What is the mechanism of action of this treatment?

Common treatments for low blood cell count often target the underlying mechanisms that impair blood cell production. For instance, oral Vitamin C is being studied for its potential to reverse epigenetic changes by activating TET2, an enzyme crucial for DNA demethylation, which can improve the function of hematopoietic stem cells. Erythropoiesis-stimulating agents (ESAs) like erythropoietin (Epo) work by stimulating the bone marrow to produce more red blood cells, addressing anemia directly. These treatments are vital for patients with low blood cell counts as they can improve blood cell production, reduce the need for transfusions, and alleviate symptoms, thereby enhancing the patient's quality of life.

What side effects are associated with this type of intervention?

Common treatments for low blood cell count include oral Vitamin C, erythropoiesis-stimulating agents (ESAs), and hydroxyurea. Oral Vitamin C, which is being studied for its potential to reverse epigenetic changes via TET2 activation, generally has minimal side effects but can cause gastrointestinal discomfort and kidney stones at high doses. ESAs, used to stimulate red blood cell production, can lead to hypertension, headaches, joint pain, and an increased risk of thromboembolic events. Hydroxyurea, which is used to manage various blood disorders, can cause cytopenias, mucocutaneous ulcers, diarrhea, peripheral neuropathy, and in rare cases, severe pulmonary toxicity.

What prior FDA approvals exist?

The FDA has approved several treatments for low blood cell count, particularly in conditions like myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Azacitidine, a hypomethylating agent, is approved in both oral and parenteral forms and works by modifying DNA methylation. Erythropoiesis-stimulating agents such as erythropoietin and darbepoetin alfa are also approved to manage anemia by stimulating red blood cell production. These treatments are somewhat related to the ongoing study of oral Vitamin C, which aims to reverse epigenetic changes via TET2 activation.

What other types of research exist?

Promising novel alternatives to oral vitamin C for low blood cell count patients include Lenalidomide (often combined with erythropoietin), Azacitidine (sometimes combined with lenalidomide or vorinostat), and Erythropoietin (EPO). These treatments have shown efficacy in clinical trials for conditions like myelodysplastic syndromes and anemia.

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Dietary Supplement

Vitamin C for Myelodysplastic Syndrome (EVITA Trial)

N/A

Waitlist Available

Led By Kirsten Grønbæk, Professor

Research Sponsored by Rigshospitalet, Denmark

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

A diagnosis of CMML-0 or -1 as according to WHO 2016 diagnostic criteria AND The presence of a detectable mutation in genes recurrently affected in myeloid malignancy representing a clonal marker (excluding germline mutations)

A diagnosis of MDS as according to World Health Organization (WHO) 2016 diagnostic criteria • Revised international prognostic scoring system (IPSS-R) risk score ≤ 3 AND bone marrow blast percentage < 5 defining low-risk

Must not have

Treatment with chemotherapy within the past 6 months

Lack of ability to understand the information given, or lack of willingness to sign a written informed consent document

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at baseline and at 3 months

Summary

This trial tests if taking vitamin C daily can help patients with certain low-risk blood cancers. These patients often have low vitamin C levels, which might affect their DNA. By boosting vitamin C, the study aims to see if it can improve DNA health and slow cancer progression. Vitamin C has been studied for its potential effects on cancer prevention and treatment, with some evidence suggesting it may improve quality of life and defense reactions in cancer patients.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Vitamin C is generally safe but may cause side effects like stomach upset, heartburn, nausea, and diarrhea. Allergic reactions could occur in rare cases.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have not had chemotherapy in the last 6 months.

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I understand the trial information and am willing to sign the consent form.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at baseline and at 3 months and 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at baseline and at 3 months and 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and at baseline and at 3 months and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Median Change from Baseline in Variant Allele Frequency at 12 Months

Secondary study objectives

Change in intestinal permeability and gut microbiota

Mean Change from Baseline in 5-hmC/5-mC Level at 3 Months and 12 months

Mean Change from Baseline in 5-mC at Selected Sites at 12 Months

+6 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Vitamin CExperimental Treatment1 Intervention

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

Group II: PlaceboPlacebo Group1 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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vitamin C (ascorbic acid)

2010

Completed Phase 3

~520

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for low blood cell count often target the underlying mechanisms that impair blood cell production. For instance, oral Vitamin C is being studied for its potential to reverse epigenetic changes by activating TET2, an enzyme crucial for DNA demethylation, which can improve the function of hematopoietic stem cells. Erythropoiesis-stimulating agents (ESAs) like erythropoietin (Epo) work by stimulating the bone marrow to produce more red blood cells, addressing anemia directly. These treatments are vital for patients with low blood cell counts as they can improve blood cell production, reduce the need for transfusions, and alleviate symptoms, thereby enhancing the patient's quality of life.

Erythropoiesis in multiple myeloma: defective red cell production due to inappropriate erythropoietin production.Erythropoiesis and erythropoietin in multiple myeloma.Efficacy of quadruple treatment on different types of pre-operative anaemia: secondary analysis of a randomised controlled trial.