Friedreich's AtaxiaPhase 1NCT01898884

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

A Phase 1 study testing VP 20629 in people with Friedreich's Ataxia, run by Shire at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial 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… Read more about this study

Who is it for?: Ages 18 to 45 years
Conditions the study is for: Friedreich's Ataxia
What is being tested?: VP 20629
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 46 participants
Where: 5 sitesUnited States
Study dates: 13 Aug 2013to 18 Jun 2015
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 45 years
All sexes
No healthy volunteers
Adult

Who may be able to join

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.

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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.

Who cannot join

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.

Trial locations and contacts

5 sites in 1 country.

City, postcode or hospital

5 sites in 1 country

United States5 sites

Emory University
Atlanta, Georgia, 30329
University of Iowa Children's Hospital
Iowa City, Iowa, 52242
UCLA Medical Center
Los Angeles, California, 90095
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of South Florida
Tampa, Florida, 33612

Other trials in United States

About this study

Official title: 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

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
Being tested: VP 20629, Placebo
Medical subject headings: Friedreich Ataxia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

VP 20629Drug
Also called Indole-3-propionic acid
PlaceboDrug

Study groups in NCT01898884
Study group	Role	What they receive
Single dose of VP 20629 or placebo	Experimental	Four groups of 8 subjects each will receive a single dose of VP 20629 (150 mg, 450 mg, 900 mg, or 1200 mg) or placebo. Receives: Drug: VP 20629; Drug: Placebo
Multiple doses of VP 20629 or placebo	Experimental	Three groups of 8 subjects each will receive multiple doses of VP 20629 (300 mg, 600 mg, or 900 mg total daily dose) or placebo. VP 20629 or placebo will be administered every 8 hours for 7 days with a single morning dose on Day 8. Receives: Drug: VP 20629; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs (TEAEs), defined as all AEs that start during study drug treatment (and up to 7 days after the last dose of the study drug) and were not seen at baseline, or were seen at baseline but increased in frequency and/or severity during study drug treatment (and up to 7 days after the last dose of study drug).
Measured: From Start of Study Treatment up to Day 19
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
An abnormal laboratory finding which required an action or intervention by the investigator, or a finding judged by the investigator to represent a change beyond the range of normal physiologic fluctuation were reported as an adverse event. Treatment-emergent were events between first dose of study drug and 7 days after the last dose that were absent before treatment or that worsened relative to pretreatment state. Number of participants with Grade 3 or higher treatment-emergent adverse events for laboratory abnormalities were reported as clinically relevant laboratory changes.
Measured: From Start of Study Treatment up to Day 19
Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
Vital sign assessments included systolic blood pressure, diastolic blood pressure, heart rate, and temperature. Vital signs abnormalities reported as TEAEs were reported.
Measured: From Start of Study Treatment up to Day 19
Number of Participants With Clinically Relevant Electrocardiogram (ECG) Abnormalities Recorded as Adverse Events (AEs)
ECG included PR interval, QRS interval, QTcB interval, QTcF interval were considered as clinically significant ECG abnormalities.
Measured: From Start of Study Treatment up to Day 19

Secondary outcomes (27)

Maximum Observed Serum Concentration (Cmax) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
The Cmax is the maximum observed plasma concentration of single dose of VP 20629 and VP 20631.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Time of Maximum Observed Plasma Concentration (Tmax) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
The Tmax is the time to reach maximum observed plasma concentration of single dose of VP 20629 and VP 20631.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Area Under the Plasma Concentration Versus Time Curve (AUC) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
The AUC is the area under the plasma concentration-time curve observed.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Area Under the Plasma Concentration Versus Time Curve (AUC[0-8]) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
The AUC(0-8) is the area under the plasma concentration-time curve from time zero to 8 hours postdose.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, and 8 hours Postdose on Day 1
Area Under the Plasma Concentration Versus Time Curve to the Last Measurable Plasma Concentration (AUCt) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
The AUCt is the measure of the plasma drug concentration from time zero to time t.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Terminal Plasma Half-Life (t1/2) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
The elimination half-life (t1/2) is the time measured for the plasma concentration to decrease by 1 half to its original concentration. It is associated with the terminal slope of the semi logarithmic drug concentration-time curve, and is calculated as 0.693/lambda(z).
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Volume of Distribution (Vz/F) of VP 20629 for Single Dose Groups
The Vz is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Total Body Drug Clearance (CL/F) of VP 20629 for Single Dose Groups
Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. Clearance obtained after oral dose (apparent oral clearance) is influenced by the fraction of the dose absorbed. Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Elimination Rate Constant (Lambda[z]) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
Lambda(z) is first-order elimination rate constant associated with the terminal portion of the curve, determined as the negative slope of the terminal log-linear phase of the drug concentration-time curve.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Cumulative Amount Excreted Into the Urine (Ae) for Unchanged VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
The Ae is the amount of drug excreted in urine. It is calculated by multiplying the urinary volume with the urinary drug concentration.
Measured: 0-4, 4-8, 8-16, 16-24, 24-36 and 36-48 hours postdose on Day 1
Percentage of Drug Excreted in Urine (Ae%) of VP 20629 for Single Dose Groups
The Ae% is the percentage of drug dose excreted into the urine calculated as (Ae divided by dose)∗100.
Measured: -4, 4-8, 8-16, 16-24, 24-36 and 36-48 hours postdose on Day 1
Renal Clearance (CLR) of VP 20629 for Single Dose Groups
The CLR is the renal clearance of the drug, calculated as Ae/AUC(0-infinity) on Day 1 or Ae(0-24)/AUC(0-24) on Day 1.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Maximum Observed Serum Concentration (Cmax) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
The Cmax is the maximum observed plasma concentration of Multiple Dose of VP 20629 and VP 20631.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 24 and 72 hours Postdose on Day 1
Maximum Observed Serum Concentration at Steady State (Cmax,ss) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
The Cmax,ss is the maximum observed plasma concentration at steady state.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Time of Maximum Observed Plasma Concentration (Tmax) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
The Tmax is the time to reach maximum observed plasma concentration of multiple dose of VP 20629 and VP 20631.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 24 and 72 hours Postdose on Day 1
Time of Maximum Observed Plasma Concentration at Steady State (Tmax,ss) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
The Tmax,ss is the time to reach maximum observed plasma concentration at steady state of multiple dose of VP 20629 and VP 20631.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Area Under the Plasma Concentration Versus Time Curve (AUC) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
The AUC is the area under the plasma concentration-time curve observed.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Area Under the Plasma Concentration Versus Time Curve at Steady State (AUCss) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
The AUCss is the area under the plasma concentration time curve observed during a dosing at steady state.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, and 8 hours Postdose on Day 8
Area Under the Plasma Concentration Versus Time Curve (AUC[0-8]) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
The AUC(0-8) is the area under the plasma concentration-time curve from time zero to 8 hours postdose.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6 and 8 hours Postdose on Day 1
Area Under the Plasma Concentration Versus Time Curve (AUCt) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
The AUCtau is the measure of the plasma drug concentration from time zero to time t.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Terminal Plasma Half-Life (t1/2) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
The elimination half-life (t1/2) is the time measured for the plasma concentration to decrease by 1 half to its original concentration. It is associated with the terminal slope of the semi logarithmic drug concentration-time curve, and is calculated as 0.693/lambda(z).
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Volume of Distribution (Vz/F) of VP 20629 for Multiple Dose Groups
The Vz is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Total Body Drug Clearance at Steady State (CLss/F) of VP 20629 for Multiple Dose Groups
Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. Clearance obtained after oral dose (apparent oral clearance) is influenced by the fraction of the dose absorbed. Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Elimination Rate Constant (Lambda[z]) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Lambda(z) is first-order elimination rate constant associated with the terminal portion of the curve, determined as the negative slope of the terminal log-linear phase of the drug concentration-time curve.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Cumulative Amount Excreted Into the Urine at Steady State (Ae,ss) of Unchanged VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
The Ae,ss is the amount of drug excreted in urine. It is calculated by multiplying the urinary volume with the urinary drug concentration.
Measured: 0-4, 4-8, 8-16, 16-24, 24-36, and 36-48 hours postdose on Day 8
Percentage of Drug Excreted in Urine at Steady-State (Ae%,ss) of VP 20629 for Multiple Dose Groups
The Ae%,ss is the percentage of drug dose excreted into the urine calculated as (Ae divided by dose)∗100.
Measured: 0-4, 4-8, 8-16, 16-24, 24-36, and 36-48 hours postdose on Day 8
Renal Clearance at Steady State (CLR,ss) of VP 20629 for Multiple Dose Groups
The CLR,ss is the renal clearance of the drug, calculated as Ae/AUC(0-infinity) on Day 8.
Measured: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

