NCT01241500

Brief Summary

The primary objective of this study is to compare overall survival (OS) in patients receiving ON 01910.Na + best supportive care (BSC) to OS of patients receiving BSC in a population of patients with myelodysplastic syndrome (MDS) with excess blasts (5% to 30% bone marrow blasts) who have failed azacitidine or decitabine treatment. This patient population has no available therapy and a short life expectancy (approximately 4 months). The high level of bone marrow activity of ON 01910.Na documented in Phase 1 and 2 studies has the potential to delay substantially the transition of MDS to Acute Myeloid Leukemia(AML), a very significant and severe complication, which shortens survival of these MDS patients.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
299

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Nov 2010

Longer than P75 for phase_3

Geographic Reach
6 countries

87 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 1, 2010

Completed
Same day until next milestone

Study Start

First participant enrolled

November 1, 2010

Completed
15 days until next milestone

First Posted

Study publicly available on registry

November 16, 2010

Completed
7.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 3, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 3, 2018

Completed
Last Updated

June 30, 2020

Status Verified

July 1, 2018

Enrollment Period

7.9 years

First QC Date

November 1, 2010

Last Update Submit

June 29, 2020

Conditions

Keywords

Myelodysplastic syndromesMDS

Outcome Measures

Primary Outcomes (1)

  • Overall survival

    Overall survival (OS) is defined as the time from randomization to death from any cause. All patients will be followed until death or progression, even if they have discontinued treatment for whatever cause. Patients lost to follow-up will be censored at the time last known alive. The OS primary analysis will compare the active ON 01910.Na regimen to BSC once a total number of 223 deaths has been reached.

    Up to 18 months

Secondary Outcomes (8)

  • Overall response (complete and partial remission) according to 2006 IWG criteria

    Changes measured at Week 4 from Baseline and every 8 Weeks thereafter

  • Complete bone marrow response according to 2006 IWG criteria

    Changes measured at Week 4 from Baseline and every 8 Weeks thereafter

  • Hematological improvements according to 2006 IWG criteria

    Weekly

  • Scores of Quality of Life Questionnaire

    Measured at Baseline and every 4 Weeks

  • Adverse events

    Weekly

  • +3 more secondary outcomes

Study Arms (2)

ON 01910.Na + best supportive care (BSC)

EXPERIMENTAL

Patients will receive ON 01910.Na 1800 mg/24 hr as a continuous intravenous infusion for 72 hours every other week for the first 16 weeks then every 4 weeks afterwards and best supportive care (BSC).

Drug: ON 01910.Na

Best supportive care (BSC)

NO INTERVENTION

Patients will receive best supportive care (BSC).

Interventions

The dose of ON 01910.Na will be 1800 mg/24 hr as a continuous intravenous infusion for 72 hours every other week for the first 16 weeks then every 4 weeks afterwards. Infusion bags must be changed every 24 hours and a new infusion bag must be used for each of the subsequent 24 hours until completion of the total 72-hour infusion time.

Also known as: rigosertib
ON 01910.Na + best supportive care (BSC)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • MDS diagnosis confirmed within 6 weeks prior to entry according to WHO or FAB classification
  • MDS classified as follows, according to WHO and FAB classification:
  • RAEB-1 (5% - 9% BM blasts)
  • RAEB-2 (10% - 19% BM blasts)
  • CMML (10% - 20% BM blasts) and WBC \< 13,000/μL
  • RAEB-t (20% - 30% BM blasts), with following criteria:
  • o WBC \< 25 x 10E9/L at entry
  • o Stable WBC at least 4 weeks prior to entry and not requiring intervention for WBC control with hydroxyurea, chemotherapy, or leukopheresis.
  • At least one cytopenia (ANC \< 1800/µL or platelet count \< 100,000/µL or hemoglobin \<10 g/dL)
  • Progression according to 2006 International Working Group (IWG) criteria any time after start of azacitidine or decitabine during past 2 years; or failure to achieve complete or partial response or hematological improvement (according to 2006 IWG) after at least six 4-week cycles of azacitidine or four 6-week cycles of decitabine during past 2 years; or relapse after initial complete or partial response or hematological improvement (according to 2006 IWG criteria) observed after at least six 4-week cycles of azacitidine or four 6-week cycles of decitabine during past 2 years; or, intolerance to azacitidine or decitabine defined by drug-related ≥Grade 3 liver or renal toxicity leading to discontinuation during the past 2 years.
  • Did not respond to, relapsed after, not eligible for, or opted not to do bone marrow transplantation
  • Off other MDS treatments for at least 4 weeks; Filgrastim (G-CSF) and erythropoietin allowed before and during the study as clinically indicated.
  • No need for induction chemotherapy
  • ECOG status 0, 1 or 2
  • Willing to adhere to protocol prohibitions and restrictions
  • +1 more criteria

You may not qualify if:

