate of this regimen is lower than 26%, this has not a merit than 200mg/m2 melphalan conditioning chemotherapy. Based upon the above assumption, this trial was designed by using Simon's optimal two-stage testing procedure. Assuming a target level of interest, p1=0.4, and a lower activity level, p0=0.26 and drop rate 0.1. Initially 44 patients will be accrued. If 13 or more CR rate were observed, the trial will be continued. Accrual will be planned to a total of 105 patients, If total 35 or more patients were assessed as CR, busulfan and melphalan conditioning regimen will be accepted as active regimen, This design provides probability ≤ 0.05 of accepting drugs worse than p0 and probability ≤ 0.20 of rejecting drugs better than p1.if we assume that drop-out rate is 10%, total accrual patient will be 105
Treatment Schedule
- Busulfan 3.2 mg/kg/day iv once daily over 3 hours(day-6 ~ day-4)
- Melphalan 70 mg/m2/day iv once daily over 30 minutes(day-3 ~ day-1). If Creatinine Clearance (mL/min) < 50, reduce to 50 mg/m2. If Creatinine Clearance (mL/min) < 30, excluded from the this trial
Informed consent
- Written informed consent must be obtained before any study-specific screening procedures are performed
Screening
- Baseline assessments should be made within 28 days before treatment start:
- Demographic data (date of birth and sex)
- Eastern Cooperative Oncology Group performance status
- Vital signs and physical examination (including height, and weight)
- Medical history (including previous diseases/treatments and concomitant diseases/ treatments)
- Hematology - complete blood counts with differential count examination
- Serum electrolytes (Na, K, Cl, Ca, phosphorus)
- Serum lactate dehydrogenase
- Hepatitis B virus/hepatitis C virus/HIV serology (If serologic tests were conducted within 6 months prior to screening, retests are not required)
- Serum Beta2-microglobulin
- Quantitative serum M-protein (Serum protein electrophoresis), including immunofixation or immune electrophoresis
- Quantitative urine M-protein in 24 hrs urine (Urine protein electrophoresis), including immunofixation or immune electrophoresis
- Serum free light chain assay
- Creatinine clearance if increased serum creatinine
- ECG
- multigated radionuclide angiography or 2D ECHO
- Chest X-ray, Radiographic skeletal survey including skull, pelvis, vertebral column and long bones
- Bone marrow aspiration and biopsy with chromosome study, and flow cytometry or immunohistochemistry
Assessment
- Primary outcome measure
- To evaluate the objective responses after ASCT, the guidelines from the International Myeloma Working (IMW) Group uniform response criteria will be used
- Response Criteria for Multiple Myeloma the guidelines from the IMW Group uniform response criteria + Add for criteria of near CR (Immunofixation positive CR)
- Serum free light chain study will be added at the every evaluation of response
- To confirm the stringent complete response (sCR) after ASCT, flow cytometry or immunohistochemistry will be used
- NCI Common Toxicity Criteria for Adverse Effects version 4.0 will be used for the examination of toxicities
- Secondary outcome measure
- PFS will be defined from the time of ASCT to the time of first sign of disease progression or death
- OS will be defined as the period from the time of ASCT to the date of the last follow-up or death from any cause
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BUSULFEX®, Alkeran® | Experimental | Drug: BUSULFEX®, Drug: Alkeran®BUSULFEX® 3.2 mg/kg/day iv once daily over 3 hours (day-6~day-4) Alkeran® 70 mg/m2/day iv once daily over 30 minutes (day-3~day-2) |
Who may qualify
- Ages
- 20 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Patients with a confirmed diagnosis of MM
- Symptomatic MM
- Age 20 - 65 years
- The MM patients who performed the stem cell collection with appropriate stem cell counts, cluster of differentiation 34 positive cells more than 2 x 10^6 /kg
- Eastern Cooperative Oncology Group 0 - 2
Key exclusion criteria (8 listed)
- Systemic amyloid light chain amyloidosis, smoldering multiple myeloma or monoclonal gammopathy of undetermined significance
- Patient with plasma cell leukemia
- Patients who received an extensive radiation therapy within 4 weeks
- Patient is known to be Human Immunodeficiency Virus positive
- Patient has known clinically active Hepatitis B or C
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BUSULFEX®BUSULFEX® 3.2 mg/kg/day iv once daily over 3 hours (day-6~day-4) | Drug | BUSULFEX®, Alkeran® |
| Alkeran®Alkeran® 70 mg/m2/day iv once daily over 30 minutes (day-3~day-2) | Drug | BUSULFEX®, Alkeran® |
Primary outcome
Treatment response up to 2 months after ASCT
Time frame: 2 months later after last patent received ASCT
1. To evaluate the objective responses after ASCT, the guidelines from the International Myeloma Working (IMW) Group uniform response criteria will be used. 2. Serum free light chain study will be added at the every evaluation of response. 3. To confirm the stringent complete response (sCR) after ASCT, flow cytometry or immunohistochemistry will be used Response Criteria for Multiple Myeloma: the guidelines from the IMW Group uniform response criteria + Add for criteria of near CR (Immunofixation positive CR)
Safety and toxicity (frequency of grade 3 or worse toxicities) of the conditioning regimen
Time frame: 2 months later after last patent received ASCT
NCI Common Toxicity Criteria for Adverse Effects version 4.0 will be used for the examination of toxicities.
2 secondary outcomes
Progression free survival(PFS)
Time frame: 2 months later after last patient received ASCT
Overall survival (OS)
Time frame: 2 months later after last patient received ASCT
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2013.
- Background: PubMed 6137651McElwain TJ, Powles RL. High-dose intravenous melphalan for plasma-cell leukaemia and myeloma. Lancet. 1983 Oct 8;2(8354):822-4. doi: 10.1016/s0140-6736(83)90739-0.
- Background: PubMed 3593657Selby PJ, McElwain TJ, Nandi AC, Perren TJ, Powles RL, Tillyer CR, Osborne RJ, Slevin ML, Malpas JS. Multiple myeloma treated with high dose intravenous melphalan. Br J Haematol. 1987 May;66(1):55-62. doi: 10.1111/j.1365-2141.1987.tb06890.x.
- Background: PubMed 16525139Barlogie B, Tricot G, Anaissie E, Shaughnessy J, Rasmussen E, van Rhee F, Fassas A, Zangari M, Hollmig K, Pineda-Roman M, Lee C, Talamo G, Thertulien R, Kiwan E, Krishna S, Fox M, Crowley J. Thalidomide and hematopoietic-cell transplantation for multiple myeloma. N Engl J Med. 2006 Mar 9;354(10):1021-30. doi: 10.1056/NEJMoa053583.
- Background: PubMed 8649495Attal M, Harousseau JL, Stoppa AM, Sotto JJ, Fuzibet JG, Rossi JF, Casassus P, Maisonneuve H, Facon T, Ifrah N, Payen C, Bataille R. A prospective, randomized trial of autologous bone marrow transplantation and chemotherapy in multiple myeloma. Intergroupe Francais du Myelome. N Engl J Med. 1996 Jul 11;335(2):91-7. doi: 10.1056/NEJM199607113350204.
- Background: PubMed 9787148Fermand JP, Ravaud P, Chevret S, Divine M, Leblond V, Belanger C, Macro M, Pertuiset E, Dreyfus F, Mariette X, Boccacio C, Brouet JC. High-dose therapy and autologous peripheral blood stem cell transplantation in multiple myeloma: up-front or rescue treatment? Results of a multicenter sequential randomized clinical trial. Blood. 1998 Nov 1;92(9):3131-6.
- Background: PubMed 12736280Child JA, Morgan GJ, Davies FE, Owen RG, Bell SE, Hawkins K, Brown J, Drayson MT, Selby PJ; Medical Research Council Adult Leukaemia Working Party. High-dose chemotherapy with hematopoietic stem-cell rescue for multiple myeloma. N Engl J Med. 2003 May 8;348(19):1875-83. doi: 10.1056/NEJMoa022340.
- Background: PubMed 16105975Blade J, Rosinol L, Sureda A, Ribera JM, Diaz-Mediavilla J, Garcia-Larana J, Mateos MV, Palomera L, Fernandez-Calvo J, Marti JM, Giraldo P, Carbonell F, Callis M, Trujillo J, Gardella S, Moro MJ, Barez A, Soler A, Font L, Fontanillas M, San Miguel J; Programa para el Estudio de la Terapeutica en Hemopatia Maligna (PETHEMA). High-dose therapy intensification compared with continued standard chemotherapy in multiple myeloma patients responding to the initial chemotherapy: long-term results from a prospective randomized trial from the Spanish cooperative group PETHEMA. Blood. 2005 Dec 1;106(12):3755-9. doi: 10.1182/blood-2005-03-1301. Epub 2005 Aug 16.
