S0629, Observation or Combination Chemotherapy, Bortezomib, Thalidomide, and Rituximab Followed By Two Autologous Peripheral Blood Stem Cell Transplants in Treating Patients With Waldenstrom Macroglobulinemia

NCT ID: NCT00723658

Last Updated: 2015-03-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2008-09-30

Brief Summary

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RATIONALE: Sometimes the cancer may not need treatment until it progresses. In this case, observation may be sufficient. Giving combination chemotherapy together with bortezomib, thalidomide, and rituximab before an autologous peripheral stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as G-CSF, helps stem cells move from the bone marrow to the blood so they can be collected and stored. More chemotherapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

PURPOSE: This observational and phase II trial is studying how well giving combination chemotherapy together with bortezomib, thalidomide, and rituximab followed by two autologous peripheral blood stem cell transplants works in treating patients with Waldenstrom macroglobulinemia.

Detailed Description

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OBJECTIVES:

Primary

* To assess the progression-free and overall survival of patients with symptomatic Waldenstrom macroglobulinemia treated with bortezomib, dexamethasone, thalidomide, cisplatin, doxorubicin hydrochloride, cyclophosphamide, and etoposide (VDT-PACE) in combination with rituximab, followed by single or tandem autologous peripheral blood stem cell transplantation and maintenance therapy.
* To assess the confirmed and unconfirmed response in patients treated with this regimen.

Secondary

* To evaluate the feasibility and toxicity of this regimen in these patients.
* To correlate the time to symptom development and overall survival with standard prognostic factors and cytopenias.
* To examine the natural history of Waldenstrom macroglobulinemia.
* To identify, in a preliminary fashion, biological correlates that may relate to progression or to symptomatic disease.

OUTLINE: This is a multicenter study. Patients with asymptomatic disease at study entry proceed directly to observation. Patients with symptomatic disease at study entry proceed directly to induction therapy.

* Observation: Patients with asymptomatic disease undergo observation monthly for 3 months and then every 3 months for up to 3 years. Patients who develop symptomatic disease proceed to induction therapy within 28 days of onset of disease symptoms. Patients who continue to have asymptomatic disease after 3 years of observation are removed from the study.
* Induction therapy: Patients receive oral dexamethasone and oral thalidomide on days 1-4; cisplatin IV, doxorubicin hydrochloride IV, cyclophosphamide IV, and etoposide IV continuously on days 1-4; bortezomib IV on days 1, 4, 8, and 11; and rituximab IV on days 1, 8, and 15. Treatment repeats every 6-8 weeks for 2 courses in the absence of disease progression or unacceptable toxicity.
* Peripheral blood stem cell (PBSC) collection: Patients receive filgrastim (G-CSF) IV beginning on day 9 of course 1 of induction therapy and continuing until WBC counts are adequate for apheresis. Patients also receive G-CSF IV beginning on day 6 of course 2 of induction therapy and continuing until apheresis is complete.
* First autologous PBSC transplantation\*: Beginning approximately 4-6 weeks after the completion of induction therapy, patients receive conditioning therapy comprising high-dose melphalan IV and bortezomib IV on days -4 and -1. Patients undergo autologous PBSC transplantation on day 0 NOTE: \*Patients who will receive a single transplant (for medical, insurance, or other reasons) will not receive melphalan and bortezomib, but will receive conditioning with carmustine, etoposide, cytarabine, and melphalan (BEAM) and will proceed to Maintenance Therapy.
* Second autologous PBSC transplantation: Beginning approximately 56-90 days after the first transplant, patients receive conditioning therapy comprising carmustine IV over 2 hours on day -5; etoposide IV over 1 hour and cytarabine IV over 1 hour on days -5 to -2; and melphalan IV on day -1. Patients undergo autologous PBSC transplantation on day 0.
* Maintenance therapy: Beginning after platelet counts recover, patients receive bortezomib IV on days 1, 4, 8, and 11 and rituximab IV over 2 hours on day 11. Treatment repeats every 3 months for 2 years in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed every 6 months for up to 5 years.

Conditions

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Lymphoma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Observation

Non-symptomatic patients are monitored monthly for 3 months, then every 3 months thereafter.

