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A Two-tiered, Phase II, Rule-based, Intra-patient Dose Escalation Study to Investigate Safety and Feasibility of Vactosertib (TEW-7197) for the Treatment of Anemic Patients with Philadelphia Chromosome-negative Myeloproliferative Neoplasms

Clinical Trial Details

Currently, the treatments of anemia in patients with myeloproliferative neoplasms (MPNs) include blood transfusions (receiving red blood cells intravenously to replace the lack of functioning red blood cells), erythropoietin stimulating agents given as injections which will help stimulate red blood cell production in the bone marrow, and sometimes steroids. These interventions are supportive but are often insufficient and temporary as they are not capable of blocking the effects of the cancer cells causing anemia. 
   
The purpose of this study is to assess how well vactosertib works in MPNs to improve anemia. This study will also provide new information about how vactosertib blocks TGFβ signaling in MPN cells and how this, in turn, can block MPN cancer cells themselves and/or their ill-effects on the bone marrow such as fibrosis.

Vactosertib is considered investigational which means it has not yet been approved by the U.S. Food and Drug Administration (FDA). 

The the dose of vactosertib will be adjusted during the study to evaluate how well vactosertib works and to ensure that the dose used is not causing unexpected toxicities. The research team will monitor patients closely during the study. 

Total study participation will last between 6 months-1 year. 

   

Key Eligibility: 
  1. Patients must be at least 18 years old
  2. Patients must meet the WHO 2016 criteria for a Ph-neg myeloproliferative neoplasm (MPN), including PV, ET, MF, MDS/MPN, MPN-U
  3. Patients with MF must have DIPSS+ Intermediate or High-risk MF (primary of post-PV/ET)
  4. Patients should be on a stable dose of current cytoreductive therapy for at least 3 months prior to C1D1

Detailed eligibility will be reviewed when you contact the study team

Study contact by location

Upper East Side - Manhattan

Contact(s)

Tania J. Curcio, NP, FNP-BC
(212) 746-2571
tjc9003@med.cornell.edu

Primary Investigator(s)

Protocol ID(s)

Weill Cornell Medicine IRB #:

1906020285

ClinicalTrials.gov:

NCT04103645

Sponsor:

Weill Cornell Medical College

Status

Open to Enrollment

Age Group

Adult

Sponsor