LeukemiaPhase 2NCT02730884
Single-Arm Study of the Efficacy and Safety of Oral Rigosertib in Patients With Myelofibrosis (MF) and Anemia
A Phase 2 study testing Rigosertib and Questionnaire in people with Leukemia and Myelofibrosis, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Myelofibrosis, Anemia
- What is being tested?
- Rigosertib and Questionnaire
- Type of treatment
- Pill or oral medicine, Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Aug 2017to 22 Aug 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- >/= 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.
Who cannot join
- 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;
Show all 20Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: A Phase II Single-Arm Study of the Efficacy and Safety of Oral Rigosertib in Patients With Myelofibrosis (MF) and Anemia
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.
Read the detailed description
Study Drug Administration:
If participant is found to be eligible to take part in this study, participant will take rigosertib capsules by mouth 2 times each day (1 morning dose, 1 afternoon dose) for 48 weeks. Participant will take participant's morning dose on an empty stomach. Participant's afternoon dose should be taken at about 3:00 pm (±1 hour), at least 2 hours after lunch. Participant should drink at least a ½ gallon of water each day. Rigosertib should be refrigerated.
Participant will also be given a study drug diary. Participant should write down each dose of study drug that participant takes, when participant takes it, and if participant misses or vomit any doses. If participant misses or vomits a dose of rigosertib, participant should not make up the dose. Participant should take the next scheduled dose as planned. Every 4 weeks for 12 weeks, then every 8 weeks thereafter, participant should bring any leftover rigosertib capsules and participant's study drug diary with participant to the clinic.
Study Visits:
On Cycle 1 Day 1:
- Participant will have a physical exam.
- Blood (about 2-3 teaspoons) and urine will be collected for routine tests.
- Blood (about 2 teaspoons) will be drawn for pharmacokinetic (PK) testing about 1 hour after participant's dose of study drug. PK testing measures the amount of study drug in the body at different time points.
- Participant will complete a questionnaire about participant's quality of life. It should take about 10-15 minutes.
- If the doctor thinks it is needed, participant will have a computed tomography (CT) or magnetic resonance imaging (MRI) scan to check the status of the disease.
Each week, blood (about 2-3 teaspoons) will be drawn for routine tests. If the study doctor approves it, participant may have these weekly blood draws at a local lab closer to participant's home. The results from these routine tests will be sent to the study doctor.
On Day 1 of Week 4, blood (about 2 teaspoons each time) will be drawn for PK testing before and 1 hour after participant's dose of study drug.
At Weeks 24 and 48 (± 1 week):
- Participant will have a bone marrow aspirate/biopsy performed to check the status of the disease. This sample may also be used for cytogenetic testing.
- If participant had a CT/MRI scan performed during Week 1, participant will have another CT/MRI scan to check the status of the disease.
- If participant can become pregnant, urine will be collected for a pregnancy test.
Every 4 weeks until Week 12 and then every 8 weeks after that:
- Participant will have a physical exam.
- Blood (about 2-3 teaspoons) and urine will be collected for routine tests.
- Participant will complete a questionnaire about participant's quality of life.
Length of Study:
Participant may continue taking the study drug for up to 48 weeks. Participant will no longer be able to take the study drugs if the disease gets worse, if intolerable side effects occur, or if participant is unable to follow study directions.
If the doctor thinks it is in participant's best interest, participant may be able to continue taking the study drug after 48 weeks. The study doctor will discuss this with participant.
Participation on the study will be over after the end-of-study visit.
End-of-Study Visit:
About 4 weeks after participant's last dose of study drug, participant will have an end-of-study visit. At this visit:
- Participant will have a physical exam.
- Blood (about 2-3 teaspoons) and urine will be collected for routine tests. This routine blood and urine collection will also include a pregnancy test if participant can become pregnant.
- Conditions
- Leukemia, Myelofibrosis, Anemia, Splenomegaly
- Being tested
- Rigosertib, Questionnaire
- Keywords
- Leukemia, Myelofibrosis, MF, Anemia, Splenomegaly, Post-polycythemia vera MF, post-PV, post-essential thrombocythemia MF, post-ET, Rigosertib, ON 01910.Na, Questionnaire, Survey
- Medical subject headings
- Leukemia, Primary Myelofibrosis, Anemia, Splenomegaly
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RigosertibDrug
Also called ON 01910.Na
Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon.
QuestionnaireBehavioural
Also called Survey
Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Rigosertib; Behavioral: Questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Baseline and 48 weeks
Secondary outcomes (1)
Symptoms Response
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.
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2016 (estimated)
- Study start16 Aug 2017
- Primary completion22 Aug 2019
- Study completion22 Aug 2019
Why it stopped: Terminated Per PIs request due to low accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Traws Pharma, Inc.
