Vitamin C for Myelodysplastic Syndrome

(EVITA Trial)

Not currently recruiting at 4 trial locations

Overseen By Stine Ulrik Mikkelsen, MD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: Rigshospitalet, Denmark

Must not be taking: Vitamin C

Disqualifiers: Chemotherapy, Active treatment, Allergic reactions, others

Compensation: Varies

Number of Visits: 3

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether vitamin C can help manage certain blood disorders by reversing specific genetic changes. The focus includes conditions such as clonal cytopenia of undetermined significance (CCUS), low-risk myelodysplastic syndromes (MDS), and early-stage chronic myelomonocytic leukemia (CMML). Participants will take either vitamin C (ascorbic acid) capsules or a placebo daily for a year to determine if vitamin C can improve their condition. This trial suits individuals with persistent low blood cell counts for over six months, diagnosed with these specific conditions, and not currently receiving active treatment for their myeloid malignancy. As an unphased trial, this study offers an opportunity to contribute to groundbreaking research that could lead to new treatment options.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must stop any vitamin C supplements or multivitamins at least 24 hours before the study begins.

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

Research has shown that vitamin C is generally safe as a treatment. In one study, patients maintained safe blood levels of vitamin C. Another study examined patients at higher risk for myelodysplastic syndrome and found manageable, non-serious side effects. Although some research has explored high doses of vitamin C, it remains safe overall.

This trial focuses on oral vitamin C. Previous studies suggest that this form is well-tolerated, with no major safety concerns. Participants in those studies took vitamin C without experiencing severe side effects, making it a viable option to consider. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike standard treatments for myelodysplastic syndrome, which often involve medications like azacitidine, decitabine, or lenalidomide, this approach uses vitamin C, a common nutrient. Researchers are excited about vitamin C because it has the potential to work by improving the function of dysfunctional blood cells, which is a different and potentially less toxic mechanism compared to traditional chemotherapy-like treatments. Additionally, vitamin C is taken orally and is usually well-tolerated, making it an attractive option for patients who may struggle with the side effects of conventional therapies.

What evidence suggests that vitamin C might be an effective treatment for myelodysplastic syndrome?

This trial will compare Vitamin C with a placebo to evaluate its effects on myelodysplastic syndrome (MDS). Studies have shown that vitamin C can influence MDS by altering the behavior of affected cells. Research suggests that vitamin C may slow the growth and survival of MDS cells by inducing cell death and influencing DNA changes. In lab studies, vitamin C stopped the growth of certain leukemia cells. Its ability to alter gene function suggests it might reverse some changes seen in these blood disorders. These findings indicate that vitamin C could be a promising treatment option worth further exploration. ^{1 2 3 5 6}

Who Is on the Research Team?

Kirsten Grønbæk, Professor

Principal Investigator

Rigshospitalet, Denmark

Are You a Good Fit for This Trial?

This trial is for adults with certain low-risk blood disorders like CCUS, MDS, or CMML-0/1. They must have specific types of low blood counts and genetic markers without other causes for their condition. People can't join if they've had recent chemo, are on active treatment (except some supportive care), are allergic to vitamin C, or can't follow the study rules.

Inclusion Criteria

I have low blood counts for more than 6 months, not caused by common conditions or other cancers.

I have CMML-0 or -1 with a specific gene mutation linked to my cancer.

My MDS is low-risk with a bone marrow blast percentage under 5.

Exclusion Criteria

Unwillingness to comply with all aspects of the protocol

I am willing to stop taking vitamin C and multivitamins 24 hours before tests.

I have not had chemotherapy in the last 6 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either vitamin C or placebo for 12 months to assess changes in epigenetic markers and disease progression

12 months

Visits every 3 months for assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• Vitamin C (ascorbic acid)

Trial Overview The study tests if oral vitamin C can reverse epigenetic changes in patients with low-risk myeloid malignancies. Participants will be randomly given either vitamin C or a placebo without knowing which one they're getting to see if it should be added to standard treatments.

How Is the Trial Designed?

Group I: Vitamin C Experimental Treatment 1 Intervention

Vitamin C (ascorbic acid) 500 mg/capsule. Ingestion of 2 capsules (1000 mg) daily for 12 months.

Group II: Placebo Placebo Group 1 Intervention

Placebo capsule. Ingestion of 2 capsules daily for 12 months. Placebo will be prepared as capsules that look and taste identical to the vitamin C supplement capsules. The content of the placebo is lactose, potato starch, gelatin, magnesium stearate, and talc.

Who Is Running the Clinical Trial?

Rigshospitalet, Denmark

Lead Sponsor

Trials

1,343

Recruited

2,453,000+

Van Andel Institute - Stand Up To Cancer Epigenetics Dream Team

Collaborator

Trials

2

Recruited

310+

University of Southern California

Collaborator

Trials

956

Recruited

1,609,000+

Imperial College London

Collaborator

Trials

1,052

Recruited

15,030,000+

University of Copenhagen

Collaborator

Trials

963

Recruited

13,350,000+

Odense University Hospital

Collaborator

Trials

808

Recruited

1,272,000+

Technical University of Denmark

Collaborator

Trials

87

Recruited

31,100+

Aalborg University Hospital

Collaborator

Trials

406

Recruited

575,000+

Van Andel Research Institute

Collaborator

Trials

28

Recruited

5,200+

Published Research Related to This Trial

In a study involving 14 patients with stage IV pancreatic cancer, intravenous ascorbic acid was administered alongside standard chemotherapy (gemcitabine and erlotinib) for eight weeks, with nine patients completing the treatment.

The addition of ascorbic acid did not increase toxicity compared to standard treatment alone, and seven out of nine patients showed stable disease, indicating potential benefits that warrant further investigation in a phase II study.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase I evaluation of intravenous ascorbic acid in combination with gemcitabine and erlotinib in patients with metastatic pancreatic cancer. Monti, DA., Mitchell, E., Bazzan, AJ., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03433781

NCT03433781 | A Phase Ib Study Evaluating the Safety ... The primary objectives phase 1 study is to establish safety and confirm a steady level of Vitamin C on ≥1 mM in > 75% of the patients is achieved. All patients ...

2. cancer.gov cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2018-00603

Ascorbic Acid in Treating Patients with Intermediate or High ... This phase Ib trial studies the side effects of ascorbic acid and to see how well it works in treating patients with intermediate or high risk myelodysplastic ...

3. 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.

4. withpower.com withpower.com/trial/phase-2-preleukemia-4-2018-7122d

High-Dose Vitamin C for Myelodysplastic Syndrome Research suggests that Vitamin C can decrease the growth and survival of myelodysplastic syndrome cells by inducing cell death and affecting DNA changes, which ...

5. brio-medical.com brio-medical.com/iv-vitamin-c-high-dose-myelodysplastic-syndrome/

High Dose IV Vitamin C For Myelodysplastic Syndrome This natural remedy has shown the ability to selectively kill cancer cells associated with MDS while sparing normal cells.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8397678/

Clinical efficacy and safety of oral and intravenous vitamin ... Oral intake of vitamin C does not appear to have any effect in patients with malignancies. Data are heterogeneous for intravenous administration.