Single high-dose vitamin A given with allogeneic stem cell transplant
A Randomized Double Blinded Trial of Vitamin A Supplementation in Allogeneic Stem Cell Transplantation.
This study tests whether giving one large oral dose of vitamin A to adults having an allogeneic stem cell transplant can lower the chance of moderate-to-severe chronic graft-versus-host disease.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 190 (estimated) |
| Sex | All |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Locations | 3 sites (Tampa, Florida and 2 other locations) |
| Trial ID | NCT06450925 on ClinicalTrials.gov |
What this trial studies
This is a randomized, double-blind Phase 2 trial comparing a single large oral dose of vitamin A to a placebo in adults undergoing allogeneic hematopoietic stem cell transplantation who receive peripheral blood stem cell grafts. The trial enrolls patients with acceptable liver tests and a vitamin A level below the age-based upper limit of normal and excludes those with raised intracranial pressure, liver cirrhosis, or pregnancy. Investigators will monitor for the development of moderate-to-severe chronic graft-versus-host disease, with particular attention to gastrointestinal GVHD, and will track safety and liver function after dosing. The design builds on preliminary data linking lower vitamin A levels to increased GI GVHD risk and tests whether supplementation at transplant can reduce that risk.
Who should consider this trial
Good fit: Adults (18+) scheduled for allogeneic stem cell transplant who are receiving PBSC grafts, can tolerate enteral dosing, have vitamin A below the age-based upper limit of normal, and meet specified liver function limits are ideal candidates.
Not a fit: Patients with active raised intracranial pressure, liver cirrhosis, current pregnancy, or those not receiving PBSC grafts are excluded and would not be expected to benefit from this intervention.
Why it matters
Potential benefit: If successful, a single dose of vitamin A could reduce the incidence and severity of chronic GVHD, especially GI complications, after allogeneic transplant.
How similar studies have performed: Observational and preliminary data have linked low vitamin A to higher GI GVHD risk, but randomized trials of high-dose vitamin A for GVHD prevention are limited, so this approach remains relatively untested in randomized settings.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Be 18 years of age or older * Be scheduled for allogeneic stem cell transplant. * Have a vitamin A level \< upper limit of normal for age. * Be able to tolerate enteral vitamin dose administration. * Have a total bilirubin level \< 1.5x ULN and an AST and/or ALT\< 3xULN for age * Receiving PBSCs as stem cell graft Exclusion Criteria: * Ongoing raised intracranial pressure * Liver cirrhosis * Patients will be excluded if they are currently pregnant.
Where this trial is running
Tampa, Florida and 2 other locations
- Moffitt Cancer Center — Tampa, Florida, United States (Recruiting)
- Roswell Park Comprehensive Cancer Center — Buffalo, New York, United States (Recruiting)
- The Ohio State University — Columbus, Ohio, United States (Recruiting)
Study contacts
- Principal investigator: Pooja Khandelwal, MD — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Celeste Dourson, MS, CCRP
- Email: celeste.dourson@cchmc.org
- Phone: 513-636-7679
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.