This is a phase II, multi-center study of pomalidomide in adult patients with Primary myelofibrosis, Secondary myelofibrosis, and unclassifiable Myeloproliferative Neoplasms showing at least grade 1 bone marrow fibrosis and requiring therapy.
Conditions
Interventions
Product Name: Pomalidomide
Product Code: CC-4047
Pharmaceutical Form: Capsule
INN or Proposed INN: Pomalidomide
CAS Number: 19171-19-8
Current Sponsor code: CC-4047
Concentration unit: mg milligram(s)
Sponsors
Universitätsklinikum Ulm
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age =50 years at the time of voluntarily signing an IRB/IEC-approved informed consent 2. Diagnosis of Myeloproliferative Neoplasms (MPN) either de novo myelofibrosis according to WHO criteria (PMF), secondary myelofibrosis (post-PV MF and post-ET MF according to the IWG-MRT consensus terminology) or unclassifiable MPN with biopsy proven myelofibrosis 3. Anemia with hemoglobin level of 11 cm diameter) and/or leukoerythroblastosis 5. Adequate organ function, i.e. ALT and/or AST =65 years) yes F.1.3.1 Number of subjects for this age range 71
Exclusion criteria
Exclusion criteria: 1. Females of childbearing potentials, pregnant or breast feeding females 2. BCR/ABL-positivity 3. Diagnosis of ET (according to WHO 2008 criteria) 4. Diagnosis of PV (according to WHO 2008 criteria) 5. >20% blasts in peripheral blood or bone marrow 6. Known positive status for HIV, HBV or HCV 7. Prior treatment with IMiDs (thalidomide, lenalidomide) or with Interferon-alpha within a 3 month time period before screening 8. History of thrombosis or pulmonary embolism 9. Peripheral neuropathy >grade 1 CTC 10. No consent for registration, storage and processing of the individual disease-characteristics and course as well as information of the family physician about study participation. 11. Presence of any medical/psychiatric condition or laboratory abnormalities which may limit full compliance with the study, increase the risk associated with study participation or study drug administration, or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the patient inappropriate for entry into this study 12. Drug or alcohol abuse within the last 6 months 13. Patients with a “currently active” second malignancy other than non-melanoma skin cancers. Patients are not considered to have a “currently active” malignancy if they have completed therapy and are considered by their physician to be at less than 30% risk of relapse within one year.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To evaluate clinical efficacy (disease response) of pomalidomide in MF patients based on the consensus criteria of the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT), extended by the criterion RBC-transfusion independence (TI);Secondary Objective: • To evaluate the safety profile of pomalidomide in MF patients • To assess predictive response parameters using cytogenetic and molecular genetic analyses including microarray-based techniques • To assess overall clinical outcome ;Primary end point(s): • Objective disease response, as defined by the IWG-MRT criteria for response in MF patients,extended by the criterion RBC-transfusion independence (TI);Timepoint(s) of evaluation of this end point: at the end of study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Overall safety profile of pomalidomide 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 • Event-free, relapse-free and overall survival ;Timepoint(s) of evaluation of this end point: Safety will be monitored by internal and external supervision. After cohorts of 38 patients,the analyses of the safety end points was performed. Twelve-monthly, a study report comprising the safety end point report, a summary as well as a complete list of serious adverse events (SAE), results of blinded central versus on-site disease assessment and a summary of study conduct to assess protocol adherence will be performed. | — |
Countries
Germany
Contacts
Public ContactInnere Medizin III Studienzentrale
Universitätsklinikum Ulm
Outcome results
None listed