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

NCT03132324 | Terminated Phase 1 FDA Drug

• 1 other identifier: INCB 59872-102
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 7

Brief 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.

Conditions

Sickle Cell Disease

Keywords

Sickle cell disease (SCD); sickle cell SS; lysine demethylase 1 (LSD1) inhibition

Outcome Measures

Primary Outcomes (2)

• 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.

Secondary Outcomes (2)

• Cmax of INCB059872

  Baseline to Day 28.

• AUC0-t of INCB059872

  Baseline to Day 28.

Study Arms (3)

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.

Drug: INCB059872

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.

Drug: INCB059872

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.

Drug: INCB059872

Interventions

INCB059872 DRUG

INCB059872 tablets

INCB059872 0.5 mg; INCB059872 1 mg; INCB059872 2 mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (7)

Acevedo Clinical Research Associates

Miami, Florida, 33142, United States

Location

Advanced Pharma

Miami, Florida, 33147, United States

Location

Vita Health and Medical Center

Tamarac, Florida, 33319, United States

Location

University of Illinois at Chicago

Chicago, Illinois, 60607, United States

Location

Boston University

Boston, Massachusetts, 02215, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

Blood Centers of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Inhibition, Psychological

Interventions

INCB059872

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Behavior

Study Officials

• Fitzroy Dawkins, MD

  Incyte Corporation

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 24, 2017
• First Posted: April 27, 2017
• Study Start: April 20, 2017
• Primary Completion: October 3, 2018
• Study Completion: October 3, 2018
• Last Updated: October 28, 2019

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)