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Use of Tocofersolan (Vedrop®) to Prevent or Treat Refractive Vitamin E Deficiency in Infants and Children

NCT03582384 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2020-05-13

No results posted yet for this study

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.

Conditions

  • Short Bowel Syndrome
  • Vitamin E Deficiency

Interventions

DRUG

Tocofersolan

After baseline labs are obtained, therapy with tocofersolan will be initiated at an initial dose of 17 mg/kg of d-alpha-tocopherol in the form of tocofersolan (0.34 ml/kg/day) and titrated based on blood levels.

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
30 Days
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-12-31
Primary Completion
2022-01-31
Completion
2023-01-31
FDA Drug
Yes

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Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03582384 on ClinicalTrials.gov