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 |
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Recruitment Status :
Completed
First Posted : April 7, 2016
Last Update Posted : August 30, 2019
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Sponsor:
M.D. Anderson Cancer Center
Collaborator:
Onconova Therapeutics, Inc.
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
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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 |
Show detailed description
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 6 participants |
| 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
Genetics Home Reference related topics:
Primary myelofibrosis
MedlinePlus related topics:
Anemia
| Arm | Intervention/treatment |
|---|---|
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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.
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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 |
Primary Outcome Measures :
- Change in Spleen Volume [ 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.
- Change in 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 :
- 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.
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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:
- >/= 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 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 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 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 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.
- 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.
Exclusion Criteria:
- 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);
- New onset seizures (within 3 months prior to Screening) or poorly controlled seizures;
- Any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy;
- Chronic use (> 2 weeks) of corticosteroids (prednisone >/= 10 mg/24 hr equivalent) within 4 weeks of Screening;
- Investigational therapy within 2 weeks of Screening;
- Psychiatric illness or social situation that would limit the patient's ability to tolerate and/or comply with study requirements.
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
| 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
| Principal Investigator: | Jorge Cortes, MD | M.D. Anderson Cancer Center |
Additional Information:
| 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 |
| Last Update Posted: | August 30, 2019 |
| Last Verified: | August 2019 |
| 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:
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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 |