Multiple MyelomaPhase 1NCT00934232
Busulfan in Multiple Myeloma
A Phase 1 study testing Busulfan in people with Multiple Myeloma, run by Guido Tricot.
Trial summary
Two main objectives of the study are: Primary: To determine the MTD of Busulfex ® that can be given safely over the least number of days to myeloma patients who are either ≥65 years of age (Group 1) or have renal insufficiency (Group 2), defined as creatinine >3mg/dL or creatinine clearance <30 mL/min. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- Busulfan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- No sites listed yet
- Study dates
- Aug 2009to Feb 2012
- Phase
- Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects must have ≥ 3 x 106 CD34 cell/kg in storage for this study.
- Subjects must have symptomatic multiple myeloma at their new patient consult at HCI that, in the opinion of the enrolling physician, requires treatment.
- Subjects must be at least 65 years of age and/or diagnosed with renal insufficiency, defined as serum creatinine ≥3mg/dL or a creatinine clearance of less than 30mL/minute.
- Subjects must not have a history of chronic obstructive or chronic restrictive pulmonary disease. Subjects must demonstrate adequate pulmonary function studies defined as ≥50% of predicted on mechanical aspects (FEV1, FVC) and diffusion capacity (DLCO).
- Subjects must demonstrate adequate cardiac function (≥40% LVEF on ECHO or MUGA).
- Subjects must demonstrate adequate liver functions with total bilirubin and transaminase levels no higher than 1.5 times the institutional upper limit of normal. (If total bilirubin is > 1.5 times the upper limit of normal, a direct bilirubin needs to be assessed. Subject eligible as long as the direct bilirubin is not > 1.5 times the upper limit of normal)
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- Subjects must have at least one evaluable myeloma marker by which to judge response: serum M protein >1g/dL, free light chains in the serum that more than four times the upper limit of normal, urine M protein of ≥ 500 mg, urine free light chains of ≥ 500 mg/day, bone marrow plasmacytosis with >20% plasma cells, extramedullary plasmacytosis, or MRI/FDG-PET/CT scan demonstrating 1 or more focal lesions due to myeloma.
- Subjects must have a SWOG performance score of 0-2 unless due to myeloma-related bone pain.
- Subjects must be informed of the investigational nature of the study and must sign an IRB-approved informed consent in accordance with institutional and federal guidelines.
- Female participants of child-bearing potential must have a negative pregnancy test documented within 10 days of enrollment.
Who cannot join
- Subjects must not have serum transaminases >1.5 times the upper limit of normal and/or a direct bilirubin >1.5 time the institutional upper limit of normal (direct bilirubin to be assessed only if the total bilirubin is > 1.5 times the upper limit of normal)
- Subjects must not be HIV positive or have active Hepatitis B or Hepatitis C infection. If serology antibody studies are positive, a quantitative PCR must be done to confirm.
- Subjects must not have a prior malignancy in which life expectancy, which in the opinion of the investigator, is more likely to be determined by the prior malignancy than the myeloma. Patients must not be currently receiving therapy for the prior malignancy.
- Subjects must not have had a prior autologous or allogeneic bone marrow transplant.
- Subjects must not be pregnant or nursing. Women and men of reproductive potential may not participate unless they agree to use an effective contraceptive method.
- Patients who have < 3 million CD34 cells/kg stored for this protocol.
About this study
Official title: A Phase I Open Label Study of IV Busulfan (Busulfex®) in Multiple Myeloma Patients 65 Years of Age or Older, or With Renal Insufficiency Undergoing Autologous Transplantation
Two main objectives of the study are:
Primary:
To determine the MTD of Busulfex ® that can be given safely over the least number of days to myeloma patients who are either ≥65 years of age (Group 1) or have renal insufficiency (Group 2), defined as creatinine >3mg/dL or creatinine clearance <30 mL/min.
Secondary:
To perform pharmacokinetic (PK) studies to evaluate individual variability and the relationship to toxicities in each of the two groups at each proposed dose level.
