NCT01898884

Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia

A Phase 1, Randomized, Double-blind, Placebo-controlled, Multicenter, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Oral VP 20629 in Adult Subjects With Friedreich's Ataxia

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 46 (actual)
• Sponsor: Shire · Industry
• Sex: All
• Age: 18 Years – 45 Years
• Healthy volunteers: Not accepted

Summary

The objectives of the study are: * To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA). \[Primary\] * To characterize the pharmacokinetics of VP 20629 by investigation of the plasma concentration-time profile following single and multiple oral doses in subjects with FA. \[Secondary\] * To investigate the pharmacodynamic effects of VP 20629 on plasma 8-isoprostane and malondialdehyde and urinary 8-hydroxydeoxyguanosine concentrations following multiple oral doses in subjects with FA. \[Exploratory\]

Conditions

• Friedreich's Ataxia

Interventions

Type	Name	Description
DRUG	VP 20629
DRUG	Placebo

Timeline

• Start date: 2013-08-13
• Primary completion: 2015-06-18
• Completion: 2015-06-18
• First posted: 2013-07-15
• Last updated: 2021-06-02
• Results posted: 2016-10-06

Locations

5 sites across 1 country: United States