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New treatment option for Low Risk Myelodysplastic Syndromes
Official title Study of R289 in Patients With Lower-risk Myelodysplastic Syndromes (LR MDS)
ClinicalTrials.gov ID: NCT05308264
What this study is testing
What is R906289 Monosodium (R289 Na)?
R906289 Monosodium (R289 Na) is an investigational medicine, being studied as a potential treatment for low risk myelodysplastic syndromes.
Also referred to as R906289 Monosodium.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Phase 1b Study of R289 in Patients with Lower-risk Myelodysplastic Syndromes (LR MDS)
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Patient must be ≥ 18 years of age at the time of signing the informed consent.
- Must have definitive diagnosis of MDS with very low, low, or intermediate-1 risk (International Prognostic Scoring System (IPSS)-R ≤ 3.5) and ≤5%...
- Must be relapsed, refractory/resistant, intolerant, or have inadequate response to therapies with known clinical benefits for MDS, such as EPOs...
- DOSE ESCALATION PHASE: a. Must meet at least one of the following criteria prior to initial administration of study treatment: 1) Symptomatic anemia...
- DOSE EXPANSION PHASE:
You likely can't join if
- Prior treatment for MDS (i.e., TPOs, EPOs, luspatercept, HMAs) concluded \< 4 weeks prior to study treatment
- Clinically significant anemia resulting from iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis, or GI bleeding.
- MDS secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases.
- Diagnosis of chronic myelomonocytic leukemia.
- History of uncontrolled seizures.
- Uncontrolled bacterial or viral infection (i.e., documented HIV, hepatitis B or hepatitis C).
See the full eligibility criteria
- Patient must be ≥ 18 years of age at the time of signing the informed consent.
- Must have definitive diagnosis of MDS with very low, low, or intermediate-1 risk (International Prognostic Scoring System (IPSS)-R ≤ 3.5) and ≤5% bone marrow myeloblasts.
- Must be relapsed, refractory/resistant, intolerant, or have inadequate response to therapies with known clinical benefits for MDS, such as EPOs, luspatercept, and HMAs(i.e., azacytidine or decitabine). Patients with del...
- DOSE ESCALATION PHASE: a. Must meet at least one of the following criteria prior to initial administration of study treatment: 1) Symptomatic anemia with hemoglobin \< 9.0 g/dL and no RBC transfusion within 16 of...
- DOSE EXPANSION PHASE:
- Relapsed, refractory to or ineligible for ESAs and has previously received one or more approved therapies for LR-MDS
- Must be RBC transfusion dependent defined as receiving ≥ 2 units of packed red blood cells (PRBCs) within 8 weeks in the preceding 16 weeks for a hemoglobin \<9.0 g/dL.
- EXPLORATORY PHASE 1b COHORT:
- Transfusion-dependent LR-MDS who are refractory or intolerant to, or are ineligible for ESAs.
- No prior therapy with any approved or investigational therapies for MDS
- No del 5q cytogenetic abnormality
- RBC transfusion dependent defined as receiving ≥ 2 units of PRBCs within 8 weeks in the preceding 16 weeks for a hemoglobin \<9.0 g/dL
- All patients must have documented marrow iron stores. If marrow iron stain is not available, the transferrin saturation must be \>20% or a serum ferritin \> 100ng/100mL
- Must have Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2 at screening.
- Must have adequate organ function, defined as:
- Hepatic function:
- aspartate amino transferase (AST) and alanine aminotransferase (ALT) ≤ 1.5 × upper limit of normal (ULN)
- total bilirubin ≤ 1.5 × ULN
- Renal function defined as creatinine clearance \> 60 mL/min (using Cockcroft-Gault), or blood creatine \< 1.5 mg/dL
- Prior treatment for MDS (i.e., TPOs, EPOs, luspatercept, HMAs) concluded \< 4 weeks prior to study treatment
- Clinically significant anemia resulting from iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis, or GI bleeding.
- MDS secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases.
- Diagnosis of chronic myelomonocytic leukemia.
- History of uncontrolled seizures.
- Uncontrolled bacterial or viral infection (i.e., documented HIV, hepatitis B or hepatitis C).
- History of other malignancy that could affect compliance or interpretation of results. Patients with an malignancy other than leukemia appropriately treated with curative intent and the malignancy has been in remission...
- Adequately treated in situ carcinoma of the cervix uteri
- Adequately treated basal cell carcinoma or localized squamous cell carcinoma of the skin, or
- Low grade, early-stage prostate cancer (Gleason score 6 or below, stage 1 or 2) with no requirement for therapy at any time prior to the study, or previously resected.
- History of or active, clinically significant, cardiovascular, respiratory, GI, renal, hepatic, neurological, psychiatric, musculoskeletal, genitourinary, dermatological, or other disorder that, in the Investigator's...
- Prior history of autologous or allogeneic stem cell transplantation
- Marked baseline prolongation of QT/QTc interval (e.g., repeated demonstration of a QTc interval \> 480 milliseconds [msec]) (Common Terminology Criteria for Adverse Events [CTCAE] Grade 1) using Fridericia's QT...
- History of additional risk factors for TdP (e.g., symptomatic heart failure with left ventricular ejection fraction [LVEF] \<40%, hypokalemia, family history of Long QT Syndrome).
- Receiving any other concurrent chemotherapy, radiotherapy, or immunotherapy (within 2 weeks of initiating study treatment), or the toxicity of the relevant prior treatment has not been resolved yet. For any long-acting...
- Use of concomitant medications that prolong the QT/QTc interval during study treatment
- Use of concomitant medications that are strong CYP3A or CYP2B6 inhibitors or inducers during study treatment
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Orange, California, United States
- Palo Alto, California, United States
- Miami, Florida, United States
- Miami Beach, Florida, United States
- St Louis, Missouri, United States
- Hackensack, New Jersey, United States
- New Brunswick, New Jersey, United States
- New York, New York, United States
- Durham, North Carolina, United States
- Cleveland, Ohio, United States
- Columbus, Ohio, United States
- Dallas, Texas, United States
- Houston, Texas, United States
- Salt Lake City, Utah, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Orange, California, United States; Palo Alto, California, United States; Miami, Florida, United States; Miami Beach, Florida, United States; St Louis, Missouri, United States and 9 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.