Graft-versus-host-disease (GVHD)Early Phase 1NCT07197749
Vitamin C With Steroids for Gastrointestinal GVHD
A Early Phase 1 study testing Vitamin C (Ascorbic Acid) in people with Graft-versus-host-disease (GVHD) and Gastrointestinal, run by Baylor College of Medicine.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Graft-versus-host-disease (GVHD), Gastrointestinal
- What is being tested?
- Vitamin C (Ascorbic Acid)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- No sites listed yet
- Study dates
- Nov 2026to Oct 2029
- Phase
- Early Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients 5 years of age or older at time of enrollment.
- Patients experiencing their initial presentation of stage 2 or greater acute LGI GVHD (with or without other organ involvement) or stage 1 LGI + skin aGVHD requiring systemic therapy after allogeneic transplant for any malignant or non-malignant indication using any graft/donor source or conditioning intensity.
- KPS ≥70%
- Patients should not have received systemic immune suppressive therapy for treatment of active GVHD except for a maximum of 72 hours of steroid therapy (with or without ruxolitinib) prior to enrollment. Topical skin and GI corticosteroids (such as budesonide and oral beclomethasone diproprionate) are allowed.
- Informed Consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent.
Who cannot join
- Relapsed, progressing or persistent malignancy or evidence of minimal residual disease (MRD) requiring withdrawal of systemic immune suppression.
- Patients with acute GVHD developing after administration of a donor lymphocyte infusion (DLI) for relapse/progression of disease.
- Patients with uncontrolled infections will be excluded. Infections are considered controlled if appropriate therapy has been instituted and, at the time of enrollment, no signs of progression are present. Progression of infection is defined as hemodynamic instability attributable to sepsis, new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms
- Known or suspected hypersensitivity to vitamin C.
- De novo chronic GVHD or overlap syndrome developing before or present at the time of enrollment.
- Patients receiving other drugs for the treatment of GVHD except as noted above. GVHD prophylaxis agents (e.g., calcineurin inhibitors) may be continued at local Investigator's discretion.
Show all 15Show fewer
- Patients on renal replacement therapy.
- Patients requiring continuous supplemental oxygen > 2L/min to maintain peripheral O2 saturation [SpO2] > 90%.
- Patients with active hepatic sinusoidal obstructive syndrome (SOS) and/or clinical evidence of impaired hepatic function (ascites or encephalopathy related to liver disease.
- Patients receiving systemic corticosteroids (CS) for any indication within 7 days before enrollment, except the following:
- Corticosteroids administered as premedication for supportive care (such as before transfusion of blood products or before intravenous medications to prevent infusion reactions, fever, etc.).
- If steroid therapy has been administered for treatment of a non-GVHD related condition and tapered to < 0.6 mg/kg/day prednisone (0.5 mg/kg/day methylprednisolone) for 7 or more days prior to enrollment.
- History of G6PD deficiency, sickle cell disease or hemochromatosis.
- History of oxalate kidney stones.
- Patients unlikely to be adherent to study specific assessments at the transplant center.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Administration of Vitamin C (Ascorbic Acid) With Steroids for First Line Therapy of Gastrointestinal GVHD
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.
Read the 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
- Being tested
- Vitamin C (Ascorbic Acid)
- Keywords
- Graft-Versus-Host-Disease, GVHD, Vitamin C, Gastrointestinal
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vitamin C (Ascorbic Acid)Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | 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). Receives: Drug: Vitamin C (Ascorbic Acid) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Endpoint
The primary endpoint is overall (complete or partial) response to combination of vitamin C with steroids at day 28 post-treatment.
Measured: 28 days post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2025
- Study startNov 2026 (estimated)
- Primary completionOct 2028 (estimated)
- Study completionOct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft-versus-host-disease (GVHD)Studies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine
- Responsible party
- Principal investigator, Erin Doherty, Baylor College of Medicine
- Investigators
- LaQuisa Hill, MD, Principal investigator, Baylor College of MedicineErin Doherty, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07197749
- Sponsor's protocol ID
- H-58040 Vit C for GVHD
- Status last verified
- Sep 2026
- First submitted
- 26 Sep 2025
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No