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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01822691

Enrollment

Registered

2013-04-02

Start date

2013-07-31

Completion date

2015-02-28

Last updated

2016-01-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Myelodysplastic Syndromes

Keywords

myelodysplastic syndromes (MDS), neoplastic stem cell disorders, refractory cytopenias, dysplastic morphological features, acute myeloid leukemia (AML)

Brief summary

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.

Interventions

DRUGINCB024360

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.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Confirmed diagnosis of myelodysplastic syndromes (MDS) * Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 * Adequate organ function: * Total bilirubin ≤ 1.5 × Upper Limit of Normal (ULN) * Aspartic transaminase (AST)/alanine transaminase (ALT) ≤ 2.5 × ULN * Creatinine ≤ 2 × ULN or Creatinine clearance of \> 30 mL/min (using the Cockcroft and Gault Equation) * Females of childbearing potential must have a negative urine or serum pregnancy test at Screening. * Women of child-bearing potential and men must agree to use adequate contraception (surgical tubal ligation or vasectomy, double-barrier method of birth control condom with spermicide in conjunction with use of an intrauterine device (IUD) or diaphragm; or sexual abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant, or a male impregnate his female partner, while participating in this study, he/she should inform their treating physician immediately. * Ability to understand and the willingness to sign a written informed consent document

Exclusion criteria

* Prior MDS therapy within 4 weeks of the first dose of study medication. For erythroid stimulating agent and growth factors: prior therapy with epoetin (Procrit) or G-CSF (neupogen) or GM-CSF (leukine) within 2 weeks of the first dose of study medication. * Has participated in any other trial in which receipt of an investigational study drug occurred within 28 days. * Has undergone a stem cell, bone marrow or solid organ transplant. * Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit by the investigator opinion compliance with study requirements * History of hepatitis or positive serology as follows: * Hepatitis B (HepB) screening testing required: HepB SAg (hepatitis B surface antigen); Anti-HepB SAg (antibody against hepatitis B surface antigen); Anti-Hepatitis B core IgG (antibody against hepatitis B core antigen); Anti-Hepatitis B core IgM antibody Note: Subjects with no prior history of hepatitis B infection who have been vaccinated against hepatitis B and who have a positive anti-HepB SAg test as the only evidence of prior exposure may participate in the trial. * Hepatitis C screening required: antibody against hepatitis C virus (HCV-antibody); HCV-RNA (serum test for circulating virus, based on detecting RNA) * Known history human immunodeficiency virus (HIV) * Is receiving any compound that is known to be a potent inducer or inhibitor of CYP3A4 * Being treated with a monoamine oxidase inhibitor (MAOI), or drug which has significant monoamine oxidase inhibitory activity (meperidine, linezolid, methylene blue) within 3 weeks prior to screening * Has, by the investigator assessment, an active autoimmune process such as rheumatoid arthritis, psoriasis, multiple sclerosis, inflammatory bowel disease, etc. or is receiving therapy for an autoimmune disease. Subjects with vitiligo, hypothyroidism or eczema may be enrolled after approval by the sponsor. * Receiving any immunologically based treatment for any reason, including chronic use of systemic steroid at doses ≥ 7.5 mg/day prednisone equivalents; use of inhaled or topical steroids is acceptable. * Prior malignancies other than MDS for which the subject has not been disease free for ≤ 3 years, except treated and cured basal or squamous cell skin cancer, superficial bladder cancer, or carcinoma in situ of the cervix. * Use of any UGT1A9 inhibitor including: diclofenac, imipramine, ketoconazole, mefenamic acid, and probenecid from screening through follow-up period. * Have had prior Serotonin Syndrome * Any unresolved toxicity greater than Grade 2 from previous anticancer therapy, except for stable chronic toxicities not expected to resolve, such as peripheral neurotoxicity

Design outcomes

Primary

Measure	Time frame	Description
Overall Response Rate (ORR)	Up to 12 months	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.

Secondary

Measure	Time frame	Description
Mean Time to Acute Myeloid Leukemia (AML) Progression	Up to 12 months	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.
Median Overall Survival (OS)	Up to 24 months	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.
Number of Participants With Study Related Serious Adverse Events (SAEs)	Up to 12 months	Participants with treatment emergent Grade 3 or 4 SAEs according to the NCI Common Terminology Criteria for Adverse Events Version (CTCAE) V4.0.
Number of Participants With Study Treatment Related Adverse Events (AEs)	Up to 12 months	Participants with treatment emergent Other (not including serious) Adverse events.

Countries

United States

Participant flow

Recruitment details

Participants were enrolled at Moffitt Cancer Center between August 2013 and January 2014.

Participants by arm

Arm	Count
INCB024360 Treatment Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.	15
Total	15

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Withdrawal by Subject	2

Baseline characteristics

Characteristic	INCB024360 Treatment
Age, Categorical <=18 years	0 Participants
Age, Categorical >=65 years	13 Participants
Age, Categorical Between 18 and 65 years	2 Participants
Age, Continuous	72 years
Region of Enrollment United States	15 participants
Sex: Female, Male Female	3 Participants
Sex: Female, Male Male	12 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	14 / 15
serious Total, serious adverse events	3 / 15

Arm	Measure	Group	Value (NUMBER)
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	Respiratory Disorders	1 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	AE of any category	12 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	Metabolism and Nutrition Disorders	7 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	Gastrointestinal Disorders	6 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	General Disorders	3 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	Investigations	3 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	Immune System Disorders	2 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	Musculoskeletal Disorders	2 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	Skin and Subcutaneous Tissue Disorders	2 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	Infections and Infestations	1 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	Nervous System Disorders	1 participants
INCB024360 Treatment	Number of Participants With Study Treatment Related Adverse Events (AEs)	Psychiatric Disorders	1 participants

Source: ClinicalTrials.gov · Data processed: Mar 6, 2026