MyelofibrosisPhase 2NCT03069326
A Clinical Study to Test the Effects of Ruxolitinib And Thalidomide Combination for Patients With Myelofibrosis
A Phase 2 study testing Ruxolitinib and Thalidomide in people with Myelofibrosis, run by Memorial Sloan Kettering Cancer Center at 7 sites in United States.
Trial summary
The purpose of this study is to test any good and bad effects of the study drugs called ruxolitinib and thalidomide. Ruxolitinib and thalidomide could shrink the cancer, but it could also cause side effects. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelofibrosis
- What is being tested?
- Ruxolitinib and Thalidomide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 7 sitesUnited States
- Study dates
- 27 Feb 2017to Feb 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- Diagnosis of myelofibrosis (either primary or post essential thrombocythemia/polycythemia vera) requiring therapy, including those previously treated and relapsed or refractory, or if newly diagnosed, with intermediate-1 or -2 or high risk IPSS, DIPSS, DIPSS+, MIPSS70 or MIPSS70+ v2.0 score
- Patients taking Ruxolitinib at the time of enrollment must have been taking Ruxolitinib for a minimum of 3 months, and must have been on a stable dose of Ruxolitinib for a minimum of 4 weeks immediately prior to enrollment. However, for patients in the thrombocytopenic cohort A expansion, patients taking Ruxolitinib at the time of enrollment who are deemed to have a suboptimal response or are refractory to Ruxolitinib single-agent therapy (less than partial response per IWG criteria) must have been taking Ruxolitinib for a minimum of 6 weeks, and must have been on a stable dose of Ruxolitinib for a minimum of 4 weeks immediately prior to enrollment
- Patients taking Ruxolitinib at the time of enrollment must be deemed to have had a suboptimal response (less than partial response per IWG criteria) to Ruxolitinib single-agent therapy or deemed to have progression of disease (per IWG criteria).
- Age ≥ 18 years at the time of signing the informed consent.
- ECOG performance status 0 to 2.
- Patients must have adequate organ function as demonstrated by the following:
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- Total bilirubin ≤ 2.0 mg/dL, unless due to Gilbert's disease
- Serum creatinine ≤ 2.0 mg/dL.
- ALT and AST ≤ 3 x upper limit of normal (unless the transaminitis is considered to be related to MF, in which case ≤5 x ULN is allowed
- Females of childbearing potential (FCBP)† must have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL within 14 days prior to and again within 24 hours* of starting Thalidomide 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 4 weeks before she starts taking Thalidomide. FCBP must also agree to ongoing pregnancy testing. Men must agree to use a condom during sexual contact with a female of child bearing potential 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.
- All study participants must be registered into the mandatory REMS® program, and be willing and able to comply with the requirements of REMS®
- Platelets ≥ 50000/uL and ANC ≥ 1000. For patients enrolled in the thrombocytopenic cohorts, platelet count must be >/= 25,000 but </= 99,000/uL.
- All study participants must be able to swallow oral medication
Who cannot join
- Use of any other standard anti-neoplastic drug or growth factor (e.g., anagrelide, G-CSF, revlimid, clofarabine) except hydroxyurea or experimental drugs, with the exception of Ruxolitinib, less than 14 days or 5-half lives prior to starting study therapy and/or lack of recovery from all toxicity from previous therapy to grade 1 or better.
- Known prior clinically relevant hypersensitivity reaction to Thalidomide, including the development of erythema nodosum if characterized by a desquamating rash.
- Prior therapy with Thalidomide in combination with Ruxolitinib
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form, which places the subject at unacceptable risk if he/she were to participate in the study or which confounds the ability to interpret data from the study.
- Pregnant or lactating females.
- Known positive for HIV or hepatitis B or C per institutional standard of care
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- Participants with prior history of thromboembolic disease (i.e. deep venous thrombosis (DVT) or pulmonary embolism (PE) within the last six months, as Thalidomide has demonstrated an increased risk of DVT or PE
- Known to have a hypercoagulability syndrome (e.g.: antithrombin III, deficiency, anticardiolipin syndrome etc).
