Vitamin A Supplementation in Allogeneic Stem Cell Transplantation.

Phase 2

Not yet recruiting

• Conditions: Graft Vs Host Disease; Vitamin A Deficiency; Vitamin D Deficiency
• Interventions: Drug: Vitamin A; Other: Placebo

• Registration Number: NCT06450925
• Lead Sponsor: Children's Hospital Medical Center, Cincinnati

Brief 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.

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 Timeline

• Study First Submitted: 2024-06-05
• Study First Submitted QC: 2024-06-05
• Study First Posted: 2024-06-10
• Status Verified: 2025-03
• Study Start: 2025-03
• Last Update Submitted: 2025-03-05
• Last Update Posted: 2025-03-07
• Primary Completion: 2028-03
• Study Completion: 2030-03

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 190

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Vitamin A	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)
Placebo	Placebo	Placebo pills containing microcrystalline cellulose will be dispensed in patients who are randomized to the placebo arm.

Primary Outcome Measures

Name	Time	Method
Incidence of moderate-severe chronic graft versus host disease (GVHD)	1 year after transplant	Incidence of moderate-severe chronic GVHD

Secondary Outcome Measures

Name	Time	Method
Incidence of acute gastrointestinal graft versus host disease (GI GVHD)	2 years after transplant	Incidence of acute GI GVHD
Incidence of relapse	2 years after transplant	Incidence of relapse
Overall survival.	2 years after transplant	Overall survival.

Trial Locations

Locations (3)

Moffitt Cancer Center; 🇺🇸 Tampa, Florida, United States

The Ohio State University; 🇺🇸 Columbus, Ohio, United States

Roswell Park Comprehensive Cancer Center; 🇺🇸 Buffalo, New York, United States