Vitamin A Supplementation in Allogeneic Stem Cell Transplantation.

Cincinnati Children's Hospital Medical Center

Status and phase: Enrolling; Phase 2

Conditions: Graft Vs Host Disease; Vitamin A Deficiency; Vitamin D Deficiency

Treatments: Drug: Vitamin A; Other: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT06450925

2023-0301

Details and patient eligibility

About

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.

Full 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.

Enrollment: 190 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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190 participants in 2 patient groups, including a placebo group

Vitamin A

Active Comparator group

Description:

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)

Treatment:

Drug: Vitamin A

Placebo

Placebo Comparator group

Description:

Placebo pills containing microcrystalline cellulose will be dispensed in patients who are randomized to the placebo arm.

Treatment:

Other: Placebo