What side effects are associated with this type of intervention?

Common treatments for Myelodysplastic Syndrome (MDS) include supportive therapies like high-dose Vitamin C, azacitidine, and decitabine. High-dose Vitamin C is generally well-tolerated but can cause gastrointestinal disturbances and kidney stones. Azacitidine and decitabine, both hypomethylating agents, are associated with side effects such as cytopenias (low blood cell counts), gastrointestinal symptoms (nausea, vomiting, diarrhea), and injection site reactions. These treatments aim to manage symptoms and improve quality of life but can also lead to increased risk of infections due to immunosuppression.

What prior FDA approvals exist?

FDA-approved treatments for Myelodysplastic Syndrome (MDS) include hypomethylating agents such as azacitidine and decitabine, which are used to improve blood counts and manage symptoms. Thrombopoietin-receptor agonists like eltrombopag and romiplostim are also approved for managing thrombocytopenia in MDS patients. These treatments focus on improving hematologic parameters and reducing transfusion needs. The study on high-dose Vitamin C aims to modulate TET2 enzyme activity, which represents a novel approach not yet covered by existing FDA-approved treatments.

What other types of research exist?

Promising novel alternatives to high-dose Vitamin C for Myelodysplastic Syndrome patients include hypomethylating agents (HMAs) such as azacitidine and decitabine. Combination therapies involving HMAs with venetoclax or glasdegib are also emerging as effective options.

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Vitamin

High-Dose Vitamin C for Myelodysplastic Syndrome

Phase 1 & 2

Waitlist Available

Led By Mohammad M Abdul Hay, MD

Research Sponsored by NYU Langone Health

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 16

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects may include digestive discomfort due to high vitamin doses, possible increased risk of kidney stones for those predisposed to them because Vitamin C can increase oxalate levels in urine which might lead to stone formation.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 16

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 16

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 16 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Number of Patients Who Experienced a Dose Limiting Toxicity (DLT)

Side effects data

From 2020 Phase 2 trial • 34 Patients • NCT01905150

21%

Nausea

14%

Fatigue

14%

Depression

14%

Diarrhea

14%

Pain

14%

Anxiety

Anemia

Ascites

Insomnia

100%

80%

60%

40%

20%

Study treatment Arm

G-FLIP Alone for 4 Weeks, Then G-FLIP+VitaminC. When DP Occurred, Then G-FLIP-DM+VitaminC

G-FLIP+VitaminC. When DP Occurred, Then G-FLIP-DM+Vitamin C.

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Vitamin C 75 gExperimental Treatment1 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 gExperimental Treatment1 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 gExperimental Treatment1 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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vitamin C

2017

Completed Phase 4

~18470

Do I qualify?Apply below to find out

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

High-dose Vitamin C is being studied for its ability to modulate TET2 enzyme activity, which is important for DNA demethylation and normal hematopoiesis. This is particularly relevant for MDS patients with TET2 mutations, as it may help restore normal blood cell production. Hypomethylating agents like azacitidine and decitabine work by inhibiting DNA methylation, thereby reactivating tumor suppressor genes and promoting normal cell differentiation. Erythropoiesis-stimulating agents (ESAs) increase red blood cell production by stimulating the bone marrow. These treatments are crucial for MDS patients as they address the underlying genetic and epigenetic abnormalities, improving blood counts and reducing the need for transfusions.