Trial Outcomes & Findings for Ruxolitinib Phosphate to Treat Diffuse Large B-Cell or Peripheral T-Cell Non-Hodgkin Lymphoma After Stem Cell Transplant (NCT NCT01431209)

NCT ID: NCT01431209

Last Updated: 2023-10-05

Results Overview

Number of patients achieving overall response rate

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

71 participants

Primary outcome timeframe

24 weeks

Results posted on

2023-10-05

Participant Flow

Evaluable population - Subjects who had completed 2 cycles of therapy with response assessments or progressed before the evaluation of the primary endpoint. Primary endpoint The primary objective of the study is to evaluate subject overall response rate (ORR) after 6 cycles for each of the subject cohorts. The description provided in this section includes the primary analyses for the primary objective.

For this study 71 subjects were screened and consented for the study. Nine were considered ineligible by the principal investigator and were not treated and removed from the study. Two other subjects were also not included in the data analysis. One withdrew consent prior to treatment. One was determined to have disease progression before treatment started.

Participant milestones

Participant milestones
Measure	Diffuse Large B-cell Lymphoma (DLBCL) Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO	Peripheral T-cell Non-Hodgkin Lymphoma (PTCL) Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO
Overall Study STARTED	47	13
Overall Study COMPLETED	43	12
Overall Study NOT COMPLETED	4	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Ruxolitinib Phosphate to Treat Diffuse Large B-Cell or Peripheral T-Cell Non-Hodgkin Lymphoma After Stem Cell Transplant

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Diffuse Large B-cell Lymphoma (DLBCL) n=47 Participants Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO	Peripheral T-cell Non-Hodgkin Lymphoma (PTCL) n=13 Participants Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO	Total n=60 Participants Total of all reporting groups
Age, Continuous	64 years STANDARD_DEVIATION 11 • n=5 Participants	66 years STANDARD_DEVIATION 13 • n=7 Participants	65 years STANDARD_DEVIATION 12 • n=5 Participants
Sex: Female, Male Female	18 Participants n=5 Participants	5 Participants n=7 Participants	23 Participants n=5 Participants
Sex: Female, Male Male	29 Participants n=5 Participants	8 Participants n=7 Participants	37 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	40 Participants n=5 Participants	12 Participants n=7 Participants	52 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment United States	47 participants n=5 Participants	13 participants n=7 Participants	60 participants n=5 Participants

PRIMARY outcome

Timeframe: 24 weeks

Number of patients achieving overall response rate

Outcome measures

Outcome measures
Measure	Diffuse Large B-cell Lymphoma (DLBCL) n=47 Participants Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO	Peripheral T-cell Non-Hodgkin Lymphoma (PTCL) n=13 Participants Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO
Overall Response Rate Complete Response	1 Participants	1 Participants
Overall Response Rate Partial Response	3 Participants	1 Participants
Overall Response Rate Stable disease	5 Participants	3 Participants
Overall Response Rate Progressive disease	34 Participants	7 Participants
Overall Response Rate Not assessed	4 Participants	1 Participants

SECONDARY outcome

Timeframe: From the date of start of treatment to date of death due to any cause, assessed up to 60 months

The Kaplan-Meier method will be used to estimate the median OS time and its 95% CI.

Outcome measures

Outcome measures
Measure	Diffuse Large B-cell Lymphoma (DLBCL) n=47 Participants Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO	Peripheral T-cell Non-Hodgkin Lymphoma (PTCL) n=13 Participants Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO
Overall Survival (OS)	5.9 Months Interval 3.3 to 14.3	6.9 Months Interval 3.8 to 24.3

SECONDARY outcome

Timeframe: From the date of start of treatment to the date of event defined as the first documented progression or death due to any cause, assessed up to 60 months.

The Kaplan-Meier method will be used to estimate the median PFS time and its 95% CI.

Outcome measures

Outcome measures
Measure	Diffuse Large B-cell Lymphoma (DLBCL) n=47 Participants Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO	Peripheral T-cell Non-Hodgkin Lymphoma (PTCL) n=13 Participants Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO
Progression-free Survival	1.8 Months Interval 1.1 to 1.8	2.2 Months Interval 0.9 to 4.6

Adverse Events

Diffuse Large B-cell Lymphoma (DLBCL)

Serious events: 22 serious events

Other events: 43 other events

Deaths: 31 deaths

Peripheral T-cell Non-Hodgkin Lymphoma (PTCL)

Serious events: 7 serious events

Other events: 12 other events

Deaths: 11 deaths

Serious adverse events

Other adverse events

Additional Information

Dr. Julie M. Vose

University of Nebraska Medical Center

Phone: 402-559-3848

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Diffuse Large B-cell Lymphoma (DLBCL) n=47 participants at risk Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO	Peripheral T-cell Non-Hodgkin Lymphoma (PTCL) n=13 participants at risk Patients receive ruxolitinib phosphate PO BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Ruxolitinib Phosphate: Given PO
Respiratory, thoracic and mediastinal disorders Respiratory, thoracic and mediastinal disorder - Other	0.00% 0/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
General disorders fatigue	4.3% 2/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	15.4% 2/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Metabolism and nutrition disorders hyponatremia	0.00% 0/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	15.4% 2/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Blood and lymphatic system disorders anemia	23.4% 11/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Respiratory, thoracic and mediastinal disorders apnea	0.00% 0/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Injury, poisoning and procedural complications Fall	2.1% 1/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Blood and lymphatic system disorders febrile neutropenia	0.00% 0/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
General disorders fever	4.3% 2/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Metabolism and nutrition disorders hypercalcemia	4.3% 2/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Metabolism and nutrition disorders hypophosphatemia	4.3% 2/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Vascular disorders hypotension	4.3% 2/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Respiratory, thoracic and mediastinal disorders lung infection	2.1% 1/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Investigations lymphocyte count decreased	8.5% 4/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
General disorders malaise	0.00% 0/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Musculoskeletal and connective tissue disorders Musculoskeletal, Other	0.00% 0/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Gastrointestinal disorders nausea	2.1% 1/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Respiratory, thoracic and mediastinal disorders pneumonitis	2.1% 1/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Skin and subcutaneous tissue disorders pruritis	0.00% 0/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Skin and subcutaneous tissue disorders skin ulceration	0.00% 0/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	7.7% 1/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Investigations platelet count decreased	10.6% 5/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	0.00% 0/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Respiratory, thoracic and mediastinal disorders dyspnea	8.5% 4/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	0.00% 0/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Investigations neutrophil count decreased	8.5% 4/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	0.00% 0/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.
Infections and infestations sepsis	6.4% 3/47 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.	0.00% 0/13 • Adverse Event monitoring should be continued for at least 30 days following the last dose of study treatment. Since this treatment trial was for relapsed or refractory disease in subjects, subjects were allowed to continue study treatment as long as they received benefit and met not reason for study discontinuation. The last subject remained on treatment for 7 1/2 years.