Ruxolitinib and Pomalidomide Combination Therapy in Patients With Primary and Secondary MF (POMINC)
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase-Ib/II Study of Ruxolitinib and Pomalidomide Combination Therapy in Patients With Primary and Secondary Myelofibrosis|
- Best response rate within 12 treatment cycles according to the IWG-MRT criteria (including CR, PR, CI) and red cell transfusion (RCT) independency according to Gale et al 2010 and 2011). [ Time Frame: one year ]
- Overall safety profile of ruxolitinib and pomalidomide combination observed during treatment, as well as cumulative incidence of leukemic transformation [ Time Frame: one year ]Overall safety profile of ruxolitinib and pomalidomide combination characterized by type, frequency, severity (graded using the National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] Version 3.0), timing and relatedness of adverse events (AEs) and laboratory abnormalities observed during treatment, as well as cumulative incidence of leukemic transformation
- Progression-free survival [ Time Frame: three years ]
- duration of response [ Time Frame: three years ]
- overall survival [ Time Frame: three years ]
- Quality of life [ Time Frame: three years ]Quality of life assessed by the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF Protocol 5/25/11), change in ECOG performance status from study entry to each visit where the variable is measured.
|Study Start Date:||August 2013|
|Estimated Study Completion Date:||January 2021|
|Estimated Primary Completion Date:||January 2018 (Final data collection date for primary outcome measure)|
ruxolitinib treatment will be started at 10 mg twice daily, whereas the dose of pomalidomide will be 0.5 mg once daily.
For all patients the starting dose of ruxolitinib in this trial is 10mg twice daily po; pomalidomide will be administered at a permanent dose of 0.5 mg po once daily.
The proposed study is an open-label, single-arm, Phase-Ib/II trial to assess the efficacy of oral drug combination ruxolitinib and pomalidomide in primary and secondary MF patients. Dosages of the drugs are derived from previous Phase-I/II studies; ruxolitinib treatment will be started at 10 mg twice daily, whereas the dose of pomalidomide will be 0.5 mg once daily.
Dose reductions and discontinuations will be allowed in case of myelosuppressive effects.
Intra-patient dose escalation will be permitted for ruxolitinib to optimize efficacy of the therapeutic regimen; pomalidomide will be given in a permanent dosage of 0.5mg per day.
Treatment response will be evaluated continuously after each treatment cycle (1 cycle = 28 days) according to the IWG-MRT criteria expanded by the response criterion RCT-independency.
In case of progressive disease study therapy will be stopped; In patients showing response or stable disease, continuous therapy within the study is intended for a maximum of 12 treatment cycles; After completion of 12 treatment cycles, therapy can be continued if a measurable benefit of treatment is evident. This extension has to be discussed between the local and the principle investigator. Conditions leading to patient withdrawal from the study are detailed in the protocol "PATIENT WITHDRAWAL FROM STUDY PARTICIPATION".
Please refer to this study by its ClinicalTrials.gov identifier: NCT01644110
|Contact: Konstanze Doehner, MD||0049731500 ext email@example.com|
|Contact: Frank Stegelmann, MD||0049731500 ext firstname.lastname@example.org|
|University of Ulm||Recruiting|
|Ulm, Germany, 89081|
|Contact: Konstanze Doehner, MD email@example.com|
|Principal Investigator: konstanze Doehner, Md|
|Principal Investigator:||Konstanz Doehner, MD||University of Ulm|