R289 for Myelodysplastic Syndrome

Recruiting at 9 trial locations

Overseen By Strait Hicklin

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Rigel Pharmaceuticals

Must not be taking: QT prolonging, CYP3A inhibitors

Disqualifiers: Chronic myelomonocytic leukemia, Uncontrolled infections, Active malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

86 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new drug called R289 to see if it is safe and effective for patients with lower-risk myelodysplastic syndromes who haven't responded to other treatments.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications that prolong the QT/QTc interval or are strong CYP3A or CYP2B6 inhibitors or inducers during the study. If you are on any of these medications, you may need to stop them before participating.

Eligibility Criteria

This trial is for adults over 18 with lower-risk Myelodysplastic Syndromes (LR MDS) who haven't responded well to other treatments like TPOs, EPOs, luspatercept, and HMAs. They should have low bone marrow myeloblasts and issues like needing blood transfusions or having low platelet counts. Participants need good liver and kidney function and can't join if they've had recent MDS treatment or certain health problems.

Inclusion Criteria

My MDS hasn't responded well to standard treatments like TPOs, EPOs, or HMAs.

My MDS is classified as very low, low, or intermediate-1 risk with ≤5% bone marrow blasts.

My liver and kidney functions are within normal ranges.

Exclusion Criteria

I have had a bone marrow transplant in the past.

I haven't had cancer in the last 2 years, except for certain skin cancers or cervical cancer.

I have risk factors for a specific heart rhythm problem, including weak heart function or low potassium.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive R289 at varying doses to determine tolerability and preliminary efficacy

24 weeks

Regular visits for dose administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term follow-up

Participants are monitored for long-term safety and pharmacodynamic changes

1 year

Treatment Details

Interventions

• R906289 Monosodium (R289 Na)

Trial Overview The study tests R289 Na in patients with LR MDS to see its safety and effectiveness. It's a Phase 1b trial which means it's early in the testing process focusing on how people react to the drug at different doses.

Participant Groups

Group I: Experimental Experimental Treatment 1 Intervention

Dose Level 1: 250mg PO qd Dose Level 2: 500mg PO qd Dose Level 3: 750 mg PO qd Dose Level 4: 250 mg PO bid Dose Level 5: 500 mg PO AM/250 mg PO PM Dose Level 6: 500 mg PO bid

R906289 Monosodium (R289 Na) is already approved in United States for the following indications:

Approved in United States as R289 for:

• Lower-risk Myelodysplastic Syndromes (LR MDS)

Who Is Running the Clinical Trial?

Rigel Pharmaceuticals

Lead Sponsor

Trials

37

Recruited

4,000+