Status:
COMPLETED
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
Lead Sponsor:
Shire
Conditions:
Friedreich's Ataxia
Eligibility:
All Genders
18-45 years
Phase:
PHASE1
Brief 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 p...
Eligibility Criteria
Inclusion
- Be 18 to 45 years of age (inclusive).
- Have a body mass index between 18 and 27 kg/m\^2 (inclusive).
- Have a clinical presentation consistent with FA.
- Have a confirmed diagnosis of FA with a defined expanded guanosine, adenine, adenine (GAA) triplet repeat number.
- Have an International Cooperative Ataxia Rating Scale (ICARS) mean total score of ≤75.
- If female, be postmenopausal (cessation of menses ≥1 year), surgically sterile, or have a negative serum human chorionic gonadotropin pregnancy test within 5 days prior to the first dose of study drug. Women of child bearing potential must also be on an acceptable method of birth control, as determined by the Investigator, for 3 months prior to the first dose and must agree to continue use through 2 months after the last dose of study drug.
- If male, be surgically sterile or agree to follow an acceptable method of birth control as determined by the Investigator, from the screening visit through 2 months after the last dose of study drug.
- Be able to swallow capsules whole.
- Agree to adhere to the protocol-defined schedule of assessments and procedures.
- Be informed of the nature of the study and provide written informed consent before any study-specific procedures are performed.
Exclusion
- Have taken coenzyme Q10, idebenone, other dietary or herbal supplements (with an anti-oxidative effect), or over-the-counter medications (including homeopathic medicines and vitamins) within 1 week prior to the first dose of study drug on Day 1.
- If female, be pregnant or breastfeeding.
- Have a positive test result for human immunodeficiency virus (HIV), hepatitis B surface antigen, or hepatitis C antibody.
- Have ingested any alcohol within 48 hours before admission to the clinical study unit on Day -1. NOTE: Caffeine intake should be limited to 2 caffeine-containing beverages per day during this same time period.
- Have participated in an investigational drug trial within 30 days prior to the first dose of study drug on Day 1. NOTE: Subjects who received study drug (VP 20629 or placebo) in a single-dose group in this study and completed the Post-treatment Safety Assessment are allowed to enroll in a multiple-dose group following a 21 day washout period, provided they continue to meet protocol eligibility criteria. Subjects cannot enroll in a multiple-dose group if they have an ongoing adverse event following participation in a single-dose group or had a serious adverse event during a single-dose group (regardless of causality).
- Have a known hypersensitivity to any ingredient in the study formulation.
- Have, as determined by the Investigator and/or medical monitor, any clinically relevant medical or surgical condition that could interfere with the administration of study drug, interpretation of study results, or compromise the safety or well-being of the subject.
- Have a Columbia-Suicide Severity Rating Scale (C-SSRS) score of 4 or 5.
Key Trial Info
Start Date :
August 13 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 18 2015
Estimated Enrollment :
46 Patients enrolled
Trial Details
Trial ID
NCT01898884
Start Date
August 13 2013
End Date
June 18 2015
Last Update
June 2 2021
Active Locations (5)
Enter a location and click search to find clinical trials sorted by distance.
1
UCLA Medical Center
Los Angeles, California, United States, 90095
2
University of South Florida
Tampa, Florida, United States, 33612
3
Emory University
Atlanta, Georgia, United States, 30329
4
University of Iowa Children's Hospital
Iowa City, Iowa, United States, 52242