Leflunomide for the Treatment of High-Risk Smoldering Multiple Myeloma
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|ClinicalTrials.gov Identifier: NCT04370483|
Recruitment Status : Active, not recruiting
First Posted : May 1, 2020
Last Update Posted : August 12, 2021
|Condition or disease||Intervention/treatment||Phase|
|Smoldering Plasma Cell Myeloma||Drug: Leflunomide Other: Quality-of-Life Assessment||Early Phase 1|
I. To estimate the anti-myeloma activity of leflunomide, when given as a single agent, as assessed by 6-month progression-free response rate based on International Myeloma Working Group (IMWG) criteria.
I. To evaluate the safety and tolerability of single agent leflunomide. II. To summarize and assess toxicities by type, frequency, severity, attribution, time course and duration.
III. To estimate overall and progression-free survival probabilities. IV. To estimate response rate and duration of response. V. To describe the impact of treatment on quality of life, as assessed by the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) Score version (v)3.0.
I. To characterize the molecular evolution of the tumor cells. II. To evaluate whether specific genetic subtypes respond differently to leflunomide.
III. To evaluate the role of immune cells in the progression of smoldering multiple myeloma (SMM).
IV. To evaluate the role of leflunomide in modulating the immune system. V. To examine the relationship between immunological changes and disease progression.
Patients receive leflunomide orally (PO) once daily (QD). Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After the completion of study treatment, patients are followed up at 30 days, every 28 days until an alternative myeloma therapy has commenced or until disease progression, and then up to 6 months.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||1 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pilot Trial of Leflunomide in Patients With High-Risk Smoldering Multiple Myeloma|
|Actual Study Start Date :||October 8, 2020|
|Estimated Primary Completion Date :||June 1, 2023|
|Estimated Study Completion Date :||June 1, 2023|
Experimental: Treatment (leflunomide)
Patients receive leflunomide PO QD. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Other: Quality-of-Life Assessment
Other Name: Quality of Life Assessment
- Progressive disease [ Time Frame: Up to 48 months ]Will be defined by International Myeloma Working Group (IMWG) criteria. Progression to overt multiple myeloma is always considered progressive disease.
- Incidence of adverse events [ Time Frame: Up to 30 days after end of treatment ]Will be defined using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5 to grade toxicities. Observed toxicities will be summarized in terms of type (organ affected or laboratory determination), severity, time of onset, duration, probable association with the study treatment and reversibility or outcome.
- Incidence of toxicities [ Time Frame: Up to 30 days after the end of treatment ]Will be defined using the NCI CTCAE version 5 to grade toxicities. Will assess toxicities by type, frequency, severity, attribution, time course and duration. Observed toxicities will be summarized in terms of type (organ affected or laboratory determination), severity, time of onset, duration, probable association with the study treatment and reversibility or outcome.
- Overall response rate [ Time Frame: From the date of first documented response (confirmed complete response [CR], very good partial response [VGPR], partial response [PR] or minor response [MR]) to documented disease relapse, progression or death, assessed up to 48 months ]The overall response rate and 95% Clopper Pearson binomial confidence interval (CI) will be calculated. Response rates will also be explored based on number/type of prior therapy(ies). Response rate (CR, VGPR, PR, or MR), based on the IMWG 2016 criteria will be calculated as the percent of evaluable patients that have confirmed CR/VGPR/PR or MR.
- Overall survival [ Time Frame: From start date of therapy to date of death from any cause, assessed up to 48 months ]Overall survival will be estimated using the product-limit method of Kaplan and Meier.
- Quality of life Questionnaire [ Time Frame: At baseline and every 6 cycles thereafter up to 36 cycles (end of treatment), length of one complete cycle is 28 days ]The Quality of Life Questionnaire Core 30 (QLQ-C30) scales (five functional scales, three symptom scales, a global health status / quality of life (QoL) scale, and six single items) will be summarized using descriptive statistics. Changes in reported QOL over time from baseline will also be summarized.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04370483
|United States, California|
|City of Hope Medical Center|
|Duarte, California, United States, 91010|
|Principal Investigator:||Michael A Rosenzweig||City of Hope Medical Center|