- Concurrent use of any strong inducers or strong inhibitors of CYP3A4. (See Appendix F for a list of prohibited and cautionary CYP3A4 inhibitors and inducers)
- Patients with active malignancy of other type than required for this study are not eligible with the exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast. Patients with malignancies with indolent behavior such as prostate cancer treated with radiation or surgery can be enrolled in the study as long as they have a reasonable expectation to have been cured with the treatment modality received.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Memorial Sloan Kettering Commack
Commack, New York, 11725
Memorial Sloan Kettering Westchester
Harrison, New York, 10604
Md Anderson Cancer Center
Houston, Texas, 77030
Memorial Sloan Kettering Monmouth
Middletown, New Jersey, 07748
Memorial Sloan Kettering Bergen
Montvale, New Jersey, 07645
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Memorial Sloan Kettering Nassau
Uniondale, New York, 11553
About this study
Official title: Evaluation of Ruxolitinib And Thalidomide Combination as a Therapy for Patients With Myelofibrosis
The purpose of this study is to test any good and bad effects of the study drugs called ruxolitinib and thalidomide. Ruxolitinib and thalidomide could shrink the cancer, but it could also cause side effects.
- Conditions
- Myelofibrosis
- Being tested
- Ruxolitinib, Thalidomide
- Keywords
- Ruxolitinib, Thalidomide, 16-1498
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Ruxolitinib will be given orally in an outpatient setting unless the patient is being seen inpatient for another reason. Ruxolitinib will be given continuously orally daily in 28-day cycles.
ThalidomideDrug
Thalidomide will be given orally in an outpatient setting unless the patient is being seen inpatient for another reason. thalidomide will be given continuously orally daily in 28-day cycles.
| Study group | Role | What they receive |
|---|---|---|
| Cohort A: Ruxolitinib and Thalidomide | Experimental | After 3 cycles of ruxolitinib treatment, either prior to study enrollment or through the ruxolitinib run-in phase, patients who meet eligibility criteria will be treated with ruxolitinib and thalidomide orally on days 1-28 of a 28 day cycle. Cycles will be continued until the patient wishes to be removed from the study, unacceptable toxicity develops, disease progression, treating physician recommends removal, or termination of study occurs. Receives: Drug: Ruxolitinib; Drug: Thalidomide |
| Cohort B: Ruxolitinib and Thalidomide | Experimental | A cohort expansion, for patients with baseline thrombocytopenia, will enroll 35 additional patients
After 3 cycles of ruxolitinib treatment, either prior to study enrollment or through the ruxolitinib run-in phase, patients who meet eligibility criteria will be treated with ruxolitinib and thalidomide orally on days 1-28 of a 28 day cycle. Cycles will be continued until the patient wishes to be removed from the study, unacceptable toxicity develops, disease progression, treating physician recommends removal, or termination of study occurs. Receives: Drug: Ruxolitinib; Drug: Thalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
best objective response rate (ORR)
(ORR; complete response, partial response, and clinical improvement by IWG-MRT) in the first six cycles of the combination therapy. Clinical improvement for this endpoint will be defined as the change in anemia, spleen, and symptom response from the time of the initiation the combination therapy. The ORR will be defined as the best response by the completion of cycle 6 of combination therapy.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2017
- Study start27 Feb 2017
- Primary completionFeb 2027 (estimated)
- Study completionFeb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MyelofibrosisStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- This is a multicenter, two stage phase II trial designed to assess the effect of ruxolitinib and thalidomide combination in subjects with Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis (PMF, post-PV MF, or post-ET MF).
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Incyte Corporation, Celgene, M.D. Anderson Cancer Center, Yale University
- Responsible party
- Sponsor
- Investigators
- Raajit Rampal, MD, PhD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03069326
- Sponsor's protocol ID
- 16-1498
- Status last verified
- Apr 2026
- First submitted
- 27 Feb 2017
- Last updated
- 13 Apr 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Ruxolitinib And Thalidomide Combination as a Therapy for Patients With Myelofibrosis
- ClinicalTrials.gov ID
- NCT03069326
- Acronym / sponsor ID
- 16-1498
- Conditions
- Myelofibrosis
- Keywords
- Ruxolitinib, Thalidomide, 16-1498
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- Incyte Corporation, Celgene, M.D. Anderson Cancer Center, Yale University
- Study start
- 27 Feb 2017 (actual)
- Primary completion
- Feb 2027 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 3 Mar 2017 (actual)
- Results
- Not posted in the source record
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 test any good and bad effects of the study drugs called ruxolitinib and thalidomide. Ruxolitinib and thalidomide could shrink the cancer, but it could also cause side effects.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This is a multicenter, two stage phase II trial designed to assess the effect of ruxolitinib and thalidomide combination in subjects with Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis (PMF, post-PV MF, or post-ET MF).
