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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 26, 2018
Sponsor:
Information provided by (Responsible Party):
Incyte Corporation

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: INCB059872 Phase 1

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 27, 2017
Actual Primary Completion Date : September 17, 2018
Actual Study Completion Date : September 17, 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: INCB059872 QOD
Initial cohort dose of INCB059872 at the protocol-specified starting dose once every other day (QOD), with subsequent cohort escalations based on protocol-specific criteria.
Drug: INCB059872
INCB059872 at the protocol-defined dose administered QOD or QD based on cohort assignment.

Experimental: INCB059872 QD
After the first 3 dose cohorts have been evaluated for tolerability of a QOD schedule, a parallel cohort will evaluate a once-daily (QD) schedule with subsequent dose escalations based on protocol-specific criteria.
Drug: INCB059872
INCB059872 at the protocol-defined dose administered QOD or QD based on cohort assignment.




Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. Cmax of INCB059872 [ Time Frame: Baseline to Day 28. ]
    Defined as maximum observed plasma concentration.

  2. 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
Criteria

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.

Information from the National Library of Medicine

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


Locations
United States, Florida
Acevedo Clinical Research Associates
Miami, Florida, United States, 33142
Advanced Pharma
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 University
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
Sponsors and Collaborators
Incyte Corporation
Investigators
Study Director: Fitzroy Dawkins, MD Incyte Corporation

Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT03132324     History of Changes
Other Study ID Numbers: INCB 59872-102
First Posted: April 27, 2017    Key Record Dates
Last Update Posted: October 26, 2018
Last Verified: October 2018
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

Keywords provided by Incyte Corporation:
Sickle cell disease (SCD)
sickle cell SS
lysine demethylase 1 (LSD1) inhibition

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn