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

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ClinicalTrials.gov Identifier: NCT02093429
Recruitment Status : Terminated (Business decision.)
First Posted : March 21, 2014
Last Update Posted : January 17, 2018
Sponsor:
Information provided by (Responsible Party):
Incyte Corporation

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.

Condition or disease Intervention/treatment Phase
MDS (Myelodysplastic Syndrome) Drug: INCB047986 Phase 1 Phase 2

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 : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : January 2014
Actual Primary Completion Date : September 2014
Actual Study Completion Date : September 2014

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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.




Primary Outcome Measures :
  1. 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 ]
  2. 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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02093429


Locations
United States, California
Highland, California, United States
United States, Indiana
Indianapolis, Indiana, United States
United States, New Jersey
Morristown, New Jersey, United States
Somerville, New Jersey, United States
United States, Tennessee
Germantown, Tennessee, United States
United States, Texas
Houston, Texas, United States
United States, Vermont
Burlington, Vermont, United States
Sponsors and Collaborators
Incyte Corporation
Investigators
Study Director: William V. Williams, M.D. Incyte Corporation

Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT02093429     History of Changes
Other Study ID Numbers: INCB 47986-201
First Posted: March 21, 2014    Key Record Dates
Last Update Posted: January 17, 2018
Last Verified: January 2018

Additional relevant MeSH terms:
Syndrome
Myelodysplastic Syndromes
Preleukemia
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Hematinics