Group Type NO_INTERVENTION

No interventions assigned to this group

Treatment

Symptomatic pts: 2 cycles VTDPACE+R:

dex 40 mg PO D1-4 thalid 200 mg PO D1-4 cisplatin 10 mg/m2 IV D1-4 dox 10 mg/m2 IV D1-4 cyclophos 400 mg/m2 IV D1-4 etoposide 40 mg/m2 IV D1-4 bortezomib 1.0 mg/m2 IV D1,4,8,11 ritux 375 mg/m2 IV D1,8,15 lovenox 40 mg/d SQ D1-platelets \>50,000/mcl GCSF 10 mcg/kg/d IV D9-WBC \<2,000/mcl apheresis \>/= 20x10\^6 when WBC and CD34 within normal range, up to 4 cycles

1. st Trans: mel 200 mg/m2 IV D-1 bortezomib 1.3 mg/m2 IV D-4, -1 PBSC \>/=3.0x10\^6/kg CD34 cells IV D0 GCSF 5 mcg/kg/d SQ D6-WBC recovery D5NS 500 ml IV D-1 dex 40 mg/d PO D-4 to -1 ondansetron 24 mg OR granisetron 2 mg PO D-1
2. nd Trans: BCNU 300 mg/m2 IV D-5 etoposide 200 mg/m2 IV D-5 to -2 AraC 400 mg/m2 IV D-5 to -2 mel 140 mg/m2 IV D-1 PBSC infusion \>/=3.0x10\^6/kg CD34 cells IV D0 GCSF 5 mcg/kg/d SQ D6-WBC recovery D5NS 150 ml/hr IV D-5 to -1 dex 40 mg/d PO D-4 to -1 ondansetron 24 mg OR granisetron 2 mg PO D-1

Group Type EXPERIMENTAL

rituximab

Intervention Type BIOLOGICAL

bortezomib

Intervention Type DRUG

carmustine

Intervention Type DRUG

cisplatin

Intervention Type DRUG

cyclophosphamide

Intervention Type DRUG

cytarabine

Intervention Type DRUG

dexamethasone

Intervention Type DRUG

doxorubicin hydrochloride

Intervention Type DRUG

etoposide

Intervention Type DRUG

melphalan

Intervention Type DRUG

thalidomide

Intervention Type DRUG

autologous-autologous tandem hematopoietic stem cell transplantation

Intervention Type PROCEDURE

peripheral blood stem cell transplantation

Intervention Type PROCEDURE

Interventions

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rituximab

Intervention Type BIOLOGICAL

bortezomib

Intervention Type DRUG

carmustine

Intervention Type DRUG

cisplatin

Intervention Type DRUG

cyclophosphamide

Intervention Type DRUG

cytarabine

Intervention Type DRUG

dexamethasone

Intervention Type DRUG

doxorubicin hydrochloride

Intervention Type DRUG

etoposide

Intervention Type DRUG

melphalan

Intervention Type DRUG

thalidomide

Intervention Type DRUG

autologous-autologous tandem hematopoietic stem cell transplantation

Intervention Type PROCEDURE

peripheral blood stem cell transplantation

Intervention Type PROCEDURE

Eligibility Criteria

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Inclusion Criteria

DISEASE CHARACTERISTICS:

* Diagnosis of Waldenstrom macroglobulinemia (WM)
* Measurable disease as determined by IgM protein quantification
* Must be registered to the treatment portion of the study within 28 days of experiencing disease-related symptoms\* AND must present with ≥ 1 of the following disease-related symptoms:

* Hemoglobin ≤ 11 g/dL
* Platelet count ≤ 100,000/mm³
* Marked tumor mass, defined as lymphadenopathy \> 2 cm, palpable hepatomegaly, splenomegaly, or significant marrow involvement (\> 50%)
* Serum albumin \< 2.5 g/dL
* Persistently elevated beta-2-microglobulin \> 3.0 mg/L in the absence of renal impairment or active infections
* Presence of B symptoms (i.e., fever, night sweats, or weight loss of \> 10% from baseline)
* Appearance of new or worsening neuropathy manifested by numbness and tingling or pain
* Symptomatic cryoglobulinemia (i.e., Raynaud phenomenon, skin ulcers, cold urticaria, or skin necrosis)
* Symptoms of hyperviscosity, if measured viscosity \> 4 cp (i.e., new headaches, vertigo, ataxia, dizziness with or without evident causes of changes in funduscopic exam, including retinal vein engorgement, hemorrhages, or exudates)
* NOTE: \*Appearance of any of the above symptoms caused by WM with no other obvious cause is a trigger for treatment initiation. Symptoms need not persist for any specified time frame.

