NCT07197749

Vitamin C With Steroids for Gastrointestinal GVHD

Administration of Vitamin C (Ascorbic Acid) With Steroids for First Line Therapy of Gastrointestinal GVHD

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 35 (estimated)
• Sponsor: Baylor College of Medicine · Academic / Other
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

After a transplant from another donor, one risk is graft versus host disease (GVHD) that happens because of differences between the donated cells (graft) and the patient's body cells (host). The new cells from the donor might see the body's cells as different and attack them. GVHD can be very serious and cause death. The standard first treatment for GVHD is corticosteroids but not all patients respond and in cases where they don't, they need to go onto other treatments that may or may not be effective. In addition, when GHVD involves the gut it can damage the cells of the gastrointestinal track causing long term problems such as abdominal pain and bowel disturbance. In laboratory studies giving a vitamin C has been able to protect the gastrointestinal cells and help them recover. In this trial the investigators would like to see if vitamin C can do the same thing when given with steroids in patients with GVHD. The standard first treatment for acute GVHD is corticosteroids but not all patients respond and in cases where they don't, they need to go onto other treatments that may or may not be effective. In addition, when GHVD involves the gut it can damage the cells of the gastrointestinal track causing long term problems such as abdominal pain and bowel disturbance. In the laboratory vitamin C has been able to protect gut stem cells and help them recover and the investigators would like to learn if this happens in people too. Vitamin C is a readily available supplement. Vitamin C has NOT been approved by the FDA for the treatment of acute GVHD.

Detailed description

Patients enrolled on this trial will receive vitamin C through the vein or by mouth daily for 56 days. This will start at the same time or within 3 days of starting standard treatment for gut GVHD with steroids. Medical tests before treatment-- Before being treated, patients will receive a series of standard medical tests: * History and Physical exam * Blood tests to measure blood cells, kidney and liver function * A biopsy of the gut to look for GVHD if possible * Serum pregnancy test for female patients who are of childbearing potential * An optional stool research sample Medical tests during and after treatment: Patients will receive standard medical tests while getting the GVHD treatment and afterwards: * History and Physical exams * Monitoring for GVHD * Blood tests to measure blood cells, kidney and liver function * A follow-up biopsy of the gut to look for GVHD if possible To learn more about the way that vitamin C may work on gut stem cells an extra amount of blood will be obtained on the day vitamin C starts, 2 and 4 weeks after starting the vitamin C. The amount of blood taken will be based on the patients weight with up to a maximum of 60 mL (12 teaspoons) of blood to be obtained at any one time. This volume is considered safe but may be decreased if the patient is anemic (have a low red blood cell count). If patient has a repeat gut biopsy to check GVHD the investigators will request a sample to be used for research purposes. Patient will receive supportive care for any acute or chronic toxicities, including blood components or antibiotics, and other intervention as appropriate.

Conditions

• Graft-versus-host-disease (GVHD)
• Gastrointestinal

Interventions

Type	Name	Description
DRUG	Vitamin C (Ascorbic Acid)	All patients enrolled on this trial will receive steroids at a minimum dose of prednisone 2 mg/kg/day PO (or methylprednisolone 1.6 mg/kg/day IV) divided into 1-2 daily doses as therapy for acute GVHD. For patients that weigh over 100 kg, maximal starting dose of prednisone will be 200 mg (or methylprednisolone-equivalent). Patients will be treated with intravenous (IV) or oral (PO) vitamin C depending on the severity of symptoms, ability to tolerate PO intake, and location of treatment (inpatient versus outpatient setting): IV dosing: 50 mg/kg/day divided TID (max 4 grams total/day) PO dosing: 500 mg BID or 1000 mg daily (depending on the prescribed formulation) Vitamin C will be administered through day 56 from start of vitamin C treatment. Patients initially receiving IV administration may be transitioned to PO formulation once tolerating PO intake; per treating physician discretion.

Timeline

• Start date: 2026-06-01
• Primary completion: 2028-10-01
• Completion: 2029-10-01
• First posted: 2025-09-29
• Last updated: 2026-03-04