| Arm | Type | Receives |
|---|---|---|
| Cohort A: Ruxolitinib and Thalidomide | Experimental | Drug: Ruxolitinib, Drug: ThalidomideAfter 3 cycles of ruxolitinib treatment, either prior to study enrollment or through the ruxolitinib run-in phase, patients who meet eligibility criteria will be treated with ruxolitinib and thalidomide orally on days 1-28 of a 28 day cycle. Cycles will be continued until the patient wishes to be removed from the study, unacceptable toxicity develops, disease progression, treating physician recommends removal, or termination of study occurs. |
| Cohort B: Ruxolitinib and Thalidomide | Experimental | Drug: Ruxolitinib, Drug: ThalidomideA cohort expansion, for patients with baseline thrombocytopenia, will enroll 35 additional patients After 3 cycles of ruxolitinib treatment, either prior to study enrollment or through the ruxolitinib run-in phase, patients who meet eligibility criteria will be treated with ruxolitinib and thalidomide orally on days 1-28 of a 28 day cycle. Cycles will be continued until the patient wishes to be removed from the study, unacceptable toxicity develops, disease progression, treating physician recommends removal, or termination of study occurs. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Diagnosis of myelofibrosis (either primary or post essential thrombocythemia/polycythemia vera) requiring therapy, including those previously treated and relapsed or refractory, or if newly diagnosed, with intermediate-1 or -2 or high risk IPSS, DIPSS, DIPSS+, MIPSS70 or MIPSS70+ v2.0 score
- Patients taking Ruxolitinib at the time of enrollment must have been taking Ruxolitinib for a minimum of 3 months, and must have been on a stable dose of Ruxolitinib for a minimum of 4 weeks immediately prior to enrollment. However, for patients in the thrombocytopenic cohort A expansion, patients taking Ruxolitinib at the time of enrollment who are deemed to have a suboptimal response or are refractory to Ruxolitinib single-agent therapy (less than partial response per IWG criteria) must have been taking Ruxolitinib for a minimum of 6 weeks, and must have been on a stable dose of Ruxolitinib for a minimum of 4 weeks immediately prior to enrollment
- Patients taking Ruxolitinib at the time of enrollment must be deemed to have had a suboptimal response (less than partial response per IWG criteria) to Ruxolitinib single-agent therapy or deemed to have progression of disease (per IWG criteria).
- Age ≥ 18 years at the time of signing the informed consent.
- ECOG performance status 0 to 2.
Key exclusion criteria (10 listed)
- Use of any other standard anti-neoplastic drug or growth factor (e.g., anagrelide, G-CSF, revlimid, clofarabine) except hydroxyurea or experimental drugs, with the exception of Ruxolitinib, less than 14 days or 5-half lives prior to starting study therapy and/or lack of recovery from all toxicity from previous therapy to grade 1 or better.
- Known prior clinically relevant hypersensitivity reaction to Thalidomide, including the development of erythema nodosum if characterized by a desquamating rash.
- Prior therapy with Thalidomide in combination with Ruxolitinib
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form, which places the subject at unacceptable risk if he/she were to participate in the study or which confounds the ability to interpret data from the study.
- Pregnant or lactating females.
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 |
|---|---|---|
| RuxolitinibRuxolitinib will be given orally in an outpatient setting unless the patient is being seen inpatient for another reason. Ruxolitinib will be given continuously orally daily in 28-day cycles. | Drug | Cohort A: Ruxolitinib and Thalidomide, Cohort B: Ruxolitinib and Thalidomide |
| ThalidomideThalidomide will be given orally in an outpatient setting unless the patient is being seen inpatient for another reason. thalidomide will be given continuously orally daily in 28-day cycles. | Drug | Cohort A: Ruxolitinib and Thalidomide, Cohort B: Ruxolitinib and Thalidomide |
Primary outcome
best objective response rate (ORR)
Time frame: 1 year
(ORR; complete response, partial response, and clinical improvement by IWG-MRT) in the first six cycles of the combination therapy. Clinical improvement for this endpoint will be defined as the change in anemia, spleen, and symptom response from the time of the initiation the combination therapy. The ORR will be defined as the best response by the completion of cycle 6 of combination therapy.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2026.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
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A Study to Compare Elritercept to Placebo in Adults With Myelofibrosis and Anemia Who Are Taking Ruxolitinib
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Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis
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Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis
Primary Myelofibrosis, Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis
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.
Raajit Rampal, MD, PhD principal investigator
Memorial Sloan Kettering Cancer Center
7 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 Ruxolitinib and Thalidomide in people with Myelofibrosis, run by Memorial Sloan Kettering Cancer Center at 7 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 13 inclusion and 10 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: under way, not looking for participants, as of the sponsor's last update on 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 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?
This study is still under way. Results are not available in the source record yet.
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?
best objective response rate (ORR) (time frame: 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 13 Apr 2026.