A Prospective Study of the Disease Characteristics of Adult Patients With Long Chain Fatty Acid Oxidation Disorders

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

61 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-01-15

Study Completion Date

2022-05-04

Brief Summary

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The purpose of the study is to collect information on disease characteristics from adult patients diagnosed with fatty acid oxidation disorders (FAOD).

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Detailed Description

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In this study there will be no drug intervention. The study will include a Baseline visit and a follow up visit scheduled at Month 4. At these visits medical history, safety assessments, concomitant medications, exercise tests and quality of life questionnaire data will be collected.

Conditions

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Fatty Acid Oxidation Disorder

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

1. A genetically confirmed diagnosis of one of the following:

1. Carnitine palmitoyltransferase 2 deficiency
2. Very long-chain Acyl-CoA dehydrogenase deficiency
3. Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency
4. Trifunctional protein deficiency
2. A stable treatment regimen for at least 30 days
3. Ambulatory and able to perform the study exercise test, using walking aids if necessary
4. Willing and able to personally sign and date an informed consent document indicating that the subject has been informed of all pertinent aspects of the study

Exclusion Criteria

1. Unstable or poorly controlled disease as determined by one or more of the following:

1. Presence of symptoms of acute rhabdomyolysis with clinically significant elevations in serum CK
2. Evidence of acute crisis from their underlying disease
2. Currently taking a PPAR agonist
3. Have motor abnormalities other than those related to the fatty acid oxidation disorder that could interfere with the study procedures, as determine by the investigator
4. Evidence of significant concomitant medical or psychiatric disease that in the opinion of the Investigator may interfere with the conduct or safety of this study
5. Pregnant or nursing females

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No