A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis
This study seeks to enroll patients with AL amyloidosis, for whom treatment with one of the standard melphalan chemotherapy-based regimens is either not recommended or is not their preference.
Pomalidomide (CC-4047) is a drug given by mouth, which can change or regulate the functioning of the immune system. So, in theory, it may reduce or prevent the production of the amyloid protein. Pomalidomide is not currently FDA-approved for AL Amyloidosis. Pomalidomide is chemically similar to thalidomide and lenalidomide, both of these drugs have been approved by the FDA for treatment of patients with multiple myeloma (MM), a disease similar to AL Amyloidosis.
Participants in this study will receive pomalidomide and dexamethasone. Phase I is a dose-escalation study and dose escalation will proceed through 3 dose-levels according to standard rules in which dose levels are started sequentially after complete evaluation of the occurrence of dose-limiting toxicities. In the Phase II portion, participants will receive pomalidomide and dexamethasone using the defined maximum tolerated dose.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis|
- Determining dose-limiting toxicity and maximal tolerated dosage [ Time Frame: one month ] [ Designated as safety issue: Yes ]Determine dose-limiting toxicity (DLT) and the maximal tolerated dose (MTD) of pomalidomide combined with dexamethasone in subjects with previously- treated light-chain (AL)-amyloido
- Response to the maximal tolerated dose [ Time Frame: one year ] [ Designated as safety issue: Yes ]Patient response to treatment
|Study Start Date:||June 2012|
|Estimated Study Completion Date:||February 2030|
|Estimated Primary Completion Date:||October 2015 (Final data collection date for primary outcome measure)|
Experimental: all patients on study
All patients enrolled at a specific dose level will receive the same treatment. There will be no randomization and no placebos.
Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-28Drug: Dexamthasone
10-20 mg on days 1, 8, 15, and 22
Determine dose-limiting toxicity (DLT) and the maximal tolerated dose (MTD) of pomalidomide combined with dexamethasone in subjects with previously- treated light-chain (AL)-amyloidosis
Determine the following at the MTD:
- Hematological complete (CR) very good partial (VGPR) and partial (PR) rates
- duration of response
- organ response
Exploratory study objective:
To investigate the relationship of changes in the levels of the biomarkers BNP and troponin I to frequency of specific adverse events and the occurrence of DLT
Please refer to this study by its ClinicalTrials.gov identifier: NCT01570387
|Contact: Sally Fennesseyfirstname.lastname@example.org|
|United States, Massachusetts|
|Boston Medical Center||Recruiting|
|Boston, Massachusetts, United States, 02118|
|Contact: Sally Fennessey 617-638-8265 email@example.com|
|Principal Investigator:||Vaishali Sanchorawala, MD||Boston Medical Center|