Rhabdomyolysis and hospitalization are the most common historical adverse events reported by patients with long chain fatty acid oxidation disorder (LCFAOD), according to the results of a prospective, multicenter, noninterventional study.
These results were presented at the International Network for Fatty Acid Oxidation Research and Management (INFORM) annual meeting that was held in Freiburg, Germany on August 28 and 29, 2022.
The aim of the study, called FORWARD, was to collect information about the characteristics of the disease in adult patients.
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There were no drug interventions during the study, which consisted of a baseline visit and a follow-up visit at month 4, during which medical history, safety assessments, concomitant medications, exercise tests, and quality of life questionnaire data were collected.
Read more about the symptoms of LCFAOD
The primary outcome measure was the change in function as measured by the 12-minute walk test. The secondary outcome measure was the change in symptoms related to fatty acid oxidation disorders using a newly developed muscle symptom questionnaire specifically designed for adults with LCFAOD.
The trial recruited 58 participants from 15 centers around the world and was completed in May 2022.
The results showed that there were small improvements in the 12-minute walk distance and physical functioning scores at 4 months. These improvements are consistent with a learning effect, the researchers reported.
The majority of patients (79.3%) reported rhabdomyolysis as a historic adverse event, while 62.1% reported hospitalization.
The researchers concluded that their findings provide “important information on natural history of [fatty acid oxidation disorder] that can be used for the design of future REN001 therapeutic trials.” REN001 is a selective peroxisome proliferator-activated receptor delta (PPARδ) agonist currently in clinical development.
LCFAOD is the name given to a group of disorders characterized by impaired fat metabolism, which leads to acute crises of energy production and chronic energy deficiency.
References
Reneo Pharmaceuticals announces poster presentations at the International Network for Fatty Acid Oxidation Research and Management annual meeting. News release. Reneo Pharmaceuticals, Inc; August 29, 2022.
A prospective study of the disease characteristics of adult patients with long chain fatty acid oxidation disorders (FORWARD). ClinicalTrials.gov. July 22, 2020. Updated January 12, 2022. Accessed September 1, 2022.
