Multiple MyelomaPhase 1/2NCT01365559
Study of Carfilzomib for Multiple Myeloma Patients Who Are Relapsed/Refractory to Bortezomib-containing Treatments
A Phase 1/2 study testing Group A: Carfilzomib & Non-IMiD Regimen and Group B: Carfilzomib & IMiD containing regimen. in people with Multiple Myeloma, run by Oncotherapeutics at 9 sites in United States.
Trial summary
This is a phase I/II multicenter, open label, nonrandomized study for patients with Multiple Myeloma (MM) who will receive treatment with carfilzomib in place of bortezomib using the same bortezomib-containing combination regimen to which a MM patient has progressed while receiving. 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?
- Group A: Carfilzomib & Non-IMiD Regimen and Group B: Carfilzomib & IMiD containing regimen.
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 9 sitesUnited States
- Study dates
- 2 May 2011to 1 Mar 2016
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Disease-related:
- Have a diagnosis of MM based on standard criteria
- Currently has MM with measurable disease, defined as a monoclonal immunoglobulin spike on serum electrophoresis of at least 0.5 gm/dL and/or urine monoclonal immunoglobulin amount of at least 200 mg/24 hours.
- Have relapsed within 12 weeks of receiving or is refractory to their most recent bortezomib-containing regimen as long as they meet the following criteria:
- Progressed from bortezomib-containing regimen either as a single agent or in combination with an alkylating agent (melphalan or cyclophosphamide), an anthracycline (doxorubicin or pegylated liposomal doxorubicin), IMiDs (thalidomide or lenalidomide), and/or a glucocorticosteroid (prednisone, dexamethasone or medrol)
- Bortezomib must have been administered at 4 doses of a minimum of 1.0 mg/m2 in no more than 28 days per cycle. Subjects must have received at least one cycle meeting this definition and have shown progressive disease to be considered eligible.
Show all 23Show fewer
- Subject who have been refractory to their most recent bortezomib-containing regimen are eligible regardless of when the subject received that regimen, as long as they meet the above criteria and have been off the treatment for > 3 weeks.
- Definition of refractory disease: patients who meet criteria for progressive disease while currently receiving treatment.
- Demographics:
- Age ≥ 18 years
- Life expectancy ≥ 3 months
- ECOG performance status 0-2 at study entry
- Laboratory tests (within 14 days prior to drug dosing on Cycle 1, Day 1)
- Absolute neutrophil count (ANC) ≥ 1.5 × 10^9/L; if the bone marrow is extensively infiltrated (> 70% plasma cells) then 1.0 x 109/L
- Hemoglobin ≥ 8 g/dL (subjects may be receiving red blood cell [RBC] transfusions in accordance with institutional guidelines)
- Platelet count ≥ 75 × 109/L; if the bone marrow is extensively infiltrated (> 70% plasma cells) then 50 x 10^9/L
- Creatinine clearance (CrCl) ≥ 30 mL/minute either measured or calculated. Subject with a creatinine > 15mL/min and < 30 mL/min due to significant myelomatous involvement of the kidneys may be enrolled in the study after receipt of approval from Oncotherapeutics.
- Adequate hepatic function, with AST (SGOT) and ALT (SGPT) 3 x upper limit of normal (ULN) or 5 x ULN if hepatic metastases are present and serum total bilirubin ≤ 1.5 x ULN
- Serum potassium > 3 and < 5
- Ethical/Other
- Written informed consent in accordance with federal, local, and institutional guidelines.
- Females of childbearing potential (FCBP) must agree to ongoing pregnancy testing and to practice contraception.
- Male subjects must agree to practice contraception.
