High-Dose Vitamin C for Myelodysplastic Syndrome

Recruiting at 1 trial location

Overseen By Ariana Budhu

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: NYU Langone Health

Must not be taking: Hypomethylation agents

Disqualifiers: Concurrent malignancy, Active infections, HIV-positive, others

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 4-8 weeks

4 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests if giving high doses of Vitamin C to patients with a specific blood disorder and genetic mutation is safe and beneficial. The goal is to see if it helps improve their blood and bone marrow conditions.

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 cannot use hypomethylation agents within 4 weeks before starting the Vitamin C treatment. You can continue using Hydroxyurea, Erythrocyte stimulating agents (ESA), or granulocyte colony stimulating factors (G-CSF) if you are already on them.

What data supports the effectiveness of the treatment 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 may make it a promising treatment option. ^{1 2 3 4 5}

Is high-dose Vitamin C safe for humans?

High-dose Vitamin C has been studied in various conditions, and while it can decrease cell proliferation in certain cancer cells, excessive intake may have potentially harmful effects, especially in children. It's important to consider these risks and consult with a healthcare provider before starting high-dose Vitamin C treatment. ^{1 5 6 7 8}

How does high-dose Vitamin C treatment differ from other treatments for Myelodysplastic Syndrome?

High-dose Vitamin C is unique because it induces cell death in Myelodysplastic Syndrome cells through apoptosis (a form of programmed cell death) by increasing oxidative stress and affecting mitochondrial function, which is different from traditional chemotherapy that targets cell division. ^{1 2 5 6 7}

Research Team

Mohammad M Abdul Hay, MD

Principal Investigator

NYU Langone Health

Eligibility Criteria

This trial is for adults with Myelodysplastic Syndrome at intermediate or high risk, who have TET2 mutations. They must have adequate liver and kidney function, a certain level of blood cells, and be able to consent. Pregnant women can't join; neither can those with recent cancers (except some skin/breast/cervical), severe infections, major surgery within 2 weeks, HIV/Hepatitis B/C, poor lung function or heart issues.

Inclusion Criteria

Myeloblasts account for less than 20% of leukocytes on peripheral blood and bone marrow aspirate

Currently or previously being on hydroxyurea

Patients who have received prior allogeneic stem cell transplant will be permitted to enroll on the protocol

Exclusion Criteria

Major surgery within 2 weeks prior to first dose of study drug

Active infections including hepatitis B carrier status, hepatitis C virus (HCV) infection (patients must have a negative Hep B and Hep C viral load at screening)

Known HIV-positive status

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive high dose Vitamin C as a continuous intravenous infusion for 5 days, repeated in cycles of 4 weeks

4-8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Vitamin C

Trial Overview The study tests high-dose Vitamin C infusion therapy's safety and effects on bone marrow/blood in patients with specific genetic changes in their MDS. It's an early-phase trial where everyone gets the same treatment: Vitamin C infusions for five days every four weeks.

Participant Groups

Group I: Vitamin C 75 g Experimental Treatment 1 Intervention

Patients will receive Vitamin C as a continuous intravenous infusion (CIVI) of 75 grams (g) daily over 24 hours for 5 days for total of 375 grams over 5 days.

Group II: Vitamin C 50 g Experimental Treatment 1 Intervention

Patients will receive Vitamin C as a continuous intravenous infusion (CIVI) of 50 grams (g) daily over 24 hours for 5 days for total of 250 grams over 5 days up.

Group III: Vitamin C 100 g Experimental Treatment 1 Intervention

Patients will receive Vitamin C as a continuous intravenous infusion (CIVI) of 100 grams (g) daily over 24 hours for 5 days for total of 500 grams over 5 days.

Vitamin C is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Vitamin C for:

• Scurvy
• Dietary supplement

Approved in European Union as Vitamin C for:

• Scurvy
• Dietary supplement

Approved in Canada as Vitamin C for:

• Scurvy
• Dietary supplement

Who Is Running the Clinical Trial?

NYU Langone Health

Lead Sponsor

Trials

1,431

Recruited

838,000+

Perlmutter New York University Cancer Center

Collaborator

Trials

2

Recruited

130+

Perlmutter Cancer Center

Collaborator

Trials

1

Recruited

4+

Findings from Research

In a study of 67 children with myelomeningocele, both fracture and non-fracture groups had high ascorbic acid intake, exceeding recommended allowances from food and supplements, with no significant difference between the two groups.

The findings suggest that ascorbic acid supplementation does not prevent fractures in these patients, and caution is advised regarding excessive intake due to potential harmful effects in growing children.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ascorbic acid and fractures in children with myelomeningocele. Repasky, D., Rickard, K., Lindseth, R.[2013]

References

1. England pubmed.ncbi.nlm.nih.gov

Oxidative stress mediates apoptotic effects of ascorbate and dehydroascorbate in human Myelodysplasia cells in vitro. [2013]

2. Germany pubmed.ncbi.nlm.nih.gov

Oral vitamin C supplementation to patients with myeloid cancer on azacitidine treatment: Normalization of plasma vitamin C induces epigenetic changes. [2021]

3. England pubmed.ncbi.nlm.nih.gov

Vitamin C - A new player in regulation of the cancer epigenome. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Vitamin C in leukemia and preleukemia cell growth. [2013]

5. China pubmed.ncbi.nlm.nih.gov

[Inhibitory Effect of Ascorbic Acid on Myelodysplastic Syndrome Cells and Its Mechanism]. [2021]

6. England pubmed.ncbi.nlm.nih.gov

Depletion of L-ascorbic acid alternating with its supplementation in the treatment of patients with acute myeloid leukemia or myelodysplastic syndromes. [2016]

7. England pubmed.ncbi.nlm.nih.gov

Clinical and molecular characteristics associated with Vitamin C deficiency in myeloid malignancies; real world data from a prospective cohort. [2023]

8. United States pubmed.ncbi.nlm.nih.gov

Ascorbic acid and fractures in children with myelomeningocele. [2013]