PATIENT CHARACTERISTICS:

* Zubrod performance status 0-2 (Zubrod performance status 3 allowed provided it is based solely on morbidity due to WM)
* ANC \> 1,500/mm³ (unless more marked cytopenias can be explained by marked marrow involvement or autoimmune myelosuppression)
* Serum creatinine \< 3 mg/dL
* Creatinine clearance \> 30 mL/min
* SGOT/SGPT \< 2 times upper limit of normal
* Direct bilirubin \< 2.0 mg/dL
* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception according to the System for Thalidomide Education and Prescribing Safety (S.T.E.P.S.®) program
* Ejection fraction ≥ 50% by ECHO or MUGA scan

* Patients with evidence of amyloidosis (i.e., periorbital perforation, proteinuria not attributable to Bence-Jones protein, unexplained arrhythmias, increased liver function tests, peripheral neuropathy, carpal tunnel syndrome, and/or macroglossia) must have an ECHO, rather than MUGA, performed to evaluate for cardiac amyloidosis (septal thickness, diastolic dysfunction, granular sparkling, or low-voltage QRS complexes)
* No myocardial infarction within the past 6 months
* No unstable angina
* No difficult-to-control congestive heart failure or cardiac arrhythmias
* No uncontrolled hypertension
* No peripheral neuropathy ≥ grade 2
* No history of multi-infarced dementia or multiple strokes
* No known hypersensitivity to boron or mannitol
* No hepatitis B or C positivity
* No HIV positivity
* No other prior malignancy within the past 5 years except for adequately treated basal cell or squamous cell skin cancer or carcinoma in situ of the cervix

PRIOR CONCURRENT THERAPY:

* At least 28 days since prior chemotherapy and/or radiotherapy and recovered
* No prior bortezomib
* No concurrent glucocorticoids unless used to control autoimmune disease associated with WM
* Concurrent participation in the Myeloma Specimen Repository study allowed
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

SWOG Cancer Research Network

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Gordan Srkalovic, MD, PhD

Role: STUDY_CHAIR

Sparrow Regional Cancer Center

Locations

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Saint Francis/Mount Sinai Regional Cancer Center at Saint Francis Hospital and Medical Center

Hartford, Connecticut, United States

Site Status

St. Francis Hospital and Health Centers - Beech Grove Campus

Beech Grove, Indiana, United States

Site Status

Reid Hospital & Health Care Services

Richmond, Indiana, United States

Site Status

Lawrence Memorial Hospital

Lawrence, Kansas, United States

Site Status

Wesley Medical Center

Wichita, Kansas, United States

Site Status

Saint Joseph Mercy Cancer Center

Ann Arbor, Michigan, United States

Site Status

CCOP - Michigan Cancer Research Consortium

Ann Arbor, Michigan, United States

Site Status

Oakwood Cancer Center at Oakwood Hospital and Medical Center

Dearborn, Michigan, United States

Site Status

Barbara Ann Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Genesys Hurley Cancer Institute

Flint, Michigan, United States

Site Status

Hurley Medical Center

Flint, Michigan, United States

Site Status

Van Elslander Cancer Center at St. John Hospital and Medical Center

Grosse Pointe Woods, Michigan, United States

Site Status

Foote Memorial Hospital

Jackson, Michigan, United States

Site Status

Sparrow Regional Cancer Center

Lansing, Michigan, United States

Site Status

St. Mary Mercy Hospital

Livonia, Michigan, United States

Site Status

St. Joseph Mercy Oakland

Pontiac, Michigan, United States

Site Status

Mercy Regional Cancer Center at Mercy Hospital

Port Huron, Michigan, United States

Site Status

Seton Cancer Institute at Saint Mary's - Saginaw

Saginaw, Michigan, United States

Site Status

St. John Macomb Hospital

Warren, Michigan, United States

Site Status

Grandview Hospital

Dayton, Ohio, United States

Site Status

Good Samaritan Hospital

Dayton, Ohio, United States

Site Status

David L. Rike Cancer Center at Miami Valley Hospital

Dayton, Ohio, United States

Site Status

CCOP - Dayton

Dayton, Ohio, United States

Site Status

Blanchard Valley Medical Associates

Findlay, Ohio, United States

Site Status

Middletown Regional Hospital

Franklin, Ohio, United States

Site Status

Charles F. Kettering Memorial Hospital

Kettering, Ohio, United States

Site Status

UVMC Cancer Care Center at Upper Valley Medical Center

Troy, Ohio, United States

Site Status

Clinton Memorial Hospital

Wilmington, Ohio, United States

Site Status

Ruth G. McMillan Cancer Center at Greene Memorial Hospital

Xenia, Ohio, United States

Site Status

Countries

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United States

Other Identifiers

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S0629

Identifier Type: OTHER

Identifier Source: secondary_id

U10CA032102

Identifier Type: NIH

Identifier Source: secondary_id

View Link

S0629

Identifier Type: -

Identifier Source: org_study_id

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