Essential ThrombocythemiaPhase 2NCT00227591
Lenalidomide and Prednisone in Treating Patients With Myelofibrosis
A Phase 2 study testing lenalidomide, prednisone and laboratory biomarker analysis in people with Essential Thrombocythemia and Polycythemia Vera, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
This phase II trial is studying how well giving lenalidomide together with prednisone works in treating patients with myelofibrosis. Lenalidomide may stop the growth of myelofibrosis by blocking blood flow to the cancer. It may also stimulate the immune system in different ways and stop cancer cells from growing. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Essential Thrombocythemia, Polycythemia Vera, Primary Myelofibrosis
- What is being tested?
- lenalidomide, prednisone and laboratory biomarker analysis
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2005to Dec 2010
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient must be diagnosed with myelofibrosis with myeloid metaplasia (MMM); agnogenic myeloid metaplasia, post-polycythemic myeloid metaplasia, or post-thrombocythemic myeloid metaplasia are included
- NOTE: Diagnosis must be confirmed by central pathology review; diagnostic samples must be submitted; patient may register and begin treatment based on the local pathology review. If the central review does not confirm patient's eligibility to participate in the trial, protocol treatment must be discontinued
- Patient must have discontinued chemotherapy (hydroxyurea, alpha interferon, anagrelide, other myelosuppressive agents, thalidomide, or any other experimental therapy) as well as growth factors and systemic use of corticosteroids >= 28 days prior to starting study drug
- All non-hematologic toxicity must be resolved to =< grade 1
- Patient must be lenalidomide-naïve (never treated with lenalidomide)
- ECOG performance status (PS) of 0, 1, or 2 at study entry
Show all 19Show fewer
- Hemoglobin level =< 10 g/dL or transfusion-dependent
- Absolute neutrophil count >= 1,000 uL
- Platelet count >= 100,000 uL
- Serum creatinine =< 2.0 mg/dL
- Total bilirubin =< 2.0 mg/dL (if elevated; direct bilirubin =< 2.0 mg/dL)
- AST (SGOT) =< 3 x ULN unless attributed to hepatic extramedullary hematopoiesis
- Women must not be pregnant or breastfeeding because this study involves an investigational agent whose genotoxic, mutagenic, and teratogenetic effects on the developing fetus and newborn are unknown; women of childbearing potential who are sexually active, must use 2 accepted methods of birth control at the same time for 4 weeks prior to lenalidomide treatment, during lenalidomide treatment, and up to 4 weeks after lenalidomide treatment is finished; sexually active males must use a latex condom for contraception during the study and up to 4 weeks after treatment with lenalidomide has ended
- All females of childbearing potential must have a blood test 10-14 days prior to the start of lenalidomide treatment to rule out pregnancy; another blood test to rule out pregnancy must be done 24 hours prior to the start of treatment with lenalidomide
- Patient must not have any condition, including the presence of laboratory abnormalities, which, based on the physician's opinion, places the patient at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
- Patient must not have any known hypersensitivity to thalidomide or lenalidomide
- Patient must not have any known positive status for HIV or infectious hepatitis type A, B or C
- Patient must not have any other active malignancy
- NOTE: SWOG patients are strongly encouraged to be registered on SWOG-9007 ("Cytogenetic Studies in Leukemia Patients"); SWOG institutions registering patients to SWOG-9007 should follow the instructions for specimen submission
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Eastern Cooperative Oncology Group
Boston, Massachusetts, 02215
About this study
Official title: A Phase II Study of Lenalidomide (CC-5013) in Combination With Prednisone for the Treatment of Myelofibrosis With Myeloid Metaplasia
This phase II trial is studying how well giving lenalidomide together with prednisone works in treating patients with myelofibrosis. Lenalidomide may stop the growth of myelofibrosis by blocking blood flow to the cancer. It may also stimulate the immune system in different ways and stop cancer cells from growing. Drugs used in chemotherapy, such as prednisone, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving lenalidomide together with prednisone may kill more cancer cells.
Read the detailed description
PRIMARY OBJECTIVES:
- To evaluate the rate of complete or partial remission from treatment with a combination of lenalidomide and prednisone in patients with myelofibrosis with myeloid metaplasia.
