NCT07283900 · Prajwal Dhakal
Ascorbate in Myelodysplastic Syndrome
(AIMS)
What this study is about
This is an where both patients and doctors know the treatment given, phase II clinical trial with safety run-in evaluating the safety, tolerability, and effectiveness of IV HDA in combination with azacitidine for participants with MDS.
View original scientific description
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.
Interventions
DRUG
High-dose ascorbate
Ascorbate, or vitamin C, is a water-soluble vitamin with antioxidant properties that also functions as a cofactor for several enzymatic reactions, including collagen synthesis and the activity of dioxygenase enzymes involved in DNA and histone demethylation
DRUG
Azacitidine
Azacitidine is a pyrimidine nucleoside analog of cytidine that incorporates into RNA and DNA, inhibiting DNA methyltransferase and leading to global DNA hypomethylation
Primary outcome measures
Incidence of dose-limiting toxicities (DLTs)
Time frame: At the end of Cycle 1 (each cycle is 28 days)
Assess the safety and tolerability of intravenous (IV) high-dose ascorbate (HDA) in combination with azacitidine.
Treatment Efficacy
Time frame: At the end of Cycle 4 (each cycle is 28 days)
Proportion of participants achieving a complete response (CR) or partial response (PR)
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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).
Where
- Iowa City, Iowa
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 16, 2026 · Source of record for eligibility and locations