Vitamin C With Steroids for Gastrointestinal GVHD

ConditionsGraft-versus-host-disease (GVHD)Gastrointestinal

Trial statusNot yet recruiting

Trial phaseEarly Phase 1

Trial typeInterventional

Biological sexAll

Age5+

SponsorBaylor College of Medicine

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

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.

Treatment groups

35 Participants

are divided into 1 treatment group

Group A: TreatmentExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

Primary Endpoint

The primary endpoint is overall (complete or partial) response to combination of vitamin C with steroids at day 28 post-treatment.

Time frame

28 days post-treatment

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Baylor College of Medicine

Lead sponsor

The Methodist Hospital Research Institute

Collaborator

Center for Cell and Gene Therapy, Baylor College of Medicine