Recurrent Waldenstrom MacroglobulinemiaPhase 1NCT01198067
Pomalidomide in Treating Patients With Relapsed or Refractory Waldenstrom Macroglobulinemia
A Phase 1 study testing Pomalidomide in people with Recurrent Waldenstrom Macroglobulinemia and Refractory Waldenstrom Macroglobulinemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This phase I trial studies the side effects and best dose of pomalidomide in treating patients with Waldenstrom macroglobulinemia that has returned after a period of improvement (relapsed) or does not respond to treatment (refractory). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Recurrent Waldenstrom Macroglobulinemia, Refractory Waldenstrom Macroglobulinemia
- What is being tested?
- Pomalidomide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Oct 2010to 2 May 2025
- 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
- Understand and voluntarily sign an informed consent form
- Able to adhere to the study visit schedule and other protocol requirements
- Waldenstrom's macroglobulinemia that has relapsed and/or is refractory to at least one prior line of therapy
- All previous cancer therapy, including radiation, hormonal therapy and surgery, must have been discontinued at least 4 weeks prior to treatment in this study
- Eastern Cooperative Oncology Group (ECOG) performance status of =< 2 at study entry
- Serum creatinine =< 2.0 mg/dL
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- Creatinine clearance >= 45 ml/min
- Total bilirubin =< 3 x upper limit of normal (ULN) or direct bilirubin =< 2 x ULN
- Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 2 x ULN
- Platelet count >= 20 K/microL
- Absolute neutrophil count >= 500 K/microL
- Disease free of prior malignancies for >= 5 years with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma in situ of the cervix or breast
- Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of at least 25 mIU/mL within 10 to 14 days prior to and again within 24 hours of starting pomalidomide and must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, at least 28 days before she starts taking pomalidomide; FCBP must also agree to ongoing pregnancy testing; men must agree to practice complete abstinence or agree use a latex condom during sexual contact with a FCBP while participating in the study, during dose interruptions and for at least 90 days following study drug discontinuation, even if they have had a successful vasectomy; all patients must be counseled at a minimum of every 28 days about pregnancy precautions and risks of fetal exposure
- Able to take aspirin (325 mg) daily as prophylactic anticoagulation (patients intolerant to acetylsalicylic acid [ASA] may use therapeutic dose warfarin or low molecular weight heparin)
- All study participants must be registered into the mandatory POMALYST REMS program, and be willing and able to comply with the requirements of the POMALYST REMS program
Who cannot join
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form
- Pregnant or breast feeding females; (lactating females must agree not to breast feed while taking pomalidomide or for 28 days after stopping pomalidomide)
- Any medical or psychiatric 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
- Use of any other experimental drug or therapy within 28 days of the first dose of study drug
- Known hypersensitivity to thalidomide or lenalidomide
- The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs
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- Any prior use of pomalidomide
- Concurrent use of other anti-cancer agents or treatments
- Known positive for human immunodeficiency virus (HIV) or acute hepatitis A or acute or chronic active hepatitis B or C
- Grade > 2 peripheral neuropathy
- Neutrophil count < 1000 K/microL and/or
- Platelet count < 100 K/microL unless infiltration by Waldenström's macroglobulinemia equals or exceed 60% of bone marrow cellularity
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I Study of Pomalidomide in Relapsed or Refractory Waldenstrom Macroglobulinemia
This phase I trial studies the side effects and best dose of pomalidomide in treating patients with Waldenstrom macroglobulinemia that has returned after a period of improvement (relapsed) or does not respond to treatment (refractory). Pomalidomide may stimulate the immune system in different ways and stop cancer cells from growing.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine the maximum tolerated dose (MTD) of pomalidomide in patients with relapsed or refractory Waldenstrom macroglobulinemia.
SECONDARY OBJECTIVES:
- To evaluate the safety and toxicity profile of pomalidomide in patients with relapsed or refractory Waldenstrom macroglobulinemia.
II. To evaluate the efficacy of pomalidomide in patients with relapsed or refractory Waldenstrom macroglobulinemia.
OUTLINE: This is a dose-escalation study.
Patients receive pomalidomide orally (PO) on days 1-28 or 1-21. Courses repeat every 21 or 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 6 months.
