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

NCT02093429 | Terminated Phase 1

• 1 other identifier: INCB 47986-201
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 7

Brief 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.

Conditions

MDS (Myelodysplastic Syndrome)

Outcome Measures

Primary Outcomes (2)

• Proportion of subjects who achieve a response for Hematologic Improvement in Erythrocytes (HI-E) during any 8-week period within the first 16-week treatment period with INCB047986 Monotherapy.

  Baseline to Week 16

• Safety and tolerability of INCB047986 as assessed by summary of clinical laboratory assessments and summary of Adverse Events (AEs).

  Up to 16 weeks

Study Arms (3)

INCB047986 4 mg

EXPERIMENTAL

Participants will receive INCB047986 4 mg once daily for at least 16 weeks.

Drug: INCB047986

INCB047986 6 mg

EXPERIMENTAL

Participants will receive INCB047986 6 mg once daily for at least 16 weeks.

Drug: INCB047986

INCB047986 10 mg

EXPERIMENTAL

Participants will receive INCB047986 10 mg once daily for at least 16 weeks.

Drug: INCB047986

Interventions

INCB047986 DRUG

INCB047986 will be supplied as tablets.

INCB047986 10 mg; INCB047986 4 mg; INCB047986 6 mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (7)

Unknown Facility

Highland, California, United States

Location

Unknown Facility

Indianapolis, Indiana, United States

Location

Unknown Facility

Morristown, New Jersey, United States

Location

Unknown Facility

Somerville, New Jersey, United States

Location

Unknown Facility

Germantown, Tennessee, United States

Location

Unknown Facility

Houston, Texas, United States

Location

Unknown Facility

Burlington, Vermont, United States

Location

MeSH Terms

Conditions

Anemia, Refractory, with Excess of Blasts

Condition Hierarchy (Ancestors)

Anemia, Refractory; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Officials

• William V. Williams, M.D.

  Incyte Corporation

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 3, 2014
• First Posted: March 21, 2014
• Study Start: January 1, 2014
• Primary Completion: September 1, 2014
• Study Completion: September 1, 2014
• Last Updated: January 17, 2018

Record last verified: 2018-01

Locations

US (7)