Vitamin A Supplementation in Allogeneic Stem Cell Transplantation.

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.

Study Overview

• Status: Recruiting
• Conditions: Vitamin D Deficiency; Vitamin A Deficiency; Graft Vs Host Disease
• Intervention / Treatment: Drug: Vitamin A; Other: Placebo

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.

• Study Type: Interventional
• Enrollment (Estimated): 190
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Celeste Dourson, MS, CCRP; Phone Number: 513-636-7679; Email: celeste.dourson@cchmc.org

Study Locations

• United States
  • Florida
    • Tampa, Florida, United States, 33612
      • Recruiting
      • Moffitt Cancer Center
      • Principal Investigator: Nelli Bejanyan, MD
  • New York
    • Buffalo, New York, United States, 14203
      • Recruiting
      • Roswell Park Comprehensive Cancer Center
      • Principal Investigator: Theresa Hahn, PhD
  • Ohio
    • Columbus, Ohio, United States, 43210
      • Recruiting
      • The Ohio State University
      • Principal Investigator: Sumithira Vasu, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Vitamin A; 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)	Drug: Vitamin A; 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.
Placebo Comparator: Placebo; Placebo pills containing microcrystalline cellulose will be dispensed in patients who are randomized to the placebo arm.	Other: Placebo; Placebo pills containing microcrystalline cellulose will be dispensed in patients who are randomized to the placebo arm.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of moderate-severe chronic graft versus host disease (GVHD)	Incidence of moderate-severe chronic GVHD	1 year after transplant

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of relapse	Incidence of relapse	2 years after transplant
Overall survival.	Overall survival.	2 years after transplant
Incidence of acute gastrointestinal graft versus host disease (GI GVHD)	Incidence of acute GI GVHD	2 years after transplant

Collaborators and Investigators

• Sponsor: Children's Hospital Medical Center, Cincinnati
• Investigators: Principal Investigator: Pooja Khandelwal, MD, Children's Hospital Medical Center, Cincinnati

Study record dates

Study Major Dates

• Study Start (Actual): March 25, 2025
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): August 1, 2029

Study Registration Dates

• First Submitted: June 5, 2024
• First Submitted That Met QC Criteria: June 5, 2024
• First Posted (Actual): June 10, 2024

Study Record Updates

• Last Update Posted (Actual): August 12, 2025
• Last Update Submitted That Met QC Criteria: August 11, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nutrition Disorders; Immune System Diseases; Eye Diseases; Vision Disorders; Avitaminosis; Deficiency Diseases; Malnutrition; Vitamin D Deficiency; Graft vs Host Disease; Night Blindness; Vitamin A Deficiency; Physiological Effects of Drugs; Micronutrients; Vitamins; Vitamin A
• Other Study ID Numbers: 2023-0301

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No