Back to results

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

Primary Purpose

MDS (Myelodysplastic Syndrome)

Status

Terminated

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

INCB047986

About this trial

This is an interventional treatment trial for MDS (Myelodysplastic Syndrome)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

INCB047986 4 mg

INCB047986 6 mg

INCB047986 10 mg

Arm Description

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

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

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

Outcomes

Primary Outcome Measures

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.

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

Secondary Outcome Measures

Full Information

NCT ID

NCT02093429

First Posted

January 3, 2014

Last Updated

January 15, 2018

Sponsor

Incyte Corporation

1. Study Identification

Unique Protocol Identification Number

NCT02093429

Brief Title

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

Official Title

A Randomized, 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)

Study Type

Interventional

2. Study Status

Record Verification Date

January 2018

Overall Recruitment Status

Terminated

Why Stopped

Business decision.

Study Start Date

January 2014 (undefined)

Primary Completion Date

September 2014 (Actual)

Study Completion Date

September 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Incyte Corporation

4. Oversight

Data Monitoring Committee

5. Study Description

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.

Detailed Description

After 50% of the subjects have completed 16 weeks of treatment (10 subjects in each dose group), a planned interim analysis will be conducted to determine which, if any, dose levels warrant further investigation based on observing at least 3 responses for hematologic improvement in erythrocytes (HI-E) and adequate safety and tolerability. Subjects who do not meet the response criteria for erythroid improvement may be treated with ESA in combination with INCB047986 for an additional 16 weeks. The study comprises: Screening: up to 4 weeks. Treatment Phase 1: at least 16 weeks of INCB047986. Treatment Phase 2: at least 8 and up to 16 weeks of INCB047986 in combination with ESA if failed treatment in Phase 1. Extension Phase: Subjects receiving benefit from either therapy may continue indefinitely or until the study is terminated. Follow-Up: 30 (± 7) days after the last dose of INCB047986 is taken.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

MDS (Myelodysplastic Syndrome)

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

6 (Actual)

8. Arms, Groups, and Interventions

Arm Title

INCB047986 4 mg

Arm Type

Experimental

Arm Description

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

Arm Title

INCB047986 6 mg

Arm Type

Experimental

Arm Description

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

Arm Title

INCB047986 10 mg

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

INCB047986

Intervention Description

INCB047986 will be supplied as tablets.

Primary Outcome Measure Information:

Title

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.

Time Frame

Baseline to Week 16

Title

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

Time Frame

Up to 16 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

William V. Williams, M.D.

Organizational Affiliation

Incyte Corporation

Official's Role

Study Director

Facility Information:

City

Highland

State/Province

California

Country

United States

City

Indianapolis

State/Province

Indiana

Country

United States

City

Morristown

State/Province

New Jersey

Country

United States

City

Somerville

State/Province

New Jersey

Country

United States

City

Germantown

State/Province

Tennessee

Country

United States

City

Houston

State/Province

Texas

Country

United States

City

Burlington

State/Province

Vermont

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs