Waldenstrom's MacroglobulinemiaPhase 1NCT01078974
Pomalidomide, Dexamethasone and Rituximab in Waldenstrom's Macroglobulinemia
A Phase 1 study testing pomalidomide, dexamethasone and rituximab in people with Waldenstrom's Macroglobulinemia, run by Steven P. Treon, MD, PhD at 1 site in United States.
Trial summary
Pomalidomide is a newly discovered drug that may stop cancer cells from growing abnormally. Pomalidomide may also stimulate the immune system to fight the cancer cells and possibly improve the effectiveness of dexamethasone and rituximab to fight the Waldenstrom's Macroglobulinemia (WM) cancer cells. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Waldenstrom's Macroglobulinemia
- What is being tested?
- pomalidomide, dexamethasone 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
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- May 2010to Mar 2016
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18 years of age or older
- Able to adhere to the study visit schedule and other protocol requirements
- Clinicopathological diagnosis of Waldenstrom's macroglobulinemia using consensus panel criteria
- CD20 positive based on any previous performed bone marrow immunohistochemistry or flow cytometric analysis
- Meet criteria to treat based on consensus panel criteria
- Patient must have received at least one previous therapy for WM
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- All previous cancer therapy, including radiation, hormonal therapy and surgery, must have been discontinued at least 4 weeks prior to treatment in this study
- Measurable disease, defined as presence of immunoglobulin M (IgM) paraprotein with a minimum IgM level of 2 times (or greater) the upper limit of each institution's normal value is required
- ECOG Performance status of 0, 1 or 2
- Laboratory tests within ranges outlined in the protocol
- Disease free of prior malignancies for 5 years or more with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma in situ of the cervix or breast
- Screening of patients at high risk of HBV or HCV infection
- Willing and able to take aspirin or alternate prophylactic anticoagulants
Who cannot join
- Any serious medical condition, laboratory abnormality, or psychiatric illness
- Pregnant or lactating females
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
- Resistance or intolerance to prior rituximab therapy
- Previous therapy with thalidomide or lenalidomide
- Known hypersensitivity to thalidomide, lenalidomide or pomalidomide
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- The development of erythema nodosum if characterized by a desquamating rash while taking similar drugs
- Concurrent use of other anti-cancer agents or treatments
- History of non-compliance to medical regimens
- Patients unwilling to or unable to comply with the protocol
- Known positive for HIV or hepatitis infection
- Any history of CVA (Cerebral Vascular Accident/stroke) or clots
- Active DVT or PE that has not been therapeutically anticoagulated
- NYHA classification III and greater heart failure
- Any patient that is unable to ingest or process pomalidomide
Trial locations and contacts
1 site in 1 country.
United States1 site
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
About this study
Official title: Phase I Study of Pomalidomide, Dexamethasone and Rituximab (PDR) in Relapsed or Refractory Waldenstrom's Macroglobulinemia
Pomalidomide is a newly discovered drug that may stop cancer cells from growing abnormally. Pomalidomide may also stimulate the immune system to fight the cancer cells and possibly improve the effectiveness of dexamethasone and rituximab to fight the Waldenstrom's Macroglobulinemia (WM) cancer cells. This drug have been used in multiple myeloma and information from these other research studies suggests that Pomalidomide may help to reduce or prevent the growth of cancer cells.
Read the detailed description
- Participants will be given a study drug-dosing calendar for each treatment cycle. Each treatment cycle lasts 28 days during which time participants will take Pomalidomide orally once a day. Dexamethasone and rituximab will be administered intravenously on weeks 1, 2, 3, 4 and on weeks 12, 13, 14, 15.
- Since we are looking for the highest dose of Pomalidomide in combination with dexamethasone and rituximab which can be administered safely without severe or unmanageable side effects, not everyone who participates will receive the same dose of the study drug. The dose participants will get will depend on the number of participants who have been enrolled in the study and how well they have tolerated their doses.
- As long as there is no evidence that the participant's Waldenstrom's Macroglobulinemia has progressed, they can continue to receive Pomalidomide for up to 52 weeks. Participants will be asked to return to the clinic for follow-up tests at least every three months for four years.
- Conditions
- Waldenstrom's Macroglobulinemia
- Being tested
- pomalidomide, dexamethasone, rituximab
- Keywords
- WM, pomalidomide, dexamethasone, rituximab
- Medical subject headings
- Waldenstrom Macroglobulinemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pomalidomideDrug
Also called CC-4047, Pomalyst
Taken orally once a day
dexamethasoneDrug
Also called Decadron
Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
rituximabDrug
Also called Rituxan
Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
| Study group | Role | What they receive |
|---|---|---|
| pomalidomide, dexamethasone, rituximab | Experimental | Drug: pomalidomide Taken orally once a day
Drug: dexamethasone Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
Drug: rituximab Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 Receives: Drug: pomalidomide; Drug: dexamethasone; Drug: rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose of Pomalidomide
To determine the MTD of pomalidomide administered orally in patients with Waldenstrom's Macroglobulinemia in combination with dexamethasone and rituximab. Because maximum tolerated dose was not determined due to study termination, the highest dose of pomalidomide administered is presented below.
