Dose Escalation for Low Risk Myelodysplastic Syndrome

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Low Risk Myelodysplastic SyndromeR906289 Monosodium (R289 Na) - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

The study will be an open-label, Phase 1b study of R289 to determine tolerability and preliminary efficacy in patients with LR MDS who are relapsed, refractory/resistant, intolerant, or have inadequate response to prior therapies such as erythropoietin (EPO), thrombopoietin (TPO), luspatercept, or hypomethylating agents (HMAs) for MDS.

Eligible Conditions
  • Low Risk Myelodysplastic Syndrome

Treatment Effectiveness

Effectiveness Progress

1 of 3

Similar Trials

Study Objectives

1 Primary · 2 Secondary · Reporting Duration: 2 Year

1 year
Characterize pharmacodynamic (PD)
2 Year
Safety and Tolerability
24 Weeks
Red Blood Cell Transfusion
8 Weeks
Characterize pharmacokinetics (PK)

Trial Safety

Safety Progress

1 of 3

Similar Trials

Trial Design

1 Treatment Group

Dose Escalation
1 of 1

Experimental Treatment

22 Total Participants · 1 Treatment Group

Primary Treatment: Dose Escalation · No Placebo Group · Phase 1 & 2

Dose Escalation
Drug
Experimental Group · 1 Intervention: R906289 Monosodium (R289 Na) · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 2 year

Who is running the clinical trial?

Rigel PharmaceuticalsLead Sponsor
28 Previous Clinical Trials
3,411 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
Your total bilirubin is within 1.5 times the upper limit of normal.
You have attained the legal age of majority and are thus eligible to provide informed consent.
You have a definite diagnosis of myelodysplastic syndrome (MDS) with an International Prognostic Scoring System-Revised (IPSS-R) score of 3.5 or lower and no more than 5 percent bone marrow myeloblasts.
You must have been unresponsive, resistant, or unable to tolerate therapies with established clinical effectiveness in MDS (e.g., TPOs, EPOs, luspatercept and HMAs like azacytidine or decitabine)
You have symptomatic anemia that has not been transfused with a hemoglobin of less than 9.0 g/dL within the past 8 weeks, or you are dependent on RBC transfusions having received at least two units of packed red blood cells (PRBCs) in the preceding 16 weeks when your hemoglobin was below 9.0 g/dL.
You have experienced a clinically significant decrease in platelet count below 100 × 109/L at least twice prior to beginning the study, and require blood transfusions.
All participants must have recorded evidence of marrow iron staining; if no such record is available, then the transferrin saturation must exceed 20% or serum ferritin levels should be greater than 100ng/100mL.
You must have an ECOG performance status ranging from 0 to 2 at the start of screening.
Your AST or ALT is at most 1.5 times the upper limit of what is considered normal.
Your renal function is within the normal range, evidenced by a creatinine clearance greater than 60 milliliters per minute (using Cockcroft-Gault) or blood creatine less than 1.5 milligrams per deciliter.