Clinical Trial from Shire Tests Anti-Oxidant Molecule in Friedreich’s Ataxia

Biopharmaceutical company Shire, is sponsoring a clinical trial for adults with Friedreich’s ataxia (FA) and is currently enrolling participants. The phase 1 clinical trial is evaluating VP 20629 (indole-3-propionic acid) against a placebo to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of treatment in FA patients 18 to 45 years old.

As explained by the registered clinical trial, “Safety and Pharmacology Study of VP 20629 in Adults With Friedreich’s Ataxia,” enrollment is estimated to reach 56, and participants will be split among seven groups. Four groups, each with eight subjects, will receive a single dose of VP20629 or placebo in a quantity of 150, 450, 900, or 1,200 mg. Three other groups, each with eight subjects, will receive multiple doses of VP 20629 or placebo in doses of 300, 600, or 900 mg. This group will be treated every eight hours for seven days until day eight, when the group will receive a single morning dose.

Vital signs and a 12-lead electrocardiogram will be used to evaluate the safety of treatment in a time frame of 10 days for the single-dose group and 17 days for the multiple-dose group. Blood and urine samples will be collected to determine the pharamacokinetics of treatment, as well as to evaluate oxidative stress and damage in the multiple-dose group by measuring levels of plasma 8-isoprostane and malondialdehyde and urinary 8-hydroxydeoxyguanosine.

Recruiting institutions include UCLA Medical Center in Los Angeles, University of South Florida in Tampa, Emory University in Atlanta, University of Iowa Children’s Hospital in Iowa City, and Children’s Hospital of Philadelphia in Philadelphia. Gwendolyn Niebler, DO, from ViroPharma (which was purchased by Shire in 2013), is directing the study, which is slated to be complete in February 2015.

According to a news release from ViroPharma in 2011, indole-3-propionic acid is a potent antioxidant. This may alleviate the free radical damage that results from iron buildup in the cytoplasm of cells of patients with FA. In the news release, Jennifer Farmer, MS, CGC, Executive Director of FA Research Alliance, stated, “We are very pleased the ViroPharma is advancing the clinical research of [indole-3-propionic acid] in FA.” Continued Vincent Milano, ViroPharma’s Chief Executive Officer, “The mission of ViroPharma is to improve the health of patients suffering from serious diseases and unmet medical needs, and FA clearly fits in that class.”