Randomized Open-Label Study of INCB047986 in Subjects With Primary Myelodysplastic Syndrome (MDS)

• Status: Terminated
• Trial end date: 2014-09-01
• Target enrollment: 0
• Participant gender: All

Summary

The study design includes a 3-dose randomization phase to determine effective doses of INCB047986 in patients with myelodysplastic syndrome (MDS) who are refractory or unlikely to respond to erythropoiesis-stimulating agents (ESAs) followed by an extension phase.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Incyte Corporation
• Treatments: Hematinics

Criteria

Inclusion Criteria:

• Subjects 18 years of age or older.

• Subjects must be diagnosed with MDS according to the World Health Organization (WHO) classification for de novo or primary MDS (Vardiman et al 2009).

• Subjects who require RBC transfusions or are either refractory to or unlikely to respond to ESA therapy should meet one of the following criteria:

• ESA failure as defined by no improvement in Hgb of at least 1.5 g/dL after 8 weeks of at least 40,000 IU per week of EPO (or equivalent).

• Have a serum erythropoietin (EPO) of ≥ 500 IU and Hgb level < 10.0 g/dL.

• Transfusion dependence defined as requiring at least 4 units of packed red blood cells (RBCs) for a Hgb of < 9 g/dL over the 8 weeks prior to screening.

• Subjects may not have received hypomethylating agents or immunosuppressive therapy for their MDS prior to this study.

Exclusion Criteria:

• Subjects at high risk for transformation to acute leukemia as evidenced by poor karyotype or peripheral blood blasts > 10%.

• Subjects with severely compromised bone marrow function as evidenced by trilineage cytopenias with anemia (Hgb < 10 g/L, platelets < 100 × 109/L, and absolute neutrophil count (ANC) < 1.8 × 109/L).

• Subjects who harbor the 5q deletion chromosomal aberration.

• Subjects with chronic myelomonocytic leukemia (CMML).

• Women who are pregnant or breastfeeding, and men and women who cannot comply with requirements to avoid fathering a child or becoming pregnant, respectively.

• Subjects with impaired liver function, end stage renal disease on dialysis, or clinically significant concurrent infections requiring therapy.

• Subjects with unstable cardiac function.

• Invasive malignancies over the previous 2 years except treated basal or squamous carcinomas of the skin, completely resected intraepithelial carcinoma of the cervix, Stage 1 or 2 treated prostate