A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease
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|ClinicalTrials.gov Identifier: NCT03132324|
Recruitment Status : Terminated (This study is terminated due to a business decision not to pursue INCB059782 in Sickle Cell Disease indication.)
First Posted : April 27, 2017
Last Update Posted : October 28, 2019
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|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: INCB059872||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1 Open-Label, Dose-Escalation Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease|
|Actual Study Start Date :||April 20, 2017|
|Actual Primary Completion Date :||October 3, 2018|
|Actual Study Completion Date :||October 3, 2018|
Experimental: INCB059872 0.5 mg
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.
Experimental: INCB059872 1 mg
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.
Experimental: INCB059872 2 mg
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.
- Safety and tolerability of INCB059872 assessed by monitoring frequency, duration, and severity of adverse events [ Time Frame: Screening through 35 days after end of treatment, up to approximately 3 months per participant. ]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.
- Change in fetal hemoglobin (HbF) from baseline [ Time Frame: Baseline through 2 weeks after end of treatment, up to approximately 2.5 months per participant. ]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.
- Cmax of INCB059872 [ Time Frame: Baseline to Day 28. ]Defined as maximum observed plasma concentration.
- AUC0-t of INCB059872 [ Time Frame: Baseline to Day 28. ]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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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
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): NCT03132324
|United States, Florida|
|Acevedo Clinical Research Associates|
|Miami, Florida, United States, 33142|
|Miami, Florida, United States, 33147|
|Vita Health and Medical Center|
|Tamarac, Florida, United States, 33319|
|United States, Illinois|
|University of Illinois at Chicago|
|Chicago, Illinois, United States, 60607|
|United States, Massachusetts|
|Boston, Massachusetts, United States, 02215|
|United States, Virginia|
|Virginia Commonwealth University|
|Richmond, Virginia, United States, 23298|
|United States, Wisconsin|
|Blood Centers of Wisconsin|
|Milwaukee, Wisconsin, United States, 53226|
|Study Director:||Fitzroy Dawkins, MD||Incyte Corporation|
|Responsible Party:||Incyte Corporation|
|Other Study ID Numbers:||
|First Posted:||April 27, 2017 Key Record Dates|
|Last Update Posted:||October 28, 2019|
|Last Verified:||October 2019|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Sickle cell disease (SCD)
sickle cell SS
lysine demethylase 1 (LSD1) inhibition
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn