Status:
TERMINATED
Intra-patient Dose Escalation Study to Investigate Safety and Feasibility of Vactosertib in Treating Anemic MPN Patients
Lead Sponsor:
Weill Medical College of Cornell University
Conditions:
Myeloproliferative Neoplasm
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
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 w...
Detailed Description
This is a two-tiered multi arm Phase 2 trial of vactosertib (TEW-7197) for the treatment of anemia in Ph-neg MPNs. Both tiers use a rule-based, accelerated dose escalation scheme to efficiently assess...
Eligibility Criteria
Inclusion
- Patients who meet the WHO 2016 criteria for a Ph-neg MPN (including PV, ET, MF, MDS/MPN, MPN-U).
- Patients with MF must have DIPSS+ Intermediate or High-risk MF (primary of post-PV/ET).
- For patients receiving cytoreductive therapy, they should be on a stable dose of current cytoreductive therapy for at least 3 months prior to C1D1.
- Anemia as defined by HGB \< 10 g/dL, or transfusion of ≥ 2 packed red blood cell (PRBC) unit within the past 4 weeks with HGB ≤8.5g/dL.
- Ineligible, unsuitable or refractoriness to ESA therapy defined as any of the following:
- Serum erythropoietin (EPO) \>125 U/L.
- Proven ESA unsuitability is defined by history of any of the following:
- Loss of erythroid hematologic improvement while receiving stable or increased ESA dose; or
- ESA-attributed toxicity that, in the treating physician's opinion, makes ESA therapy unsuitable for subject.
- ESA refractoriness defined by lack of erythroid hematologic improvement to ESA:27
- Less than 1.5 g/dL increase in hemoglobin after at least 6 weeks of ESA therapy; or
- Ongoing transfusion dependence that has not been reduced by \> 4U over an 8-week period compared to ESA pre-treatment 8 weeks.
- Acceptable Cardiovascular status
Exclusion
- Any other serious medical condition which in the Investigator's opinion would preclude safe participation in the study.
- Patients with history of TIA or stroke within the past 12 months are excluded.
- Female subjects who are breastfeeding, or intend to breastfeed, during the study or in the 30 days following the last dose of study drug are excluded.
Key Trial Info
Start Date :
November 22 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 10 2024
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT04103645
Start Date
November 22 2019
End Date
July 10 2024
Last Update
July 2 2025
Active Locations (1)
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1
Weill Medical College of Cornell University
New York, New York, United States, 10021