Trial Outcomes & Findings for Efficacy and Safety of Lenalidomide in Combination With Cyclosporine A in Patients With Myelodysplastic Syndromes (NCT NCT00840827)

Results Overview

Efficacy of lenalidomide + CSA will be evaluated as a function of red blood cell transfusion needs improvement (≥ 50% decrease in RBC transfusion requirements after 16 weeks of study treatment).

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

16 weeks

Results posted on

2019-11-19

Participant Flow

Participant milestones

Participant milestones
Measure	All Patients Lenalidomide 10mg po daily/ CSA 250mg orally twice daily lenalidomide: Lenalidomide 10mg po daily cyclosporine A: CSA 250mg orally twice daily
Overall Study STARTED	6
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	All Patients Lenalidomide 10mg po daily/ CSA 250mg orally twice daily lenalidomide: Lenalidomide 10mg po daily cyclosporine A: CSA 250mg orally twice daily
Overall Study Adverse Event	1
Overall Study Lack of Efficacy	4
Overall Study Physician Decision	1

Baseline Characteristics

Efficacy and Safety of Lenalidomide in Combination With Cyclosporine A in Patients With Myelodysplastic Syndromes

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Patients n=6 Participants Lenalidomide 10mg po daily/ CSA 250mg orally twice daily lenalidomide: Lenalidomide 10mg po daily cyclosporine A: CSA 250mg orally twice daily
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=93 Participants
Age, Categorical >=65 years	4 Participants n=93 Participants
Sex: Female, Male Female	2 Participants n=93 Participants
Sex: Female, Male Male	4 Participants n=93 Participants

PRIMARY outcome

Timeframe: 16 weeks

Population: 6 patients were enrolled, the target enrollment was not met. Study was terminated by the PI and no statistical analysis was performed. None of the patients completed the 16 weeks of study treatment, therefore red blood cell transfusion could not be evaluated.

Efficacy of lenalidomide + CSA will be evaluated as a function of red blood cell transfusion needs improvement (≥ 50% decrease in RBC transfusion requirements after 16 weeks of study treatment).

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 12 months

Population: 6 patients were enrolled, the target enrollment was not met. Study was terminated by the PI and no statistical analysis was performed. None of the patients completed the 12 months of study treatment, therefore red blood cell transfusion independence could not be evaluated.

Efficacy of lenalidomide + CSA will be evaluated as a function of red blood cell transfusion independence.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Baseline and 12 months

Population: 6 patients were enrolled, the target enrollment was not met. Study was terminated by the PI and no statistical analysis was performed. None of the patients completed the 12 months of study treatment, therefore the change of hemoglobin concentration from baseline to 12 weeks could not be evaluated.

Efficacy of lenalidomide + CSA will be evaluated as a function of change of hemoglobin concentration from baseline to 12 months.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Population: 6 patients were enrolled, the target enrollment was not met. Study was terminated by the PI and no statistical analysis was performed. None of the patients completed the 12 months of study treatment, therefore the type of adverse events to lenalidomide in combination with CSA, could not be evaluated.

Safety will be evaluated by type of adverse events to lenalidomide in combination with CSA

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Population: 6 patients were enrolled, the target enrollment was never met. Study was terminated by the PI and no statistical analysis was performed. None of the patients completed the 12 months of study treatment, therefore tolerability could not be evaluated.

Tolerability will be evaluated by the frequency of adverse events to lenalidomide in combination with CSA

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Population: 6 patients were enrolled, the target enrollment was never met. Study was terminated by the PI and no statistical analysis was performed. None of the patients completed the 12 months of study treatment, therefore tolerability could not be evaluated.

Tolerability will be evaluated by the severity of adverse events to lenalidomide in combination with CSA

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Population: 6 patients were enrolled, the target enrollment was never met. Study was terminated by the PI and no statistical analysis was performed. None of the patients completed the 12 months of study treatment, therefore tolerability could not be evaluated.

Tolerability will be evaluated by the relatedness of adverse events to lenalidomide in combination with CSA

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Population: 6 patients were enrolled, the target enrollment was never met. Study was terminated by the PI and no statistical analysis was performed. None of the patients completed the 12 months of study treatment, therefore safety could not be evaluated.

Safety will be evaluated by frequency of each type of adverse event to lenalidomide in combination with CSA

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Population: 6 patients were enrolled, the target enrollment was never met. Study was terminated by the PI and no statistical analysis was performed. None of the patients completed the 12 months of study treatment, therefore safety could not be evaluated.

Safety will be evaluated by the relatedness of each type of adverse event to lenalidomide in combination with CSA

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Population: 6 patients were enrolled, the target enrollment was never met. Study was terminated by the PI and no statistical analysis was performed. None of the patients completed the 12 months of study treatment, therefore safety could not be evaluated.

Safety will be evaluated by the severity of each type of adverse event to lenalidomide in combination with CSA

Outcome measures

Outcome data not reported

Adverse Events

All Patients

Serious events: 4 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Patients n=6 participants at risk Lenalidomide 10mg po daily/ CSA 250mg orally twice daily lenalidomide: Lenalidomide 10mg po daily cyclosporine A: CSA 250mg orally twice daily
Respiratory, thoracic and mediastinal disorders Pneumonia	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
Cardiac disorders Myocardial Infarction	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
Blood and lymphatic system disorders Thrombocytopenia	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
Blood and lymphatic system disorders Cytopenia	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.

Other adverse events

Other adverse events
Measure	All Patients n=6 participants at risk Lenalidomide 10mg po daily/ CSA 250mg orally twice daily lenalidomide: Lenalidomide 10mg po daily cyclosporine A: CSA 250mg orally twice daily
Blood and lymphatic system disorders Anemia	66.7% 4/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
General disorders Fatigue	50.0% 3/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
Musculoskeletal and connective tissue disorders Leg Cramps	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
Gastrointestinal disorders Constipation	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
Skin and subcutaneous tissue disorders Skin rash	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
Gastrointestinal disorders GERD	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
Gastrointestinal disorders Intermittent Diarrhea	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
General disorders Nausea	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
General disorders Lip tingling	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.
Eye disorders Decreased Vision	16.7% 1/6 • AEs were collected from the 8th day of the study drug administration till discontinuation of study drug. An additional safety assessment was done 30 days (+/- 2 days) following the last dose of study drug.

Additional Information

Clinical Trials Administrator

Weill Cornell Medicine

Phone: 212-746-0284

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place