This study is being done to learn more about the drug, pomalidomide and to gather data on its safety and side effects when used in combination with commercially available cyclophosphamide and dexamethasone. This combination is considered experimental and has not been approved by the FDA. Pomalidomide is a third generation immunomodulatory (IMiDs) agent, which is a more potent version of thalidomide and lenalidomide drugs that have been approved by the United States Food and Drug Administration \[FDA\] for the treatment of MM. In February 2013, pomalidomide was also approved by the FDA for patients with MM who have had more than 2 types of therapy. Pomalidomide is taken orally as capsules, and cyclophosphamide and dexamethasone are also taken orally as tablets in this study. Cyclophosphamide and dexamethasone are commercially available and are often used in combination with other drugs to treat Multiple Myeloma. Preliminary data from both the laboratory and patient studies suggest that this combination of drugs is more effective than pomalidomide and dexamethasone alone. However, the regimen being used in this study, which consists of daily cyclophosphamide, also permits support of low blood counts with either injections or transfusions as needed.
This is an open label, single center, phase II study of a combination of pomalidomide, daily low dose oral cyclophosphamide, and dexamethasone in patients with relapsed/refractory multiple myeloma. The three oral drugs will be given in 28-day cycles: Pomalidomide 4 mg daily x 21 days; cyclophosphamide 50 mg BID x 21 days; and dexamethasone 40 mg weekly x 3 (20 mg weekly if the patient aged ≥ 75 years old). Subjects meeting eligibility criteria with ANC \< 1000/µL and platelet count \< 50,000/µL will start at dose level -1 of both pomalidomide (3 mg daily) and cyclophosphamide (50 mg daily). G-CSF and platelet transfusion support is permitted if needed. Dose reduction for hematologic toxicity will begin with cyclophosphamide and then subsequently alternate with pomalidomide until a subject cannot tolerate dose level -2 of both agents - in which case subject would come off study. If subject has 2 or more concurrent toxicities that are potentially attributable to both agents (e.g. hematologic toxicity) then dose modification guidelines will be followed with dose reduction being done sequentially with one agent at a time, unless in the opinion of the investigator, both agents required concurrent dose reduction.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
35
4 mg PO x 21 days
low dose cyclophosphamide 50 mg PO BID x 21 days
dexamethasone 40 mg PO weekly (or 20 mg if ≥ 75 years old).
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Best overall response rate (ORR)
disease response
Time frame: up to 24 months
Stringent complete response (sCR)
Time frame: up to 24 months
Complete response (CR)
Time frame: up to 24 months
Very good partial response (VGPR)
Time frame: up to 24 months
Partial response (PR)
Time frame: up to 24 months
Time to progression (TTP)
TTP is defined as beginning with the time the first dose of PCD regimen is administered until disease progression on PCD regimen.
Time frame: up to 24 months
Duration of response (DOR)
DOR is defined as the time from first evidence of PR or better to confirmation of disease progression.
Time frame: up to 24 months
Clinical benefit response (CBR)
CBR is the combination of the ORR and minimal response (MR) i.e. this includes sCR, CR, VGPR, PR, and MR.
Time frame: up to 24 months
Progression free survival (PFS)
PFS is defined as the duration of time from start of treatment to the first occurrence of disease progression or death on study from any cause, whichever occurs earlier.
Time frame: up to 24 months
Overall survival (OS)
OS is defined as the time from the first dose of study treatment to the date of death (whatever the cause).
Time frame: up to 24 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.