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

Sickle Cell Disease Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03132324
• Other study ID #: INCB 59872-102
• Status: Terminated
• Phase: Phase 1
• Start date: April 20, 2017
• Est. completion date: October 3, 2018

Study information

• Verified date: October 2019
• Source: Incyte Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 12
• Est. completion date: October 3, 2018
• Est. primary completion date: October 3, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• INCB059872
INCB059872 tablets

Locations

Country	Name	City	State
United States	Boston University	Boston	Massachusetts
United States	University of Illinois at Chicago	Chicago	Illinois
United States	Acevedo Clinical Research Associates	Miami	Florida
United States	Advanced Pharma	Miami	Florida
United States	Blood Centers of Wisconsin	Milwaukee	Wisconsin
United States	Virginia Commonwealth University	Richmond	Virginia
United States	Vita Health and Medical Center	Tamarac	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Incyte Corporation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.
Primary	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.
Secondary	Cmax of INCB059872	Defined as maximum observed plasma concentration.	Baseline to Day 28.
Secondary	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.	Baseline to Day 28.