- Being tested
- Pomalidomide
- 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 4-Aminothalidomide, Actimid, CC-4047, Imnovid
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (pomalidomide) | Experimental | Patients receive pomalidomide PO on days 1-28 or 1-21. Courses repeat every 21 or 28 days in the absence of disease progression or unacceptable toxicity. Receives: Drug: Pomalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients Experiencing MTD
Maximum Tolerated Dose
Measured: Participants Experiencing Study Medication Maximum Tolerated Dose through Study Completion (Avg. 1 Year)
Secondary outcomes (3)
Cycles Completed
Length of Treatment
Measured: Study Completion (Avg. 1 Year)
Changes in Waldestrom Biomarkers
Average Paraprotein1 gm/dL Change Cycle 1 thru Study
Measured: Through Study Completion (Avg. 1 Year)
Changes in Waldestrom Biomarkers
Average Reduction in IgM Protein mg/dL from Cycle 1 thru Study
Measured: Through Study Completion (Avg. 1 Year)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2010 (estimated)
- Study start6 Oct 2010
- Primary completion2 May 2025
- Study completion2 May 2025
Why it stopped: \<75% participant accrual
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 Recurrent Waldenstrom MacroglobulinemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- 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
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Sheeba K Thomas, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01198067
- Sponsor's protocol ID
- 2009-0972
- Other IDs
- NCI-2012-01882, NCI-2010-02076, 2009-0972
- Status last verified
- Sep 2025
- First submitted
- 8 Sep 2010
- Last updated
- 4 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Pomalidomide in Relapsed or Refractory Waldenstrom Macroglobulinemia
- ClinicalTrials.gov ID
- NCT01198067
- Acronym / sponsor ID
- 2009-0972
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: \<75% participant accrual
- Enrolment
- 15 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 6 Oct 2010 (actual)
- Primary completion
- 2 May 2025 (actual)
- Study completion
- 2 May 2025 (actual)
- First posted
- 9 Sep 2010 (estimated)
- Results
- Posted 4 Sep 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase I trial studies the side effects and best dose of pomalidomide in treating patients with Waldenstrom macroglobulinemia that has returned after a period of improvement (relapsed) or does not respond to treatment (refractory). Pomalidomide may stimulate the immune system in different ways and stop cancer cells from growing.
Detailed description
PRIMARY OBJECTIVES:
- To determine the maximum tolerated dose (MTD) of pomalidomide in patients with relapsed or refractory Waldenstrom macroglobulinemia.
SECONDARY OBJECTIVES:
- To evaluate the safety and toxicity profile of pomalidomide in patients with relapsed or refractory Waldenstrom macroglobulinemia.
II. To evaluate the efficacy of pomalidomide in patients with relapsed or refractory Waldenstrom macroglobulinemia.
OUTLINE: This is a dose-escalation study.
Patients receive pomalidomide orally (PO) on days 1-28 or 1-21. Courses repeat every 21 or 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 6 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (pomalidomide) | Experimental | Drug: PomalidomidePatients receive pomalidomide PO on days 1-28 or 1-21. Courses repeat every 21 or 28 days in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Understand and voluntarily sign an informed consent form
- Able to adhere to the study visit schedule and other protocol requirements
- Waldenstrom's macroglobulinemia that has relapsed and/or is refractory to at least one prior line of therapy
- All previous cancer therapy, including radiation, hormonal therapy and surgery, must have been discontinued at least 4 weeks prior to treatment in this study
- Eastern Cooperative Oncology Group (ECOG) performance status of =< 2 at study entry
Key exclusion criteria (12 listed)
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form
- Pregnant or breast feeding females; (lactating females must agree not to breast feed while taking pomalidomide or for 28 days after stopping pomalidomide)
- Any medical or psychiatric 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
- Use of any other experimental drug or therapy within 28 days of the first dose of study drug
- Known hypersensitivity to 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: 4-Aminothalidomide, Actimid, CC-4047, ImnovidGiven PO | Drug | Treatment (pomalidomide) |
Primary outcome
Number of Patients Experiencing MTD
Time frame: Participants Experiencing Study Medication Maximum Tolerated Dose through Study Completion (Avg. 1 Year)
Maximum Tolerated Dose
3 secondary outcomes
Cycles Completed
Time frame: Study Completion (Avg. 1 Year)
Changes in Waldestrom Biomarkers
Time frame: Through Study Completion (Avg. 1 Year)
Changes in Waldestrom Biomarkers
Time frame: Through Study Completion (Avg. 1 Year)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- MD Anderson Cancer CenterLink provided by the sponsor.
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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.
Sheeba K Thomas principal investigator
M.D. Anderson Cancer Center
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 in people with Recurrent Waldenstrom Macroglobulinemia and Refractory Waldenstrom Macroglobulinemia, run by M.D. Anderson Cancer Center 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 15 inclusion and 12 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 4 Sep 2025. 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 4 Sep 2025). 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?
Number of Patients Experiencing MTD (time frame: Participants Experiencing Study Medication Maximum Tolerated Dose through Study Completion (Avg. 1 Year)). 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 4 Sep 2025.