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NCT01375140

Ruxolitinib and Lenalidomide for Patients With Myelofibrosis

Completed Phase 2 Results posted Last updated 8 June 2025
What this trial tests

Phase 2 trial testing Ruxolitinib in Myeloproliferative Diseases in 31 participants. Completed in 5 September 2018.

Timeline
22 September 2011
Primary endpoint
5 September 2018
5 September 2018

Quick facts

Lead sponsorM.D. Anderson Cancer Center
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment31
Start date22 September 2011
Primary completion5 September 2018
Estimated completion5 September 2018
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

M.D. Anderson Cancer Center — full company profile →

Who can join

18 and older, any sex, with Myeloproliferative Diseases. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Participants With Objective Response Primary · 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

GroupValue95% CI
Ruxolitinib + Lenalidomide7

Adverse events — posted to ClinicalTrials.gov

Time frame: 6 years, 5 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Ruxolitinib + Lenalidomide
Serious: 19/31 (61%)
Deaths: 2/31

Serious adverse events (27 terms)

ReactionSystemRuxolitinib + Lenalidomide
Lung InfectionInfections and infestations
Skin InfectionInfections and infestations
FeverGeneral disorders
Thromboembolic eventVascular disorders
Neutropenic FeverInfections and infestations
AnemiaBlood and lymphatic system disorders
Multi-Organ FailureGeneral disorders
Pleural EffusionRespiratory, thoracic and mediastinal disorders
Superior Vena Cava SyndromeVascular disorders
Chest painCardiac disorders
Duodenal InfectionInfections and infestations
Myocardial InfarctionCardiac disorders
Unspecified neoplasmNeoplasms benign, malignant and unspecified (incl cysts and polyps)
NeutropeniaBlood and lymphatic system disorders
Soft Tissue InfectionInfections and infestations
Device related infectionInfections and infestations
Back PainMusculoskeletal and connective tissue disorders
HyperuricemiaMetabolism and nutrition disorders
Tumor Lysis SyndromeMetabolism and nutrition disorders
ArthralgiaMusculoskeletal and connective tissue disorders
HypercalcemiaMetabolism and nutrition disorders
Acute kidney injuryRenal and urinary disorders
Respiratory FailureRespiratory, thoracic and mediastinal disorders
SepsisInfections and infestations
Transient ischemic AttacksNervous system disorders
Other adverse events (16 terms — click to expand)

ReactionSystemRuxolitinib + Lenalidomide
ThrombocytopeniaBlood and lymphatic system disorders
AnemiaBlood and lymphatic system disorders
DiarrheaGastrointestinal disorders
Elevated ASTInvestigations
NeutropeniaBlood and lymphatic system disorders
Elevated ALTInvestigations
HyperbilirubinemiaInvestigations
Elevated CreatinineInvestigations
Abdominal PainGastrointestinal disorders
ConstipationGastrointestinal disorders
HyperglycemiaMetabolism and nutrition disorders
Dry SkinSkin and subcutaneous tissue disorders
Ecchymosis-upper extremityBlood and lymphatic system disorders
Edema limbGeneral disorders
NauseaGastrointestinal disorders
Peripheral Neuropathy MotorNervous system disorders

Most-reported serious reactions: Lung Infection, Skin Infection, Fever, Thromboembolic event, Neutropenic Fever, Anemia, Multi-Organ Failure, Pleural Effusion.

Data from ClinicalTrials.gov NCT01375140 adverse events section.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. A phase II study of decitabine and gemtuzumab ozogamicin in newly diagnosed and relapsed acute myeloid leukemia and high-risk myelodysplastic syndrome.
    Daver N, Kantarjian H, Ravandi F, Estey E, et al · · 2016 · cited 63× · PMID 26365212 · DOI 10.1038/leu.2015.244
  2. Myelofibrosis in 2019: moving beyond JAK2 inhibition.
    Schieber M, Crispino JD, Stein B. · · 2019 · cited 54× · PMID 31511492 · DOI 10.1038/s41408-019-0236-2
  3. Ruxolitinib in combination with lenalidomide as therapy for patients with myelofibrosis.
    Daver N, Cortes J, Newberry K, Jabbour E, et al · · 2015 · cited 47× · PMID 26088933 · DOI 10.3324/haematol.2015.126821
  4. Beyond Ruxolitinib: Fedratinib and Other Emergent Treatment Options for Myelofibrosis.
    Bewersdorf JP, Jaszczur SM, Afifi S, Zhao JC, et al · · 2019 · cited 28× · PMID 31920387 · DOI 10.2147/cmar.s212559
  5. Historical views, conventional approaches, and evolving management strategies for myeloproliferative neoplasms.
    Stein BL, Gotlib J, Arcasoy M, Nguyen MH, et al · · 2015 · cited 19× · PMID 25870379 · DOI 10.6004/jnccn.2015.0058
  6. Guidelines for the management of myeloproliferative neoplasms.
    Choi CW, Bang SM, Jang S, Jung CW, et al · · 2015 · cited 18× · PMID 26552452 · DOI 10.3904/kjim.2015.30.6.771
  7. Finding a Jill for JAK: Assessing Past, Present, and Future JAK Inhibitor Combination Approaches in Myelofibrosis.
    Kuykendall AT, Horvat NP, Pandey G, Komrokji R, et al · · 2020 · cited 15× · PMID 32823910 · DOI 10.3390/cancers12082278
  8. Targeting phosphatidylinositol-3-kinase pathway for the treatment of Philadelphia-negative myeloproliferative neoplasms.
    Pandey R, Kapur R. · · 2015 · cited 15× · PMID 26062813 · DOI 10.1186/s12943-015-0388-z

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