Read the detailed description
This study is for first line treatment for multiple myeloma patients in patients >65 years or have renal insufficiency Busulfan introduces cytopenia much slower than melphalan while the time to recover after transplantation is the same. The duration of cytopenia with busulfan should therefore be shorter. Busulfan given in myeloma patients with renal failure has proven to be effective.
The initial proposed dose level in our study will be 3.2mg/kg of body weight over 6 hours for 3 days. The next levels will be 3.2mg/kg of body weight over 6 hours for 4 days, 4.3 mg/kg of body weight over 6 hours for 3 days, 5.6 mg/kg of body weight over 6 hours for 2 days, and 6.4 mg/kg of body weight over 6 hours for 2 days. Three patients per group will be entered at the first level. Further enrollment will be dependent upon toxicities observed. All dose escalations will be initiated after the PI has reviewed the toxicity data available on the prior dose levels and confirms that it is safe to proceed to subsequent dose level(s). Maximum tolerated dose will defined as the dose level immediately below the dose level at which ≥ 2 of 6 patients experiences a grade 4 non-hematological toxicity that is unexpected, related, and serious, and it will be evaluated separately for patients > 65 years of age or in patients with renal insufficiency.
- Conditions
- Multiple Myeloma
- Being tested
- Busulfan
- Keywords
- cancer, myeloma
- Medical subject headings
- Multiple Myeloma, Neoplasms, Neoplasms, Plasma Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Also called Busulfex ®
Busulfan (Bu) is a bifunctional alkylating agent currently used almost exclusively as a component of conditioning regimens for both autologous and allogeneic stem-cell transplants.
| Study group | Role | What they receive |
|---|---|---|
| Busulfan | Experimental | Busulfex given to patients who are either ≥65 years or have renal insufficiency Receives: Drug: Busulfan |
What is being measured
The primary outcome is the main question the study is designed to answer.
determine MTD of Busulfex given safely over least number of days patients who are either ≥65 years Group 1) or have renal insufficiency (Group 2), defined as creatinine >3mg/dL or creatinine clearance <30 mL/min.
Measured: 1 year
Secondary outcomes (1)
To perform pharmacokinetic (PK) studies to evaluate individual variability and the relationship to toxicities in each of the two groups at each proposed dose level.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2009 (estimated)
- Study startAug 2009
- Primary completionFeb 2012
- Study completionFeb 2012
Why it stopped: PI left the institution
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Guido Tricot
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Guido Tricot, University of Iowa
- Investigators
- Guido Tricot, MD, PhD, Principal investigator, University of Iowa
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00934232
- Sponsor's protocol ID
- 32857
- Status last verified
- May 2017
- First submitted
- 4 Jun 2009
- Last updated
- 23 May 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Open Label Study of IV Busulfan (Busulfex®) in Multiple Myeloma Patients 65 Years of Age or Older, or With Renal Insufficiency Undergoing Autologous Transplantation
- ClinicalTrials.gov ID
- NCT00934232
- Acronym / sponsor ID
- 32857
- Conditions
- Multiple Myeloma
- Keywords
- cancer, myeloma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: PI left the institution
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Guido TricotUniversity, hospital or other
- Collaborators
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Study start
- Aug 2009 (actual)
- Primary completion
- Feb 2012 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 8 Jul 2009 (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.
Two main objectives of the study are:
Primary:
To determine the MTD of Busulfex ® that can be given safely over the least number of days to myeloma patients who are either ≥65 years of age (Group 1) or have renal insufficiency (Group 2), defined as creatinine >3mg/dL or creatinine clearance <30 mL/min.
Secondary:
To perform pharmacokinetic (PK) studies to evaluate individual variability and the relationship to toxicities in each of the two groups at each proposed dose level.
Detailed description
This study is for first line treatment for multiple myeloma patients in patients >65 years or have renal insufficiency Busulfan introduces cytopenia much slower than melphalan while the time to recover after transplantation is the same. The duration of cytopenia with busulfan should therefore be shorter. Busulfan given in myeloma patients with renal failure has proven to be effective.
The initial proposed dose level in our study will be 3.2mg/kg of body weight over 6 hours for 3 days. The next levels will be 3.2mg/kg of body weight over 6 hours for 4 days, 4.3 mg/kg of body weight over 6 hours for 3 days, 5.6 mg/kg of body weight over 6 hours for 2 days, and 6.4 mg/kg of body weight over 6 hours for 2 days. Three patients per group will be entered at the first level. Further enrollment will be dependent upon toxicities observed. All dose escalations will be initiated after the PI has reviewed the toxicity data available on the prior dose levels and confirms that it is safe to proceed to subsequent dose level(s). Maximum tolerated dose will defined as the dose level immediately below the dose level at which ≥ 2 of 6 patients experiences a grade 4 non-hematological toxicity that is unexpected, related, and serious, and it will be evaluated separately for patients > 65 years of age or in patients with renal insufficiency.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Busulfan | Experimental | Drug: BusulfanBusulfex given to patients who are either ≥65 years or have renal insufficiency |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Subjects must have ≥ 3 x 106 CD34 cell/kg in storage for this study.
- Subjects must have symptomatic multiple myeloma at their new patient consult at HCI that, in the opinion of the enrolling physician, requires treatment.
- Subjects must be at least 65 years of age and/or diagnosed with renal insufficiency, defined as serum creatinine ≥3mg/dL or a creatinine clearance of less than 30mL/minute.
- Subjects must not have a history of chronic obstructive or chronic restrictive pulmonary disease. Subjects must demonstrate adequate pulmonary function studies defined as ≥50% of predicted on mechanical aspects (FEV1, FVC) and diffusion capacity (DLCO).
- Subjects must demonstrate adequate cardiac function (≥40% LVEF on ECHO or MUGA).
Key exclusion criteria (6 listed)
- Subjects must not have serum transaminases >1.5 times the upper limit of normal and/or a direct bilirubin >1.5 time the institutional upper limit of normal (direct bilirubin to be assessed only if the total bilirubin is > 1.5 times the upper limit of normal)
- Subjects must not be HIV positive or have active Hepatitis B or Hepatitis C infection. If serology antibody studies are positive, a quantitative PCR must be done to confirm.
- Subjects must not have a prior malignancy in which life expectancy, which in the opinion of the investigator, is more likely to be determined by the prior malignancy than the myeloma. Patients must not be currently receiving therapy for the prior malignancy.
- Subjects must not have had a prior autologous or allogeneic bone marrow transplant.
- Subjects must not be pregnant or nursing. Women and men of reproductive potential may not participate unless they agree to use an effective contraceptive method.
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 |
|---|---|---|
| BusulfanAlso: Busulfex ®Busulfan (Bu) is a bifunctional alkylating agent currently used almost exclusively as a component of conditioning regimens for both autologous and allogeneic stem-cell transplants. | Drug | Busulfan |
Primary outcome
determine MTD of Busulfex given safely over least number of days patients who are either ≥65 years Group 1) or have renal insufficiency (Group 2), defined as creatinine >3mg/dL or creatinine clearance <30 mL/min.
Time frame: 1 year
1 secondary outcome
To perform pharmacokinetic (PK) studies to evaluate individual variability and the relationship to toxicities in each of the two groups at each proposed dose level.
Time frame: 18 months
Results and safety
The study was stopped early (reason given: PI left the institution). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2017.
- Individual participant dataSharing planned: No
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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.
Guido Tricot, MD, PhD principal investigator
University of Iowa
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Busulfan in people with Multiple Myeloma, run by Guido Tricot.
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 10 inclusion and 6 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: stopped early, as of the sponsor's last update on 23 May 2017. Status can change between updates, so confirm with the study contact before making plans.
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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: PI left the institution). Results are not available in the source record.
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?
determine MTD of Busulfex given safely over least number of days patients who are either ≥65 years Group 1) or have renal insufficiency (Group 2), defined as creatinine >3mg/dL or creatinine clearance <30 mL/min. (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 May 2017.