The purpose of this study is to assess the hematologic effects of IMG-7289 therapy in ET and PV patients who require platelet, White Blood Cell (WBC) or Red Blood Cell (RBC) control, and have failed at least one standard therapy.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
4
Daily oral dose of 0.6 mg/kg/day IMG-7829 capsules. Dose escalation an de-escalation rules applied as necessary.
University of Miami
Miami, Florida, United States
Hematologic Response Rates
As evaluated by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European Leukemia Net (ELN) response criteria.
Time frame: 24 Weeks
Incidence of Treatment-Related Toxicity
As evaluated by the treating physician using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0.
Time frame: Up to 3 Years
Change in Total Symptom Score (TSS) as Measured by the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF)
As measured using the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) that includes 14 disease related symptoms each scored from 0 (absent) to 10 (worst imaginable).
Time frame: Baseline, Up to 3 Years
Change in Mutational Allele Burden
Evaluated via Next Generation Sequencing (NGS) molecular profiling from serum blood sample.
Time frame: Baseline, Up to 3 Years
Change in Spleen Size in Centimeters
Measured via physical examination and radiologic imaging measurement.
Time frame: Baseline, Up to 3 Years
Change in Fibrosis Score
Assessed using a slightly modified version of European Consensus Criteria for Grading Myelofibrosis from bone marrow/aspirate sample, as presented in Thiele et al, 2005. Myelofibrosis (MF) scores are graded on a four-point scale, from MF-0 to MF-3, grading the reticulin and collagen content of bone marrow, with MF-0 being the lowest and MF-3 the highest.
Time frame: Baseline, Up to 3 Years
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