Graft Vs Host DiseasePhase 2NCT06450925
Vitamin A Supplementation in Allogeneic Stem Cell Transplantation.
A Phase 2 study testing Vitamin A in people with Graft Vs Host Disease and Vitamin A Deficiency, run by Children's Hospital Medical Center, Cincinnati at 3 sites in United States.
Trial summary
The investigators hypothesize that single oral high dose supplementation with vitamin A will reduce the incidence of moderate-severe chronic graft-versus-host disease (GVHD) compared with placebo. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Graft Vs Host Disease, Vitamin A Deficiency, Vitamin D Deficiency
- What is being tested?
- Vitamin A
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 190 participants
- Where
- 3 sitesUnited States
- Study dates
- 25 Mar 2025to Aug 2029
- Phase
- Phase 2
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Ongoing raised intracranial pressure (see appendix D for definition of raised intracranial pressure)
- Liver cirrhosis (see appendix C for definition of liver cirrhosis)
- Patients will be excluded if they are currently pregnant.
- Patients will be excluded if they have a fish (not shellfish) allergy.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14203
Theresa Hahn, PhD
- Recruiting
The Ohio State University
Columbus, Ohio, 43210
Sumithira Vasu, MD
- Recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
Nelli Bejanyan, MD
About this study
Official title: A Randomized Double Blinded Trial of Vitamin A Supplementation in Allogeneic Stem Cell Transplantation.
The investigators hypothesize that single oral high dose supplementation with vitamin A will reduce the incidence of moderate-severe chronic graft-versus-host disease (GVHD) compared with placebo.
Read the detailed description
Hematopoietic stem cell transplantation (HSCT) is an effective treatment strategy for many malignancies, marrow failure syndromes, and immune deficiencies in children, adolescents, and adults. Vitamin A and its derivatives regulate growth and differentiation of intestinal cells, and vitamin A deficiency is associated with increased susceptibility to infection in both human and animal models. The investigators' preliminary data suggest lower vitamin A levels were associated with an increased incidence of gastrointestinal graft versus host disease (GI GVHD) in patients undergoing HSCT.
This study is a randomized double blinded comparison of vitamin A supplementation comparing a single large dose of vitamin A with a placebo.
- Being tested
- Vitamin A, Placebo
- Medical subject headings
- Graft vs Host Disease, Vitamin A Deficiency, Vitamin D Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vitamin ADrug
Enrolled subjects will receive one observed oral vitamin A dose, prior to their HSCT, in the outpatient clinic or inpatient Bone Marrow Transplant (BMT) floor.
PlaceboOther
Placebo pills containing microcrystalline cellulose will be dispensed in patients who are randomized to the placebo arm.
| Study group | Role | What they receive |
|---|---|---|
| Vitamin A | Active comparator | Route of administration: Oral. Frequency: Once. Timing: Pre-transplant Dose of Vit A: 1.2 mg/kg, max dose 75 mg Formulation of Vit A: 2.5 mg liquid filled oral capsules.
Vitamin A level assessment: Vitamin A levels will be measured pre-transplant and again at day +30 (± 10 days) Receives: Drug: Vitamin A |
| Placebo | Placebo comparator | Placebo pills containing microcrystalline cellulose will be dispensed in patients who are randomized to the placebo arm. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of moderate-severe chronic graft versus host disease (GVHD)
Incidence of moderate-severe chronic GVHD
Measured: 1 year after transplant
Secondary outcomes (3)
Incidence of acute gastrointestinal graft versus host disease (GI GVHD)
Incidence of acute GI GVHD
Measured: 2 years after transplant
Incidence of relapse
Incidence of relapse
Measured: 2 years after transplant
Overall survival.
Overall survival.
Measured: 2 years after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2024
- Study start25 Mar 2025
- Primary completionAug 2027 (estimated)
- Study completionAug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft Vs Host DiseaseStudies of the same condition, open and under way.
- 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 190 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Pooja Khandelwal, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06450925
- Sponsor's protocol ID
- 2023-0301
- Status last verified
- Aug 2026
- First submitted
- 5 Jun 2024
- Last updated
- 24 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No