Clinical Trial: A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis

Brief Summary:

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.


Detailed Summary:

Primary objective:

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

Secondary objectives:

Determine the following at the MTD:

  • Hematological complete (CR) very good partial (VGPR) and partial (PR) rates
  • duration of response
  • organ response
  • Time-to-event
  • Survival

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


Sponsor: Vaishali Sanchorawala

Current Primary Outcome: Determining dose-limiting toxicity and maximal tolerated dosage [ Time Frame: one month ]

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


Original Primary Outcome: Same as current

Current Secondary Outcome: Response to the maximal tolerated dose [ Time Frame: one year ]

Patient response to treatment


Original Secondary Outcome: Same as current

Information By: Boston Medical Center

Dates:
Date Received: February 27, 2012
Date Started: June 2012
Date Completion: February 2030
Last Updated: January 3, 2017
Last Verified: January 2017