Clinical trial NCT00142116: Thalidomide and Rituximab in Waldenstrom's Macroglobulinemia
Official title: Phase II Study of Thalidomide and Rituximab in Waldenstrom's Macroglobulinemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Waldenstrom's Macroglobulinemia
Lymphoplasmacytic Lymphoma
Keywords: thalidomide
rituximab
Waldenstrom's
Lead sponsor: Dana-Farber Cancer Institute
Sponsor class: OTHER
Collaborators: name: Dana-Farber Cancer Institute
name: Massachusetts General Hospital
name: Beth Israel Deaconess Medical Center
name: Brigham and Women's Hospital
name: Cape Cod Hospital
First posted: 2005-09-02
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the percentage of people who can attain remission and the length of time such responses to therapy are sustained, as well as the side effects that might result from rituximab and thalidomide in people with lymphoplasmacytic lymphoma.

Detailed description: * Patients will receive thalidomide(200mg) orally once daily for two weeks. If after two weeks of thalidomide, the patient is doing well the dose of thalidomide will increase (400mg) and they will remain on it for up to 50 additional weeks. The length of time a patient is on thalidomide will depend upon how they are responding to therapy.
* During the second week of the study patients will also begin receiving rituximab intravenously once weekly for 4 weeks, which may then be repeated 8 weeks later depending upon the response.
* A determination of how the patient is responding will be made based on testing conducted at 12 weeks. This testing includes blood tests and possibly a bone marrow biopsy. If it is determined that the disease is not progressing, patients will begin a second phase of treatment which includes 4 additional weekly infusions of rituximab and the continuation of oral thalidomide.
* If it is determined at the 12-week evaluation, or at any time thereafter, that the disease has progressed (by studying serum immunoglobulin M (IgM) levels, bone marrow involvement, tumor cells, and/or development of new signs and symptoms) then the patient will be removed from the study.
* Periodic examinations and tests will be done to determine how the patient is doing, what response and side effects (if any) the patient may be having from the study drugs. If patient is responding to therapy then they will remain on this study and followed for a period of two years.
* Bone marrow biopsies and aspirations will be obtained at 3-6 month intervals extending for 2 years following the last treatment.

Eligibility criteria: Inclusion Criteria:

* Clinicopathological diagnosis of Waldenstrom's macroglobulinemia requiring therapy
* Baseline staging requirements
* Absolute Neutrophil Count \> 500/microliter (uL)
* Platelet Count \> 25,000/uL
* Serum creatinine \< 2.5mg/dL
* Total bilirubin and transaminase (SGOT) \< 2.5 X Upper Limit of Normal (ULN)
* Greater than 18 years of age
* Life expectancy of 3 months or greater
* Eastern Cooperative Oncology Group (ECOG) status performance of 0-2

Exclusion Criteria:

* Chemotherapy, steroid therapy, or radiation therapy within 30 days of study entry
* Pregnant or lactating women
* Serious co-morbid disease
* Uncontrolled bacterial, fungal or viral infection
* Active second malignancy

Primary outcomes:
measure: Objective Response Rate
timeFrame: 3 years
measure: Time to Progression
timeFrame: 49.1 months
Secondary outcomes:
measure: To Identify the Mechanism(s) of Action for Combined Thalidomide and Rituximab Activity.
timeFrame: 3 years
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
Study officials:
affiliation: Dana-Farber Cancer Institute
name: Steven P. Treon, MD, MA, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Treon SP, Soumerai JD, Branagan AR, Hunter ZR, Patterson CJ, Ioakimidis L, Briccetti FM, Pasmantier M, Zimbler H, Cooper RB, Moore M, Hill J 2nd, Rauch A, Garbo L, Chu L, Chua C, Nantel SH, Lovett DR, Boedeker H, Sonneborn H, Howard J, Musto P, Ciccarelli BT, Hatjiharissi E, Anderson KC. Thalidomide and rituximab in Waldenstrom macroglobulinemia. Blood. 2008 Dec 1;112(12):4452-7. doi: 10.1182/blood-2008-04-150854. Epub 2008 Aug 19.