Measured: 2 years
Tolerability of Pomalidomide
Number of participants with dose limiting toxicities which resulted in being removed from pomalidomide therapy
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2010 (estimated)
- Study startMay 2010
- Primary completionSep 2015
- Study completionMar 2016
Why it stopped: Safety concerns (IgM Flare)
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.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Steven P. Treon, MD, PhD
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene Corporation
- Responsible party
- Sponsor-investigator, Steven P. Treon, MD, PhD, Dana-Farber/Brigham and Women's Cancer Center
- Investigators
- Steven P. Treon, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01078974
- Sponsor's protocol ID
- 10-007
- Other IDs
- PO-WM-PI-0005
- Status last verified
- Jun 2016
- First submitted
- 1 Mar 2010
- Last updated
- 25 Jul 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Pomalidomide, Dexamethasone and Rituximab (PDR) in Relapsed or Refractory Waldenstrom's Macroglobulinemia
- ClinicalTrials.gov ID
- NCT01078974
- Acronym / sponsor ID
- 10-007
- Conditions
- Waldenstrom's Macroglobulinemia
- Keywords
- WM, pomalidomide, dexamethasone, rituximab
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Safety concerns (IgM Flare)
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Steven P. Treon, MD, PhDUniversity, hospital or other
- Collaborators
- Celgene Corporation
- Study start
- May 2010
- Primary completion
- Sep 2015 (actual)
- Study completion
- Mar 2016 (actual)
- First posted
- 2 Mar 2010 (estimated)
- Results
- Posted 25 Jul 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Pomalidomide is a newly discovered drug that may stop cancer cells from growing abnormally. Pomalidomide may also stimulate the immune system to fight the cancer cells and possibly improve the effectiveness of dexamethasone and rituximab to fight the Waldenstrom's Macroglobulinemia (WM) cancer cells. This drug have been used in multiple myeloma and information from these other research studies suggests that Pomalidomide may help to reduce or prevent the growth of cancer cells.
Detailed description
- Participants will be given a study drug-dosing calendar for each treatment cycle. Each treatment cycle lasts 28 days during which time participants will take Pomalidomide orally once a day. Dexamethasone and rituximab will be administered intravenously on weeks 1, 2, 3, 4 and on weeks 12, 13, 14, 15.
- Since we are looking for the highest dose of Pomalidomide in combination with dexamethasone and rituximab which can be administered safely without severe or unmanageable side effects, not everyone who participates will receive the same dose of the study drug. The dose participants will get will depend on the number of participants who have been enrolled in the study and how well they have tolerated their doses.
- As long as there is no evidence that the participant's Waldenstrom's Macroglobulinemia has progressed, they can continue to receive Pomalidomide for up to 52 weeks. Participants will be asked to return to the clinic for follow-up tests at least every three months for four years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| pomalidomide, dexamethasone, rituximab | Experimental | Drug: pomalidomide, Drug: dexamethasone, Drug: rituximabDrug: pomalidomide Taken orally once a day Drug: dexamethasone Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 Drug: rituximab Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 18 years of age or older
- Able to adhere to the study visit schedule and other protocol requirements
- Clinicopathological diagnosis of Waldenstrom's macroglobulinemia using consensus panel criteria
- CD20 positive based on any previous performed bone marrow immunohistochemistry or flow cytometric analysis
- Meet criteria to treat based on consensus panel criteria
Key exclusion criteria (15 listed)
- Any serious medical condition, laboratory abnormality, or psychiatric illness
- Pregnant or lactating females
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
- Resistance or intolerance to prior rituximab therapy
- Previous therapy with thalidomide or lenalidomide
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 |
|---|---|---|
| pomalidomideAlso: CC-4047, PomalystTaken orally once a day | Drug | pomalidomide, dexamethasone, rituximab |
| dexamethasoneAlso: DecadronGiven intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 | Drug | pomalidomide, dexamethasone, rituximab |
| rituximabAlso: RituxanGiven intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 | Drug | pomalidomide, dexamethasone, rituximab |
Primary outcome
Maximum Tolerated Dose of Pomalidomide
Time frame: 2 years
To determine the MTD of pomalidomide administered orally in patients with Waldenstrom's Macroglobulinemia in combination with dexamethasone and rituximab. Because maximum tolerated dose was not determined due to study termination, the highest dose of pomalidomide administered is presented below.
Tolerability of Pomalidomide
Time frame: 2 years
Number of participants with dose limiting toxicities which resulted in being removed from pomalidomide therapy
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Waldenstrom's Macroglobulinemia
RecruitingPhase 2
Testing the Combination of Venetoclax and Rituximab, in Comparison to the Usual Treatment (Ibrutinib Plus Rituximab or Zanubrutinib Alone) for Waldenstrom's Macroglobulinemia/Lymphoplasmacytic Lymphoma
Lymphoplasmacytic Lymphoma, Waldenstrom Macroglobulinemia
Active, not recruitingPhase 2
A Study to Investigate Efficacy and Safety of BCL2 Inhibitor Sonrotoclax as Monotherapy and in Combination With Zanubrutinib in Adults With Waldenström Macroglobulinemia
Waldenstrom Macroglobulinemia, Waldenstrom's Macroglobulinemia Recurrent
Active, not recruitingPhase 2
Bendamustine, Rituximab and Acalabrutinib in Waldenstrom's Macroglobulinemia
Waldenstrom Macroglobulinemia
Not yet recruitingPhase 2
Teclistamab In Waldenstrom's Macroglobulinemia
Waldenstrom's Macroglobulinemia
Not yet recruitingPhase 3
Evaluation of Iopofosine I 131 vs. R-CD in WM Patients
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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 principal investigator
Dana-Farber Cancer Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing pomalidomide, dexamethasone and rituximab in people with Waldenstrom's Macroglobulinemia, run by Steven P. Treon, MD, PhD at 1 site 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 13 inclusion and 15 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: stopped early, as of the sponsor's last update on 25 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 25 Jul 2016). 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?
Maximum Tolerated Dose of Pomalidomide (time frame: 2 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?
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 25 Jul 2016.