A Pilot Study of Infusional Cyclophosphamide and Pulse Dexamethasone With Rapamycin or Hydroxychloroquine in Patients With Relapsed or Refractory Multiple Myeloma
Multiple myeloma is a plasma cell neoplasm with median survival of 3 to 5 years. Recent
advances have improved patient outlook, but the disease remains incurable and patients
become refractory to treatments, develop organ dysfunction from myeloma itself, and
encounter severe toxicities from therapies, limiting further treatment options. Subjects
over the age of 18 with relapsed or refractory multiple myeloma who have had at least one
prior therapy, have a life expectancy of at least 4 weeks and an ECOG performance status of
0,1 or 2 will be included. The investigators propose an initial pilot trial to test the
feasibility of giving standard chemotherapy with infusional cyclophosphamide and pulse
dexamethasone (cy/dex) for four days, together with the investigational agents, oral
rapamycin (cohort A), and in a separate parallel arm, oral hydroxychloroquine (HCQ) (cohort
B). The investigators will enroll 3 patients on cohort A and 3 patients on cohort B, both
at the planned 1st dose level for the overall phase I/II trial which will succeed this trial
if feasibility is demonstrated. The 6 patients from this pilot trial will be included in
total number of patients for the overall study which will be completed when further funding
is secured. Feasibility will be defined by the ability to deliver the regimen as planned
and the ability to perform the planned correlative studies. Subjects will receive up to 12
cycles and will be followed for 12 months after the last cycle. Data collected will include
toxicity, response rates, correlative studies to examine the pharmacodynamics of mTOR
inhibition and autophagy and the pharmacokinetics of rapamycin and HCQ.
Interventional
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Primary Purpose: Treatment
Number of Participants with Adverse Events
The feasibility of administering rapamycin and hydroxychloroquine (HCQ), with standard doses of infusional cyclophosphamide and pulse dexamethasone (cy/dex) for patients with relapsed/refractory multiple myeloma.
Yes
Dan Vogl, MD
Principal Investigator
Abramson Cancer Center of the University of Pennsylvania
United States: Institutional Review Board
UPCC 09411
NCT01396200
June 2011
October 2012
Name | Location |
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Abramson Cancer Center of the University of Pennsylvania | Philadelphia, Pennsylvania 19104-4283 |