Single-Arm Study of the Efficacy and Safety of Oral Rigosertib in Patients With Myelofibrosis (MF) and Anemia

NCT02730884 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: 2014-0546NCI-2016-00761
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical research study is to learn if rigosertib can help to control MF in patients with anemia. The safety of this drug will also be studied. This is an investigational study. Rigosertib is not FDA-approved or commercially available. It is currently being used for research purposes only. The study doctor can explain how the study drug is designed to work. Up to 35 participants will be enrolled in this study. All will be enrolled at MD Anderson.

Conditions

Leukemia; Myelofibrosis; Anemia; Splenomegaly

Keywords

Leukemia; Myelofibrosis; MF; Anemia; Splenomegaly; Post-polycythemia vera MF; post-PV; post-essential thrombocythemia MF; post-ET; Rigosertib; ON 01910.Na; Questionnaire; Survey

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Spleen Volume Response

  Spleen response defined as ≥ 35% spleen volume reduction from Baseline, which must be confirmed by MRI or CT measurement per revised International Working Group for Myelofibrosis Research and Treatment (IWG MRT) response criteria.

  Baseline and 48 weeks

• Participants With Anemia Response

  Anemia response defined as the proportion of transfusion-independent patients with Hgb increase of at least 2 g/dL from Baseline or the proportion of transfusion-dependent patients becoming transfusion independent for at least 12 weeks as defined in 2013 International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria.

  Baseline and 48 weeks

Secondary Outcomes (1)

• Symptoms Response

  48 weeks

Study Arms (1)

Rigosertib

EXPERIMENTAL

Participants receive oral Rigosertib under fasting conditions twice a day on a continuous basis. Quality of life questionnaire completed on Day 1 of Cycle 1.

Drug: Rigosertib; Behavioral: Questionnaire

Interventions

Rigosertib DRUG

Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon.

Also known as: ON 01910.Na

Rigosertib

Questionnaire BEHAVIORAL

Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.

Also known as: Survey

Rigosertib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \>/= 18 years of age;
• Diagnosis of primary myelofibrosis (PMF) or post-polycythemia vera (post-PV) MF or post-essential thrombocythemia (post-ET) MF based on the World Health Organization (WHO) criteria or the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria, which must be confirmed by BM aspirate and/or biopsy within 6 weeks prior to Screening. Measurement of JAK2 V617F allele burden in Bone Marrow (BM) samples, if not done within 6 months prior to Screening, must be provided with the Screening BM biopsy/aspirate report (patients are eligible regardless of JAK2 mutation status);
• Anemia or RBC-transfusion dependence defined as follows: a) Anemia: defined for the purpose of this protocol as 1) a hemoglobin level \<10 g/L on every determination over 84 days before study-entry, without red blood cell (RBC)-transfusions, or 2) a hemoglobin level \<10 g/L on a patient that is receiving RBC-transfusions periodically but not meeting criteria for transfusion-dependent patient as defined below. The baseline hemoglobin value for these subjects is the lowest hemoglobin level during the antecedent 84 days; b) RBC-transfusion-dependence: RBC-transfusion-frequency of \>/=2 units packed red blood cells (PRBC)/28 days averaged over 84 days immediately pre-study-entry. There must not be any consecutive 42 days without an RBC-transfusion during this interval.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2;
• Willing to adhere to the prohibitions and restrictions specified in this protocol (Notation: the subject's willingness to adhere to prohibitions and restrictions must be clearly communicated in the on-study note);
• 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 known iron, vitamin B12, or folate deficiencies, auto-immune or hereditary hemolysis, or gastrointestinal (GI) bleeding;
• Serum ferritin \< 50 ng/mL;
• Any active malignancy within the past year, except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix or breast; patients with history of prior malignancies should be free of disease for at least 3 years to be eligible for this study.
• 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;
• Direct 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 \>/= 2.5 mg/dL;
• Ascites requiring active medical management including paracentesis;
• Hyponatremia (defined as serum sodium level \< 130 mEq/L);
• Female patients who are pregnant or lactating;
• Patients of childbearing potential (ie, women of childbearing potential and men with female partners of childbearing potential) who are unwilling to follow strict contraception requirements (including 2 reliable methods in combination: 1 non-hormonal, highly-reliable method \[diaphragm, condoms with spermicidal foam or jelly, or sterilization\] plus 1 additional reliable method \[birth control pills, intrauterine device, contraceptive injections, or contraceptive patches\]) before entry and throughout the study, up to and including the 30-day non-treatment follow-up period;
• Female patients of childbearing potential who have a positive 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 sustained systolic pressure \>/= 160 mmHg and/or a diastolic pressure \>/= 110 mmHg);

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• Traws Pharma, Inc.: collaborator

Study Sites (1)

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• University of Texas MD Anderson Cancer Center Website

MeSH Terms

Conditions

Leukemia; Primary Myelofibrosis; Anemia; Splenomegaly

Interventions

ON 01910; Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders; Bone Marrow Diseases; Hypertrophy; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Results Point of Contact

• Title: Jorge Cortes, MD/Professor
• Organization: The University of Texas MD Anderson Cancer Center

jcortes@mdanderson.org

713-794-5783

Study Officials

• Jorge Cortes, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 24, 2016
• First Posted: April 7, 2016
• Study Start: August 16, 2017
• Primary Completion: August 22, 2019
• Study Completion: August 22, 2019
• Last Updated: August 7, 2020
• Results First Posted: August 7, 2020

Record last verified: 2020-08

Locations

US (1)