High-Dose Vitamin C for Myelodysplastic Syndrome

No longer 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether high doses of Vitamin C can safely treat individuals with myelodysplastic syndrome (a bone marrow disorder) who have a specific genetic mutation called TET2. The goal is to observe how the body processes Vitamin C and its effects on blood cells. Participants will receive one of three different doses of Vitamin C through an IV over five days. This trial suits those with myelodysplastic syndrome who have the TET2 mutation and do not have certain health conditions like active infections or heart issues. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this potentially groundbreaking therapy.

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.

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

Research has shown that high-dose Vitamin C is usually well-tolerated, even in large amounts. One study found that people taking Vitamin C experienced mostly mild side effects. Another study examined high-dose Vitamin C administered through an IV in individuals with a specific genetic mutation and found it was safe for most participants. Out of 10 people in the study, 8 continued without major problems.

This treatment is currently in the early stages of clinical trials, where researchers are primarily assessing its safety before conducting larger studies. While early trials suggest it is generally safe, further research is needed to confirm these results in a larger population. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about high-dose Vitamin C as a treatment for Myelodysplastic Syndrome (MDS) because it offers a unique approach compared to standard treatments like chemotherapy, immunosuppressive therapy, or bone marrow transplants. Unlike these options, which often come with significant side effects, high-dose Vitamin C is believed to enhance the immune system and potentially target cancer cells without harming healthy cells. It is delivered through a continuous intravenous infusion, which could lead to more consistent therapeutic effects. This treatment's potential to offer a less toxic and innovative mechanism of action is what makes it particularly intriguing to researchers.

What evidence suggests that high-dose Vitamin C could be an effective treatment for myelodysplastic syndrome?

Research has shown that high-dose Vitamin C might help treat myelodysplastic syndrome, particularly in individuals with a TET2 mutation. Studies have found that Vitamin C can slow the growth of these cancer cells by inducing cell death and affecting their DNA. In one study, high-dose Vitamin C treatment led to positive results in eight out of ten patients. Additionally, Vitamin C has been observed to help normalize blood cell production and promote the death of abnormal cells. This trial will evaluate different dosages of Vitamin C—50 g, 75 g, and 100 g—to determine its effectiveness as a treatment option for this condition. ^{1 2 3 6 7}

Who Is on the Research Team?

Mohammad M Abdul Hay, MD

Principal Investigator

NYU Langone Health

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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.

ashpublications.org

ashpublications.org/blood/article/144/23/2456/518017/High-dose-IV-ascorbic-acid-therapy-for-patients

High-dose IV ascorbic acid therapy for patients with CCUS ...

This phase 2 trial assessed high-dose IV ascorbic acid in TET2 mutant clonal cytopenia. Eight of 10 patients were eligible for response assessment.

3.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC6798470/

Oral vitamin C supplementation to patients with myeloid ...

Both studies show that vitamin C can normalize myeloid differentiation and induce cell death [19, 20]. In vitro studies of solid cancers have ...

4.

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 ...

5.

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 ...

6.

pmc.ncbi.nlm.nih.gov

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

Clinical efficacy and safety of oral and intravenous vitamin ...

Stephenson et al. (2013) came to the conclusion that vitamin C was tolerable even in higher doses and that side effects were mostly mild. The ...

7.

sciencedirect.com

sciencedirect.com/science/article/pii/S2452336415000114

High-dose vitamin C and cancer

The general consensus among practitioners is that high-dose intravenous vitamin C (HDIVC) is greater than 10 g/infusion and low-dose intravenous vitamin C ( ...