Who cannot join
- Disease-related
- Plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein (M-protein) and skin changes (POEMS) syndrome
- Plasma cell leukemia
- Severe hypercalcemia, i.e., serum calcium 12 mg/dL (3.0 mmol/L) corrected for albumin
- Received the following prior therapy:
- Chemotherapy within 21 days of enrollment (6 wks for nitrosoureas)
Show all 29Show fewer
- Corticosteroids (>10 mg/day prednisone or equivalent) within 21 days of enrollment
- Immunotherapy or antibody therapy as well as thalidomide, lenalidomide, arsenic trioxide, or bortezomib within 21 days before enrollment
- Radiation therapy within 21 days before enrollment, receipt of localized radiation therapy does not preclude enrollment
- Use of any other experimental drug or therapy within 28 days of enrollment
- Concurrent Conditions
- Impaired cardiac function or clinically significant cardiac diseases, including any one of the following:
- Unstable angina or myocardial infarction within 4 months prior to enrollment
- NYHA Class III or IV heart failure
- Uncontrolled angina
- Clinically significant pericardial disease
- Severe uncontrolled ventricular arrhythmias, sick sinus syndrome, or electrocardiographic evidence of acute ischemia or Grade 3 conduction system abnormalities unless subject has a pacemaker. Prior to study entry, any ECG abnormality at Screening has to be documented by the investigator as not medically relevant.
- Pregnant or lactating females
- Major surgery within 28 days prior to enrollment or has not recovered from side effects of such therapy (Kyphoplasty is not considered to be a major surgical procedure; however, the investigator is to discuss enrollment of a patient with a recent history of kyphoplasty with Oncotherapeutics).
- Acute active infection requiring treatment with systemic antibiotics, antivirals, or antifungals within 14 days prior to receiving first dose of study drug
- Known human immunodeficiency virus infection; baseline testing is not required
- Active hepatitis B or C infection; baseline testing is not required
- Uncontrolled hypertension or uncontrolled diabetes within 14 days prior to enrollment
- Nonhematologic malignancy within the past 5 years with the exception of a) adequately treated basal cell carcinoma, squamous cell skin cancer, or thyroid cancer; b) carcinoma in situ of the cervix or breast; c) prostate cancer of Gleason Grade 6 or less with stable prostate-specific antigen levels; or d) cancer considered cured by surgical resection or unlikely to impact survival during the duration of the study, such as localized transitional cell carcinoma of the bladder or benign tumors of the adrenal or pancreas
- Concurrent use of other anti-cancer agents or treatments
- Significant neuropathy (Grades 3-4, or Grade 2 with pain) within 14 days prior to enrollment
- Known history of allergy to Captisol® (a cyclodextrin derivative used to solubilize carfilzomib)
- Subjects with pleural effusions requiring thoracentesis or ascites requiring paracentesis within 14 days prior to enrollment
- Any other clinically significant medical disease or condition that, in the Investigator's opinion, may interfere with protocol adherence or a subject's ability to give informed
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Center for Cancer and Blood Disorders
Bethesda, Maryland, 20817
Pacific Oncology and Hematology
Encinitas, California, 92024
Virginia Cancer Specialists
Fairfax, Virginia, 233031
Franciscan St. Francis Health
Indianapolis, Indiana, 46237
Family Cancer Center Foundation, Inc.
Memphis, Tennessee, 38119
Pacific Cancer Care
Salinas, California, 93901
Central Coast Medical Oncology
Santa Maria, California, 93454
James R. Berenson, MD, Inc.
West Hollywood, California, 90069
Cancer Centers of America
Zion, Illinois, 60099
About this study
Official title: A Phase I/II Study of Carfilzomib as a Replacement for Multiple Myeloma Patients Failing Bortezomib-containing Regimens
This is a phase I/II multicenter, open label, nonrandomized study for patients with Multiple Myeloma (MM) who will receive treatment with carfilzomib in place of bortezomib using the same bortezomib-containing combination regimen to which a MM patient has progressed while receiving. This study will enroll 45 patients total. These patients will be resistant to bortezomib as demonstrated by progressive disease while on bortezomib or have relapsed within 12 weeks of the last dose of bortezomib in a combination regimen. Patients will be sub-divided into 2 groups in this study, treatments containing (Group A) or not containing immunomodulatory drugs (IMiDs) (Group B). Thirty patient will be enrolled into Group A and 15 patients into Group B for a total of 45 patients. Patients must have received 4 doses of a minimum of 1.0 mg/m^2 of bortezomib in no more than 4 weeks per cycle. Patients must have received at least one cycle meeting this definition and have shown progressive disease to be considered eligible. Patients who have been refractory to or relapsed within 12 weeks of the last dose of bortezomib in their most recent bortezomib-containing regimen that does not include either thalidomide or lenalidomide are eligible regardless of when patients received that regimen, as long as they meet the above criteria.
