Waldenstrom's MacroglobulinemiaPhase 2NCT00142116
Thalidomide and Rituximab in Waldenstrom's Macroglobulinemia
A Phase 2 study testing Thalidomide and Rituximab in people with Waldenstrom's Macroglobulinemia and Lymphoplasmacytic Lymphoma, run by Steven P. Treon, MD, PhD at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Waldenstrom's Macroglobulinemia, Lymphoplasmacytic Lymphoma
- What is being tested?
- Thalidomide and Rituximab
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 2 sitesUnited States
- Study dates
- May 2003to Feb 2008
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Show all 9Show fewer
- Greater than 18 years of age
- Life expectancy of 3 months or greater
- Eastern Cooperative Oncology Group (ECOG) status performance of 0-2
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
About this study
Official title: Phase II Study of Thalidomide and Rituximab in Waldenstrom's Macroglobulinemia
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.
Read the 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.
- Being tested
- Thalidomide, Rituximab
- Keywords
- thalidomide, rituximab, Waldenstrom's
- Medical subject headings
- Waldenstrom Macroglobulinemia, Lymphoma, B-Cell, Marginal Zone
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThalidomideDrug
Also called Thalomid
200mg orally once a day for 14 weeks if that dosage is tolerated well, it will be increased to 400mg for up to 50 weeks.
RituximabDrug
Also called Rituxan
Given intravenously once weekly for 4 weeks beginning the second week of study treatment. If tolerated well, this may be repeated 8 weeks later.
| Study group | Role | What they receive |
|---|---|---|
| Thalidomide and Rituximab | Experimental | Thalidomide 200mg orally once a day for 14 weeks if that dosage is tolerated well, it will be increased to 400mg for up to 50 weeks
Rituximab Given intravenously once weekly for 4 weeks beginning the second week of study treatment. If tolerated well, this may be repeated 8 weeks later. Receives: Drug: Thalidomide; Drug: Rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate
Response determinations were made using modified consensus panel criteria from the Third International Workshop on WM, and response rates were determined on an evaluable basis. A complete response was defined as having resolution of all symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly. Patients achieving a partial response and a minor response were defined as achieving a more than or equal to 50% and more than or equal to 25% reduction in serum IgM levels, respectively. Patients with stable disease were defined as having less than 25% change in serum IgM levels, in the absence of new or increasing adenopathy or splenomegaly and/or other progressive signs or symptoms of WM. Progressive disease was defined as a greater than 25% increase in serum IgM level occurred from the lowest attained response value or progression of clinically significant disease-related symptom(s).
Measured: 3 years
Time to Progression
Time to disease progression (TTP) was calculated from the start of therapy using the Kaplan-Meier method.
Measured: 49.1 months
Secondary outcomes (1)
To Identify the Mechanism(s) of Action for Combined Thalidomide and Rituximab Activity.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2005 (estimated)
- Study startMay 2003
- Primary completionFeb 2004
- Study completionFeb 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Waldenstrom's MacroglobulinemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Steven P. Treon, MD, PhD
- Sponsor type
- University, hospital or other
- Collaborators
- Dana-Farber Cancer Institute, Massachusetts General Hospital, Beth Israel Deaconess Medical Center, Brigham and Women's Hospital, Cape Cod Hospital
- Responsible party
- Sponsor-investigator, Steven P. Treon, MD, PhD, Dana-Farber Cancer Institute
- Investigators
- Steven P. Treon, MD, MA, PhD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00142116
- Sponsor's protocol ID
- 03-077
- Status last verified
- May 2014
- First submitted
- 1 Sep 2005
- Last updated
- 2 Jun 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 18713945
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Thalidomide and Rituximab in Waldenstrom's Macroglobulinemia
- ClinicalTrials.gov ID
- NCT00142116
- Acronym / sponsor ID
- 03-077
- Keywords
- thalidomide, rituximab, Waldenstrom's
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Steven P. Treon, MD, PhDUniversity, hospital or other
- Collaborators
- Dana-Farber Cancer Institute, Massachusetts General Hospital, Beth Israel Deaconess Medical Center, Brigham and Women's Hospital, Cape Cod Hospital
- Study start
- May 2003
- Primary completion
- Feb 2004 (actual)
- Study completion
- Feb 2008 (actual)
- First posted
- 2 Sep 2005 (estimated)
- Results
- Posted 2 Jun 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Thalidomide and Rituximab | Experimental | Drug: Thalidomide, Drug: RituximabThalidomide 200mg orally once a day for 14 weeks if that dosage is tolerated well, it will be increased to 400mg for up to 50 weeks Rituximab Given intravenously once weekly for 4 weeks beginning the second week of study treatment. If tolerated well, this may be repeated 8 weeks later. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ThalidomideAlso: Thalomid200mg orally once a day for 14 weeks if that dosage is tolerated well, it will be increased to 400mg for up to 50 weeks. | Drug | Thalidomide and Rituximab |
| RituximabAlso: RituxanGiven intravenously once weekly for 4 weeks beginning the second week of study treatment. If tolerated well, this may be repeated 8 weeks later. | Drug | Thalidomide and Rituximab |
Primary outcome
Objective Response Rate
Time frame: 3 years
Response determinations were made using modified consensus panel criteria from the Third International Workshop on WM, and response rates were determined on an evaluable basis. A complete response was defined as having resolution of all symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly. Patients achieving a partial response and a minor response were defined as achieving a more than or equal to 50% and more than or equal to 25% reduction in serum IgM levels, respectively. Patients with stable disease were defined as having less than 25% change in serum IgM levels, in the absence of new or increasing adenopathy or splenomegaly and/or other progressive signs or symptoms of WM. Progressive disease was defined as a greater than 25% increase in serum IgM level occurred from the lowest attained response value or progression of clinically significant disease-related symptom(s).
Time to Progression
Time frame: 49.1 months
Time to disease progression (TTP) was calculated from the start of therapy using the Kaplan-Meier method.
1 secondary outcome
To Identify the Mechanism(s) of Action for Combined Thalidomide and Rituximab Activity.
Time frame: 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 18713945Treon 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Steven P. Treon, MD, MA, PhD principal investigator
Dana-Farber Cancer Institute
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Thalidomide and Rituximab in people with Waldenstrom's Macroglobulinemia and Lymphoplasmacytic Lymphoma, run by Steven P. Treon, MD, PhD at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Jun 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Jun 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (time frame: 3 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00142116, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jun 2014.