Stopped: Low accrual
This study assesses the potential of using a TGFβ receptor inhibitor for the treatment of anemic patients with myeloproliferative neoplasms. TGFβ signaling is known to be abnormally high in patients with myeloproliferative neoplasms and it is thought that abnormal TGFβ signals cause many of the problems with blood cell formation in these diseases. The study design allows all patients to receive the study drug, vactosertib. The dose of vactosertib is individualized within a pre-set range based upon its effectiveness and tolerability. A total of up to 37 patients will be treated.
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Identify the Safest, Minimally Effective Starting Dose Level for Patients on Tier 1
Timeframe: Baseline to week 16
Identify Dose Limiting Toxicities (DLTs) in Patients With MPN Enrolled on Tier 1
Timeframe: Baseline to week 12
Identify the Maximum Tolerated Dose (MTD) of Vactosertib in Patients With MPN Enrolled on Tier 1
Timeframe: Baseline to week 12
Number of Tier 2 Patients Who Have Achieved Erythropoietic Response as Defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Criteria
Timeframe: baseline to week 16
Number of Tier 2 Patients Who Have Achieved Clinical Response in Symptoms as Defined by International Working Group (IWG) Criteria
Timeframe: baseline to week 16
Number of Tier 2 Patients Who Have Achieved Splenic Response in Symptoms as Defined by International Working Group (IWG) Criteria
Timeframe: baseline to week 16
Identify Dose Limiting Toxicities (DLTs) in Patients With MPN Enrolled on Tier 2
Timeframe: baseline to week 12
Identify the Maximum Tolerated Dose (MTD) of Vactosertib in Patients With MPN Enrolled on Tier 2
Timeframe: baseline to week 12