  • Anemia due to factors other than MDS (including hemolysis or gastrointestinal bleeding) unless stabilized for 1 week after RBC transfusion.
  • 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
  • Total bilirubin ≥1.5 mg/dL not related to hemolysis or Gilbert's disease.
  • Alanine transaminase (ALT)/aspartate transaminase (AST) ≥2.5 x upper limit of normal (ULN)
  • Serum creatinine ≥2.0 mg/dL
  • Ascites requiring active medical management including paracentesis, or hyponatremia (defined as serum sodium value of \<130 mEq/L)
  • Pregnant or lactating females
  • Patients unwilling to follow strict contraception requirements (including condom use for males with sexual partners, and for females: prescription oral contraceptives \[birth control pills\], contraceptive injections, intrauterine device, double-barrier method \[spermicidal jelly or foam with condoms or diaphragm\], contraceptive patch, or surgical sterilization) before entry and throughout the study
  • Females with reproductive potential who do not have a negative urine beta-human chorionic gonadotropin pregnancy test at screening
  • Major surgery without full recovery or major surgery within 3 weeks of ON 01910.Na treatment start
  • Uncontrolled hypertension (defined as systolic pressure ≥160 mmHg and/or diastolic pressure ≥110 mmHg)
  • New onset seizures (within 3 months prior to first dose of ON 01910.Na) or poorly controlled seizures
  • Any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (87)

Virginia G. Piper Cancer Center

Scottsdale, Arizona, 85258, United States

Location

University of California San Diego Moores Cancer Center

La Jolla, California, 92093, United States

Location

Stanford Cancer Center

Stanford, California, 94305, United States

Location

Yale Cancer Center

New Haven, Connecticut, 06520, United States

Location

Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States

Location

Integrated Community Oncology Network

Jacksonville, Florida, 32256, United States

Location

Innovative Medical Research of South Florida, Inc.

Miami, Florida, 33169, United States

Location

Mount Sinai Comprehensive Cancer Centers

Miami Beach, Florida, 33140, United States

Location

Woodlands Medical Specialists

Pensacola, Florida, 32503, United States

Location

Martin Memorial Cancer Center

Stuart, Florida, 34994, United States

Location

Cleveland Clinic Florida

Weston, Florida, 33331, United States

Location

Emory University Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

University of Chicago Medical Center

Chicago, Illinois, 60637, United States

Location

North Shore Medical Center

Evanston, Illinois, 60201, United States

Location

Cardinal Bernardin Cancer Center

Maywood, Illinois, 60153, United States

Location

Edward H. Kaplan MD & Associates

Skokie, Illinois, 60076, United States

Location

University of Kansas Medical Center

Westwood, Kansas, 66205, United States

Location

Mary Bird Perkins Cancer Center

Baton Rouge, Louisiana, 70809, United States

Location

University of Maryland Greenebaum Cancer Center

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21231, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 48109, United States

Location

Providence Cancer Center

Southfield, Michigan, 48075, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Overlook Hospital

Summit, New Jersey, 07901, United States

Location

North Shore - LIJ Health System

Lake Success, New York, 11042, United States

Location

Weill Cornell Medical College

New York, New York, 10021, United States

Location

Mount Sinai Medical Center

New York, New York, 10029, United States

Location

Albert Einstein College of Medicine

The Bronx, New York, 10461, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

University of Oklahoma Health Science Center

Oklahoma City, Oklahoma, 73104, United States

Location

University of Pennsylvania Health System

Philadelphia, Pennsylvania, 19104, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Bon Secours St. Francois Health System

Greenville, South Carolina, 29601, United States

Location

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, 75390, United States

Location

University of Texas M. D. Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Cancer Care Centers of South Texas

