Date: 2015-08-17
Type of information: Initiation of patient enrollment
phase: 1-2
Announcement: initiation of patient enrollment
Company: Retrotope (USA - CA)
Product: RT001
Action
mechanism: Retrotope has discovered that a mechanism common to many degenerative diseases, namely, the free radical degradation of lipids in mitochondrial and cellular membranes, may actually cause disease. Free radicals attack and degrade the polyunsaturated fats (PUFAs) that are essential components of cellular membranes. Degradation products of these fats are associated with many diseases of neurodegeneration and aging, and create further damage cascades that are toxic to cells. Retrotope’s lead compound (RT001) is a patented, orally available, stabilized fatty-acid that shuts down this degradation and stabilizes (“fireproofs”) cellular membranes against further attack.
Disease: Friedreich\'s ataxia
Therapeutic area: Rare diseases - Genetic diseases - Neurodegenerative diseases
Country: USA
Trial
details: The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich\'s ataxia. (NCT02445794)
Latest
news: * On August 17, 2015, Retrotope announces the opening of enrollment for a 28-day, first-in-human, randomized, double-blind, controlled, ascending dose study of orally dosed RT001 to evaluate the safety, tolerability, pharmacokinetics (PK), disease
state, and exploratory endpoints in patients with Friedreich’s ataxia.
The RT001 protocol is a two center study planned for 18 patients with Friedreich’s ataxia who are ambulatory (with or without an assistive device). The primary endpoints are to evaluate the safety and tolerability of two dose levels of RT001 when administered orally to patients with Friedreich’s ataxia for 28 consecutive days; to determine the PK profile of RT001 at both dose levels following a single and multiple oral administration; and to determine the dose for future studies. The secondary endpoints are to evaluate the effects of RT001 on disease state endpoints using the Friedreich’s Ataxia Rating Scale neurological subscore and the Timed 25-Foot Walk (T25FW) performance measurement relevant to ataxia.
Two of Friedreich\'s Ataxia Research Alliance (FARA)\'s Collaborative Clinical Research Network sites, the University of South Florida and the University of California Los Angeles, will be conducting the study and FARA will utilize the Patient Registry to assist the sites in recruitment. The University of South Florida is open for enrollment, the University of California Los Angeles will be opening in the early fall for enrollment.