Carfilzomib will subsequently replace bortezomib using the patient's most recent bortezomib-containing regimen to which the patient progressed while receiving. Patients will be eligible if they progressed from bortezomib with an alkylating agent (melphalan or cyclophosphamide), an anthracycline (doxorubicin or pegylated liposomal doxorubicin) and/or a glucocorticosteroid (prednisone, dexamethasone or medrol)and IMiD (thalidomide or lenalidomide). The study will consist of a screening period, followed by up to eight open label treatment cycles, a final assessment to occur 28 days after the end of the last treatment cycle, a follow-up period and maintenance cycles of single agent carfilzomib.
Patient who complete the combination treatment period without progressive disease will be eligible for maintenance therapy with single-agent carfilzomib. During maintenance therapy carfilzomib will be administered at the same dose given during the last cycle of combination treatment.
- Conditions
- Multiple Myeloma
- Being tested
- Group A: Carfilzomib & Non-IMiD Regimen, Group B: Carfilzomib & IMiD containing regimen.
- Keywords
- multiple myeloma, carfilzomib, relapsed, refractory, bortezomib, Oncotherapeutics
- Medical subject headings
- Multiple Myeloma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Group A: Carfilzomib & Non-IMiD RegimenDrug
Carfilzomib will be administered intravenously starting at a dose of 20 mg/m^2 over 30 minutes for the first cycle and will then be increased to 27, 36 and 45 mg/m2 during cycles 2-4, respectively. Doses will be administered IV once daily on days 1, 2, 8, 9, 15, and 16 of each cycle. Cycles will be 28 days in length.
Combination non-IMiD drug is dosed on the identical schedule and dosage as patient was in previous bortezomib containing regimen.
Maintenance regimen maybe administered if patient does not progress while on study.
Group B: Carfilzomib & IMiD containing regimen.Drug
Carfilzomib will be administered intravenously starting at a dose of 20 mg/m^2 over 30 minutes for the first cycle and will then be increased to 27, 36 and 45 mg/m2 during cycles 2-4, respectively. Doses will be administered IV once daily on days 1, 2, 8, 9, 15, and 16 of each cycle. Cycles will be 28 days in length.
Combination IMiD drug is dosed on the identical schedule and dosage as patient was in previous bortezomib containing regimen.
Maintenance regimen maybe administered if patient does not progress while on study.
| Study group | Role | What they receive |
|---|---|---|
| Group A: Carfilzomib & Non-IMiD containing regimen | Experimental | Bortezomib is replaced with carfilzomib in a combined regimen identical to the patient's previous regimen. Regimen cannot include thalidomide or lenalidomide. Receives: Drug: Group A: Carfilzomib & Non-IMiD Regimen |
| Group B: Carfilzomib & IMiD containing regimen. | Experimental | Bortezomib is replaced with carfilzomib in a regimen that includes IMiDs (lenalidomide or thalidomide). Thus, the regimen is carfilzomib in an IMiD-containing regimen. Receives: Drug: Group B: Carfilzomib & IMiD containing regimen. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Establish MTD, determine DLT and to determine the efficacy as assessed by the overall response rate.
Phase I:
- To establish the maximum tolerated dose (MTD) and determine the dose limiting toxicities (DLT) following treatment.
Phase II:
- To determine the efficacy as assessed by the overall response rate [CR + VGPR + PR + MR] and the Time to Progression (TTP) of disease.
Measured: Montly
Secondary outcomes (1)
To establish safety and efficacy following treatment.
