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Myelodysplastic SyndromePhase 2ONTARGETNCT01584531

Efficacy and Safety of Oral Rigosertib in Transfusion-dependent, Low or Int-1 or Trisomy 8 Int-2 Myelodysplastic Syndrome

A Phase 2 study testing rigosertib in people with Myelodysplastic Syndrome and MDS, run by Traws Pharma, Inc. at 5 sites in United States.

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 primary objectives of this study are to determine if rigosertib sodium, given orally in the form of soft gel capsules, is safe and is associated with a reduction in the number of blood transfusion units that are needed in patients with myelodysplastic syndrome (MDS) classified as Low or Intermediate-1 (Int-1) (any… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndrome, MDS, Trisomy 8
What is being tested?
rigosertib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
82 participants
Where
5 sitesUnited States
Study dates
May 2012to Nov 2015
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 confirmed by bone marrow aspirate and/or biopsy within 6 weeks prior to first dose of study drug according to World Health Organization (WHO) or French-American-British (FAB) classification
  • MDS classified as Low risk or Int-1 risk (any cytogenetics) or Trisomy 8 Int-2 risk, according to IPSS classification
  • Transfusion dependency defined by at least 4 units of RBC administered within 8 weeks before baseline
  • Off all other treatments for MDS (azacitidine, decitabine, lenalidomide, chemotherapy, immunosuppressive agents) for at least 4 weeks
  • ECOG performance status of 0, 1 or 2

Who cannot join

  • Ongoing clinically significant anemia due to factors such as iron, B12, or folate deficiencies, auto-immune or hereditary hemolysis, or gastrointestinal (GI) bleeding, unless stabilized for 1 week after RBC transfusion
  • 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 21Show fewer
  • Total bilirubin ≥1.5 mg/dL not related to hemolysis or Gilbert's disease
  • ALT/AST ≥2.5 x 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 who are unwilling to follow strict contraception requirements
  • Female patients with reproductive potential who do not have a negative urine beta-human chorionic gonadotropin (bHCG) pregnancy test at Screening
  • Major surgery without full recovery or major surgery within 3 weeks of rigosertib treatment start
  • 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 starting rigosertib
  • Investigational therapy within 4 weeks of starting rigosertib
  • Psychiatric illness or social situation that would limit the patient's ability to tolerate and/or comply with study requirements

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • Winship Cancer Institute, Emory University

    Atlanta, Georgia, 30322

  • Bon Secours St. Francis Hospital

    Greenville, South Carolina, 29601

  • Columbia University Medical Center

    New York, New York, 10032

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • Mayo Clinic

    Scottsdale, Arizona, 85259

Other trials in United States

About this study

Official title: A Phase II, Multicenter, Single-arm Study to Assess the Efficacy and Safety of Oral Rigosertib in Transfusion-dependent Low or Intermediate-1 (Any Cytogenetics) or Trisomy 8 Intermediate-2 Myelodysplastic Syndrome Patients Based on IPSS Classification

The primary objectives of this study are to determine if rigosertib sodium, given orally in the form of soft gel capsules, is safe and is associated with a reduction in the number of blood transfusion units that are needed in patients with myelodysplastic syndrome (MDS) classified as Low or Intermediate-1 (Int-1) (any cytogenetics) or trisomy 8 Intermediate 2 (Int-2) in the International Prognostic Scoring System (IPSS) who are transfusion-dependent. Rigosertib will be taken on days 1 to 21 of a 21-day cycle.

Read the detailed description

This will be a Phase II open-label, multicenter (up to 5 centers), single-arm study. Sixty transfusion-dependent patients with MDS classified as Low or Int-1 risk (any cytogenetics) or trisomy 8 Int-2 by International Prognostic Scoring System (IPSS) will be enrolled to receive rigosertib BID for 21 consecutive days of a 21-day cycle.

Patients will be stratified on prior treatment with azacitidine and/or decitabine and/or lenalidomide and/or erythropoietin.

Patients will remain treated on study until 2006 Internation Working Group (IWG) progression criteria are met or until death from any cause.

All study participants will be allowed, as medically justified, access to RBC and platelet transfusions, and to filgrastim [G-CSF]. Erythropoiesis-stimulating agents (ESAs) will not be allowed during the initial 3 cycles. Rigosertib dosing adjustment policies are described in Protocol.

Being tested
rigosertib
Keywords
rigosertib sodium, rigosertib, ON 01910.Na, oral rigosertib
Medical subject headings
Myelodysplastic Syndromes, Chromosome 8, trisomy

What participants receive

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

  • rigosertibDrug

    Also called rigosertib sodium, ON 01910.Na, oral rigosertib

    Rigosertib sodium will be available as soft gel capsules in strengths of 280 mg and 70 mg. Rigosertib will be administered on an outpatient basis.

    Patients will take a 560 mg dose (e.g., 2 x 280 mg capsules) of oral rigosertib in the morning and 280 mg dose (e.g., 1 x 280 mg capsules) of oral rigosertib every day of 21-day cycles. Rigosertib should be taken in a fasting state (defined by at least 30 minutes before next meal) BID at 12 hr intervals (with a window of 2 hr). Any vomited dose will be reported as a missed dose.

    The patient will fill a diary indicating the day and time of drug intake.

Study groups in NCT01584531
Study groupRoleWhat they receive
21-Day RegimenExperimental560 mg oral rigosertib in the morning and 280 mg rigosertib in the afternoon on days 1 to 21 of 21-day cycle
Receives: Drug: rigosertib

What is being measured

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

  1. Number of units of red blood cell transfusions

    Number of units of red blood cell transfusions will be compared with the pretreatment transfusion number in the previous 8 weeks.

    Measured: 8 weeks

Secondary outcomes (3)

  1. Number of Adverse Events (AEs)

    AEs reported by the patient or observed by the Investigator or study site personnel Safety assessments will be counted and documented on Case Report Forms and source documents. All AEs from signature of the ICF through 30 days after a patient discontinues from the study will be included.

    Measured: From date of randomization until 30 days after last dose of study drug

  2. Bone marrow blasts

    Change in number of bone marrow blasts will be compared to pretreatment.

    Measured: 4 weeks

  3. Complete blood count

    Complete blood count with differential.

    Measured: 4 weeks

Timeline

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

  1. First posted25 Apr 2012 (estimated)
  2. Study startMay 2012
  3. Primary completionMay 2015
  4. Study completionNov 2015

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
82 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01584531
Sponsor's protocol ID
Onconova 09-05
Other IDs
AAAJ0151
Acronym
ONTARGET
Status last verified
Jun 2017
First submitted
23 Apr 2012
Last updated
26 Jun 2017
Results posted
No
Data monitoring committee
No

Publications

  • 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 rigosertib in people with Myelodysplastic Syndrome and MDS, run by Traws Pharma, Inc. at 5 sites 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 5 inclusion and 21 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 26 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites 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?

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?

Number of units of red blood cell transfusions (time frame: 8 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 NCT01584531, which also lists 2 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 26 Jun 2017.

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