Trial Outcomes & Findings for Single-Arm Study of the Efficacy and Safety of Oral Rigosertib in Patients With Myelofibrosis (MF) and Anemia (NCT NCT02730884)

Last Updated: 2020-08-07

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Baseline and 48 weeks

Results posted on

2020-08-07

Participant Flow

Recruitment Period: August 2017 to April 2019

Participant milestones

Participant milestones
Measure	Rigosertib 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. Rigosertib: Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon. Questionnaire: Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Single-Arm Study of the Efficacy and Safety of Oral Rigosertib in Patients With Myelofibrosis (MF) and Anemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Rigosertib n=3 Participants 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. Rigosertib: Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon. Questionnaire: Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=93 Participants
Age, Categorical >=65 years	2 Participants n=93 Participants
Age, Continuous	67 years n=93 Participants
Sex: Female, Male Female	2 Participants n=93 Participants
Sex: Female, Male Male	1 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	3 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	3 participants n=93 Participants

PRIMARY outcome

Timeframe: Baseline and 48 weeks

Population: of the three participants, one participant was not evaluable for response.

Outcome measures

Outcome measures
Measure	Rigosertib n=2 Participants 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. Rigosertib: Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon. Questionnaire: Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.
Number of Participants With Spleen Volume Response	0 Participants

PRIMARY outcome

Timeframe: Baseline and 48 weeks

Population: of the three participants, one participant was not evaluable for 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.

Outcome measures

Outcome measures
Measure	Rigosertib n=2 Participants 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. Rigosertib: Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon. Questionnaire: Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.
Participants With Anemia Response	0 Participants

SECONDARY outcome

Timeframe: 48 weeks

Population: of the three participants, one participant was not evaluable for 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.

Outcome measures

Outcome measures
Measure	Rigosertib n=2 Participants 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. Rigosertib: Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon. Questionnaire: Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.
Symptoms Response	0 Participants

Adverse Events

Rigosertib

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Rigosertib n=3 participants at risk 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. Rigosertib: Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon. Questionnaire: Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.
Blood and lymphatic system disorders Anemia	33.3% 1/3 • Number of events 1 • Up to 1 year

Other adverse events

Other adverse events
Measure	Rigosertib n=3 participants at risk 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. Rigosertib: Participants take 560 mg Rigosertib by mouth in the morning (two 280 mg capsules) and 560 mg Rigosertib in the afternoon. Questionnaire: Quality of life questionnaire completed on Day 1 of Cycle 1. It should take about 10-15 minutes to complete.
Investigations Neutrophil Count Decrease	33.3% 1/3 • Number of events 1 • Up to 1 year
Infections and infestations Would Infection	33.3% 1/3 • Number of events 1 • Up to 1 year
Musculoskeletal and connective tissue disorders Myalgia	33.3% 1/3 • Number of events 1 • Up to 1 year
Gastrointestinal disorders Nausea	33.3% 1/3 • Number of events 1 • Up to 1 year
Respiratory, thoracic and mediastinal disorders Dyspnea	33.3% 1/3 • Number of events 1 • Up to 1 year
Investigations Thrombocytopenia	33.3% 1/3 • Number of events 1 • Up to 1 year
Gastrointestinal disorders Dirrhea	33.3% 1/3 • Number of events 1 • Up to 1 year
Cardiac disorders Acute Congestive Heart Failure	33.3% 1/3 • Number of events 1 • Up to 1 year
Renal and urinary disorders Urinary Tract Pain	33.3% 1/3 • Number of events 1 • Up to 1 year
Eye disorders Gastroparesis	33.3% 1/3 • Number of events 1 • Up to 1 year

Additional Information

Jorge Cortes, MD/Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-794-5783

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place