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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03582384
Recruitment Status : Withdrawn (Study never started)
First Posted : July 11, 2018
Last Update Posted : May 13, 2020
Sponsor:
Information provided by (Responsible Party):
Daniel Elman, Boston Children’s Hospital

Brief 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.


Condition or disease Intervention/treatment Phase
Short Bowel Syndrome Vitamin E Deficiency Drug: Tocofersolan Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Use of Tocofersolan (Vedrop®) to Prevent or Treat Refractive Vitamin E Deficiency in Infants and Children
Estimated Study Start Date : December 2020
Estimated Primary Completion Date : January 2022
Estimated Study Completion Date : January 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Vitamin E
Drug Information available for: Vitamin E

Arm Intervention/treatment
Experimental: Treatment 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.




Primary Outcome Measures :
  1. Vitamin E Level [ Time Frame: 1 Year ]
    Primary outcomes measured will include whether the vitamin E level increased to > 5 mg/L



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

1. Patients with intestinal failure or fat malabsorption who have refractory vitamin E deficiency (defined as vitamin E level < 5 mg/L) despite receiving vitamin E supplementation with currently available enteral vitamin E products for at least 6 months or requiring the provision of vitamin parenterally as part of a multivitamin infusion.

Exclusion Criteria:

  1. Pregnancy
  2. Use of warfarin or any related vitamin K antagonist.
  3. Enrollment in any other clinical trial involving an investigational agent (unless approved by the principal investigators of the other trial)
  4. The parent or guardian or child unwilling to provide consent or assent
Publications of Results:
Other Publications:
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Responsible Party: Daniel Elman, Clinical Research Specialist, Boston Children’s Hospital
ClinicalTrials.gov Identifier: NCT03582384    
Other Study ID Numbers: IRB-P00026926
First Posted: July 11, 2018    Key Record Dates
Last Update Posted: May 13, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Short Bowel Syndrome
Vitamin E Deficiency
Malabsorption Syndromes
Intestinal Diseases
Gastrointestinal Diseases
Digestive System Diseases
Postoperative Complications
Pathologic Processes
Avitaminosis
Deficiency Diseases
Malnutrition
Nutrition Disorders