Recruiting
Phase 2

HDA & Azacitidine

Sponsor:

Prajwal Dhakal

Code:

NCT07283900

Conditions

Myelodysplastic Syndromes

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

High-dose ascorbate

Azacitidine

Study Details

Brief summary:

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.

Conditions

Myelodysplastic Syndromes

Study ID

NCT07283900

Start date

Mar 11, 2026

Status verified date

Mar, 2026

Completion date

May 1, 2029

Anticipated

Primary completion date

May 1, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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).

Study Design

Enrollment

38 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: High-Dose Ascorbate + Azacitidine

All participants receive the combination of high-dose intravenous ascorbate (75 g on days 1, 3, 5, and 7) and azacitidine (75 mg/m² intravenous or subcutaneous on days 1-7) in 28-day treatment cycles.

Interventions

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

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 measure

  • Incidence of dose-limiting toxicities (DLTs) [ Time Frame: At the end of Cycle 1 (each cycle is 28 days) ]
  • Treatment Efficacy [ Time Frame: At the end of Cycle 4 (each cycle is 28 days) ]

Central Contacts and Locations

Central contacts

Locations

University of Iowa

Recruiting

Iowa City, Iowa, United States, 52242

More Information

Sponsor

Prajwal Dhakal

Last update posted

Mar 16, 2026

Last verified

Mar, 2026

Keywords

  • Vitamin C
  • Ascorbate
  • Myelodysplastic Syndrome
  • AIMS

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Prajwal Dhakal on 2026-03-16.