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

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ClinicalTrials.gov Identifier: NCT02730884
Recruitment Status : Terminated (Terminated Per PIs request due to low accrual)
First Posted : April 7, 2016
Results First Posted : August 7, 2020
Last Update Posted : August 7, 2020
Sponsor:
Collaborator:
Onconova Therapeutics, Inc.
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Study Description
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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.


Condition or disease Intervention/treatment Phase
Leukemia Myelofibrosis Anemia Splenomegaly Drug: Rigosertib Behavioral: Questionnaire Phase 2

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Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Single-Arm Study of the Efficacy and Safety of Oral Rigosertib in Patients With Myelofibrosis (MF) and Anemia
Actual Study Start Date : August 16, 2017
Actual Primary Completion Date : August 22, 2019
Actual Study Completion Date : August 22, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: Rigosertib
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
Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon.
Other Name: ON 01910.Na

Behavioral: Questionnaire
Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.
Other Name: Survey


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Spleen Volume Response [ Time Frame: Baseline and 48 weeks ]
    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.

  2. Participants With Anemia Response [ Time Frame: Baseline and 48 weeks ]
    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.


Secondary Outcome Measures :
  1. Symptoms Response [ Time Frame: 48 weeks ]
    Symptoms response defined as the proportion of patients achieving ≥ 50% reduction in the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) at any time before Week 48.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. >/= 18 years of age;
  2. 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);
  3. 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.
  4. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2;
  5. 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);
  6. 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.

Exclusion Criteria:

  1. 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;
  2. Serum ferritin < 50 ng/mL;
  3. 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.
  4. Uncontrolled intercurrent illness, including, but not limited to symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia;
  5. Active infection not adequately responding to appropriate therapy;
  6. Direct bilirubin >/= 2.0 mg/dL not related to hemolysis or Gilbert's disease;
  7. Alanine transaminase (ALT) or aspartate transaminase (AST)>/= 2.5 x the upper limit of normal (ULN);
  8. Serum creatinine >/= 2.5 mg/dL;
  9. Ascites requiring active medical management including paracentesis;
  10. Hyponatremia (defined as serum sodium level < 130 mEq/L);
  11. Female patients who are pregnant or lactating;
  12. 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;
  13. Female patients of childbearing potential who have a positive blood or urine pregnancy test at Screening;
  14. Major surgery without full recovery or major surgery within 3 weeks of Screening;
  15. Uncontrolled hypertension (defined as a sustained systolic pressure >/= 160 mmHg and/or a diastolic pressure >/= 110 mmHg);
  16. New onset seizures (within 3 months prior to Screening) or poorly controlled seizures;
  17. Any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy;
  18. Chronic use (> 2 weeks) of corticosteroids (prednisone >/= 10 mg/24 hr equivalent) within 4 weeks of Screening;
  19. Investigational therapy within 2 weeks of Screening;
  20. Psychiatric illness or social situation that would limit the patient's ability to tolerate and/or comply with study requirements.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02730884


Locations
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United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Onconova Therapeutics, Inc.
Investigators
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Principal Investigator: Jorge Cortes, MD M.D. Anderson Cancer Center
  Study Documents (Full-Text)

Documents provided by M.D. Anderson Cancer Center:
Study Protocol and Statistical Analysis Plan  [PDF] August 15, 2017

More Information
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Additional Information:

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Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT02730884    
Other Study ID Numbers: 2014-0546
NCI-2016-00761 ( Registry Identifier: NCI CTRP )
First Posted: April 7, 2016    Key Record Dates
Results First Posted: August 7, 2020
Last Update Posted: August 7, 2020
Last Verified: August 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by M.D. Anderson Cancer Center:
Leukemia
Myelofibrosis
MF
Anemia
Splenomegaly
Post-polycythemia vera MF
post-PV
 post-essential thrombocythemia MF
post-ET
Rigosertib
ON 01910.Na
Questionnaire
Survey
Additional relevant MeSH terms:
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Anemia
Primary Myelofibrosis
Splenomegaly
Hematologic Diseases
Myeloproliferative Disorders
Bone Marrow Diseases
Hypertrophy
 Pathological Conditions, Anatomical
ON 01910
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action