13 Aug 201318 Jun 2015

First posted15 Jul 2013 (estimated)
Study start13 Aug 2013
Primary completion18 Jun 2015
Study completion18 Jun 2015

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 46 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01898884
Sponsor's protocol ID: 20629-100
Status last verified: May 2021
First submitted: 3 Jul 2013
Last updated: 2 Jun 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
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Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
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Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
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RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders

All Friedreich's Ataxia trials

Study at a glance

From the registry record, last updated by the sponsor on 2 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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
ClinicalTrials.gov ID: NCT01898884
Acronym / sponsor ID: 20629-100
Conditions: Friedreich's Ataxia
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 46 participants (actual)
Lead sponsor: ShireIndustry (pharma, biotech, devices)
Study start: 13 Aug 2013 (actual)
Primary completion: 18 Jun 2015 (actual)
Study completion: 18 Jun 2015 (actual)
First posted: 15 Jul 2013 (estimated)
Results: Posted 6 Oct 2016

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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]

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
Single dose of VP 20629 or placebo	Experimental	Drug: VP 20629, Drug: PlaceboFour groups of 8 subjects each will receive a single dose of VP 20629 (150 mg, 450 mg, 900 mg, or 1200 mg) or placebo.
Multiple doses of VP 20629 or placebo	Experimental	Drug: VP 20629, Drug: PlaceboThree groups of 8 subjects each will receive multiple doses of VP 20629 (300 mg, 600 mg, or 900 mg total daily dose) or placebo. VP 20629 or placebo will be administered every 8 hours for 7 days with a single morning dose on Day 8.

Who may qualify

Ages: 18 to 45 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (10 listed)

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.

Key exclusion criteria (8 listed)

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).

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
VP 20629Also: Indole-3-propionic acid	Drug	Multiple doses of VP 20629 or placebo, Single dose of VP 20629 or placebo
Placebo	Drug	Multiple doses of VP 20629 or placebo, Single dose of VP 20629 or placebo

Primary outcome

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Time frame: From Start of Study Treatment up to Day 19
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs (TEAEs), defined as all AEs that start during study drug treatment (and up to 7 days after the last dose of the study drug) and were not seen at baseline, or were seen at baseline but increased in frequency and/or severity during study drug treatment (and up to 7 days after the last dose of study drug).
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
Time frame: From Start of Study Treatment up to Day 19
An abnormal laboratory finding which required an action or intervention by the investigator, or a finding judged by the investigator to represent a change beyond the range of normal physiologic fluctuation were reported as an adverse event. Treatment-emergent were events between first dose of study drug and 7 days after the last dose that were absent before treatment or that worsened relative to pretreatment state. Number of participants with Grade 3 or higher treatment-emergent adverse events for laboratory abnormalities were reported as clinically relevant laboratory changes.
Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
Time frame: From Start of Study Treatment up to Day 19
Vital sign assessments included systolic blood pressure, diastolic blood pressure, heart rate, and temperature. Vital signs abnormalities reported as TEAEs were reported.
Number of Participants With Clinically Relevant Electrocardiogram (ECG) Abnormalities Recorded as Adverse Events (AEs)
Time frame: From Start of Study Treatment up to Day 19
ECG included PR interval, QRS interval, QTcB interval, QTcF interval were considered as clinically significant ECG abnormalities.