Phase I:
Obtain preliminary evidence of efficacy following treatment based on:
- SPEP, UPEP and quantification of serum immunoglobulins
- Bone marrow aspirates \& biopsies
- B2M
- A roentgenographic skeletal survey of bones
Phase II:
Establish the safety \& tolerability following treatment based on:
- Adverse events
- Clinical lab tests
- Vital signs
- Medical history \& body weight measurements
- ECOG performance status
- Concomitant medication usage
Both phases:
Progression Free Survival among patients who continue onto maintenance treatment
Measured: Montly
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2011 (estimated)
- Study start2 May 2011
- Primary completion13 Jun 2014
- Study completion1 Mar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oncotherapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Amgen
- Responsible party
- Sponsor
- Investigators
- James R Berenson, MD, Principal investigator, James R. Berenson, MD., Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01365559
- Sponsor's protocol ID
- IST-CAR-516
- Status last verified
- Mar 2018
- First submitted
- 31 May 2011
- Last updated
- 5 Mar 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
Berenson JR, Hilger JD, Yellin O, Dichmann R, Patel-Donnelly D, Boccia RV, Bessudo A, Stampleman L, Gravenor D, Eshaghian S, Nassir Y, Swift RA, Vescio RA. Replacement of bortezomib with carfilzomib for multiple myeloma patients progressing from bortezomib combination therapy. Leukemia. 2014 Jul;28(7):1529-36. doi: 10.1038/leu.2014.27. Epub 2014 Jan 16.
PubMed 24429497
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of Carfilzomib as a Replacement for Multiple Myeloma Patients Failing Bortezomib-containing Regimens
- ClinicalTrials.gov ID
- NCT01365559
- Acronym / sponsor ID
- IST-CAR-516
- Conditions
- Multiple Myeloma
- Keywords
- multiple myeloma, carfilzomib, relapsed, refractory, bortezomib, Oncotherapeutics
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- OncotherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Amgen
- Study start
- 2 May 2011 (actual)
- Primary completion
- 13 Jun 2014 (actual)
- Study completion
- 1 Mar 2016 (actual)
- First posted
- 3 Jun 2011 (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.
This is a phase I/II multicenter, open label, nonrandomized study for patients with Multiple Myeloma (MM) who will receive treatment with carfilzomib in place of bortezomib using the same bortezomib-containing combination regimen to which a MM patient has progressed while receiving. This study will enroll 45 patients total. These patients will be resistant to bortezomib as demonstrated by progressive disease while on bortezomib or have relapsed within 12 weeks of the last dose of bortezomib in a combination regimen. Patients will be sub-divided into 2 groups in this study, treatments containing (Group A) or not containing immunomodulatory drugs (IMiDs) (Group B). Thirty patient will be enrolled into Group A and 15 patients into Group B for a total of 45 patients. Patients must have received 4 doses of a minimum of 1.0 mg/m^2 of bortezomib in no more than 4 weeks per cycle. Patients must have received at least one cycle meeting this definition and have shown progressive disease to be considered eligible. Patients who have been refractory to or relapsed within 12 weeks of the last dose of bortezomib in their most recent bortezomib-containing regimen that does not include either thalidomide or lenalidomide are eligible regardless of when patients received that regimen, as long as they meet the above criteria.
Carfilzomib will subsequently replace bortezomib using the patient's most recent bortezomib-containing regimen to which the patient progressed while receiving. Patients will be eligible if they progressed from bortezomib with an alkylating agent (melphalan or cyclophosphamide), an anthracycline (doxorubicin or pegylated liposomal doxorubicin) and/or a glucocorticosteroid (prednisone, dexamethasone or medrol)and IMiD (thalidomide or lenalidomide). The study will consist of a screening period, followed by up to eight open label treatment cycles, a final assessment to occur 28 days after the end of the last treatment cycle, a follow-up period and maintenance cycles of single agent carfilzomib.
