Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Rare Diseases

Syndrome

Leukemia

Cytopenia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Diseases

Hematologic Diseases

Precancerous Conditions

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.

INCB024360 is an inhibitor of the enzyme indoleamine 2,3-dioxygenase (IDO) that is proposed for development for the treatment of malignant diseases. Participants were to receive the study drug in 28 day (4 week) cycles of treatment.

INCB024360

Rami Komrokji, M.D.

The primary purpose of this research study is to assess whether the participant's disease, Myelodysplastic Syndromes (MDS), responds favorably to INCB024360. The study will also evaluate the long-term outcomes of the participant's disease after they have finished taking INCB024360.

Phase II INCB024360 Study for Patients With Myelodysplastic Syndromes (MDS)

A Phase II Study to Determine the Safety and Efficacy of INCB024360 in Patients With Myelodysplastic Syndromes

ORR, measured by Response Criteria for Patients with Myelodysplastic Syndrome (MDS). According International Working Group (IWG) 2006 criteria (Cheson et al, 2006). Complete Remission (CR), Partial Remission (PR), Marrow CR, and Hematological Improvement (HI)(any cell line). Stable Disease (SD): Failure to achieve at least PR, but no evidence of progression for \> 8 weeks.

Disease progression defined as progression to int-2 or high risk International Prognostic Scoring System (IPSS) score or AML, based on World Health Organization (WHO) AML Criteria.

Overall Survival (OS) defined as the time between the start of treatment and death. Treatment Duration is 17 weeks plus optional continuation phase. Study Duration is Treatment Phase followed by survival follow-up.

Participants with treatment emergent Grade 3 or 4 SAEs according to the NCI Common Terminology Criteria for Adverse Events Version (CTCAE) V4.0.

Participants with treatment emergent Other (not including serious) Adverse events.

Incyte Corporation

H. Lee Moffitt Cancer Center and Research Institute

INCB024360 Treatment

Age, Categorical

Age, Continuous

Sex: Female, Male

United States

Region of Enrollment

The study was closed prior to meeting the overall survival criteria.

Dr. Rami Komrokji

Stable Disease

Complete Response

Marrow Complete Response

Hematological Improvement

Progressive Disease

Overall Response Rate (ORR)

Mean Time to Acute Myeloid Leukemia (AML) Progression

Median Overall Survival (OS)

Number of Participants With Study Related Serious Adverse Events (SAEs)

AE of any category

Metabolism and Nutrition Disorders

Gastrointestinal Disorders

General Disorders

Investigations

Immune System Disorders

Musculoskeletal Disorders

Skin and Subcutaneous Tissue Disorders

Infections and Infestations

Nervous System Disorders

Psychiatric Disorders

Respiratory Disorders

Number of Participants With Study Treatment Related Adverse Events (AEs)

Overall Study

Spots Global Cancer Trial Database for dysplastic morphological features