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Myelodysplastic SyndromesPhase 2AIMSNCT07283900

Ascorbate in Myelodysplastic Syndrome

A Phase 2 study testing High-dose ascorbate and Azacitidine in people with Myelodysplastic Syndromes, run by Prajwal Dhakal at 1 site in United States.

Suspended

Paused. Enrolment is on hold for now and may resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 99 years
Conditions the study is for
Myelodysplastic Syndromes
What is being tested?
High-dose ascorbate and Azacitidine
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
38 participants
Where
1 siteUnited States
Study dates
11 Mar 2026to 1 May 2029
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 99 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • 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.
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  • 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).

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of Iowa

    Iowa City, Iowa, 52242

Other trials in United States

About this study

Official title: A Phase II Trial of High Dose Ascorbate in Combination With Azacitidine in Adults With Myelodysplastic Syndrome

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.

Read the detailed description

This Phase II clinical trial investigates the combination of high-dose intravenous ascorbate (vitamin C) with azacitidine in adults with higher-risk myelodysplastic syndrome (MDS). The study includes a small safety run-in followed by an efficacy phase, enrolling a total of 38 participants. It aims to determine whether adding high-dose ascorbate can safely enhance the therapeutic response to azacitidine, a standard hypomethylating agent used in MDS treatment.

Being tested
High-dose ascorbate, Azacitidine
Keywords
Vitamin C, Ascorbate, Myelodysplastic Syndrome, AIMS
Medical subject headings
Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • High-dose ascorbateDrug

    Also called Vitamin C

    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

  • AzacitidineDrug

    Also called Vidaza

    Azacitidine is a pyrimidine nucleoside analog of cytidine that incorporates into RNA and DNA, inhibiting DNA methyltransferase and leading to global DNA hypomethylation

Study groups in NCT07283900
Study groupRoleWhat they receive
High-Dose Ascorbate + AzacitidineExperimentalAll 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.
Receives: Drug: High-dose ascorbate; Drug: Azacitidine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of dose-limiting toxicities (DLTs)

    Assess the safety and tolerability of intravenous (IV) high-dose ascorbate (HDA) in combination with azacitidine.

    Measured: At the end of Cycle 1 (each cycle is 28 days)

  2. Treatment Efficacy

    Proportion of participants achieving a complete response (CR) or partial response (PR)

    Measured: At the end of Cycle 4 (each cycle is 28 days)

Secondary outcomes (8)

  1. Overall Survival (OS)

    Time from treatment initiation to death from any cause.

    Measured: From treatment initiation until death from any cause or up to 24 months, whichever comes first

  2. Event-Free Survival (EFS)

    Time from treatment initiation to progression, relapse, treatment failure, or death.

    Measured: From treatment initiation until disease progression, disease relapse, treatment failure, or death from any cause, whichever came first, assessed up to 24 months

  3. Transfusion Requirements

    Changes in red blood cell and platelet transfusion needs during treatment.

    Measured: At baseline, assessed throughout the treatment up to the end of cycle 4 (each cycle is 28 days)

  4. Hematologic Parameters

    Changes in hemoglobin, platelet, and neutrophil counts.

    Measured: At baseline, assessed throughout the treatment up to the end of cycle 4 (each cycle is 28 days)

  5. Composite Complete Response (cCR) Rate

    Proportion of participants achieving CR, CRh, CRL, or CR-equivalent per IWG 2023 criteria.

    Measured: At the end of cycle 4 (each cycle is 28 days)

  6. Overall Response Rate (ORR)

    Proportion of participants achieving CR, CRh, CRL, PR, or hematologic improvement (HI) per IWG 2023 criteria after four treatment cycles

    Measured: At the end of cycle 4 (each cycle is 28 days)

  7. Health-Related Quality of Life (HRQOL) Using European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30)

    HRQOL will be assessed using the EORTC QLQ-C30 questionnaire. This outcome measure will evaluate participants' overall quality of life, functional status, and symptom burden at various time points throughout the study. Scoring Range: 0 to 100 for each scale. Functioning Scales: Higher scores = better functioning. Symptom Scales: Higher scores = worse symptoms. Global Health Status/QOL Scale: Higher scores = better overall QOL.

    Measured: At baseline, at the end of cycle 4, every 3 months after the end of cycle 4 up to 24 months (each cycle is 28 days)

  8. Health-Related Quality of Life (HRQOL) using EuroQol (EQ-5D-5L) questionnaire

    HRQOL will be assessed using the EQ-5D-5L questionnaire, which uses a descriptive system and a visual analogue scale. The descriptive system will evaluate participants' mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Scoring range: 11111 to 55555. Higher scores= worse problems. The visual analog scale asks patients to rate their current health on a scale of 0 to 100. Higher scores= better health status.

    Measured: At baseline, at the end of cycle 4, every 3 month after end of cycle 4 up to 24 months (each cycle is 28 days)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Dec 2025
  2. Study start11 Mar 2026
  3. Primary completion1 May 2028 (estimated)
  4. Study completion1 May 2029 (estimated)

Why it stopped: temporary suspension

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
38 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07283900
Sponsor's protocol ID
202508099
Acronym
AIMS
Status last verified
Oct 2026
First submitted
19 Nov 2025
Last updated
9 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing High-dose ascorbate and Azacitidine in people with Myelodysplastic Syndromes, run by Prajwal Dhakal at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: paused, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of dose-limiting toxicities (DLTs) (time frame: At the end of Cycle 1 (each cycle is 28 days)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07283900.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2026.

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