Study of the Absorption of Vitamin E Water-soluble Form (Pegylated) in the Familial Hypocholesterolemia With Chylomicron Retention (VEDROP)

To evaluate the kinetics of intestinal absorption of vitamin E water-soluble form from the classical lipid-soluble form in a population of patients with intestinal malabsorption (hypocholesterolemias family by retention of chylomicrons).

Study Overview

• Status: Completed
• Conditions: Hypobetalipoproteinemias
• Intervention / Treatment: Drug: Tocofersolan; Drug: tocopherol alpha; Drug: Tocofersolan and tocopherol alpha

• Study Type: Interventional
• Enrollment (Actual): 14
• Phase: Phase 3

Contacts and Locations

Study Locations

• France
  • Bron, France, 69677
    • Hopital Femme Mere Enfant-Hospices Civils de Lyon

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Patient suffering from familial hypocholesterolemia by retention of chylomicrons
• Systematically followed in the department of Gastroenterology and Paediatric Nutrition in Women Mother Child's Hospital or in the endocrinology department of GHE (Louis Pradel Hospital);
• During treatment with oral vitamin E;
• over the age of 3 years and weighing over 16 kg at the time of inclusion
• For which there is a signed informed consent from the patient or parents / guardians in the case of a minor patient, and benefit from social security coverage.

Exclusion Criteria:

• Patient with encephalopathy
• Hypersensitivity to the active substance or the excipients of Vedrop
• A suspected allergy to local anaesthetics (including xylocaine)
• Patients who may not be compliant to treatment (psychiatric);
• In case of refusal to participate in the study from the patient and / or parents or legal guardian;
• Patients unable to consent (if patients with encephalopathy)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tocofersolan: Vitamin E water-soluble; 2 months off the conventional treatment before the study. Administration of a daily dose of vitamin E for 4 months	Drug: Tocofersolan; Administration of a daily dose of vitamin E for 4 months:50 IU/kg
Active Comparator: Tocopherol alpha: Vitamin E conventional fat-soluble form; 2 months off the conventional treatment before the study. Administration of a daily dose of vitamin E for 4 months	Drug: tocopherol alpha; Administration of a daily dose of vitamin E for 4 months:50 IU/kg
Active Comparator: volunteers	Drug: Tocofersolan and tocopherol alpha; Administration of a single dose of vitamin E (Vitamin E water-soluble then Vitamin E conventional fat-soluble form)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Measurement the area under the curve of absorption of vitamin E according to its dosage form (fat-soluble versus water-soluble) in the beginning of each treatment period. versus water-soluble) in the beginning of each treatment period.	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Measurement of serum (membranes erythrocytic) of vitamin E at the beginning and after each treatment.	Measurement of serum (membranes erythrocytic) of vitamin E at the beginning and after each treatment. Search for a correlation between the rates in adipose tissue and those in the membrane of erythrocytes.	12 months
Measurement of tissue (subcutaneous adipose tissue) of vitamin E at the beginning and after each treatment.	Measurement of tissue (subcutaneous adipose tissue) of vitamin E at the beginning and after each treatment. Search for a correlation between the rates in adipose tissue and those in the membrane of erythrocytes.	12 months

Collaborators and Investigators

• Sponsor: Hospices Civils de Lyon
• Investigators: Principal Investigator: Noel PERETTI, MD, PhD, Hôpital Femme Mère Enfant, Hospices civils de Lyon

Study record dates

Study Major Dates

• Study Start: October 1, 2011
• Primary Completion (Actual): August 1, 2015
• Study Completion (Actual): August 1, 2015

Study Registration Dates

• First Submitted: October 18, 2011
• First Submitted That Met QC Criteria: October 20, 2011
• First Posted (Estimate): October 24, 2011

Study Record Updates

• Last Update Posted (Estimate): November 13, 2015
• Last Update Submitted That Met QC Criteria: November 12, 2015
• Last Verified: November 1, 2015

More Information

Terms related to this study

• Keywords: Vitamin E pegylated; chylomicron retention; Familial Hypocholesterolemia; Hypobetalipoproteinemias
• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Dyslipidemias; Lipid Metabolism, Inborn Errors; Hypolipoproteinemias; Hypobetalipoproteinemias; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Protective Agents; Micronutrients; Vitamins; Antioxidants; Vitamin E; Tocopherols; alpha-Tocopherol; Tocotrienols
• Other Study ID Numbers: 2010.634