Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

Phase 2 Completed 23 enrolled Baylor Research Institute Ultragenyx Pharmaceutical Inc · collab

RandomizedCrossoverDouble-blindTreatment

Summary

The purpose of the study is to determine if triheptanoin is an effective treatment for the symptoms of Adult Polyglucosan Body Disease.

Timeline

Start: 2009-06
Primary completion: 2015-07
Completion: 2015-07

Outcome

Missed primary endpoint

paper The overall mean difference between patients on triheptanoin versus placebo was 6 m; 95% confidence interval (CI) -11 to 22; p = 0.50. PMID 29110179 ↗

Publications

Schiffmann R, Wallace ME, Rinaldi D, Ledoux I, Luton MP, Coleman S, Akman HO, Martin K, Hogrel JY, Blankenship D, Turner J, Mochel F. A double-blind, placebo-controlled trial of triheptanoin in adult polyglucosan body disease and open-label, long-term outcome. J Inherit Metab Dis. 2018 Sep;41(5):877-883. doi: 10.1007/s10545-017-0103-x. Epub 2017 Nov 6.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Triheptanoin	Other / unclassified	1 g/kg	Oral
Subject	Triheptanoin	Other / unclassified	2 g/kg	Oral