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

Incyte

Status and phase: Terminated; Phase 1

Conditions: Sickle Cell Disease

Treatments: Drug: INCB059872

Study type: Interventional

Funder types: Industry

Identifiers

NCT03132324

INCB 59872-102

Details and patient eligibility

About

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.

Enrollment: 12 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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12 participants in 3 patient groups

INCB059872 0.5 mg

Experimental group

Description:

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.

Treatment:

Drug: INCB059872

INCB059872 1 mg

Experimental group

Description:

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.

Treatment:

Drug: INCB059872

INCB059872 2 mg

Experimental group

Description:

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.

Treatment:

Drug: INCB059872