Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia (NCT01898884) | Clinical Trial Compass
CompletedPhase 1
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
United States46 participantsStarted 2013-08-13
Plain-language 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. \[Exploratory\]
Who can participate
Age range18 Years – 45 Years
SexALL
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Inclusion criteria
✓. 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.
✓. Be able to swallow capsules whole.
✓. Agree to adhere to the protocol-defined schedule of assessments and procedures.
Exclusion criteria
What they're measuring
1
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Timeframe: From Start of Study Treatment up to Day 19
2
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
Timeframe: From Start of Study Treatment up to Day 19
3
Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
Timeframe: From Start of Study Treatment up to Day 19
4
Number of Participants With Clinically Relevant Electrocardiogram (ECG) Abnormalities Recorded as Adverse Events (AEs)
Timeframe: From Start of Study Treatment up to Day 19
✕. 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.