- Responsible party
- Sponsor
- Investigators
- Jorge Cortes, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02730884
- Sponsor's protocol ID
- 2014-0546
- Other IDs
- NCI-2016-00761
- Status last verified
- Aug 2020
- First submitted
- 24 Mar 2016
- Last updated
- 7 Aug 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Single-Arm Study of the Efficacy and Safety of Oral Rigosertib in Patients With Myelofibrosis (MF) and Anemia
- ClinicalTrials.gov ID
- NCT02730884
- Acronym / sponsor ID
- 2014-0546
- Conditions
- Leukemia, Myelofibrosis, Anemia, Splenomegaly
- Keywords
- Leukemia, Myelofibrosis, MF, Anemia, Splenomegaly, Post-polycythemia vera MF, post-PV, post-essential thrombocythemia MF
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated Per PIs request due to low accrual
- Enrolment
- 3 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Traws Pharma, Inc.
- Study start
- 16 Aug 2017 (actual)
- Primary completion
- 22 Aug 2019 (actual)
- Study completion
- 22 Aug 2019 (actual)
- First posted
- 7 Apr 2016 (estimated)
- Results
- Posted 7 Aug 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Study Drug Administration:
If participant is found to be eligible to take part in this study, participant will take rigosertib capsules by mouth 2 times each day (1 morning dose, 1 afternoon dose) for 48 weeks. Participant will take participant's morning dose on an empty stomach. Participant's afternoon dose should be taken at about 3:00 pm (±1 hour), at least 2 hours after lunch. Participant should drink at least a ½ gallon of water each day. Rigosertib should be refrigerated.
Participant will also be given a study drug diary. Participant should write down each dose of study drug that participant takes, when participant takes it, and if participant misses or vomit any doses. If participant misses or vomits a dose of rigosertib, participant should not make up the dose. Participant should take the next scheduled dose as planned. Every 4 weeks for 12 weeks, then every 8 weeks thereafter, participant should bring any leftover rigosertib capsules and participant's study drug diary with participant to the clinic.
Study Visits:
On Cycle 1 Day 1:
- Participant will have a physical exam.
- Blood (about 2-3 teaspoons) and urine will be collected for routine tests.
- Blood (about 2 teaspoons) will be drawn for pharmacokinetic (PK) testing about 1 hour after participant's dose of study drug. PK testing measures the amount of study drug in the body at different time points.
- Participant will complete a questionnaire about participant's quality of life. It should take about 10-15 minutes.
- If the doctor thinks it is needed, participant will have a computed tomography (CT) or magnetic resonance imaging (MRI) scan to check the status of the disease.
Each week, blood (about 2-3 teaspoons) will be drawn for routine tests. If the study doctor approves it, participant may have these weekly blood draws at a local lab closer to participant's home. The results from these routine tests will be sent to the study doctor.
On Day 1 of Week 4, blood (about 2 teaspoons each time) will be drawn for PK testing before and 1 hour after participant's dose of study drug.
At Weeks 24 and 48 (± 1 week):
- Participant will have a bone marrow aspirate/biopsy performed to check the status of the disease. This sample may also be used for cytogenetic testing.
- If participant had a CT/MRI scan performed during Week 1, participant will have another CT/MRI scan to check the status of the disease.
- If participant can become pregnant, urine will be collected for a pregnancy test.
Every 4 weeks until Week 12 and then every 8 weeks after that:
- Participant will have a physical exam.
- Blood (about 2-3 teaspoons) and urine will be collected for routine tests.
- Participant will complete a questionnaire about participant's quality of life.
Length of Study:
Participant may continue taking the study drug for up to 48 weeks. Participant will no longer be able to take the study drugs if the disease gets worse, if intolerable side effects occur, or if participant is unable to follow study directions.
If the doctor thinks it is in participant's best interest, participant may be able to continue taking the study drug after 48 weeks. The study doctor will discuss this with participant.
Participation on the study will be over after the end-of-study visit.
End-of-Study Visit:
About 4 weeks after participant's last dose of study drug, participant will have an end-of-study visit. At this visit:
- Participant will have a physical exam.
- Blood (about 2-3 teaspoons) and urine will be collected for routine tests. This routine blood and urine collection will also include a pregnancy test if participant can become pregnant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rigosertib | Experimental | Drug: Rigosertib, Behavioral: QuestionnaireParticipants receive oral Rigosertib under fasting conditions twice a day on a continuous basis. Quality of life questionnaire completed on Day 1 of Cycle 1. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- >/= 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);
Key exclusion criteria (20 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RigosertibAlso: ON 01910.NaParticipants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon. | Drug | Rigosertib |
| QuestionnaireAlso: SurveyQuality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete. | Behavioural | Rigosertib |
Primary outcome
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.
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.
1 secondary outcome
Symptoms Response
Time frame: 48 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jorge Cortes, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rigosertib and Questionnaire in people with Leukemia and Myelofibrosis, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 7 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Aug 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Spleen Volume Response (time frame: Baseline and 48 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2020.