Triheptanoin
Sources réglementaires consultées
Indications approuvées
- Source de calories et d'acides gras chez l'adulte et l'enfant atteints d'un trouble de l'oxydation des acides gras à longue chaîne confirmé par analyse moléculaire.
Mises en garde cliniques
- Mise en garde majeure · Ne pas administrer en cas d'insuffisance pancréatique : une quantité faible ou absente d'enzymes pancréatiques peut réduire l'absorption et l'effet clinique. — https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5644d49f-08b9-e806-e063-6294a90a80f9
- Mise en garde majeure · Surveiller le fonctionnement et l'intégrité de la sonde ; ne pas utiliser de sondes ou composants en PVC et ne pas administrer le liquide seul. — https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5644d49f-08b9-e806-e063-6294a90a80f9
Interactions médicamenteuses
- SévèreInhibiteurs de la lipase pancréatique, tels que l'orlistatA08AB01
Mécanisme: Ils peuvent réduire l'exposition au métabolite heptanoate et l'effet clinique de la triheptanoïne.
Recommandation: Éviter l'administration concomitante.
https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5644d49f-08b9-e806-e063-6294a90a80f9
Effets indésirables
Communs (≥1%)
Douleur abdominale · Diarrhée · Vomissements · Nausées
Grossesse et allaitement
Catégorie FDA: Datos humanos insuficientes
Les données sont insuffisantes pour évaluer le risque pendant la grossesse ; un registre d'exposition est disponible. Le passage dans le lait est inconnu ; mettre en balance les bénéfices de l'allaitement et le besoin clinique maternel.
Bibliographie récente (PubMed)
Paroxysmal movement disorders are common in Glut1 deficiency syndrome (Glut1DS). Not all patients respond to or tolerate ketogenic diets. The objective was to evaluate the effectiveness and safety of triheptanoin in reducing the frequency of disabling movement disorders in patients with Glut1DS not receiving a ketogenic diet. UX007G-CL301 was a randomized, double-blind, placebo-controlled, phase 3 crossover study. After a 6-week run-in, eligible patients were randomized 1:1 to the first sequence (triheptanoin/placebo or placebo/triheptanoin) titration plus maintenance, followed by washout and the opposite sequence titration plus maintenance. The placebo (safflower oil) matched the appearance, taste, and smell of triheptanoin. Open-label triheptanoin was administered in the extension. The frequency of disabling paroxysmal movement disorder events per 4 weeks (recorded by diary during maintenance; primary endpoint) was assessed by Wilcoxon rank-sum test. Forty-three patients (children, n = 16; adults, n = 27) were randomized and treated. There was no difference between triheptanoin and placebo in the mean (interquartile range) number of disabling paroxysmal movement disorder events (14.3 [4.7-38.3] vs. 11.8; [3.2-28.7]; Hodges-Lehmann estimated median difference: 1.46; 95% confidence interval, -1.12 to 4.36; P = 0.2684). Treatment-emergent adverse events were mild/moderate in severity and included diarrhea, vomiting, upper abdominal pain, headache, and nausea. Two patients discontinued the study because of non-serious adverse events that were predominantly gastrointestinal. The study was closed early during the open-label extension because of lack of effectiveness. Seven patients continued to receive triheptanoin compassionately. There were no significant differences between the triheptanoin and placebo groups in the frequency of disabling movement disorder events during the double-blind maintenance period. © 2024 The Authors. Movement Disorders published by Wiley Per