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DS Stage I Plasma Cell MyelomaPhase 2NCT01605032

Busulfan, Melphalan, and Bortezomib Before First-Line Stem Cell Transplant in Treating Patients With Multiple Myeloma

A Phase 2 study testing Busulfan, Melphalan and Bortezomib in people with DS Stage I Plasma Cell Myeloma and DS Stage II Plasma Cell Myeloma, run by Albert Einstein College of Medicine at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This phase II trial studies how well busulfan, melphalan, and bortezomib before first-line stem cell transplant works in treating patients with multiple myeloma. Giving chemotherapy before a peripheral blood stem cell transplant may stop the growth of cancer cells by stopping them from dividing or killing them. Read more about this study

Who is it for?
Ages 18 to 72 years
What is being tested?
Busulfan, Melphalan and Bortezomib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
4 sitesUnited States
Study dates
Feb 2012to Mar 2018
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 18 to 72 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients must have histologically or cytologically confirmed multiple myeloma
  • Measurable disease must be present as defined by protein criteria (quantifiable M-component in serum, urine or serum free light chains) in order to evaluate response as per IMWG; non-secretory patients are eligible provided the patient has > 20% plasmacytosis OR multiple (> 3) focal plasmacytomas or focal lesions on magnetic resonance imaging (MRI)
  • Patients must have received induction chemotherapy for myeloma, but not more than 12 months of prior chemotherapy for this disease, and must be eligible for the first planned autologous transplant
  • A minimum stem cell dose of 2.0 x 10^6 cluster of differentiation 34-positive (CD34+) cells/kg has been collected
  • Life expectancy of greater than 12 months
  • Eastern Cooperative Oncology Group (ECOG) performance status =< 2 (Karnofsky >= 60%)
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  • Leukocytes >= 3,000/mcL (unless myeloma related)
  • Absolute neutrophil count >= 1,500/mcL (unless myeloma related)
  • Platelets >= 50,000/mcL (unless myeloma related)
  • Total bilirubin =< 2 x institutional upper limit of normal unless 2nd to Gilbert's disease
  • Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 3 x institutional upper limit of normal
  • Creatinine =< 1.5 x institutional upper limit of normal OR creatinine clearance >= 60 mL/min/1.73 m^2 for patients with creatinine levels above institutional normal
  • Ejection fraction by echocardiogram (ECHO) or multi gated acquisition scan (MUGA) >= 40% performed within 60 days prior to registration
  • Patients must have adequate pulmonary function studies: > 50% of predicted on mechanical aspects (forced expiratory volume in one second [FEV1], forced vital capacity [FVC]) and diffusion capacity (diffusing capacity of the lung for carbon monoxide [DLCO]) > 50% of predicted, within 60 days of registration; if the patients is unable to complete pulmonary function tests due to multiple myeloma (MM) related pain or condition, exception may be granted if the principal investigator (PI) documents that the patient is a candidate for high dose therapy
  • Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for at least six months following the stem cell transplantation; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately
  • Ability to understand and the willingness to sign a written informed consent document

Who cannot join

  • Patients who have had chemotherapy or radiotherapy within 4 weeks prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier
  • Prior treatment history of autologous hematopoietic stem cell transplant (HSCT) or high-dose chemotherapy with stem cell rescue for any medical reason, not limited to myeloma treatment
  • Patients may not be receiving any other investigational agents
  • Patients with known brain metastases should be excluded from this clinical trial
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to or other agents used in the study, such as busulfan, melphalan, bortezomib, boron, or mannitol
  • Grade 2 or greater peripheral neuropathy within 14 days prior to enrollment
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  • Unresolved grade >= 3 non-hematologic toxicity from previous therapy; patients with grade 2 toxicity will be eligible at the discretion of the PI
  • Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ; cancer treated with curative intent < 5 years will not be allowed unless approved by the PI; cancer treated with curative intent > 5 years will be allowed
  • Patients must not have significant co-morbid medical condition
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements; patients must not have suffered recent (< 6 months) myocardial infarction, unstable angina, difficult to control congestive heart failure, uncontrolled hypertension, or difficult to control cardiac arrhythmias
  • Pregnant women are excluded from this study; breastfeeding should be discontinued if the mother is treated with busulfan
  • Human immunodeficiency virus (HIV)-positive patients on combination antiretroviral therapy are ineligible
  • Patients found to have an active hepatitis B infection (hepatitis B surface antigen +) are not eligible unless they meet ONE of the following criteria:
  • Patient is able to start dual anti-hepatitis (Hep) B therapy prior to enrollment with adefovir and telbivudine
  • Patient is already on dual anti-hepatitis B therapy
  • Consultation and co-management with a hepatitis expert regarding hepatitis B treatment is strongly encouraged before and during the trial
  • Patients, who are positive for hepatitis B core antibody, but negative for the hepatitis B surface antigen, should be started on lamivudine 100 mg daily until at least 3 months post stem cell transplant

