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Myelodysplastic SyndromesPhase 2NCT01904682

Oral Rigosertib in Low Risk MDS Patients Refractory to ESAs

A Phase 2 study testing Oral rigosertib in people with Myelodysplastic Syndromes, run by Traws Pharma, Inc. at 14 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndromes
What is being tested?
Oral rigosertib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
45 participants
Where
14 sitesin 3 countries
Study dates
Jul 2013to May 2021
Phase
Phase 2
Are there results?
None posted in the registry

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

  • 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.

Who cannot join

  • 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.
Show all 22Show fewer
  • 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.

Trial locations and contacts

14 sites in 3 countries.

14 sites in 3 countries

United States10 sites

  • Anschutz Cancer Pavilion University of Colorado

    Aurora, Colorado, 80045

  • Greenbaum Cancer Center University of Maryland

    Baltimore, Maryland, 21201

  • The University of Chicago

    Chicago, Illinois, 60637

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

  • The University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

  • Rutgers Cancer Institute of New Jersey

    New Brunswick, New Jersey, 08903

  • Mount Sinai Medical Center

    New York, New York, 10029

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Stanford University School of Medicine

    Stanford, California, 94305

  • Washington Cancer Institute at Medstar Washington Hospital Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

Germany3 sites

  • Universitätsklinikum Köln

    Cologne, North Rhine-Westphalia, 50937

  • Universitätsklinikum Carl Gustav Carus an der Technischen Universität Dresden

    Dresden, Saxony, 01307

  • Heinrich Heine Universität

    Düsseldorf, North Rhine-Westphalia, 40225

Other trials in Germany

France1 site

  • Hôpital Saint-Louis, Service d'Hématologie

    Paris, IDF, 75475

Other trials in France

About this study

Official title: A Single-arm Study to Assess the Efficacy and Safety of Oral Rigosertib in Transfusion-dependent, Low or Intermediate-1, Myelodysplastic Syndrome Patients Based on the International Prognostic Scoring System

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.

Read the detailed description

This will be a Phase II, single-arm, multicenter study (approximately 15 centers). Approximately 40 transfusion-dependent patients with Low- or Int-1 risk Myelodysplastic Syndrome (MDS) by International Prognostic Scoring System (IPSS) will be enrolled and treated with oral rigosertib administered twice daily for 21 consecutive days of a 21-day cycle (continuous regimen) in order to obtain at least 35 evaluable patients treated for at least 8 weeks. Patients will take 560 mg rigosertib (two 280 mg capsules) in the morning and 280 mg (one 280 mg capsule) in the afternoon, in fasting conditions. All patients on intermittent regimen at the time of Amendment 2 of the Protocol will be switched to the continuous regimen, including patients on reduced doses.

Being tested
Oral rigosertib
Keywords
Erythrocyte transfusion, Erythropoiesis-stimulating agent, International Prognostic Scoring System
Medical subject headings
Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Oral rigosertibDrug

    Also called ON 01910.Na, rigosertib sodium

    Dose of 560 mg consists of two (2) 280 mg soft gel capsules of rigosertib.

Study groups in NCT01904682
Study groupRoleWhat they receive
Oral rigosertibExperimentalPatients will take 560 mg oral rigosertib (two 280 mg capsules) in the morning and 280 mg (one 280 mg capsule) in the afternoon, in fasting conditions, for 21 consecutive days of 21-day cycle (continuous regimen).
Receives: Drug: Oral rigosertib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Hematologic Improvement

    The number of patients who achieve hematologic improvement will be documented. Hematologic improvement is defined by the 2006 International Working Group (IWG) response criteria for the erythroid, platelet and neutrophil lineages.

    Measured: 24 Weeks

Secondary outcomes (3)

  1. Overall Response

    The number of patients with a complete remission or a partial remission will be documented. Complete remission and partial remission are defined according to 2006 IWG response criteria for MDS. Overall response = complete remission + partial remission.

    Measured: Up to 2 years

  2. Duration of Response

    The number of weeks a complete remission or a partial remission is observed in a patient will be documented.

    Measured: Up to 2 years

  3. Number of Adverse Events

    Specific safety parameters and procedures will include recording of medical history, medication history, physical examination, measurement of vital signs (blood pressure, temperature, respiration rate, and pulse), weight, laboratory evaluations, and toxicity and AE assessments.

    Measured: Up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Jul 2013 (estimated)
  2. Study startJul 2013
  3. Primary completionDec 2020
  4. Study completionMay 2021

Resources

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
45 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01904682
Sponsor's protocol ID
Onconova 09-07
Other IDs
2013-000672-15
Status last verified
Jun 2021
First submitted
14 Jul 2013
Last updated
16 Jun 2021
Results posted
No
Data monitoring committee
No

Publications

  • Seetharam M, Fan AC, Tran M, Xu L, Renschler JP, Felsher DW, Sridhar K, Wilhelm F, Greenberg PL. Treatment of higher risk myelodysplastic syndrome patients unresponsive to hypomethylating agents with ON 01910.Na. Leuk Res. 2012 Jan;36(1):98-103. doi: 10.1016/j.leukres.2011.08.022. Epub 2011 Sep 14.

    PubMed 21924492

  • Navada SC, Silverman LR. The safety and efficacy of rigosertib in the treatment of myelodysplastic syndromes. Expert Rev Anticancer Ther. 2016 Aug;16(8):805-10. doi: 10.1080/14737140.2016.1209413. Epub 2016 Jul 15.

    PubMed 27400247

  • Garcia-Manero G, Fenaux P. Comprehensive Analysis of Safety: Rigosertib in 557 Patients with Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML). Blood Dec 2016, 128 (22) 2011; ASH 2016.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Oral rigosertib in people with Myelodysplastic Syndromes, run by Traws Pharma, Inc. at 14 sites in 3 countries.

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 8 inclusion and 22 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: finished, as of the sponsor's last update on 16 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in United States, Germany, France. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Hematologic Improvement (time frame: 24 Weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01904682, which also lists 3 linked publications.

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 16 Jun 2021.

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