Randomized 01-Dec-2015 Open-Label Study of T42486 in with Primary Myelodysplastic Syndrome (MDS) 01-Dec-2015

Study identifier:T42486

ClinicalTrials.gov identifier:N/A

EudraCT identifier:N/A

CTIS identifier:N/A

Terminated/Withdrawn

Official Title

A Randomized, 01-Dec-2015 Open-Label, 2-Stage Study of INCB047986 Administered Orally to Subjects With Primary Myelodysplastic Syndrome (MDS) Refractory to or Unlikely to Respond to Erythropoiesis-Stimulating Agents (ESAs) 01-Dec-2015

Medical condition

MDS (Myelodysplastic syndrome)

Phase

Phase 1/2

Healthy volunteers

Study drug

INCB047986

Sex

All

Actual Enrollment

Study type

Interventional

Age

18 Years +

Date

Study Start Date: 01 Jan 2014

Primary Completion Date: 01 Sept 2014

Study Completion Date: 01 Sept 2014

Study design

Allocation: Randomized
Endpoint Classification: -
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment

Verification:

Verified 01 Dec 2023 by Intrasphere

Collaborators

Inclusion and exclusion 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

No locations available

Arms	Assigned Interventions
Experimental: INCB047986 4 mg Participants will receive INCB047986 4 mg once daily for at least 16 weeks.	Drug: INCB047986 INCB047986 will be supplied as tablets.
Experimental: INCB047986 6 mg Participants will receive INCB047986 6 mg once daily for at least 16 weeks.	Drug: INCB047986 INCB047986 will be supplied as tablets.
Experimental: INCB047986 10 mg Participants will receive INCB047986 10 mg once daily for at least 16 weeks.	Drug: INCB047986 INCB047986 will be supplied as tablets.

Contacts

Contact

Intrasphere Call Center

1-855-463-3463

Investigators

Principal Investigator

William V. Williams

Intrasphere