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

Completed · Phase 1 · Has a placebo group

Conditions studied: Friedreich's Ataxia

In brief

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. \[Exploratory\]

Key facts

Study ID

NCT01898884

Run by

Shire

People needed

46

Starts

2013-08-13

Expected to finish

2015-06-18

Last updated by the study team

2021-06-02

Who can join

Age: 18 and older, up to 45. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• UCLA Medical Center — Los Angeles, California, United States
• University of South Florida — Tampa, Florida, United States
• Emory University — Atlanta, Georgia, United States
• University of Iowa Children's Hospital — Iowa City, Iowa, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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