Vitamin C + Standard Care for Acute Myeloid Leukemia

Overseen By Kittika Poonsombudlert, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Kittika Poonsombudlert

Must not be taking: Warfarin, Strong CYP3A4 drugs

Disqualifiers: Prior AML therapy, Diabetes, Kidney disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether adding high-dose vitamin C to standard treatment can effectively and safely assist individuals with newly diagnosed acute myeloid leukemia (AML), a type of blood cancer. The study compares the standard treatment of azacitidine and venetoclax with the same treatment plus high-dose vitamin C (also known as high-dose ascorbate). It seeks participants with a recent non-APL AML diagnosis who cannot undergo intensive chemotherapy due to existing health conditions, such as heart or lung issues. Participants must understand and sign a consent form and agree to use contraception during the study. The trial aims to discover new ways to treat AML in individuals who are not candidates for more aggressive treatments. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants a chance to contribute to groundbreaking medical advancements.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but if you are on warfarin or other strong CYP3A4 drugs, you may need to switch to a different medication.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that high-dose vitamin C is safe and well-tolerated in cancer patients, including those with acute myeloid leukemia (AML). Studies indicate that patients taking vitamin C supplements during chemotherapy experienced fewer severe side effects. Specifically, one study found that these patients had a lower rate of serious complications. Additionally, using high-dose vitamin C alone has proven safe, with few complications reported.

While this study is in an early phase, focusing on safety, previous findings offer reassurance about the treatment's safety when used with standard care drugs like azacitidine and venetoclax. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatment for acute myeloid leukemia, which typically involves azacitidine and venetoclax, this new approach includes high-dose ascorbate, also known as vitamin C. Researchers are excited because high-dose ascorbate may enhance the effectiveness of standard chemotherapy by targeting cancer cells more precisely. This could potentially improve outcomes and offer a new mechanism of action against leukemia cells, adding a natural and easily accessible component to the treatment arsenal.

What evidence suggests that high-dose ascorbate might be an effective treatment for acute myeloid leukemia?

Research has shown that high-dose vitamin C might help treat cancer. Studies have found that it can stop the growth of acute myeloid leukemia (AML) cells by inducing cell death. High doses of vitamin C can also alter cancer cell behavior and enhance the immune system's ability to fight tumors. Although patients with AML who took vitamin C supplements experienced fewer complications, there was no improvement in overall survival. In this trial, some participants will receive high-dose vitamin C alongside standard treatments like azacitidine and venetoclax to further explore its potential benefits. ^{1 3 5 6 7}

Who Is on the Research Team?

Kittika Poonsombudlert, MD

Principal Investigator

University of Iowa

Are You a Good Fit for This Trial?

This trial is for adults with newly diagnosed Acute Myeloid Leukemia (AML). Participants should not have received prior treatment for AML. Specific eligibility criteria are not provided, but typically include factors like age, overall health status, and the subtype of AML.

Inclusion Criteria

Ability to understand and sign a written informed consent document

I am a woman who can have children and have a negative pregnancy test.

I have myelodysplasia and haven't had chemotherapy or hypomethylating agents.

Exclusion Criteria

Known hypersensitivity or allergy to certain medications

Other major co-morbidities deemed unsuitable by the treating physician

I have kidney disease, iron overload, diabetes, or another cancer.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive high-dose ascorbate in combination with azacitidine and venetoclax. Safety and dose-limiting toxicities are assessed.

4 weeks

Multiple visits for treatment and monitoring

Expansion

Additional patients are added to assess the composite complete remission rate.

Up to 3 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• High-dose ascorbate

Trial Overview The study tests high-dose ascorbate (vitamin C) combined with standard treatments azacitidine and venetoclax in patients with AML. It's a Phase I trial to see if this mix is safe and works better than current treatments.

How Is the Trial Designed?

Group I: B. High-dose ascorbate administered concomitantly with azacitidine and venetoclax Experimental Treatment 4 Interventions

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.

Group II: A. Standard of Care (azacitidine and venetoclax) Active Control 3 Interventions

Twelve patients will be randomized to Arm A.

Who Is Running the Clinical Trial?

Kittika Poonsombudlert

Lead Sponsor

Citations

1. sciencedirect.com sciencedirect.com/science/article/pii/S2352304225002314

High-dose vitamin C: A promising anti-tumor agent, insight ... High-dose vitamin C exhibits selective anti-tumor effects, including pro-oxidative cytotoxicity, anti-cancer epigenetic regulation, and immune modulation.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3798917/

The Effects of High Concentrations of Vitamin C on Cancer ... Vitamin C at concentrations of 0.25–1.0 mM induced a dose- and time-dependent inhibition of proliferation in acute myeloid leukemia (AML) cell lines.

3. journals.sagepub.com journals.sagepub.com/doi/10.1177/09603271251345656

High-dose vitamin C potently induces apoptosis in acute ... High-dose VC has been shown to have anti-tumor effects in various types of cancers, including acute myeloid leukemia (AML). In this study, we ...

4. jeccr.biomedcentral.com jeccr.biomedcentral.com/articles/10.1186/s13046-021-02134-y

High-dose intravenous vitamin C, a promising multi-targeting ... Mounting evidence indicates that vitamin C has the potential to be a potent anti-cancer agent when administered intravenously and in high doses (high-dose IVC).

5. hematology.org hematology.org/newsroom/press-releases/2023/patients-with-aml-who-received-vitamin-cd-supplements-had-fewer-complications

Patients with AML Who Received Vitamin C/D ... Patients with AML who received vitamin C/D supplements had fewer complications, but no overall survival benefit seen.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10685150/

Vitamin C and D supplementation in acute myeloid leukemia In this study, we showed that vitamin C and D supplementation during intensive chemotherapy for AML was safe, associated with lower rate of grade 3 to 4 adverse ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8557029/

High-dose intravenous vitamin C, a promising multi-targeting ... Overall, high dose VitC administered as a single agent has not only been shown to be safe and well-tolerated in cancer patients, but also to ...