NCT01904682

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

Completed Phase 2 Last updated 15 June 2021

What this trial tests

Phase 2 trial testing Oral rigosertib in Myelodysplastic Syndromes in 45 participants. Completed in 1 May 2021.

Timeline

1 July 2013

Primary endpoint
1 December 2020

1 May 2021

Quick facts

Lead sponsor	Traws Pharma, Inc.
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	45
Start date	1 July 2013
Primary completion	1 December 2020
Estimated completion	1 May 2021
Sites	14 locations across United States, France, Germany

Drugs / interventions tested

• Oral rigosertib — full drug profile →

Conditions studied

• Myelodysplastic Syndromes — all drugs for Myelodysplastic Syndromes →

Sponsor

Traws Pharma, Inc. — full company profile →

Who can join

18 and older, any sex, with Myelodysplastic Syndromes. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Hematologic Improvement
  Time frame: 24 Weeks
  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.

Sponsor's own description

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.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Targeting PI3K in cancer: mechanisms and advances in clinical trials.
   Yang J, Nie J, Ma X, Wei Y, et al · · 2019 · cited 1142× · PMID 30782187 · DOI 10.1186/s12943-019-0954-x
2. Targeting Polo-Like Kinases: A Promising Therapeutic Approach for Cancer Treatment.
   Liu X. · · 2015 · cited 132× · PMID 26055176 · DOI 10.1016/j.tranon.2015.03.010
3. Risk-Adapted, Individualized Treatment Strategies of Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML).
   Bewersdorf JP, Zeidan AM. · · 2021 · cited 21× · PMID 33807279 · DOI 10.3390/cancers13071610
4. Identification of a novel Polo-like kinase 1 inhibitor that specifically blocks the functions of Polo-Box domain.
   Chen Y, Zhang J, Li D, Jiang J, et al · · 2017 · cited 16× · PMID 27902479 · DOI 10.18632/oncotarget.13603
5. Lights and Shadows on the Cancer Multi-Target Inhibitor Rigosertib (ON-01910.Na).
   Monfort-Vengut A, de Cárcer G. · · 2023 · cited 12× · PMID 37111716 · DOI 10.3390/pharmaceutics15041232
6. Molecular Insights and Therapeutic Advances in Low-Risk Myelodysplastic Neoplasms: A Clinical Review.
   Dhillon V, Maciejewski J, Balasubramanian SK. · · 2025 · cited 1× · PMID 41300977 · DOI 10.3390/cancers17223610