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Ruxolitinib and Lenalidomide for Patients With Myelofibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01375140
Recruitment Status : Completed
First Posted : June 17, 2011
Results First Posted : July 31, 2019
Last Update Posted : July 31, 2019
Incyte Corporation
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:

The goal of this clinical research study is to learn if the combination of ruxolitinib and lenalidomide can help to control MF. The safety of this study drug combination will also be studied.

Ruxolitinib is designed to stop certain proteins (called JAK1 and JAK2) that are found in MF cells from sending signals that may lead to the growth of cancer cells.

Lenalidomide is designed to change the body's immune system. It may also interfere with the development of tiny blood vessels that help support tumor growth. This may decrease the growth of cancer cells.

Condition or disease Intervention/treatment Phase
Myeloproliferative Diseases Drug: Ruxolitinib Drug: Lenalidomide Drug: Prednisone Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 31 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Evaluation of Ruxolitinib and Lenalidomide Combination as a Therapy for Patients With Myelofibrosis
Actual Study Start Date : September 22, 2011
Actual Primary Completion Date : September 5, 2018
Actual Study Completion Date : September 5, 2018

Arm Intervention/treatment
Experimental: Ruxolitinib + Lenalidomide
Ruxolitinib 15 mg orally twice daily continuously + Lenalidomide orally 5 mg/day on days 1-21, followed by 7 days of no therapy (28-day cycle). Prednisone will be added for patients who have not responded after 3 cycles of therapy. Prednisone 30 mg by mouth a day during cycle 4, 15 mg/day during cycle 5, and 15 mg every other day during cycle 6, and then it will be discontinued.
Drug: Ruxolitinib
15 mg by mouth twice daily (BID), continuously in 28-day cycles.
Other Name: INCB018424

Drug: Lenalidomide
5 mg by mouth each day on days 1-21, followed by 7 days of no therapy of each 28 day cycle.
Other Names:
  • CC-5013
  • Revlimid

Drug: Prednisone

Prednisone will be added for patients who have not responded after 3 cycles of therapy.

30 mg by mouth a day during cycle 4, 15 mg/day during cycle 5, and 15 mg every other day during cycle 6, and then it will be discontinued.

Primary Outcome Measures :
  1. Participants With Objective Response [ Time Frame: 3 cycles (28 days each) up to 3 months ]
    To determine the efficacy of the combination of Ruxolitinib + Lenalidomide in patients with Myelofibrosis (MF). Objective response rate equals Complete and Partial Response, and Clinical Improvement as defined by International Working Group for Myelofibrosis Research and Treatment (IWG-MRT). Objective response rate (ORR), defined as a clinical improvement (CI), partial remission (PR), and complete remission (CR) according to the International Working Group (IWG) Criteria. Complete remission (CR): bone marrow blasts <5%, hemoglobin >/= 10, absolute neutrophil count (ANC) >/= 1000, platelets >/= 100, <2% immature myeloid cell, spleen and liver not palpable. Partial Response (PR): CR plus one or more of the following: ANC >/= 1000, decreased platelets by 50%, hemoglobin >/= 8.5 but < 10, <2% immature myeloid cells. Clinical improvement (CI): hemoglobin increase of 2g/dl, transfusion independence or reduction splenomegaly and/or hepatomegaly >/= 50%, >/=50% reduction in MPN-SAF TSS

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. 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 according to International Working Group (IWG) criteria.
  2. Understanding and voluntary signing an IRB-approved informed consent form.
  3. Age equal to or greater than 18 years at the time of signing the informed consent.
  4. Disease-free of prior malignancies for equal to or greater than 2 years with exception of basal cell or squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast.
  5. ECOG performance status 0 to 2.
  6. Patients must have adequate organ function as demonstrated by the following: a. Direct bilirubin equal to or less than 2.0 mg/dL, b. Serum creatinine equal to or less than 2.0 mg/dL., c. SGPT equal to or less than 3 x upper limit of normal
  7. Females of childbearing potential (FCBP)(A female of childbearing potential is a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months)
  8. Cont. from 7: must have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL within 10 to 14 days prior to and again within 24 hours of starting lenalidomide 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 lenalidomide. 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.
  9. All study participants must be registered into the mandatory RevAssist® program, and be willing and able to comply with the requirements of RevAssist®.
  10. Platelets >/= 100000/uL
  11. ANC >/= 1000/uL

Exclusion Criteria:

  1. Use of any other standard (e.g. hydroxyurea, anagrelide, growth factors) or experimental drug or therapy within 14 days or 5-half lives, whichever is longer, of starting study therapy and/or lack of recovery from all toxicity from previous therapy to grade 1 or better.
  2. Known prior clinically relevant hypersensitivity reaction to thalidomide, including the development of erythema nodosum if characterized by a desquamating rash.
  3. Prior therapy with lenalidomide or ruxolitinib.
  4. Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
  5. Suspected Pregnancy, Pregnant or lactating females.
  6. 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.
  7. Known positive for HIV or infectious hepatitis, type A, B or C.
  8. Known prior clinically relevant hypersensitivity to prednisone.
  9. Participants with prior history of thromboembolic disease (i.e. deep venous thrombosis (DVT) or pulmonary embolism (PE) within the last six months, as Lenalidomide has demonstrated a significantly increased risk of DVT or PE.
  10. Known to have a hypercoagulability syndrome (eg: antithrombin III, deficiency, anticardiolipin syndrome etc…)
  11. Concurrent use of strong inducers or strong inhibitors of CYP3A4 (strong inducers are rifampin and St. John's Worth, carbamazepine, phenytoin, and barbiturates such as phenobarbital; strong inhibitors are HIV-antivirals, clarythromycin, itraconazole, ketoconazole, nefazodone, and telithromycin).
  12. Incarcerated persons are excluded from the protocol.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01375140

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United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Incyte Corporation
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Principal Investigator: Srdan Verstovsek, MD M.D. Anderson Cancer Center
  Study Documents (Full-Text)

Documents provided by M.D. Anderson Cancer Center:
Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: M.D. Anderson Cancer Center Identifier: NCT01375140    
Other Study ID Numbers: 2011-0269
NCI-2011-01126 ( Registry Identifier: NCI CTRP )
First Posted: June 17, 2011    Key Record Dates
Results First Posted: July 31, 2019
Last Update Posted: July 31, 2019
Last Verified: July 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by M.D. Anderson Cancer Center:
Myeloproliferative Diseases
Post essential thrombocythemia/polycythemia vera
Interactive Voice Response System
Additional relevant MeSH terms:
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Primary Myelofibrosis
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Anti-Inflammatory Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Immunologic Factors
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors