Acute Myeloid LeukemiaPhase 1NCT07177079
High-dose Ascorbate (HDA) in Combination With Standard of Care Azacitidine and Venetoclax in Acute Myeloid Leukemia (AML)
A Phase 1 study testing Azacitidine, Venetoclax and High-dose ascorbate in people with Acute Myeloid Leukemia, run by Kittika Poonsombudlert at 1 site in United States.
Trial summary
This is a randomized, open-label, Phase I clinical study with expansion. It will assess the safety and efficacy of high-dose ascorbate administered concomitantly with azacitidine and venetoclax in newly diagnosed AML. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Azacitidine, Venetoclax and High-dose ascorbate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Mar 2026to 31 Dec 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults aged ≥ 18 who are deemed unfit for intensive chemotherapy by meeting at least one of the following criteria:
- age ≥ 75
- Eastern Cooperative Oncology Group (ECOG) performance of 2-3
- Severe cardiac disorder (e.g., congestive heart failure requiring treatment, ejection fraction ≤ 50%, or chronic stable angina)
- Severe pulmonary disorder (e.g., DLCO ≤ 65% or FEV1 ≤ 65%)
- Calculated Creatinine clearance 25 - 45 mL/min using the Cockcroft-Gault formula
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- Hepatic disorder with total bilirubin > 1.5 times the upper limit of normal
- Any other comorbidity that the investigators determine to be incompatible with intensive chemotherapy
- Newly diagnosed (non-APL) acute myeloid leukemia except those with cytogenetic/molecular abnormalities in the exclusion criteria
- Participants must have adequate organ function, defined as:
- Aspartate aminotransferase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤ 3.0 x upper limit of normal (ULN)
- International normalized ratio (INR) < 1.5 x ULN and partial thromboplastin time (PTT) < 1.5 x ULN (patient could be eligible if they respond appropriately to correction with FFP or cryoprecipitate)
- Patients with a history of antecedent myelodysplasia (MDS) are eligible if they have not had prior chemotherapy/hypomethylating agent (e.g., azacitidine or decitabine). Prior exposure to other investigational agents could be considered at PI's discretion
- Patients who have developed therapy-related AML after prior radiation or chemotherapy for other malignancy(ies) are eligible if they have not been exposed to hypomethylating agent (e.g., azacitidine or decitabine) and/or venetoclax
- Patients presenting with marked leukocytosis (WBC > 25 k/mm3) should receive cytoreduction with hydroxyurea or cytarabine dose ≤ 1 g/m2 to mitigate the risk of tumor lysis syndrome before initiation of therapy with venetoclax
- For female participants of childbearing potential, a negative serum or urine pregnancy test (sensitivity of at least 25 mIU/mL) at screening
- Ability to understand and the willingness to sign a written informed consent document.
- Both male and female participants of childbearing potential agree to use an adequate method of contraception from screening through 6 months after the last dose of study treatment.
- Patients with evidence of Central Nervous System (CNS) disease involvement or who are at high risk of developing CNS disease at baseline/screening (defined as: presenting WBC > 200k/mm3, or monocytic differentiation or active CSN symptoms concerning CNS involvement per the treating physician) are eligible for this trial and may continue to receive or initiate intrathecal chemotherapy as treatment or prophylaxis, respectively, per institutional practice
Who cannot join
- Patients who have received prior therapy to treat their AML (except for cytoreductive hydroxyurea or cytarabine dose ≤ 1 g/m2 for hyperleukocytosis)
- Known hypersensitivity or allergy to ascorbate, azacitidine/decitabine, or venetoclax
- AML patients with the following cytogenetic/molecular aberrations are not eligible i. t(8;21)(q22;q22.1)/RUNX1::RUNX1T1 ii. inv(16)(p13.1q22) or t(16;16)(p13.1;q22)/ CBFB::MYH11 iii. bZIP in-frame mutated CEBPA without any adverse mutations iv. KMT2A rearrangement v. NPM1 or IDH1 or IDH2 or FLT3-ITD or FLT3-TKD mutation
- Patients with kidney disease needing dialysis, diabetic nephropathy, renal transplant recipients, and those with history of acute or chronic oxalate nephropathy
- Patients with primary hemochromatosis or transfusional iron overload as defined as persistently elevated Ferritin > 1000 ng/mL.
- Patients with type I or type II diabetes mellitus on treatment with short acting insulin who need at least a daily blood glucose monitoring test via finger stick
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- Patients with a prior or concurrent malignancy whose natural history or treatment has the potential to interfere with the safety or efficacy assessment of the investigational regimen
- Other major co-morbidities as determined unsuitable per the treating physician
- HIV-infection that is uncontrolled by anti-retroviral therapy. (HIV-infected patients on effective anti- retroviral therapy with undetectable viral load within 6 months are eligible for this study)
- Patients with G6PD (glucose-6-phosphate dehydrogenase) deficiency
- Patients who are on warfarin or other strong CYP3A4 inducer/inhibitor and cannot have a drug substitution or who decline the drug substitution
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Iowa Health Care
Iowa City, Iowa, 52242
Kittika Poonsombudlert, MD, +1 319 356 1770, kittika-poonsombudlert@uiowa.edu
Kittika Poonsombudlert, +1 319 356 1770, kittika-poonsombudlert@uiowa.edu
About this study
Official title: High-dose Ascorbate (HDA) in Combination With Azacitidine and Venetoclax (Aza/Ven) in Newly Diagnosed Acute Myeloid Leukemia (AML)
This is a randomized, open-label, Phase I clinical study with expansion. It will assess the safety and efficacy of high-dose ascorbate administered concomitantly with azacitidine and venetoclax in newly diagnosed AML.
Read the detailed description
The purpose of this research study is to see if adding high dose ascorbate (vitamin c) intravenous infusion (IV) to the standard treatment regimen for AML (azacitidine/venetoclax or decitabine/venetoclax) is safe, and also to see if the addition of high dose ascorbate enhances the anti-AML impact of the azacitidine given with standard care.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Azacitidine, Venetoclax, High-dose ascorbate, Decitabine
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzacitidineDrug
A chemotherapy drug known as a hypomethylating agent
VenetoclaxDrug
Targeted cancer therapy used to treat certain blood cancers. It specifically targets a protein called BCL-2 to trigger the self-destruction of cancer cells.
High-dose ascorbateDrug
Also called Vitamin C
Administering vitamin C intravenously to achieve very high concentrations in the bloodstream. In contrast to low doses, which act as antioxidants, these pharmacological doses can function as a pro-oxidant, killing cancer cells while leaving healthy cells unharmed.
DecitabineDrug
Azacitidine may be substituted with decitabine 20 mg/m2 daily, on days 1-5, at PI discretion in the event of toxicity/drug supply shortage.
| Study group | Role | What they receive |
|---|---|---|
| A. Standard of Care (azacitidine and venetoclax) | Active comparator | Twelve patients will be randomized to Arm A. Receives: Drug: Azacitidine; Drug: Venetoclax; Drug: Decitabine |
| B. High-dose ascorbate administered concomitantly with azacitidine and venetoclax | Experimental | As this is the first time high-dose ascorbate has been administered in combination with standard of care (azacitidine and venetoclax), the safety of the combination will be assessed in the first 6 patients randomized to Arm B. These patients will continue with the expansion portion of the study, and an additional 6 patients will be added for a total of 12. Receives: Drug: Azacitidine; Drug: Venetoclax; Drug: High-dose ascorbate; Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase I: Dose-limiting toxicities (DLTs) according to CTCAE version 5.0
As this is the first time high-dose ascorbate has been administered in combination with standard of care aza/ven, the safety of the combination will be assessed in the first 6 patients randomized to Arm B. An initial DLT assessment will be made when 3 participants have been randomized to the Investigational Arm B and are evaluable for DLTs. If at most 1 out of 3 participants experience a DLT, an additional cohort of 3 participants will be evaluated for DLTs. If at most 1 out of 6 participants experience a DLT, the combination will be deemed safe. Should greater than or equal to 2 (≥2) out of 3 or 6 participants experience a DLT, the combination will be deemed unsafe, and accrual will be terminated.
Measured: Days 1 through 28
Expansion: Composite complete remission rate defined as the proportion of patients with a complete remission (CR or CRi)
The primary objective of the expansion is to estimate the composite complete remission rate defined as the proportion of patients with a complete remission (CR or CRi) by the end of study treatment for each arm separately.
Measured: Three years from initiation of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2025
- Study start18 Mar 2026
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kittika Poonsombudlert
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Kittika Poonsombudlert, University of Iowa
- Investigators
- Kittika Poonsombudlert, MD, Principal investigator, University of Iowa
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07177079
- Sponsor's protocol ID
- 202506290
- Status last verified
- Aug 2026
- First submitted
- 9 Sep 2025
- Last updated
- 31 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- High-dose Ascorbate (HDA) in Combination With Azacitidine and Venetoclax (Aza/Ven) in Newly Diagnosed Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT07177079
- Acronym / sponsor ID
- 202506290
- Conditions
- Acute Myeloid Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Kittika PoonsombudlertUniversity, hospital or other
- Study start
- 18 Mar 2026 (actual)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 16 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a randomized, open-label, Phase I clinical study with expansion. It will assess the safety and efficacy of high-dose ascorbate administered concomitantly with azacitidine and venetoclax in newly diagnosed AML.
Detailed description
The purpose of this research study is to see if adding high dose ascorbate (vitamin c) intravenous infusion (IV) to the standard treatment regimen for AML (azacitidine/venetoclax or decitabine/venetoclax) is safe, and also to see if the addition of high dose ascorbate enhances the anti-AML impact of the azacitidine given with standard care.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| A. Standard of Care (azacitidine and venetoclax) | Active comparator | Drug: Azacitidine, Drug: Venetoclax, Drug: DecitabineTwelve patients will be randomized to Arm A. |
| B. High-dose ascorbate administered concomitantly with azacitidine and venetoclax | Experimental | Drug: Azacitidine, Drug: Venetoclax, Drug: High-dose ascorbate, Drug: DecitabineAs this is the first time high-dose ascorbate has been administered in combination with standard of care (azacitidine and venetoclax), the safety of the combination will be assessed in the first 6 patients randomized to Arm B. These patients will continue with the expansion portion of the study, and an additional 6 patients will be added for a total of 12. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Adults aged ≥ 18 who are deemed unfit for intensive chemotherapy by meeting at least one of the following criteria:
- Newly diagnosed (non-APL) acute myeloid leukemia except those with cytogenetic/molecular abnormalities in the exclusion criteria
- Participants must have adequate organ function, defined as:
- Patients with a history of antecedent myelodysplasia (MDS) are eligible if they have not had prior chemotherapy/hypomethylating agent (e.g., azacitidine or decitabine). Prior exposure to other investigational agents could be considered at PI's discretion
- Patients who have developed therapy-related AML after prior radiation or chemotherapy for other malignancy(ies) are eligible if they have not been exposed to hypomethylating agent (e.g., azacitidine or decitabine) and/or venetoclax
Key exclusion criteria (11 listed)
- Patients who have received prior therapy to treat their AML (except for cytoreductive hydroxyurea or cytarabine dose ≤ 1 g/m2 for hyperleukocytosis)
- Known hypersensitivity or allergy to ascorbate, azacitidine/decitabine, or venetoclax
- AML patients with the following cytogenetic/molecular aberrations are not eligible i. t(8;21)(q22;q22.1)/RUNX1::RUNX1T1 ii. inv(16)(p13.1q22) or t(16;16)(p13.1;q22)/ CBFB::MYH11 iii. bZIP in-frame mutated CEBPA without any adverse mutations iv. KMT2A rearrangement v. NPM1 or IDH1 or IDH2 or FLT3-ITD or FLT3-TKD mutation
- Patients with kidney disease needing dialysis, diabetic nephropathy, renal transplant recipients, and those with history of acute or chronic oxalate nephropathy
- Patients with primary hemochromatosis or transfusional iron overload as defined as persistently elevated Ferritin > 1000 ng/mL.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AzacitidineA chemotherapy drug known as a hypomethylating agent | Drug | A. Standard of Care (azacitidine and venetoclax), B. High-dose ascorbate administered concomitantly with azacitidine and venetoclax |
| VenetoclaxTargeted cancer therapy used to treat certain blood cancers. It specifically targets a protein called BCL-2 to trigger the self-destruction of cancer cells. | Drug | A. Standard of Care (azacitidine and venetoclax), B. High-dose ascorbate administered concomitantly with azacitidine and venetoclax |
| High-dose ascorbateAlso: Vitamin CAdministering vitamin C intravenously to achieve very high concentrations in the bloodstream. In contrast to low doses, which act as antioxidants, these pharmacological doses can function as a pro-oxidant, killing cancer cells while leaving healthy cells unharmed. | Drug | B. High-dose ascorbate administered concomitantly with azacitidine and venetoclax |
| DecitabineAzacitidine may be substituted with decitabine 20 mg/m2 daily, on days 1-5, at PI discretion in the event of toxicity/drug supply shortage. | Drug | A. Standard of Care (azacitidine and venetoclax), B. High-dose ascorbate administered concomitantly with azacitidine and venetoclax |
Primary outcome
Phase I: Dose-limiting toxicities (DLTs) according to CTCAE version 5.0
Time frame: Days 1 through 28
As this is the first time high-dose ascorbate has been administered in combination with standard of care aza/ven, the safety of the combination will be assessed in the first 6 patients randomized to Arm B. An initial DLT assessment will be made when 3 participants have been randomized to the Investigational Arm B and are evaluable for DLTs. If at most 1 out of 3 participants experience a DLT, an additional cohort of 3 participants will be evaluated for DLTs. If at most 1 out of 6 participants experience a DLT, the combination will be deemed safe. Should greater than or equal to 2 (≥2) out of 3 or 6 participants experience a DLT, the combination will be deemed unsafe, and accrual will be terminated.
Expansion: Composite complete remission rate defined as the proportion of patients with a complete remission (CR or CRi)
Time frame: Three years from initiation of study
The primary objective of the expansion is to estimate the composite complete remission rate defined as the proportion of patients with a complete remission (CR or CRi) by the end of study treatment for each arm separately.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Kittika Poonsombudlert, MD principal investigator
University of Iowa
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Azacitidine, Venetoclax and High-dose ascorbate in people with Acute Myeloid Leukemia, run by Kittika Poonsombudlert 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 19 inclusion and 11 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: looking for participants now, as of the sponsor's last update on 31 Aug 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kittika Poonsombudlert, MD (+1 319 356 1770, kittika-poonsombudlert@uiowa.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase I: Dose-limiting toxicities (DLTs) according to CTCAE version 5.0 (time frame: Days 1 through 28). 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?
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 31 Aug 2026.