Randomized, clinical trial of RT001: Early signals of efficacy in Friedreich's ataxia

Abstract

RT001 was found to be safe and tolerable over 28 days, and improved peak workload. Further research into the effect of RT001 in Friedreich's ataxia is warranted. © 2018 International Parkinson and Movement Disorder Society.

Corneal confocal microscopy: Neurologic disease biomarker in Friedreich ataxia

“…Healthy matched subjects were identified by advertisements. Written informed consent was obtained under Age at 1st symptom, yr N/A 11.5 ± 5.0 (5)(6)(7)(8)(9)(10)(11)(12)(13)(14)(15)(16)(17)(18)(19)(20)(21) Age at diagnosis, yr N/A 14.7 ± 6.5 Duration from 1st symptom, yr N/A 13.0 ± 5.2 (3)(4)(5)(6)(7)(8)(9)(10)(11)(12)(13)(14)(15)(16)(17)(18)(19)(20)(21) Duration from diagnosis, yr N/A 10.0 ± 5.4 (2)(3)(4)(5)(6)(7)(8)(9)(10)(11)(12)(13)(14)(15)…”

“…4,5 The recent focus on disease-modifying therapies for FRDA has highlighted the need for validated biomarkers of disease progression. 6,7 The current standards to assess FRDA disease severity are the two clinical scales Friedreich's Ataxia Rating Scale (FARS) 8,9 and Scale for the Assessment and Rating of Ataxia (SARA). 10 Based on a body of literature demonstrating the presence of a distal axonopathy and abnormal quantitative sensory thresholds in FRDA, indicative of unmyelinated nerve fiber pathology, 11 we hypothesized that quantification of View this article online at wileyonlinelibrary.com.…”

ALSUntangled No. 47: RT001

“…D-PUFA) groups relative to the PUFA groups. Positive trends in walking speed and other measures were also noted in the RT001 groups relative to the PUFA groups (22).…”

“…In the Friedreich's ataxia trial, the dose of 1.8 grams daily was well-tolerated with no reported adverse events. However, over half of the patients taking 9 g of RT001 per day had diarrhea and one patient of very low body weight required hospitalization due to diarrhea (22). RT001 is enzymatically metabolized to arachidonic acid with deuterium on the 13 th carbon in humans (22).…”

“…However, over half of the patients taking 9 g of RT001 per day had diarrhea and one patient of very low body weight required hospitalization due to diarrhea (22). RT001 is enzymatically metabolized to arachidonic acid with deuterium on the 13 th carbon in humans (22). This form of deuterated arachidonic acid has been shown in a mouse cell line to decrease the production of important cell signaling molecules known as prostaglandins and leukotrienes (23).…”