SECONDARY OBJECTIVES:
- To examine drug toxicity. II. To examine duration of response. III. To examine the effect of treatment on bone marrow fibrosis, angiogenesis, and cytogenetics.
OUTLINE:
For courses 1 and 2, patients receive oral lenalidomide once daily and oral prednisone once daily on days 1-28. For course 3, patients receive oral lenalidomide once daily on days 1-28 and oral prednisone once on days 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, and 27. Patients with stable or responding disease after course 3 receive oral lenalidomide alone once daily on days 1-28 for courses 4-6. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed every 3 months for 1 year and then every 6 months for up to 5 years from study entry.
- Being tested
- lenalidomide, prednisone, laboratory biomarker analysis
- Medical subject headings
- Thrombocythemia, Essential, Polycythemia Vera, Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
lenalidomideDrug
Also called CC-5013, IMiD-1, Revlimid
Given orally (PO)
prednisoneDrug
Also called DeCortin, Deltra
Given PO
laboratory biomarker analysisOther
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (lenalidomide, prednisone) | Experimental | For courses 1 and 2, patients receive oral lenalidomide once daily and oral prednisone once daily on days 1-28. For course 3, patients receive oral lenalidomide once daily on days 1-28 and oral prednisone once on days 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, and 27. Patients with stable or responding disease after course 3 receive oral lenalidomide alone once daily on days 1-28 for courses 4-6. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Receives: Drug: lenalidomide; Drug: prednisone; Other: laboratory biomarker analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate
Response was evaluated for Anemia and Spleen:
Major anemia response: hemoglobin increase to within normal limits in the absence of transfusion. Minor anemia response: hemoglobin improvement of at least 2 grams per deciliter independent of transfusion support, or achievement of transfusion independence in transfusion-dependent patients. Major spleen response: normalization of spleen size to the range of 12-14 centimeters by ultrasound. Minor spleen response: a 50% or more decrease in excess spleen size by ultrasound. Complete remission (CR): complete resolution of disease-related symptoms, splenomegaly, normalization of peripheral blood count, white cell differential and smear, and normalization of bone marrow histology. Partial remission (PR): a major or minor response in anemia or splenomegaly. Overall Response (OR)=CR + PR, assessed among eligible, treated patients.
Measured: Assessed at the end of cycle 3
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2005 (estimated)
- Study startDec 2005
- Primary completionDec 2010
- Study completionDec 2010
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 Essential ThrombocythemiaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Ayalew Tefferi, Principal investigator, Eastern Cooperative Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00227591
- Sponsor's protocol ID
- NCI-2012-02976
- Other IDs
- NCI-2012-02976, ECOG-E4903, E4903, E4903, U10CA021115
- Status last verified
- Dec 2012
- First submitted
- 26 Sep 2005
- Last updated
- 21 May 2014
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Mesa RA, Yao X, Cripe LD, Li CY, Litzow M, Paietta E, Rowe JM, Tefferi A, Tallman MS. Lenalidomide and prednisone for myelofibrosis: Eastern Cooperative Oncology Group (ECOG) phase 2 trial E4903. Blood. 2010 Nov 25;116(22):4436-8. doi: 10.1182/blood-2010-05-287417. Epub 2010 Jul 22.
PubMed 20651074
Study at a glance
From the registry record, last updated by the sponsor on 21 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Lenalidomide (CC-5013) in Combination With Prednisone for the Treatment of Myelofibrosis With Myeloid Metaplasia
- ClinicalTrials.gov ID
- NCT00227591
- Acronym / sponsor ID
- NCI-2012-02976
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- Dec 2005
- Primary completion
- Dec 2010 (actual)
- Study completion
- Dec 2010 (actual)
- First posted
- 28 Sep 2005 (estimated)
- Results
- Posted 26 Sep 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial is studying how well giving lenalidomide together with prednisone works in treating patients with myelofibrosis. Lenalidomide may stop the growth of myelofibrosis by blocking blood flow to the cancer. It may also stimulate the immune system in different ways and stop cancer cells from growing. Drugs used in chemotherapy, such as prednisone, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving lenalidomide together with prednisone may kill more cancer cells.