San Antonio, Texas, 78229, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

H. Hartziekenhuis Roeselare-Menen vzw

Roeselare, West-vlaanderen, 8800, Belgium

Location

Ziekenhuis Netwerk Antwerpen

Antwerp, 2060, Belgium

Location

Universitair Ziekenhuis Gent

Ghent, 9000, Belgium

Location

CHU de Mont-Godinne

Yvoir, 5530, Belgium

Location

CHU Angers Service de Medecine D - Maladies du Sang

Angers, 49033, France

Location

CHU Avignon Centre Hospitalier Henri Dufaut

Avignon, 84000, France

Location

Hôpital Avicenne Hématologie Clinique

Bobigny, 93009, France

Location

CHU Caen Hématologie Clinique

Caen, 14000, France

Location

CHU Estaing Service d'hématologie

Clermont-Ferrand, 63000, France

Location

CHU Lille Hôpital Claude Huriez

Lille, 59037, France

Location

CHU Limoges Hopital Dupuytren

Limoges, 87042, France

Location

Institute Paoli Calmettes

Marseille, 13009, France

Location

Hôpital de L'archet I

Nice, 6202, France

Location

Hôtel Dieu Sce Hématologie Clinique

Paris, 75004, France

Location

Hôpital Saint-Antoine

Paris, 75571, France

Location

CHU Perpignan Centre Hospitalier Hôpital Saint-Jean

Perpignan, 66046, France

Location

CRLCC Henri Becquerel

Rouen, 76038, France

Location

Chu-Strasbourg-Hopital Civil

Strasbourg, 67091, France

Location

Hôpital Purpan

Toulouse, 31059, France

Location

Universitätsklinikum Bonn

Bonn, North Rhine-Westphalia, 53127, Germany

Location

Universitätsklinikum zu Köln

Cologne, 50924, Germany

Location

Universitätsklinikum Dresden

Dresden, 01307, Germany

Location

Heinrich-Heine-Universität Düsseldorf

Düsseldorf, 40225, Germany

Location

Klinikum der Johann Wolfgang-Goethe-Universität

Frankfurt am Main, 60590, Germany

Location

Universitätsklinikum Hamburg-Eppendorf

Hamburg, 20246, Germany

Location

Universitätsmedizin Mannheim

Mannheim, 68167, Germany

Location

Johannes-Wesling-Klinikum Minden

Minden, 32429, Germany

Location

Klinikum Rechts der Isar der Technischen Universität München

München, 81675, Germany

Location

Universitätsklinikum Ulm

Ulm, 89081, Germany

Location

Azienda Ospedaliera Universitaria Senese Policlinico Santa Maria alle Scotte

Siena, SI, 53100, Italy

Location

Azienda Ospedaliera Santi Antonio e Biagio e Cesare Arrigo

Alessandria, 15100, Italy

Location

Azienda Ospedaliero-Universitaria di Bologa Policlinico S. Orsola-Malpighi

Bologna, 40138, Italy

Location

Azienda Ospedaliera-Universitairia Vittorio Emanuele-Ferrarotto-Santo Bambino

Catania, 95124, Italy

Location

Azienda Ospedaliera Universitaria Careggi di Firenze

Florence, 50134, Italy

Location

Azienda Ospedaliera Universitaria San Martino

Genova, 16132, Italy

Location

Azienda Osperdaliera Universitaria Maggiore della Carità

Novara, 28100, Italy

Location

Università degli Studi La Sapienza

Roma, 00161, Italy

Location

Azienda Ospedaliero Universitaria San Giovanni Battista di Torino

Torino, 10126, Italy

Location

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, 33006, Spain

Location

Hospital Universitario La Princesa

Madrid, 28006, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Clínico Universitario Virgen de la Victoria

Málaga, 29010, Spain

Location

Hospital Universitario Son Espases

Palma de Mallorca, 07012, Spain

Location

Hospital Clínico Universitario de Salamanca

Salamanca, 37007, Spain

Location

Hospital Universitari i Politècnic La Fe

Valencia, 46009, Spain

Location

Hospital Clínico Universitario de Valencia

Valencia, 46010, Spain

Location

Related Publications (6)

  • 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.

    PMID: 21924492BACKGROUND
  • Athuluri-Divakar SK, Vasquez-Del Carpio R, Dutta K, Baker SJ, Cosenza SC, Basu I, Gupta YK, Reddy MV, Ueno L, Hart JR, Vogt PK, Mulholland D, Guha C, Aggarwal AK, Reddy EP. A Small Molecule RAS-Mimetic Disrupts RAS Association with Effector Proteins to Block Signaling. Cell. 2016 Apr 21;165(3):643-55. doi: 10.1016/j.cell.2016.03.045.

    PMID: 27104980BACKGROUND
  • Garcia-Manero G, Fenaux P, Al-Kali A, Baer MR, Sekeres MA, Roboz GJ, Gaidano G, Scott BL, Greenberg P, Platzbecker U, Steensma DP, Kambhampati S, Kreuzer KA, Godley LA, Atallah E, Collins R Jr, Kantarjian H, Jabbour E, Wilhelm FE, Azarnia N, Silverman LR; ONTIME study investigators. Rigosertib versus best supportive care for patients with high-risk myelodysplastic syndromes after failure of hypomethylating drugs (ONTIME): a randomised, controlled, phase 3 trial. Lancet Oncol. 2016 Apr;17(4):496-508. doi: 10.1016/S1470-2045(16)00009-7. Epub 2016 Mar 9.

  • 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.

  • Al-Kali A. Relationship of bone marrow blast (BMBL) response to overall survival (OS) in a multicenter study of rigosertib (Rigo) in patients (pts) with myelodysplastic syndrome (MDS) with excess blasts progressing on or after treatment with a hypomethylating agent (HMA). Journal of Clinical Oncology 2017 35:15_suppl, 7056-7056 ASCO 2017

    RESULT
  • 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.

    RESULT

Related Links

MeSH Terms

Conditions

Myelodysplastic SyndromesAnemia, Refractory, with Excess of BlastsLeukemia, Myelomonocytic, Chronic

Interventions

ON 01910

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesAnemia, RefractoryAnemiaLeukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsMyelodysplastic-Myeloproliferative DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Steven M. Fruchtman, MD

    Traws Pharma, Inc.

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 1, 2010

First Posted

November 16, 2010

Study Start

November 1, 2010

Primary Completion

October 3, 2018

Study Completion

October 3, 2018

Last Updated

June 30, 2020

Record last verified: 2018-07

Locations