- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02093429
Randomized Open-Label Study of INCB047986 in Subjects With Primary Myelodysplastic Syndrome (MDS)
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 Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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California
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Highland, California, United States
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Indiana
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Indianapolis, Indiana, United States
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New Jersey
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Morristown, New Jersey, United States
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Somerville, New Jersey, United States
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Tennessee
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Germantown, Tennessee, United States
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Texas
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Houston, Texas, United States
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Vermont
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Burlington, Vermont, United States
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: INCB047986 4 mg
Participants will receive INCB047986 4 mg once daily for at least 16 weeks.
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INCB047986 will be supplied as tablets.
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Experimental: INCB047986 6 mg
Participants will receive INCB047986 6 mg once daily for at least 16 weeks.
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INCB047986 will be supplied as tablets.
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Experimental: INCB047986 10 mg
Participants will receive INCB047986 10 mg once daily for at least 16 weeks.
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INCB047986 will be supplied as tablets.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
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Baseline to Week 16
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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
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Up to 16 weeks
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: William V. Williams, M.D., Incyte Corporation
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- INCB 47986-201
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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