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This study is terminated due to a business decision not to pursue INCB059782 in Sickle Cell Disease indication.
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The purpose of this study was to evaluate the safety and tolerability, and the pharmacokinetic and biologic activity of INCB059872 in participants with sickle cell disease.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| INCB059872 0.5 mg | Experimental | INCB059872 0.5 mg tablet administered orally every other day (QOD) for 28 days on an empty stomach. If dose was well tolerated, once daily (QD) administration was evaluated independently and in parallel with QOD administration. |
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| INCB059872 1 mg | Experimental | INCB059872 1 mg tablet administered orally QOD for 28 days on an empty stomach. If dose was well tolerated, QD administration was evaluated independently and in parallel with QOD administration. |
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| INCB059872 2 mg | Experimental | INCB059872 2 mg tablet administered orally QOD for 28 days on an empty stomach. If dose was well tolerated, QD administration was evaluated independently and in parallel with QOD administration. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| INCB059872 | Drug | INCB059872 tablets |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety and tolerability of INCB059872 assessed by monitoring frequency, duration, and severity of adverse events | An adverse event is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurs after a participant provides informed consent. | Screening through 35 days after end of treatment, up to approximately 3 months per participant. |
| Change in fetal hemoglobin (HbF) from baseline | Pharmacodynamic activity assessed by measuring changes of HbF from baseline and their correlation to INCB059872 treatment. The HbF (F cells) in human whole blood will be characterized using flow cytometry. | Baseline through 2 weeks after end of treatment, up to approximately 2.5 months per participant. |
| Measure | Description | Time Frame |
|---|---|---|
| Cmax of INCB059872 | Defined as maximum observed plasma concentration. | Baseline to Day 28. |
| AUC0-t of INCB059872 | Defined as area under the single-dose plasma concentration-time curve from Hour 0 to the last quantifiable measurable plasma concentration. |
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Inclusion Criteria:
Diagnosis of SCD (sickle cell SS) confirmed through hemoglobin electrophoresis.
Must be red blood cell (RBC) transfusion-independent (not currently on regularly scheduled transfusions) for ≥ 3 months from the time of first dose of study drug.
No RBC transfusion within 30 days of first dose of study drug.
Hydroxyurea (HU) refractory
-Must not have received HU therapy during the 3 months before receiving study drug.
Creatinine clearance ≥ 60 mL/min based on the institutional formula.
Willingness to avoid pregnancy or fathering children.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Fitzroy Dawkins, MD | Incyte Corporation | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Acevedo Clinical Research Associates | Miami | Florida | 33142 | United States | ||
| Advanced Pharma |
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| Baseline to Day 28. |
| Miami |
| Florida |
| 33147 |
| United States |
| Vita Health and Medical Center | Tamarac | Florida | 33319 | United States |
| University of Illinois at Chicago | Chicago | Illinois | 60607 | United States |
| Boston University | Boston | Massachusetts | 02215 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| Blood Centers of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D007266 | Inhibition, Psychological |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D001519 | Behavior |
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| ID | Term |
|---|---|
| C000730036 | INCB059872 |
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