- Background: PubMed 16275936Fermand JP, Katsahian S, Divine M, Leblond V, Dreyfus F, Macro M, Arnulf B, Royer B, Mariette X, Pertuiset E, Belanger C, Janvier M, Chevret S, Brouet JC, Ravaud P; Group Myelome-Autogreffe. High-dose therapy and autologous blood stem-cell transplantation compared with conventional treatment in myeloma patients aged 55 to 65 years: long-term results of a randomized control trial from the Group Myelome-Autogreffe. J Clin Oncol. 2005 Dec 20;23(36):9227-33. doi: 10.1200/JCO.2005.03.0551. Epub 2005 Nov 7.
- Background: PubMed 16432076Barlogie B, Kyle RA, Anderson KC, Greipp PR, Lazarus HM, Hurd DD, McCoy J, Moore DF Jr, Dakhil SR, Lanier KS, Chapman RA, Cromer JN, Salmon SE, Durie B, Crowley JC. Standard chemotherapy compared with high-dose chemoradiotherapy for multiple myeloma: final results of phase III US Intergroup Trial S9321. J Clin Oncol. 2006 Feb 20;24(6):929-36. doi: 10.1200/JCO.2005.04.5807. Epub 2006 Jan 23.
- Background: PubMed 2301376Alexanian R, Barlogie B, Tucker S. VAD-based regimens as primary treatment for multiple myeloma. Am J Hematol. 1990 Feb;33(2):86-9. doi: 10.1002/ajh.2830330203.
- Background: PubMed 9864146Barlogie B, Jagannath S, Desikan KR, Mattox S, Vesole D, Siegel D, Tricot G, Munshi N, Fassas A, Singhal S, Mehta J, Anaissie E, Dhodapkar D, Naucke S, Cromer J, Sawyer J, Epstein J, Spoon D, Ayers D, Cheson B, Crowley J. Total therapy with tandem transplants for newly diagnosed multiple myeloma. Blood. 1999 Jan 1;93(1):55-65.
- Background: PubMed 2571813Samson D, Gaminara E, Newland A, Van de Pette J, Kearney J, McCarthy D, Joyner M, Aston L, Mitchell T, Hamon M, et al. Infusion of vincristine and doxorubicin with oral dexamethasone as first-line therapy for multiple myeloma. Lancet. 1989 Oct 14;2(8668):882-5. doi: 10.1016/s0140-6736(89)91549-3.
Related trials for Multiple Myeloma
RecruitingPhase 2
A Study to Evaluate the Efficacy of Cemsidomide + Dexamethasone in Participants With Relapsed/Refractory Multiple Myeloma
Multiple Myeloma, Relapsed/Refractory Multiple Myeloma
RecruitingPhase 1/2
AZD0305 as Monotherapy or in Combination With Anticancer Agents in Participants With Multiple Myeloma
Multiple Myeloma
RecruitingPhase 1
Study to Assess Safety and Tolerability of OPN-6602 in Subjects With Relapsed and/or Refractory Multiple Myeloma
Relapsed Multiple Myeloma, Refractory Multiple Myeloma
RecruitingPhase 1
A Study to Learn About the Effects of Cemsidomide in Combination With Elranatamab in Relapsed/Refractory Multiple Myeloma Subjects
Multiple Myeloma (MM)
Active, not recruiting
Real World Treatment Experience of Patients With Breast, Lung, or GI Cancer or Multiple Myeloma Using Remote Symptom Monitoring
Breast Cancer, Lung Cancer
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Je-Jung Lee, M.D., Ph.D. principal investigator
Chonnam National University Hospital
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BUSULFEX® and Alkeran® in people with Multiple Myeloma, run by Chonnam National University Hospital at 8 sites in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Aug 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Je-Jung Lee, M.D., PH.D. (82-61-379-7638, f0115@chonnam.ac.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment response up to 2 months after ASCT (time frame: 2 months later after last patent received ASCT). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01923935, which also lists 32 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2013.