Effects of triheptanoin (UX007) in patients with long‐chain fatty acid oxidation disorders: Results from an open‐label, long‐term extension study

• DOI: 10.1002/jimd.12313
• Publication Date: 2020-09-04T10:18:55Z
• Authors: Jerry Vockley; Barbara Burton; Gerard Berry; Nicola Longo; John Phillips; Amarilis Sanchez‐...; Kimberly Chapman; Pranoot Tanpaiboon; Stephanie Grunewald; Elaine Murphy; Xiaoxiao Lu; Jason Cataldo
• Keywords: Rollover (web design)

Abstract Long‐chain fatty acid oxidation disorders (LC‐FAOD) are autosomal recessive conditions that impair conversion of long‐chain acids into energy, leading to significant clinical symptoms. Triheptanoin is a highly purified, 7‐carbon chain triglyceride approved in the United States as source calories and for treatment pediatric adult patients with molecularly confirmed LC‐FAOD. CL202 an open‐label, long‐term extension study evaluating triheptanoin (Dojolvi) safety efficacy Patients rolled over from CL201 trial (rollover); were triheptanoin‐naïve (naïve); or had participated investigator‐sponsored trials/expanded access programs (IST/other). Results focus on rollover naïve groups, pretreatment data allow comparison. Primary outcomes annual rate duration major events (MCEs; rhabdomyolysis, hypoglycemia, cardiomyopathy events). Seventy‐five enrolled (24 rollover, 20 naïve, 31 IST/other). Mean was 23.0 months 15.7 34.7 IST/other. In group, mean annualized MCE decreased 1.76 events/year pre‐triheptanoin 0.96 ( P = .0319). Median reduced by 66%. median 2.33 0.71 .1072). 80%. The most common related adverse (AEs) diarrhea, abdominal pain/discomfort, vomiting, mild moderate. Three serious AEs (diverticulitis, ileus, rhabdomyolysis) possibly drug; all resolved. Two death; neither drug related. MCEs. Safety consistent previous observations.