What's the purpose of this trial?
This is an open-label, phase II clinical trial with safety run-in evaluating the safety, tolerability, and efficacy of IV HDA in combination with azacitidine for participants with MDS.
This trial is currently open and accepting patients.
You may be able to join this trial if you:
The following criteria is a partial list of reasons why patients may be
eligible to participate in this clinical trial. Further evaluation with a medical professional is
required.
Inclusion Criteria
* Age ≥ 18 years.
* Diagnosis of myelodysplastic syndrome (MDS) requiring treatment with a hypomethylating agent (HMA).
* Higher-risk MDS per the Molecular International Prognostic Scoring System (IPSS-M) - Moderate High, High, or Very High risk categories.
* No prior MDS-directed therapy, except:
≤ 1 prior cycle of azacitidine, decitabine, or oral decitabine-cedazuridine; or prior use of ESA, luspatercept, or imetelstat. Prior hydroxyurea use is allowed but continuation beyond Cycle 1 requires PI approval.
* ECOG performance status 0-2.
* Adequate organ function: Creatinine clearance \>45 mL/min; total bilirubin ≤1.5 × ULN; ALT and AST ≤3 × ULN.
* Ability to provide written informed consent.
* Willingness to comply with study visits, treatment, and contraception requirements.
* Negative pregnancy test for women of childbearing potential at screening.
Exclusion Criteria
* MDS with isolated del(5q) eligible for lenalidomide therapy.
* MDS/MPN overlap syndromes other than MDS.
* Known hypersensitivity or allergy to ascorbate or azacitidine.
* Pregnant or nursing individuals.
* Inability or unwillingness to use adequate contraception.
* Uncontrolled intercurrent illness including active infection, recent myocardial infarction (≤6 months), uncontrolled heart failure or arrhythmia, pulmonary edema, unstable angina, or significant psychiatric illness.
* Renal disease requiring dialysis, diabetic nephropathy, renal transplant recipients, or history of oxalate nephropathy.
* Paroxysmal nocturnal hemoglobinuria.
* Uncontrolled HIV infection (patients on effective antiretroviral therapy with undetectable viral load within 6 months are eligible).
* G6PD deficiency.
* Use of warfarin (due to potential interaction with high-dose ascorbate).
* Diabetic patients using fingerstick or continuous glucose monitors to adjust insulin doses (ascorbate can cause false readings).
* Concurrent active malignancy, except adequately treated nonmelanoma skin cancer or curatively treated in situ cancers with \>2 years disease-free.
* Systemic immunosuppressive therapy with prednisone ≥20 mg/day (or equivalent), except for inhaled or topical steroids.
* Primary hemochromatosis or transfusion-related iron overload (ferritin \>1000 ng/mL).
Additional Trial Information
Phase 2
Enrollment: 38 patients (estimated)
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