Oral Rigosertib in Low Risk MDS Patients Refractory to ESAs

Completed · Phase 2

Conditions studied: Myelodysplastic Syndromes

In brief

The study will enroll low risk MDS patients who need red blood cell transfusions and who are refractory to or are not using erythropoiesis-stimulating agents. The purpose of the study is to determine whether oral rigosertib treatment results in hematological improvements according to the 2006 International Working Group criteria in these patients. The study will also record any side effects that may occur during the study.

Key facts

Study ID

NCT01904682

Run by

Traws Pharma, Inc.

People needed

45

Starts

2013-07-01

Expected to finish

2021-05-01

Last updated by the study team

2021-06-16

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of MDS according to World Health Organization (WHO) criteria (Appendix 2) or French-American-British (FAB) classification that must be confirmed by bone marrow (BM) aspirate and/or biopsy within 6 weeks prior to Screening.
• Myelodysplastic syndrome (MDS) classified as Low risk or Int-1 risk, according to International Prognostic Scoring System (IPSS) classification; in addition, patients should never have been classified as Int-2 or High-risk since their MDS was diagnosed;
• Transfusion dependency defined by transfusion of at least 4 units of Red blood cells (RBC) within 56 days before Screening (pre-transfusion Hgb values values must be ≤ 9 g/dL to be taken into account).
• Refractory to 8- to 12-week course of Erythropoiesis-stimulating agent (ESA) administered within the past 2 years before enrollment, or erythropoietin (EPO) level ˃ 500 mU/mL and off ESA for at least 8 weeks before Screening.
• Off all other treatments for MDS (azacitidine, decitabine, lenalidomide, chemotherapy, immunotherapy) for at least 2 weeks prior to Screening.
• Eastern Cooperative Oncology Group(ECOG) performance status of 0, 1 or 2.
• Willing to adhere to the prohibitions and restrictions specified in this protocol.
• The patient must signed an informed consent form (ICF) indicating that s/he understands the purpose of, and procedures required for, the study and is willing to participate.

You may not qualify if…

• Ongoing clinically significant anemia due to factors such as iron, vitamin B12, or folate deficiencies, auto-immune or hereditary hemolysis, or gastrointestinal (GI) bleeding.
• Serum ferritin < 50 ng/mL.
• Hypoplastic MDS (cellularity <10%)
• Any active malignancy within the past year, except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix or breast.
• Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
• Active infection not adequately responding to appropriate therapy.
• Total bilirubin ≥ 2.0 mg/dL not related to hemolysis or Gilbert's disease.
• Alanine transaminase (ALT) or aspartate transaminase (AST) ≥ 2.5 x the upper limit of normal (ULN).
• Serum creatinine ≥ 2.0 mg/dL.
• Ascites requiring active medical management including paracentesis.
• Hyponatremia (defined as serum sodium value of < 130 mEq/L).
• Female patients who are pregnant or lactating.
• Patients of childbearing potential who are unwilling to follow strict contraception requirements.
• Female patients with reproductive potential who do not have a negative blood or urine pregnancy test at Screening.
• Major surgery without full recovery or major surgery within 3 weeks of Screening.
• Uncontrolled hypertension (defined as a systolic pressure ≥ 160 mmHg and/or a diastolic pressure ≥ 110 mmHg).
• New onset seizures (within 3 months prior to the first dose of rigosertib) or poorly controlled seizures.
• Any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy.
• Chronic use (˃ 2 weeks) of corticosteroids (˃ 10 mg/24 hr equivalent prednisone) within 4 weeks of Screening.
• Investigational therapy within 4 weeks of Screening.
• Allergy to a local anaesthetic.
• Psychiatric illness or social situation that would limit the patient's ability to tolerate and/or comply with study requirements.

Where it is running

• Stanford University School of Medicine — Stanford, California, United States
• Anschutz Cancer Pavilion University of Colorado — Aurora, Colorado, United States
• Washington Cancer Institute at Medstar Washington Hospital Center — Washington D.C., District of Columbia, United States
• The University of Chicago — Chicago, Illinois, United States
• Greenbaum Cancer Center University of Maryland — Baltimore, Maryland, United States
• Mayo Clinic — Rochester, Minnesota, United States
• Rutgers Cancer Institute of New Jersey — New Brunswick, New Jersey, United States
• Mount Sinai Medical Center — New York, New York, United States
• Cleveland Clinic Foundation — Cleveland, Ohio, United States
• The University of Texas MD Anderson Cancer Center — Houston, Texas, United States
• Hôpital Saint-Louis, Service d'Hématologie — Paris, IDF, France
• Universitätsklinikum Köln — Cologne, North Rhine-Westphalia, Germany
• Heinrich Heine Universität — Düsseldorf, North Rhine-Westphalia, Germany
• Universitätsklinikum Carl Gustav Carus an der Technischen Universität Dresden — Dresden, Saxony, Germany

Full record on ClinicalTrials.gov

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