Recurrent Plasma Cell MyelomaPhase 1/2NCT02509052
Leflunomide in Treating Patients With Relapsed or Refractory Multiple Myeloma
A Phase 1/2 study testing Laboratory Biomarker Analysis, Leflunomide and Pharmacological Study in people with Recurrent Plasma Cell Myeloma and Refractory Plasma Cell Myeloma, run by City of Hope Medical Center at 1 site in United States.
Trial summary
This phase I/II trial studies the side effects and best dose of leflunomide in treating patients with multiple myeloma that has come back (relapsed) or has not responded to previous treatment (refractory). Leflunomide may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Recurrent Plasma Cell Myeloma, Refractory Plasma Cell Myeloma
- What is being tested?
- Laboratory Biomarker Analysis, Leflunomide and Pharmacological Study
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Dec 2015to 9 Jun 2022
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- All subjects must have the ability to understand and the willingness to sign a written informed consent
- Patients must have a life expectancy of > 3 months
- Patients must exhibit an Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
- Patients must have a diagnosis of multiple myeloma
- Serum M-protein >= 0.5 g/dL
- Urine M-protein >= 200 mg/24 hr
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- Serum free light chain >=10 mg/dL provided the free light chain (FLC) ratio is abnormal
- 10% plasma cells in bone marrow
- Patients must be relapsed or are refractory to at least 3 prior lines of therapy, including both a proteasome inhibitor an immunomodulatory drug (IMiD), and for whom a transplant is not recommended (induction therapy and stem cell transplant +/- maintenance will be considered as one regimen)
- At least 2 weeks from prior therapy to time of start of treatment; prior therapy includes steroids (except prednisone or equivalent - up to 10 mg per day is allowed)
- Platelet count >= 50,000/uL; platelet transfusions are not allowed within 14 days of platelet assessment
- Absolute neutrophil count (ANC) >= 1000/mm^3; growth factor is not permitted within 14 days of neutrophil assessment
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 2.0 x upper limit of normal (ULN)
- Total Bilirubin < 1.5 x ULN
- Calculated creatinine clearance (CrCl) >= 30 mL/min per 24 hour urine collection or the Cockcroft-Gault formula
- Negative serum or urine beta-human chorionic gonadotropin (B-HCG) test (female patient of childbearing potential* only), to be performed locally within the screening period
- Negative for tuberculosis antigen (e.g. T-Spot test)
- Negative for hepatitis A, B, or C infection
- Adequate pulmonary function as defined by forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLCO) >= 50% of predicted by pulmonary function testing
- Agreement by females of childbearing potential* and sexually active males to use an effective method of contraception (hormonal or barrier method of birth control or abstinence) prior to study entry and for three months following duration of study participation; should a woman become pregnant or suspect that she is pregnant while participating on the trial, she should inform her treating physician immediately * A female of childbearing potential is defined as a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months
Who cannot join
- Prior treatment with leflunomide
- Current or planned use of other investigational agents, or concurrent biological, chemotherapy, or radiation therapy during the study treatment period
- Current or planned growth factor or transfusion support until after initiation of treatment; if growth factor or transfusion support is provided between screening and start of treatment, the participant will no longer be eligible
- Prior diagnosis of rheumatoid arthritis
- Prior allogenic transplant
- Acute active infection requiring systemic therapy within 2 weeks prior to enrollment
Show all 18Show fewer
- Pre-existing liver disease
- Known human immunodeficiency virus (HIV) infection
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to leflunomide or cholestyramine
- Non-hematologic malignancy within the past 3 years aside from the following exceptions:
- Adequately treated basal cell or squamous cell skin cancer
- Carcinoma in situ of the cervix
- Prostate cancer < Gleason grade 6 with a stable prostate specific antigen (PSA)
- Successfully treated in situ carcinoma of the breast
- Clinically significant medical disease or condition that, in the investigator's opinion, may interfere with protocol adherence or the patient's ability to give informed consent
- Pregnant women and women who are lactating; breastfeeding should be discontinued if the mother is enrolled on this study
- Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns or compliance with clinical study procedures, e.g., infection/inflammation, intestinal obstruction, unable to swallow medication, social/ psychological issues, etc
- NONCOMPLIANCE: Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)
Trial locations and contacts
1 site in 1 country.
United States1 site
City of Hope Medical Center
Duarte, California, 91010
About this study
Official title: A Phase I/II Dose-Escalation Trial of Leflunomide in Patients With Relapsed or Relapsed/Refractory Multiple Myeloma
This phase I/II trial studies the side effects and best dose of leflunomide in treating patients with multiple myeloma that has come back (relapsed) or has not responded to previous treatment (refractory). Leflunomide may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Read the detailed description
PRIMARY OBJECTIVES: I. To determine the maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of leflunomide, when given as a single agent. (Phase I) II. To assess the safety and tolerability of leflunomide at each dose level by evaluation of toxicities including: type, frequency, severity, attribution, time course and duration. (Phase I) III. To evaluate the anti-myeloma activity of leflunomide, when given as a single agent, as assessed by overall response rate (ORR). (Phase II)
SECONDARY OBJECTIVES: I. To obtain estimates of: response duration, clinical benefit response, overall survival, progression-free survival. (Phase II)
TERTIARY OBJECTIVES: I. To characterize the relationship between serum concentration of the active leflunomide metabolite, teriflunomide (A77 1726), and toxicity. (Phase I/II) II. To assess the relationship between serum concentration of the active leflunomide metabolite, teriflunomide (A77 1726), and disease response. (Phase I/II) III. To explore the relationship between polymorphisms in the CYP1A2, CYP2C19, or DHODH genes and toxicity/response. (Phase I/II) IV. To explore the ex vivo cytotoxicity of leflunomide toward primary multiple myeloma (MM) cells, in order to evaluate whether individual ex vivo leflunomide response might be a useful predictor of therapeutic response. (Phase I/II) V. To explore the potential additive or synergistic effects of combining leflunomide with other classes of Food and Drug Administration (FDA)-approved drugs. (Phase I/II) VI. To generate a preliminary ribonucleic acid (RNA)/microRNA (miRNA) and deoxyribonucleic acid (DNA) methylation signature associated with response of MM cells to leflunomide in vivo (mRNA/miRNA and DNA methylation, phase II only) and teriflunomide ex vivo (messenger RNA [mRNA]/miRNA). (Phase I/II)
OUTLINE: This is a dose-escalation study. Patients receive leflunomide orally (PO) once daily (QD) on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 30 days and then every 28 days until disease progression (active follow-up) or every 3 months (long term follow-up).
- Being tested
- Laboratory Biomarker Analysis, Leflunomide, Pharmacological Study
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Laboratory Biomarker AnalysisOther
Correlative studies
LeflunomideDrug
Also called Arava, SU101
Given PO
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Arm 1: 20 mg leflunomide | Experimental | Patients receive 20 mg leflunomide PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Receives: Other: Laboratory Biomarker Analysis; Drug: Leflunomide; Other: Pharmacological Study |
| Arm 2: 40 mg leflunomide | Experimental | Patients receive 40 mg leflunomide PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Receives: Other: Laboratory Biomarker Analysis; Drug: Leflunomide; Other: Pharmacological Study |
| Arm 3: 60 mg leflunomide | Experimental | Patients receive 60 mg leflunomide PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Receives: Other: Laboratory Biomarker Analysis; Drug: Leflunomide; Other: Pharmacological Study |
What is being measured
The primary outcome is the main question the study is designed to answer.
MTD, Defined as the Highest Dose in Which =< 1/6 Patients Experience a Dose-limiting Toxicity, Graded According to the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.03
Observed toxicities will be summarized, for all dose levels, in terms of type (organ affected or laboratory determination), severity, time of onset, duration, serum concentration of the active leflunomide metabolite, probable association with the study treatment and reversibility or outcome.
Measured: 28 days
Best Overall Response Rate: Proportion of Patients Reaching CR by IMWG Criteria
Stringent complete response [sCR]/complete response [CR]/very good partial response [VGPR]/or partial response [PR]), assessed by International Myeloma Working Group (IMWG) criteria.
Measured: From the start of treatment until disease progression/recurrence, assessed up to 48 months
Secondary outcomes (3)
Clinical Benefit Response Rate (sCR/CR/VGPR/Partial Response [PR]/Minimal Response [MR] or Stable Disease [SD]), Assessed by IMWG Criteria
Clinical benefit response rate (sCR/CR/VGPR/partial response [PR]/minimal response [MR] or stable disease [SD]), assessed by International Myeloma Working Group (IMWG) criteria
Measured: From the start of treatment until disease progression/recurrence, assessed up to 48 months
Response Duration
Median and range of nine patients with Complete Response (CR)
Measured: Assessed up to 48 months
Response Duration
Number of patients with Stable Disease greater than or equal to 90 days
Measured: Assessed at ninety days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2015 (estimated)
- Study start2 Dec 2015
- Primary completion18 Sep 2018
- Study completion9 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Recurrent Plasma Cell MyelomaStudies of the same condition, open and under way.
- More trials run by City of Hope Medical CenterThe sponsor's other registered studies.
- 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
- Sequential
- Model description
- The phase I portion will implement a modified rolling six dose escalation design, a more conservative version of the rolling six design of Skolnik, et al., for enrollment with dose escalation, de-escalation, or expansion of a cohort on the basis of the occurrence of dose limiting toxicities (DLTs) during cycle 1. The starting dose of leflunomide is 20mg daily, with the intention to test up to 60mg daily. Escalation will proceed in increments of 20mg/daily; de-escalation will proceed in decrements of 10mg/day. Leglunomide is administered with a loading dose of 100 mg for the 1st three doses.
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- City of Hope Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Michael Rosenzweig, Principal investigator, City of Hope Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02509052
- Sponsor's protocol ID
- 15140
- Other IDs
- NCI-2015-01181, 15140
- Status last verified
- Sep 2023
- First submitted
- 23 Jul 2015
- Last updated
- 10 Oct 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Rosenzweig M, Palmer J, Tsai NC, Synold T, Wu X, Tao S, Hammond SN, Buettner R, Duarte L, Htut M, Karanes C, Nathwani N, Pichiorri F, Sahebi F, Sanchez JF, Chowdhury A, Krishnan A, Forman SJ, Rosen ST. Repurposing leflunomide for relapsed/refractory multiple myeloma: a phase 1 study. Leuk Lymphoma. 2020 Jul;61(7):1669-1677. doi: 10.1080/10428194.2020.1742900. Epub 2020 Apr 8.
PubMed 32268821
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Dose-Escalation Trial of Leflunomide in Patients With Relapsed or Relapsed/Refractory Multiple Myeloma
- ClinicalTrials.gov ID
- NCT02509052
- Acronym / sponsor ID
- 15140
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- City of Hope Medical CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 2 Dec 2015 (actual)
- Primary completion
- 18 Sep 2018 (actual)
- Study completion
- 9 Jun 2022 (actual)
- First posted
- 27 Jul 2015 (estimated)
- Results
- Posted 10 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase I/II trial studies the side effects and best dose of leflunomide in treating patients with multiple myeloma that has come back (relapsed) or has not responded to previous treatment (refractory). Leflunomide may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Detailed description
PRIMARY OBJECTIVES: I. To determine the maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of leflunomide, when given as a single agent. (Phase I) II. To assess the safety and tolerability of leflunomide at each dose level by evaluation of toxicities including: type, frequency, severity, attribution, time course and duration. (Phase I) III. To evaluate the anti-myeloma activity of leflunomide, when given as a single agent, as assessed by overall response rate (ORR). (Phase II)
SECONDARY OBJECTIVES: I. To obtain estimates of: response duration, clinical benefit response, overall survival, progression-free survival. (Phase II)
TERTIARY OBJECTIVES: I. To characterize the relationship between serum concentration of the active leflunomide metabolite, teriflunomide (A77 1726), and toxicity. (Phase I/II) II. To assess the relationship between serum concentration of the active leflunomide metabolite, teriflunomide (A77 1726), and disease response. (Phase I/II) III. To explore the relationship between polymorphisms in the CYP1A2, CYP2C19, or DHODH genes and toxicity/response. (Phase I/II) IV. To explore the ex vivo cytotoxicity of leflunomide toward primary multiple myeloma (MM) cells, in order to evaluate whether individual ex vivo leflunomide response might be a useful predictor of therapeutic response. (Phase I/II) V. To explore the potential additive or synergistic effects of combining leflunomide with other classes of Food and Drug Administration (FDA)-approved drugs. (Phase I/II) VI. To generate a preliminary ribonucleic acid (RNA)/microRNA (miRNA) and deoxyribonucleic acid (DNA) methylation signature associated with response of MM cells to leflunomide in vivo (mRNA/miRNA and DNA methylation, phase II only) and teriflunomide ex vivo (messenger RNA [mRNA]/miRNA). (Phase I/II)
OUTLINE: This is a dose-escalation study. Patients receive leflunomide orally (PO) once daily (QD) on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 30 days and then every 28 days until disease progression (active follow-up) or every 3 months (long term follow-up).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- The phase I portion will implement a modified rolling six dose escalation design, a more conservative version of the rolling six design of Skolnik, et al., for enrollment with dose escalation, de-escalation, or expansion of a cohort on the basis of the occurrence of dose limiting toxicities (DLTs) during cycle 1. The starting dose of leflunomide is 20mg daily, with the intention to test up to 60mg daily. Escalation will proceed in increments of 20mg/daily; de-escalation will proceed in decrements of 10mg/day. Leglunomide is administered with a loading dose of 100 mg for the 1st three doses.