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Rutgers Cancer Institute of New Jersey

    New Brunswick, New Jersey, 08903

  • NYU Cancer Institute

    New York, New York, 10016

  • Memorial Sloan-Kettering Cancer Center

    New York, New York, 10065

  • Albert Einstein College of Medicine

    The Bronx, New York, 10461

Other trials in United States

About this study

Official title: Phase II Study Assessing the Efficacy and Toxicity of PK--directed Intravenous Busulfan in Combination With High--Dose Melphalan and Bortezomib as Conditioning Regimen for First--Line Autologous Hematopoietic Stem Cell Transplantation in Patients With Multiple Myeloma

This phase II trial studies how well busulfan, melphalan, and bortezomib before first-line stem cell transplant works in treating patients with multiple myeloma. Giving chemotherapy before a peripheral blood stem cell transplant may stop the growth of cancer cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient's blood and stored. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To determine the complete response rate as defined by the International Myeloma Working Group (IMWG) criteria for patients with multiple myeloma treated with high dose chemotherapy with pharmacokinetic (PK) directed intravenous (IV) busulfan, bortezomib and melphalan (Bu/BTZ/Mel140) followed by autologous hematopoietic stem cell transplantation (ASCT) as first line therapy.

SECONDARY OBJECTIVES:

  1. To determine the overall response rate of the regimen Bu/BTZ/Mel140. II. To determine the treatment related toxicity and mortality of the regimen, including 100-day mortality rates.

III. To determine the duration of response, time to progression, progression-free survival, event-free survival and overall survival for this conditioning regimen.

IV. To determine whether there is a gender or race difference in the pharmacokinetic profile of IV busulfan.

  1. To determine methylation and gene expression signatures of pre-treatment bone marrow plasma cells and explore associations of these signatures with outcome.

OUTLINE:

CONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4.

TRANSPLANT: Patients undergo autologous peripheral blood stem cell transplant (PBSCT) on day 0.

After completion of study treatment, patients are followed up for up to 5 years.

Being tested
Busulfan, Melphalan, Bortezomib, Autologous Hematopoietic Stem Cell Transplantation, Peripheral Blood Stem Cell 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.

  • BusulfanDrug

    Given IV

  • MelphalanDrug

    Also called Alkeran

    Given IV

  • BortezomibDrug

    Given IV

  • Autologous Hematopoietic Stem Cell TransplantationProcedure or surgery

    Also called Autologous Stem Cell Transplantation

    Undergo autologous PBSCT

  • Peripheral Blood Stem Cell TransplantationProcedure or surgery

    Also called PBPC transplantation, Peripheral Blood Progenitor Cell Transplantation, Peripheral Stem Cell Support, Peripheral Stem Cell Transplantation

    Undergo autologous PBSCT

Study groups in NCT01605032
Study groupRoleWhat they receive
Treatment (busulfan, melphalan, bortezomib, autologous PBSCT)ExperimentalCONDITIONING: Patients receive busulfan IV over 3 hours on days -6 to -3, melphalan IV over 20 minutes on day -2, and bortezomib IV over 3-5 seconds on days -6, -3, 1, and 4. TRANSPLANT: Patients undergo autologous PBSCT on day 0.
Receives: Drug: Busulfan; Drug: Melphalan; Drug: Bortezomib; Procedure: Autologous Hematopoietic Stem Cell Transplantation; Procedure: Peripheral Blood Stem Cell Transplantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Rate of Complete Response as Determined by the IMWG Criteria

    Number of patients achieved complete response after the treatment regimen

    Measured: Day 100

Secondary outcomes (5)

  1. Overall Response Rate

    Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

    Measured: Up to day 100

  2. Mortality

    Measured: Up to day 100

  3. Time-to-progression

    Measured: From start of treatment to disease progression with deaths, up to 2 years

  4. Progression-free Survival

    The progression free survival was assessed over a period of 2 years

    Measured: 2 years

  5. Overall Survival

    The overall survival of patients was measured of a period of 2 years.

    Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 May 2012 (estimated)
  2. Study startFeb 2012
  3. Primary completionMar 2018
  4. Study completionMar 2018

Resources

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
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01605032
Sponsor's protocol ID
11-12-434
Other IDs
NCI-2013-01207, 11-102, 11-12-434, P30CA013330
Status last verified
Aug 2020
First submitted
7 Mar 2012
Last updated
20 Aug 2020
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Busulfan, Melphalan and Bortezomib in people with DS Stage I Plasma Cell Myeloma and DS Stage II Plasma Cell Myeloma, run by Albert Einstein College of Medicine at 4 sites in United States.

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 16 inclusion and 17 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: finished, as of the sponsor's last update on 20 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. 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?

Yes. Results are posted in the registry (first posted 20 Aug 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Rate of Complete Response as Determined by the IMWG Criteria (time frame: Day 100). 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 NCT01605032.

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 20 Aug 2020; AzTrials last refreshed its copy on 6 Nov 2026.

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