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Phase II Study of Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia

Phase 2
18 Years
Open (Enrolling)
Waldenstrom's Macroglobulinemia

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Trial Information

Phase II Study of Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia

- Participants will take RAD001 orally once a day in the morning. Each treatment cycle
lasts for four weeks. Participants will receive up to 48 cycles of treatment.

- During each cycle, participants will be asked to visit the clinic for scheduled tests
and exams. They will visit the clinic on the first day of each of the first three
cycles, and then once every 3 cycles. During the visits, participants will have a
physical exam and blood tests.. Participants may also have CT scans of the chest,
abdomen and pelvis as well as a bone marrow aspirate and biopsy.

Inclusion Criteria:

- 18 years of age or older

- Adequate liver and renal function as outlined in the protocol

- Fasting serum cholesterol 300mg/dl or less OR 7.75mmol/L or less AND fasting
triglycerides 2.5 x institutional ULN or less.

- Clinicopathological diagnosis of Waldenstrom's macroglobulinemia as defined by
consensus panel of the Second International Workshop on Waldenstrom's

- No previous therapy for WM

- Measurable disease, defined as presence of immunoglobulin M (IgM) paraprotein with a
minimum IgM level of 2 times the upper limit of each institution's normal value or
greater is required

- ECOG Performance status of 0-2

- Patients must have a life expectancy of at least 3 months

- Baseline platelet and absolute neutrophil as outlined in the protocol

- INR and PTT 1.5 x normalized ratio or less

- A male subject agrees to use an acceptable method for contraception for the duration
of study and for 8 weeks after the last dose of the study drug

- Female subject either post-menopausal or surgically sterilized or willing to use
acceptable methods of birth control for the duration of the study and for 8 weeks
after the last dose of study drug

Exclusion Criteria:

- Patients experiencing symptomatic hyperviscosity and requiring plasmapheresis. This
includes any patient who, in the judgement of the investigator requires urgent
response and will not be eligible. These patients have hyperviscosity which includes
serum IgM levels of 5000 mg/dL or greater. Symptoms may include nosebleeds, visual
complications, fatigue, headaches, confusion, etc.

- Patients, who have had a major surgery or significant traumatic injury within 4 weeks
of start of study drug, patients who have not recovered from the side effects of any
major surgery or patients that may require major surgery during the course of the

- Patients receiving chronic, systemic treatment with corticosteroids or another
immunosuppressive agent. Topical or inhaled corticosteroids are allowed.

- Patients should not receive any immunization with attenuated live vaccines within one
week of study entry or during study period.

- Patients who have had any severe and/or uncontrolled medical conditions or other
conditions that would affect their participation in the study.

- Impairment of gastrointestinal function or gastrointestinal disease that may
significantly alter the absorption of RAD001.

- Female patients that are pregnant or breast feeding, or adults of reproductive
potential who are not using effective birth control methods.

- Patients with known hypersensitivity to RAD001 or other rapamycins or to its

- Patients with other malignancies within the past 3 years except for adequately
treated carcinoma of the cervix or basal or squamous cell of the skin

- Patients with known history of HIV seropositivity

- History of noncompliance to medical regimens

- Patients unwilling to or unable to comply with the protocol

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To assess the overall response and major response rates of RAD001 in patients with previously untreated WM.

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Steven Treon, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Dana-Farber Cancer Institute


United States: Institutional Review Board

Study ID:




Start Date:

November 2009

Completion Date:

December 2013

Related Keywords:

  • Waldenstrom's Macroglobulinemia
  • everolimus
  • RAD001
  • WM
  • Waldenstrom Macroglobulinemia



Dana-Farber Cancer Institute Boston, Massachusetts  02115