Trial Outcomes & Findings for Ruxolitinib and Lenalidomide for Patients With Myelofibrosis (NCT NCT01375140)

Last Updated: 2025-06-08

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

31 participants

Primary outcome timeframe

3 cycles (28 days each) up to 3 months

Results posted on

2025-06-08

Participant Flow

Recruitment Period: September 2011- August 2013

Participant milestones

Participant milestones
Measure	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. Ruxolitinib: 15 mg by mouth twice daily (BID), continuously in 28-day cycles. Lenalidomide: 5 mg by mouth each day on days 1-21, followed by 7 days of no therapy of each 28 day cycle. 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.
Overall Study STARTED	31
Overall Study COMPLETED	31
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Ruxolitinib and Lenalidomide for Patients With Myelofibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ruxolitinib + Lenalidomide n=31 Participants 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. Ruxolitinib: 15 mg by mouth twice daily (BID), continuously in 28-day cycles. Lenalidomide: 5 mg by mouth each day on days 1-21, followed by 7 days of no therapy of each 28 day cycle. 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.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	14 Participants n=5 Participants
Age, Categorical >=65 years	17 Participants n=5 Participants
Age, Continuous	66 years n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants
Race (NIH/OMB) White	27 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	31 participants n=5 Participants

PRIMARY outcome

Timeframe: 3 cycles (28 days each) up to 3 months

Outcome measures

Outcome measures
Measure	Ruxolitinib + Lenalidomide n=31 Participants 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. Ruxolitinib: 15 mg by mouth twice daily (BID), continuously in 28-day cycles. Lenalidomide: 5 mg by mouth each day on days 1-21, followed by 7 days of no therapy of each 28 day cycle. 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.
Participants With Objective Response	7 Participants

Adverse Events

Ruxolitinib + Lenalidomide

Serious events: 19 serious events

Other events: 31 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Ruxolitinib + Lenalidomide n=31 participants at risk 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. Ruxolitinib: 15 mg by mouth twice daily (BID), continuously in 28-day cycles. Lenalidomide: 5 mg by mouth each day on days 1-21, followed by 7 days of no therapy of each 28 day cycle. 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.
Infections and infestations Neutropenic Fever	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Blood and lymphatic system disorders Anemia	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Infections and infestations Skin Infection	9.7% 3/31 • Number of events 9 • 6 years, 5 months
Infections and infestations Lung Infection	25.8% 8/31 • Number of events 12 • 6 years, 5 months
General disorders Multi-Organ Failure	3.2% 1/31 • Number of events 1 • 6 years, 5 months
General disorders Fever	9.7% 3/31 • Number of events 3 • 6 years, 5 months
Respiratory, thoracic and mediastinal disorders Pleural Effusion	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Vascular disorders Superior Vena Cava Syndrome	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Vascular disorders Thromboembolic event	6.5% 2/31 • Number of events 2 • 6 years, 5 months
Cardiac disorders Chest pain	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Infections and infestations Duodenal Infection	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Cardiac disorders Myocardial Infarction	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Unspecified neoplasm	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Blood and lymphatic system disorders Neutropenia	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Infections and infestations Soft Tissue Infection	3.2% 1/31 • Number of events 2 • 6 years, 5 months
Infections and infestations Device related infection	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Musculoskeletal and connective tissue disorders Back Pain	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Metabolism and nutrition disorders Hyperuricemia	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Metabolism and nutrition disorders Tumor Lysis Syndrome	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Musculoskeletal and connective tissue disorders Arthralgia	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Metabolism and nutrition disorders Hypercalcemia	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Renal and urinary disorders Acute kidney injury	3.2% 1/31 • Number of events 2 • 6 years, 5 months
Respiratory, thoracic and mediastinal disorders Respiratory Failure	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Infections and infestations Sepsis	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Nervous system disorders Transient ischemic Attacks	3.2% 1/31 • Number of events 2 • 6 years, 5 months
General disorders Death	3.2% 1/31 • Number of events 1 • 6 years, 5 months
Infections and infestations Wound Infection	3.2% 1/31 • Number of events 1 • 6 years, 5 months

Other adverse events

Other adverse events
Measure	Ruxolitinib + Lenalidomide n=31 participants at risk 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. Ruxolitinib: 15 mg by mouth twice daily (BID), continuously in 28-day cycles. Lenalidomide: 5 mg by mouth each day on days 1-21, followed by 7 days of no therapy of each 28 day cycle. 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.
Gastrointestinal disorders Abdominal Pain	9.7% 3/31 • Number of events 3 • 6 years, 5 months
Gastrointestinal disorders Constipation	9.7% 3/31 • Number of events 3 • 6 years, 5 months
Gastrointestinal disorders Diarrhea	25.8% 8/31 • Number of events 8 • 6 years, 5 months
Skin and subcutaneous tissue disorders Dry Skin	6.5% 2/31 • Number of events 2 • 6 years, 5 months
Blood and lymphatic system disorders Ecchymosis-upper extremity	6.5% 2/31 • Number of events 2 • 6 years, 5 months
General disorders Edema limb	6.5% 2/31 • Number of events 2 • 6 years, 5 months
Blood and lymphatic system disorders Neutropenia	19.4% 6/31 • Number of events 6 • 6 years, 5 months
Blood and lymphatic system disorders Anemia	41.9% 13/31 • Number of events 13 • 6 years, 5 months
Blood and lymphatic system disorders Thrombocytopenia	51.6% 16/31 • Number of events 16 • 6 years, 5 months
Investigations Elevated ALT	16.1% 5/31 • Number of events 5 • 6 years, 5 months
Investigations Elevated AST	25.8% 8/31 • Number of events 8 • 6 years, 5 months
Investigations Hyperbilirubinemia	16.1% 5/31 • Number of events 5 • 6 years, 5 months
Investigations Elevated Creatinine	12.9% 4/31 • Number of events 4 • 6 years, 5 months
Metabolism and nutrition disorders Hyperglycemia	9.7% 3/31 • Number of events 3 • 6 years, 5 months
Gastrointestinal disorders Nausea	6.5% 2/31 • Number of events 2 • 6 years, 5 months
Nervous system disorders Peripheral Neuropathy Motor	6.5% 2/31 • Number of events 2 • 6 years, 5 months

Additional Information

Srdan Verstovsek, MD/Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-745-3429

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place