| Arm | Type | Receives |
|---|---|---|
| Arm 1: 20 mg leflunomide | Experimental | Other: Laboratory Biomarker Analysis, Drug: Leflunomide, Other: Pharmacological StudyPatients receive 20 mg leflunomide PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. |
| Arm 2: 40 mg leflunomide | Experimental | Other: Laboratory Biomarker Analysis, Drug: Leflunomide, Other: Pharmacological StudyPatients receive 40 mg leflunomide PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. |
| Arm 3: 60 mg leflunomide | Experimental | Other: Laboratory Biomarker Analysis, Drug: Leflunomide, Other: Pharmacological StudyPatients receive 60 mg leflunomide PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- All subjects must have the ability to understand and the willingness to sign a written informed consent
- Patients must have a life expectancy of > 3 months
- Patients must exhibit an Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
- Patients must have a diagnosis of multiple myeloma
- Serum M-protein >= 0.5 g/dL
Key exclusion criteria (18 listed)
- Prior treatment with leflunomide
- Current or planned use of other investigational agents, or concurrent biological, chemotherapy, or radiation therapy during the study treatment period
- Current or planned growth factor or transfusion support until after initiation of treatment; if growth factor or transfusion support is provided between screening and start of treatment, the participant will no longer be eligible
- Prior diagnosis of rheumatoid arthritis
- Prior allogenic transplant
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 |
|---|---|---|
| Laboratory Biomarker AnalysisCorrelative studies | Other | Arm 1: 20 mg leflunomide, Arm 2: 40 mg leflunomide, Arm 3: 60 mg leflunomide |
| LeflunomideAlso: Arava, SU101Given PO | Drug | Arm 1: 20 mg leflunomide, Arm 2: 40 mg leflunomide, Arm 3: 60 mg leflunomide |
| Pharmacological StudyCorrelative studies | Other | Arm 1: 20 mg leflunomide, Arm 2: 40 mg leflunomide, Arm 3: 60 mg leflunomide |
Primary outcome
MTD, Defined as the Highest Dose in Which =< 1/6 Patients Experience a Dose-limiting Toxicity, Graded According to the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.03
Time frame: 28 days
Observed toxicities will be summarized, for all dose levels, in terms of type (organ affected or laboratory determination), severity, time of onset, duration, serum concentration of the active leflunomide metabolite, probable association with the study treatment and reversibility or outcome.
Best Overall Response Rate: Proportion of Patients Reaching CR by IMWG Criteria
Time frame: From the start of treatment until disease progression/recurrence, assessed up to 48 months
Stringent complete response [sCR]/complete response [CR]/very good partial response [VGPR]/or partial response [PR]), assessed by International Myeloma Working Group (IMWG) criteria.
3 secondary outcomes
Clinical Benefit Response Rate (sCR/CR/VGPR/Partial Response [PR]/Minimal Response [MR] or Stable Disease [SD]), Assessed by IMWG Criteria
Time frame: From the start of treatment until disease progression/recurrence, assessed up to 48 months
Response Duration
Time frame: Assessed up to 48 months
Response Duration
Time frame: Assessed at ninety days.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 32268821Rosenzweig M, Palmer J, Tsai NC, Synold T, Wu X, Tao S, Hammond SN, Buettner R, Duarte L, Htut M, Karanes C, Nathwani N, Pichiorri F, Sahebi F, Sanchez JF, Chowdhury A, Krishnan A, Forman SJ, Rosen ST. Repurposing leflunomide for relapsed/refractory multiple myeloma: a phase 1 study. Leuk Lymphoma. 2020 Jul;61(7):1669-1677. doi: 10.1080/10428194.2020.1742900. Epub 2020 Apr 8.
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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.
Michael Rosenzweig principal investigator
City of Hope Medical Center
1 site 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 Laboratory Biomarker Analysis, Leflunomide and Pharmacological Study in people with Recurrent Plasma Cell Myeloma and Refractory Plasma Cell Myeloma, run by City of Hope Medical Center at 1 site 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 20 inclusion and 18 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 10 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Yes. Results are posted in the registry (first posted 10 Oct 2023). 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?
MTD, Defined as the Highest Dose in Which =< 1/6 Patients Experience a Dose-limiting Toxicity, Graded According to the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.03 (time frame: 28 days). 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 NCT02509052, 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 10 Oct 2023.