A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease



Status:Terminated
Conditions:Anemia
Therapuetic Areas:Hematology
Healthy:No
Age Range:18 - Any
Updated:3/3/2019
Start Date:April 27, 2017
End Date:September 17, 2018

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A Phase 1 Open-Label, Dose-Escalation Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease

The purpose of this study is to evaluate the safety and tolerability, and the pharmacokinetic
and biologic activity of INCB059872 in participants with sickle cell disease.


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:

- Any unresolved toxicity ≥ Grade 2 from previous therapy except for stable chronic
toxicities not expected to resolve.

- Pregnant or nursing women or participants expecting to conceive or father children
within the projected duration of the study, starting with screening visit through
completion of safety follow-up.

- Received an investigational study drug within 28 days or 5 half-lives (whichever is
longer) before receiving the first dose of study drug (requirement may be waived with
medical monitor approval).

- Chronic or current active infectious disease requiring systemic antibiotics,
antifungal, or antiviral treatment.

- Prior receipt of LSD1 inhibitor therapy for any indication.
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One Silber Way
Boston, Massachusetts 02215
(617) 353-2000
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2035 W Taylor St
Chicago, Illinois
(312) 996-4350
University of Illinois at Chicago A major research university in the heart of one of...
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2950 Northwest 83rd Street
Miami, Florida 33147
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Milwaukee, Wisconsin 53233
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Richmond, Virginia 23298
(804) 828-0100
Virginia Commonwealth University Since our founding as a medical school in 1838, Virginia Commonwealth University...
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Tamarac, Florida 33319
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