Vitamin E Treatment for Long-Chain 3-Hydroxyacyl Coenzyme A (CoA) Dehydrogenase (LCHAD) Associated Neuropathy

This study has been terminated.

(New data does not support a role of vitamin E in LCHAD associated neuropathy.)

Sponsor:

Collaborators:

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Oregon State University

Information provided by:

Oregon Health and Science University

ClinicalTrials.gov Identifier:

NCT00840112

First received: February 9, 2009

Last updated: January 25, 2013

Last verified: July 2010

History of Changes

Purpose

Purpose:

People with a genetic defect in the ability to burn fat can also develop a problem with the nerves in their feet. The nerve problem, or neuropathy, can limit their ability to walk. Part of the treatment of their genetic defect in the ability to burn fat is to eat a very low fat diet. Vitamin E is found only in fatty foods like oils and nuts. People with a genetic defect in the ability to burn fat may have low vitamin E because of their low fat diet. The purpose of this study is to test whether vitamin E supplements can improve the nerve function in the feet of people with a genetic defect in the ability to burn fat.

Procedures:

Blood samples will be drawn at the beginning of the study, after 2 months and after 6 months of vitamin E supplements. The blood will be analyzed for plasma vitamin E concentrations. Around the time of each blood draw subjects will record all the food and beverages he or she consumes for three days. The subject will send the record to the investigator. Subjects will have a physical exam by a doctor specializing in nerves, a neurologist before and after taking vitamin E. They will have nerve function measured with a test called a nerve conduction velocity or NCV. Subjects will be given 800 international units (IU) of vitamin E per day for 6 months.

Condition	Intervention
Peripheral Neuropathy Mitochondrial Trifunctional Protein Deficiency	Dietary Supplement: Vitamin E supplement

Study Type:	Interventional
Study Design:	Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Vitamin E Treatment for LCHAD Associated Neuropathy

Resource links provided by NLM:

Genetics Home Reference related topics: Charcot-Marie-Tooth disease hereditary neuropathy with liability to pressure palsies long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency mitochondrial trifunctional protein deficiency

MedlinePlus related topics: Burns Dietary Supplements Peripheral Nerve Disorders Vitamin E

Drug Information available for: alpha-Tocopherol Tocopherol Vitamin E succinate Tocopherol acetate dl-alpha-Tocopherol

U.S. FDA Resources

Further study details as provided by Oregon Health and Science University:

Primary Outcome Measures:

Plasma Vitamin E Concentrations [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Neurological examinations: Sensory exam, muscle weakness exam and deep tendon reflex exam [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Enrollment:	1
Study Start Date:	July 2010
Estimated Study Completion Date:	January 2016
Estimated Primary Completion Date:	July 2015 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: LCHAD/TFP with peripheral neuropathy Subjects diagnosed with LCHAD or TFP and with documented peripheral neuropathy	Dietary Supplement: Vitamin E supplement 400 IU (268 mg) capsules of will be provided for the subjects. Subjects will be instructed to take one capsule with meals 2 times per day. Other Name: alpha tocopherol

Show Detailed Description

Eligibility

Ages Eligible for Study:	7 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Confirmed diagnosis of TFP, or LCHAD deficiency and progressive peripheral neuropathy
Subjects must be > 7 years of age, and be willing to take vitamin E supplements.

Exclusion Criteria:

None

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00840112

Locations

United States, Oregon
Oregon Health and Sciences University
Portland, Oregon, United States, 97239

Sponsors and Collaborators

Oregon Health and Science University

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Oregon State University

Investigators

Principal Investigator:

Melanie Gillingham, Ph.D

Oregon Health and Science University

More Information

No publications provided

Responsible Party:	Melanie Gillingham, Ph.D., Oregon Health and Sciences University
ClinicalTrials.gov Identifier:	NCT00840112 History of Changes
Other Study ID Numbers:	OHSU eirb# 4929, F32DK065400
Study First Received:	February 9, 2009
Last Updated:	January 25, 2013
Health Authority:	United States: Institutional Review Board United States: Federal Government

Keywords provided by Oregon Health and Science University:

Long-chain 3-hydroxyacyl CoA dehydrogenase (LCHAD)
Mitochondrial trifunctional protein (TFP)
LCHAD/TFP deficiency with peripheral neuropathy

Additional relevant MeSH terms:

Peripheral Nervous System Diseases
Protein Deficiency
Demyelinating Diseases
Polyneuropathies
Nerve Compression Syndromes
Neurologic Manifestations
Neurotoxicity Syndromes
Neuromuscular Diseases
Nervous System Diseases
Deficiency Diseases
Malnutrition
Nutrition Disorders
Signs and Symptoms
Poisoning

Substance-Related Disorders
Vitamin E
Alpha-Tocopherol
Tocopherols
Tocotrienols
Vitamins
Antioxidants
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protective Agents
Physiological Effects of Drugs
Micronutrients
Growth Substances

ClinicalTrials.gov processed this record on May 16, 2013

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