A Prospective Study of the Disease Characteristics of Adult Patients With Long Chain Fatty Acid Oxidation Disorders
Completed
Conditions studied: Fatty Acid Oxidation Disorder
In brief
The purpose of the study is to collect information on disease characteristics from adult patients diagnosed with fatty acid oxidation disorders (FAOD).
Key facts
- Study ID
- NCT04482049
- Run by
- Reneo Pharma Ltd
- People needed
- 61
- Starts
- 2021-01-15
- Expected to finish
- 2022-05-04
- Last updated by the study team
- 2023-11-18
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- A genetically confirmed diagnosis of one of the following:
- Carnitine palmitoyltransferase 2 deficiency
- Very long-chain Acyl-CoA dehydrogenase deficiency
- Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency
- Trifunctional protein deficiency
- A stable treatment regimen for at least 30 days
- Ambulatory and able to perform the study exercise test, using walking aids if necessary
- 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
You may not qualify if…
- Unstable or poorly controlled disease as determined by one or more of the following:
- Presence of symptoms of acute rhabdomyolysis with clinically significant elevations in serum CK
- Evidence of acute crisis from their underlying disease
- Currently taking a PPAR agonist
- 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
- 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
- Pregnant or nursing females
Where it is running
- Oregon Health & Science University (OHSU) — Portland, Oregon, United States
- University of Pittsburgh — Pittsburgh, Pennsylvania, United States
- Vanderbuilt University Medical Center — Nashville, Tennessee, United States
- Medizinische Universität Innsbruck — Innsbruck, Austria
- UZ Leuven University Hospitals Leuven — Leuven, Belgium
- Vseobecna fakultni nemocnice v Praze, Klinika pediatrie a dedicnych poruch metabolizmu — Prague, Czechia
- Rigshospitalet, Klinik for nerve- og muskelsygdomme — Copenhagen, Denmark
- Hôpital Pitié-Salpêtrière — Paris, France
- Pest Megyei Flór Ferenc Kórház — Kistarcsa, Hungary
- Universita di Brescia — Brescia, Italy
- Azienda Ospedaliera Universitaria Policlinico "G. Martino" di Messina — Messina, Italy
- Uniwersyteckie Centrum Kliniczne — Gdansk, Poland
- Hospital 12 de Octubre — Madrid, Spain
- Unidad de Diagnóstico y Tratamiento de Enfermedades Metabólicas Complejo Hospitalario Universitario de Santiago Travesía de Choupana s/n — Santiago de Compostela, Spain
- Salford Royal NHS Trust — Salford, United Kingdom
Full record on ClinicalTrials.gov
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