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

Phase 2
18 Years
Not Enrolling
Myelodysplastic Syndromes

Thank you

Trial Information

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

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

- 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

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Best Overall Response Rate (ORR)

Outcome Description:

The Primary Endpoint is best Overall Response, measured by Response Criteria for Patients with 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). Treatment Duration is 17 weeks plus optional continuation phase. Study Duration is Treatment Phase followed by 24 months survival follow-up.

Outcome Time Frame:

On Study Through Follow-up - Approximately 36 Months

Safety Issue:


Principal Investigator

Rami Komrokji, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

H. Lee Moffitt Cancer Center and Research Institute


United States: Food and Drug Administration

Study ID:




Start Date:

August 2013

Completion Date:

September 2015

Related Keywords:

  • Myelodysplastic Syndromes
  • myelodysplastic syndromes (MDS)
  • neoplastic stem cell disorders
  • refractory cytopenias
  • dysplastic morphological features
  • acute myeloid leukemia (AML)
  • Myelodysplastic Syndromes
  • Preleukemia



H. Lee Moffitt Cancer Center and Research InstituteTampa, Florida  33612