Study on Increasing Doses of Vactosertib for Treating Anemia in Patients with Myeloproliferative Neoplasms

Sponsor: Weill Medical College of Cornell University

Sponsor score: 40
Inactive

ClinicalTrials ID:NCT04103645

Provider View

This clinical trial is exploring the safety and feasibility of using a drug called vactosertib to treat anemia in patients with myeloproliferative neoplasms (MPNs), a group of blood disorders. The study will adjust the dose of vactosertib for each patient to find the most effective and tolerable amount.

Patient Parameters

Program Overview

The study aims to test a new treatment for anemia in patients with myeloproliferative neoplasms (MPNs), which are blood disorders where the body makes too many blood cells. The drug being tested, vactosertib, targets a specific signal in the body that is often too high in these patients and may cause problems with blood cell production. Up to 37 patients will participate, and each will receive vactosertib at a dose adjusted to their needs.

Description

This study is divided into two parts and involves using vactosertib to treat anemia in patients with certain types of myeloproliferative neoplasms (MPNs) that do not have a specific genetic marker (Ph-negative). In the first part, 12 patients will start with a low dose of vactosertib, which will be gradually increased based on how well they tolerate it and how effective it is over 16 weeks. If this part is successful, the study will expand to include 25 more patients for 24 weeks.

Patients will continue their current treatment while taking vactosertib, as long as their treatment has been stable for at least three months before joining the study. Supportive care, like blood transfusions, will be allowed if needed, but other treatments to stimulate red blood cell production will not be permitted if they are not effective for the patient.

Eligibility Criteria

Inclusion Criteria

  • Diagnosed with a specific type of myeloproliferative neoplasm (MPN) without a certain genetic marker, including polycythemia vera (PV), essential thrombocythemia (ET), myelofibrosis (MF), myelodysplastic/myeloproliferative neoplasms (MDS/MPN), or unclassified MPN (MPN-U).
  • For patients with myelofibrosis (MF), they must be at intermediate or high risk according to specific criteria.
  • If receiving treatment to reduce blood cell production, it must be stable for at least three months before starting the study.
  • Have anemia, defined as hemoglobin levels below 10 g/dL or needing at least two blood transfusions in the past four weeks with hemoglobin levels at or below 8.5 g/dL.
  • Not eligible for or not responding to treatments that stimulate red blood cell production, based on specific criteria.
  • Have an acceptable cardiovascular health status.

Exclusion Criteria

  • Any serious medical condition that would make participating in the study unsafe, according to the study doctor.
  • History of a mini-stroke (TIA) or stroke in the past 12 months.
  • Women who are breastfeeding or plan to breastfeed during the study or within 30 days after the last dose of the study drug.

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