Use of Tocofersolan (Vedrop®) to Prevent or Treat Refractive Vitamin E Deficiency in Infants and Children
Status:
Withdrawn
Trial end date:
2023-01-01
Target enrollment:
Participant gender:
Summary
Patients with short bowel syndrome or other forms of intestinal failure/fat malabsorption are
unable to tolerate adequate oral or enteral feedings. They require that nutrition be given as
enteral nutrition that is delivered by feeding tube. Often these children take supplements
such as vitamins to help improve their nutritional status but, due to their condition, they
have difficulty absorbing the supplement sufficiently and most of it is lost in the stool.
The drug that will be studied, Tocofersolan (Vedrop®) is a form of vitamin E, a type of the
fat soluble vitamin needed in the human diet. It has been formulated in such a way that it
may be more easily absorbed by patients with this condition. The main purpose of the study is
to learn about the safety and tolerability of this form of vitamin E. Before receiving the
study drug, the severity of the child's vitamin E deficiency will be determined by a blood
sample, followed by giving them a daily dose of tocofersolan (Vedrop®) either orally or
through their feeding tube. After a 4 weeks of therapy, a second blood sample will be checked
and the child will continue either same dose of tocoferssolan or it will be adjusted in
response to the blood levels. If the study drug works as it is designed to do, there should
be an increase in the concentration of the vitamin E in the child's blood, suggesting that
the drug was absorbed. At each visit, a sample of blood will be obtained to assess the
child's vitamin E status and general health.
Patients will remain on tocofersolan for approximately one year or as long as the study
remains open. Based on the European pediatric experience, patients should be expected be on
tocofersolan a minimum of 3 months, ideally 6 months to see optimal clinical response.
Phase:
Phase 2
Details
Lead Sponsor:
Boston Children's Hospital Boston Children’s Hospital