This research study is evaluating a drug called idelalisib (formerly known as GS-1101 or CAL-101) as a possible treatment for Waldenstrom's Macroglobulinemia (WM).
This research study is a Phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational drug, idelalisib, to learn whether idelalisib works in treating a specific cancer. "Investigational" means that idelalisib is still being studied and that research doctors are trying to find out more about it-such as the safest dose to use, the side effects it may cause, and if idelalisib is effective for treating different types of cancer. Idelalisib has already been approved in the US by the FDA to treat patients with relapsed chronic lymphocytic leukemia, follicular lymphoma and small lymphocytic lymphoma. Idelalisib is a newly discovered drug that is being developed as an anti-cancer agent. This drug has been used in laboratory experiments and other research studies in B-cell malignancies and information from those other research studies suggests that idelalisib may help to target the tumor cells in B-cell malignancies, including WM. B cells are a type of white blood cell responsible for making antibodies. In this research study, the investigators are testing the safety and efficacy of idelalisib as a treatment option for relapsed or refractory Waldenstrom's Macroglobulinemia.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Oral twice daily for 6 months followed by once daily until disease progression or unacceptable toxicity.
Dana Farber Cancer Institute
Boston, Massachusetts, United States
Overall Response Rate (ORR)
ORR measured by decrease in serum IgM level by at least 25% from baseline.
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Percentage of Participants With Adverse Events
Assess the safety and tolerability of idelalisib
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Complete Response (CR)
CR measured by decrease in serum IgM levels to normal range, disappearnace of monoclonal protein by immunofixation, no evidence of bone marrow involvement, and resolution of any extramedullary disease by CT scan.
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Very Good Partial Response (VGPR)
VGPR measured by decrease in serum IgM levels of at least 90% from baseline.
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Partial Response (PR)
PR measured by decrease in serum IgM levels of between 25% and 50% from baseline.
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Minimal Response
Minimal response measured by decrease in serum IgM levels of between 25% and 50%.
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Rate of Stable Disease
Stable disease measured by serum IgM levels \<25% reduced from baseline.
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Progressive Disease
Progressive disease measured by an 25% increase in serum IgM level with an absolute increase of at least 500mg/dL from the lowest attained IgM on therapy.
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.