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

NCT00840112 | Terminated

• 2 other identifiers: OHSU eirb# 4929F32DK065400
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Peripheral Neuropathy; Mitochondrial Trifunctional Protein Deficiency

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Plasma Vitamin E Concentrations

  6 months

Secondary Outcomes (1)

• Neurological examinations: Sensory exam, muscle weakness exam and deep tendon reflex exam

  6 months

Study Arms (1)

LCHAD/TFP with peripheral neuropathy

EXPERIMENTAL

Subjects diagnosed with LCHAD or TFP and with documented peripheral neuropathy

Dietary Supplement: Vitamin E supplement

Interventions

Vitamin E supplement DIETARY_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.

Also known as: alpha tocopherol

LCHAD/TFP with peripheral neuropathy

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• None

Sponsors & Collaborators

• Oregon Health and Science University: lead
• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): collaborator
• Oregon State University: collaborator

Study Sites (1)

Oregon Health and Sciences University

Portland, Oregon, 97239, United States

Location

MeSH Terms

Conditions

Peripheral Nervous System Diseases; Trifunctional Protein Deficiency With Myopathy And Neuropathy

Interventions

alpha-Tocopherol

Condition Hierarchy (Ancestors)

Neuromuscular Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Tocopherols; Vitamin E; Benzopyrans; Pyrans; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Melanie Gillingham, Ph.D

  Oregon Health and Science University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: February 9, 2009
• First Posted: February 10, 2009
• Study Start: July 1, 2010
• Primary Completion: July 1, 2015
• Study Completion: October 1, 2015
• Last Updated: October 21, 2015

Record last verified: 2010-07

Locations

US (1)