Patient who complete the combination treatment period without progressive disease will be eligible for maintenance therapy with single-agent carfilzomib. During maintenance therapy carfilzomib will be administered at the same dose given during the last cycle of combination treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A: Carfilzomib & Non-IMiD containing regimen | Experimental | Drug: Group A: Carfilzomib & Non-IMiD RegimenBortezomib is replaced with carfilzomib in a combined regimen identical to the patient's previous regimen. Regimen cannot include thalidomide or lenalidomide. |
| Group B: Carfilzomib & IMiD containing regimen. | Experimental | Drug: Group B: Carfilzomib & IMiD containing regimen.Bortezomib is replaced with carfilzomib in a regimen that includes IMiDs (lenalidomide or thalidomide). Thus, the regimen is carfilzomib in an IMiD-containing regimen. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- Have a diagnosis of MM based on standard criteria
- Currently has MM with measurable disease, defined as a monoclonal immunoglobulin spike on serum electrophoresis of at least 0.5 gm/dL and/or urine monoclonal immunoglobulin amount of at least 200 mg/24 hours.
- Have relapsed within 12 weeks of receiving or is refractory to their most recent bortezomib-containing regimen as long as they meet the following criteria:
- Definition of refractory disease: patients who meet criteria for progressive disease while currently receiving treatment.
- Age ≥ 18 years
Key exclusion criteria (29 listed)
- Disease-related
- Plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein (M-protein) and skin changes (POEMS) syndrome
- Plasma cell leukemia
- Severe hypercalcemia, i.e., serum calcium 12 mg/dL (3.0 mmol/L) corrected for albumin
- Received the following prior therapy:
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 |
|---|---|---|
| Group A: Carfilzomib & Non-IMiD RegimenCarfilzomib will be administered intravenously starting at a dose of 20 mg/m^2 over 30 minutes for the first cycle and will then be increased to 27, 36 and 45 mg/m2 during cycles 2-4, respectively. Doses will be administered IV once daily on days 1, 2, 8, 9, 15, and 16 of each cycle. Cycles will be 28 days in length. Combination non-IMiD drug is dosed on the identical schedule and dosage as patient was in previous bortezomib containing regimen. Maintenance regimen maybe administered if patient does not progress while on study. | Drug | Group A: Carfilzomib & Non-IMiD containing regimen |
| Group B: Carfilzomib & IMiD containing regimen.Carfilzomib will be administered intravenously starting at a dose of 20 mg/m^2 over 30 minutes for the first cycle and will then be increased to 27, 36 and 45 mg/m2 during cycles 2-4, respectively. Doses will be administered IV once daily on days 1, 2, 8, 9, 15, and 16 of each cycle. Cycles will be 28 days in length. Combination IMiD drug is dosed on the identical schedule and dosage as patient was in previous bortezomib containing regimen. Maintenance regimen maybe administered if patient does not progress while on study. | Drug | Group B: Carfilzomib & IMiD containing regimen. |
Primary outcome
Establish MTD, determine DLT and to determine the efficacy as assessed by the overall response rate.
Time frame: Montly
Phase I: • To establish the maximum tolerated dose (MTD) and determine the dose limiting toxicities (DLT) following treatment. Phase II: • To determine the efficacy as assessed by the overall response rate [CR + VGPR + PR + MR] and the Time to Progression (TTP) of disease.
1 secondary outcome
To establish safety and efficacy following treatment.
Time frame: Montly
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2018.
- Derived from this study: PubMed 24429497Berenson JR, Hilger JD, Yellin O, Dichmann R, Patel-Donnelly D, Boccia RV, Bessudo A, Stampleman L, Gravenor D, Eshaghian S, Nassir Y, Swift RA, Vescio RA. Replacement of bortezomib with carfilzomib for multiple myeloma patients progressing from bortezomib combination therapy. Leukemia. 2014 Jul;28(7):1529-36. doi: 10.1038/leu.2014.27. Epub 2014 Jan 16.
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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.
James R Berenson, MD principal investigator
James R. Berenson, MD., Inc.
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Group A: Carfilzomib & Non-IMiD Regimen and Group B: Carfilzomib & IMiD containing regimen. in people with Multiple Myeloma, run by Oncotherapeutics at 9 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 23 inclusion and 29 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 5 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 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 combined 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.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Establish MTD, determine DLT and to determine the efficacy as assessed by the overall response rate. (time frame: Montly). 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 NCT01365559, which also lists 1 linked publication.
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 5 Mar 2018.