Pomalidomide With Melphalan and Dexamethasone for Untreated Systemic AL Amyloidosis
The goal of this clinical research study is to find the highest tolerable dose of pomalidomide that can be given in combination with melphalan and dexamethasone that can be given to patients with AL amyloidosis. The safety of this drug combination will also be studied.
Pomalidomide is designed to change the body's immune system. It may also interfere with the development of tiny blood vessels that help support tumor growth. This may decrease the growth of cancer cells.
Melphalan is designed to damage the DNA (genetic material) of cells, which may cause cancer cells to die.
Dexamethasone is a corticosteroid that is similar to a natural hormone made by your body. Dexamethasone is often given to Multiple Myeloma (MM) patients in combination with other chemotherapy to treat cancer.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I/II Clinical Trial of Pomalidomide With Melphalan and Dexamethasone in Patients With Newly Diagnosed Untreated Systemic AL Amyloidosis|
- Maximum Tolerated Dose (MTD) of Pomalidomide with Melphalan and Dexamethasone (PMD). [ Time Frame: 28 days ] [ Designated as safety issue: Yes ]Maximum tolerated dose defined as highest dose level at which less than 33% of patients experienced dose-limiting toxicities. Dose-limiting toxicities defined as (grade 4 neutropenia lasting more than 7 days despite G-CSF administration, any other grade 4 hematologic toxicity, any grade 3 non-hematologic toxicity, or a new cycle delay beyond a maximum of 4 weeks) in less than 33% of patients during the first cycle of therapy.
- Complete Response (CR) Rate [ Time Frame: 28 days ] [ Designated as safety issue: No ]Complete response rate defined using the consensus opinion from the 10th International Symposium on Amyloid and Amyloidosis. Student t-test/Wilcoxon rank sum test and ANOVA/Kruskal-Wallis test, or chi-square test /Fisher's exact test used to test association between response and prognostic factors. Univariate and multivariate logistic regression models will be fit to identify clinical factors associated with hematologic CR, overall response and organ response rate. Kaplan and Meier product limit methods used to estimate the progression free survival (PFS), duration of response (DOR), and TTNT with 95% confidence intervals.
|Study Start Date:||February 2014|
|Estimated Primary Completion Date:||February 2020 (Final data collection date for primary outcome measure)|
Experimental: Pomalidomide + Melphalan + Dexamethasone
Starting dose of Pomalidomide 1 mg/day by mouth on days 1-21. Melphalan 9 mg/m2 by mouth on days 1-4 of every 28-day cycle. Dexamethasone 40 mg/day by mouth on days 1-4. Questionnaires completed at different time points during study.
Phase I: Starting dose of Pomalidomide 1 mg/day by mouth on days 1-21 of a 28 day cycle.
Phase II: Starting dose of Pomalidomide maximum tolerated dose from Phase I.
Phase I and II: 9 mg/m2 by mouth on days 1-4 of a 28-day cycle.
Other Name: AlkeranDrug: Dexamethasone
Phase I and II: 40 mg/day by mouth on days 1-4 of a 28 day cycle.
Other Name: DecadronBehavioral: Questionnaires
Questionnaires about the feeling in hands and quality of life completed at different time points during the study.
Other Name: Surveys
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|Contact: Robert Orlowski, MD, PHD||713-792-2860|
|United States, Massachusetts|
|Tufts Medical Center||Not yet recruiting|
|Boston, Massachusetts, United States, 02111|
|United States, Texas|
|UT MD Anderson Cancer Center||Not yet recruiting|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Robert Orlowski, MD, PHD||UT MD Anderson Cancer Center|