Detailed description
PRIMARY OBJECTIVES:
- To evaluate the rate of complete or partial remission from treatment with a combination of lenalidomide and prednisone in patients with myelofibrosis with myeloid metaplasia.
SECONDARY OBJECTIVES:
- To examine drug toxicity. II. To examine duration of response. III. To examine the effect of treatment on bone marrow fibrosis, angiogenesis, and cytogenetics.
OUTLINE:
For courses 1 and 2, patients receive oral lenalidomide once daily and oral prednisone once daily on days 1-28. For course 3, patients receive oral lenalidomide once daily on days 1-28 and oral prednisone once on days 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, and 27. Patients with stable or responding disease after course 3 receive oral lenalidomide alone once daily on days 1-28 for courses 4-6. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed every 3 months for 1 year and then every 6 months for up to 5 years from study entry.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (lenalidomide, prednisone) | Experimental | Drug: lenalidomide, Drug: prednisone, Other: laboratory biomarker analysisFor courses 1 and 2, patients receive oral lenalidomide once daily and oral prednisone once daily on days 1-28. For course 3, patients receive oral lenalidomide once daily on days 1-28 and oral prednisone once on days 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, and 27. Patients with stable or responding disease after course 3 receive oral lenalidomide alone once daily on days 1-28 for courses 4-6. Treatment repeats every 28 days for up to 6 courses 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 (19 listed)
- Patient must be diagnosed with myelofibrosis with myeloid metaplasia (MMM); agnogenic myeloid metaplasia, post-polycythemic myeloid metaplasia, or post-thrombocythemic myeloid metaplasia are included
- Patient must have discontinued chemotherapy (hydroxyurea, alpha interferon, anagrelide, other myelosuppressive agents, thalidomide, or any other experimental therapy) as well as growth factors and systemic use of corticosteroids >= 28 days prior to starting study drug
- Patient must be lenalidomide-naïve (never treated with lenalidomide)
- ECOG performance status (PS) of 0, 1, or 2 at study entry
- Hemoglobin level =< 10 g/dL or transfusion-dependent
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| lenalidomideAlso: CC-5013, IMiD-1, RevlimidGiven orally (PO) | Drug | Treatment (lenalidomide, prednisone) |
| prednisoneAlso: DeCortin, DeltraGiven PO | Drug | Treatment (lenalidomide, prednisone) |
| laboratory biomarker analysisCorrelative studies | Other | Treatment (lenalidomide, prednisone) |
Primary outcome
Overall Response Rate
Time frame: Assessed at the end of cycle 3
Response was evaluated for Anemia and Spleen: Major anemia response: hemoglobin increase to within normal limits in the absence of transfusion. Minor anemia response: hemoglobin improvement of at least 2 grams per deciliter independent of transfusion support, or achievement of transfusion independence in transfusion-dependent patients. Major spleen response: normalization of spleen size to the range of 12-14 centimeters by ultrasound. Minor spleen response: a 50% or more decrease in excess spleen size by ultrasound. Complete remission (CR): complete resolution of disease-related symptoms, splenomegaly, normalization of peripheral blood count, white cell differential and smear, and normalization of bone marrow histology. Partial remission (PR): a major or minor response in anemia or splenomegaly. Overall Response (OR)=CR + PR, assessed among eligible, treated patients.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 20651074Mesa RA, Yao X, Cripe LD, Li CY, Litzow M, Paietta E, Rowe JM, Tefferi A, Tallman MS. Lenalidomide and prednisone for myelofibrosis: Eastern Cooperative Oncology Group (ECOG) phase 2 trial E4903. Blood. 2010 Nov 25;116(22):4436-8. doi: 10.1182/blood-2010-05-287417. Epub 2010 Jul 22.
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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.
Ayalew Tefferi principal investigator
Eastern Cooperative Oncology Group
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing lenalidomide, prednisone and laboratory biomarker analysis in people with Essential Thrombocythemia and Polycythemia Vera, run by National Cancer Institute (NCI) 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 19 inclusion and 0 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 21 May 2014. 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 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 26 Sep 2013). 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?
Overall Response Rate (time frame: Assessed at the end of cycle 3). 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 NCT00227591, 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 21 May 2014.