Myeloproliferative neoplasm in accelerated phase (MPN-AP)
Conditions
Brief summary
Phase I: physical examinations, laboratory tests, AEs, serious AEs (SAEs), ECGs, and vital signs. - DLTs (dose limiting toxicity) will be used to establish the MTD of fedratinib in combination with CC-486. The SRC will determine the RP2D and schedule based on safety data of the combination of fedratinib and CC-486. Phase II: - Best response to the combination therapy at week 24 according to the IWG-MRT and the ELN consensus report. This includes CI, PR and CR
Detailed description
duration of response - Change in MF-associated symptoms measured by MFSAF - Time from the first observation of best symptom response to first documented loss of symptom response - Spleen response by palpation - Time from the first treatment dose to disease progression, relapse or death - proportion of subjects who transit to allogeneic SCT - proportion of subjects that demonstrate an anemia - Analysis of incidence of subjects with a CTCAE Grade ≥3 of nausea, diarrhea, vomiting or occurrence o
Interventions
Sponsors
Martin-Luther-Universitaet Halle-Wittenberg
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Phase I: physical examinations, laboratory tests, AEs, serious AEs (SAEs), ECGs, and vital signs. - DLTs (dose limiting toxicity) will be used to establish the MTD of fedratinib in combination with CC-486. The SRC will determine the RP2D and schedule based on safety data of the combination of fedratinib and CC-486. Phase II: - Best response to the combination therapy at week 24 according to the IWG-MRT and the ELN consensus report. This includes CI, PR and CR | — |
Secondary
| Measure | Time frame |
|---|---|
| duration of response - Change in MF-associated symptoms measured by MFSAF - Time from the first observation of best symptom response to first documented loss of symptom response - Spleen response by palpation - Time from the first treatment dose to disease progression, relapse or death - proportion of subjects who transit to allogeneic SCT - proportion of subjects that demonstrate an anemia - Analysis of incidence of subjects with a CTCAE Grade ≥3 of nausea, diarrhea, vomiting or occurrence o | — |
Countries
Germany
Outcome results
None listed