27 secondary outcomes

Maximum Observed Serum Concentration (Cmax) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Time of Maximum Observed Plasma Concentration (Tmax) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Area Under the Plasma Concentration Versus Time Curve (AUC) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Area Under the Plasma Concentration Versus Time Curve (AUC[0-8]) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, and 8 hours Postdose on Day 1
Area Under the Plasma Concentration Versus Time Curve to the Last Measurable Plasma Concentration (AUCt) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Terminal Plasma Half-Life (t1/2) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Volume of Distribution (Vz/F) of VP 20629 for Single Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Total Body Drug Clearance (CL/F) of VP 20629 for Single Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Elimination Rate Constant (Lambda[z]) of VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Cumulative Amount Excreted Into the Urine (Ae) for Unchanged VP 20629 and Its Metabolite (VP 20631) for Single Dose Groups
Time frame: 0-4, 4-8, 8-16, 16-24, 24-36 and 36-48 hours postdose on Day 1
Percentage of Drug Excreted in Urine (Ae%) of VP 20629 for Single Dose Groups
Time frame: -4, 4-8, 8-16, 16-24, 24-36 and 36-48 hours postdose on Day 1
Renal Clearance (CLR) of VP 20629 for Single Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8,10, 12, 24 and 48 hours Postdose on Day 1
Maximum Observed Serum Concentration (Cmax) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 24 and 72 hours Postdose on Day 1
Maximum Observed Serum Concentration at Steady State (Cmax,ss) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Time of Maximum Observed Plasma Concentration (Tmax) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 24 and 72 hours Postdose on Day 1
Time of Maximum Observed Plasma Concentration at Steady State (Tmax,ss) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Area Under the Plasma Concentration Versus Time Curve (AUC) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Area Under the Plasma Concentration Versus Time Curve at Steady State (AUCss) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, and 8 hours Postdose on Day 8
Area Under the Plasma Concentration Versus Time Curve (AUC[0-8]) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6 and 8 hours Postdose on Day 1
Area Under the Plasma Concentration Versus Time Curve (AUCt) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Terminal Plasma Half-Life (t1/2) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Volume of Distribution (Vz/F) of VP 20629 for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Total Body Drug Clearance at Steady State (CLss/F) of VP 20629 for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Elimination Rate Constant (Lambda[z]) of VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8
Cumulative Amount Excreted Into the Urine at Steady State (Ae,ss) of Unchanged VP 20629 and Its Metabolite (VP 20631) for Multiple Dose Groups
Time frame: 0-4, 4-8, 8-16, 16-24, 24-36, and 36-48 hours postdose on Day 8
Percentage of Drug Excreted in Urine at Steady-State (Ae%,ss) of VP 20629 for Multiple Dose Groups
Time frame: 0-4, 4-8, 8-16, 16-24, 24-36, and 36-48 hours postdose on Day 8
Renal Clearance at Steady State (CLR,ss) of VP 20629 for Multiple Dose Groups
Time frame: Predose, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 5, 6, 8, 10, 12, 24 and 48 hours Postdose on Day 8

Results and safety

Results posted to the registry on 6 Oct 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Single Dose Placebo	8	8
Single Dose VP 20629 150 mg	6	6
Single Dose VP 20629 450 mg	6	6
Single Dose VP 20629 900 mg	6	6
Single Dose VP 20629 1200 mg	6	6
Multiple Dose Placebo	0	0
Multiple Dose VP 20629 300 mg	0	0
Multiple Dose VP 20629 600 mg	0	0
Multiple Dose VP 20629 900 mg	0	0

Primary outcome: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

Time frame: From Start of Study Treatment up to Day 19. Number. Unit: participants

Measure	Single Dose Placebo n = 8	Single Dose VP 20629 150 mg n = 6	Single Dose VP 20629 450 mg n = 6	Single Dose VP 20629 900 mg n = 6	Single Dose VP 20629 1200 mg n = 6	Multiple Dose Placebo n = 6	Multiple Dose VP 20629 300 mg n = 6	Multiple Dose VP 20629 600 mg n = 6	Multiple Dose VP 20629 900 mg n = 5
TEAE	3	0	4	5	6	4	3	6	3
TESAE	0	0	0	0	0	0	1	0	0

Population analysed: The ITT-safety (ITT-S) set consisted of all participants who were randomly assigned to an investigational product treatment group and who received at least 1 partial or complete dose of investigational product.

Primary outcome: Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)

Time frame: From Start of Study Treatment up to Day 19. Number. Unit: participants

Measure	Single Dose Placebo n = 8	Single Dose VP 20629 150 mg n = 6	Single Dose VP 20629 450 mg n = 6	Single Dose VP 20629 900 mg n = 6	Single Dose VP 20629 1200 mg n = 6	Multiple Dose Placebo n = 6	Multiple Dose VP 20629 300 mg n = 6	Multiple Dose VP 20629 600 mg n = 6	Multiple Dose VP 20629 900 mg n = 5
Blood Glucose Increased	0	0	0	0	1	0	0	0	0
Neutrophil Count Decreased	0	0	0	0	0	0	0	1	0

Population analysed: The ITT-S set consisted of all participants who were randomly assigned to an investigational product treatment group and who received at least 1 partial or complete dose of investigational product.

Primary outcome: Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)

Time frame: From Start of Study Treatment up to Day 19. Number. Unit: participants

Measure	Single Dose Placebo n = 8	Single Dose VP 20629 150 mg n = 6	Single Dose VP 20629 450 mg n = 6	Single Dose VP 20629 900 mg n = 6	Single Dose VP 20629 1200 mg n = 6	Multiple Dose Placebo n = 6	Multiple Dose VP 20629 300 mg n = 6	Multiple Dose VP 20629 600 mg n = 6	Multiple Dose VP 20629 900 mg n = 5
Value	0	0	0	0	0	0	0	0	0

Primary outcome: Number of Participants With Clinically Relevant Electrocardiogram (ECG) Abnormalities Recorded as Adverse Events (AEs)

Time frame: From Start of Study Treatment up to Day 19. Number. Unit: participants

Measure	Single Dose Placebo n = 8	Single Dose VP 20629 150 mg n = 6	Single Dose VP 20629 450 mg n = 6	Single Dose VP 20629 900 mg n = 6	Single Dose VP 20629 1200 mg n = 6	Multiple Dose Placebo n = 6	Multiple Dose VP 20629 300 mg n = 6	Multiple Dose VP 20629 600 mg n = 6	Multiple Dose VP 20629 900 mg n = 5
Value	0	1	0	0	0	0	1	0	0

27 secondary outcomes are reported in the full results.

Adverse events

Time frame: From the start of the study drug administration upto 30 days after the last dose of study drug administration.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Single Dose Placebo	0 of 8 (0%)	3 of 8 (37.5%)	Not reported
Single Dose VP 20629 150mg	0 of 6 (0%)	0 of 6 (0%)	Not reported
Single Dose VP 20629 450mg	0 of 6 (0%)	4 of 6 (66.7%)	Not reported
Single Dose VP 20629 900mg	0 of 6 (0%)	5 of 6 (83.3%)	Not reported
Single Dose VP 20629 1200mg	0 of 6 (0%)	6 of 6 (100%)	Not reported
Multiple Dose Placebo	0 of 6 (0%)	4 of 6 (66.7%)	Not reported
Multiple Dose VP 20629 300mg	1 of 6 (16.7%)	3 of 6 (50%)	Not reported
Multiple Dose VP 20629 600mg	0 of 6 (0%)	6 of 6 (100%)	Not reported
Multiple Dose VP 20629 900mg	0 of 5 (0%)	3 of 5 (60%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Study Director study director

Takeda

5 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing VP 20629 in people with Friedreich's Ataxia, run by Shire at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 6 Oct 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) (time frame: From Start of Study Treatment up to Day 19). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01898884.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jun 2021.