Clinical trial NCT03132324: A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Sickle Cell Disease
Keywords: Sickle cell disease (SCD)
sickle cell SS
lysine demethylase 1 (LSD1) inhibition
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2017-04-27
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Safety and tolerability of INCB059872 assessed by monitoring frequency, duration, and severity of adverse events
timeFrame: Screening through 35 days after end of treatment, up to approximately 3 months per participant.
measure: Change in fetal hemoglobin (HbF) from baseline
timeFrame: Baseline through 2 weeks after end of treatment, up to approximately 2.5 months per participant.
Secondary outcomes:
measure: Cmax of INCB059872
timeFrame: Baseline to Day 28.
measure: AUC0-t of INCB059872
timeFrame: Baseline to Day 28.
Other outcomes:
Locations:
city: Miami
country: United States
facility: Acevedo Clinical Research Associates
state: Florida
city: Miami
country: United States
facility: Advanced Pharma
state: Florida
city: Tamarac
country: United States
facility: Vita Health and Medical Center
state: Florida
city: Chicago
country: United States
facility: University of Illinois at Chicago
state: Illinois
city: Boston
country: United States
facility: Boston University
state: Massachusetts
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Milwaukee
country: United States
facility: Blood Centers of Wisconsin
state: Wisconsin
Study officials:
affiliation: Incyte Corporation
name: Fitzroy Dawkins, MD
role: STUDY_DIRECTOR
References: