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Omaveloxolone for Friedreich's Ataxia

Q: Has the FDA ratified Part A - Cohort 1?

This trial has limited data in support of safety and efficacy, so Part A - Cohort 1 received a score of 1.

Q: Do the eligibility requirements for this trial limit participants to those over 35 years of age?

This research requires participants aged between 2 and 15 years old. Notably, there are 16 clinical trials for minors and 28 studies available for those over 65.

Q: What type of individual is most suitable to take part in this medical investigation?

Eligibility criteria for this research trial includes possessing a diagnosable case of friedreich ataxia and being between the ages of 2 to 15. The researchers wish to recruit 20 participants in total.

Q: Are there any vacancies for potential participants in this research endeavor?

According to clinicaltrials.gov, this particular trial is not presently recruiting participants despite having been posted on November 1st 2023 and updated as recently as September 19th 2023. Fortunately, there are 40 other studies actively seeking candidates at the current moment in time.

Phase 1

Waitlist Available

Led By David Lynch, MD

Research Sponsored by Reata Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

Awards & highlights

Study Summary

This trial studies the safety & effectiveness of a drug for kids with Friedreich's Ataxia. It will be done in 3 parts based on age.

Who is the study for?

This trial is for children aged 2 to less than 16 with genetically confirmed Friedreich's Ataxia (FA). Participants must be able to follow the study plan, use birth control if of childbearing potential, and not have certain infections or recent participation in other trials. They should also have a heart function above a set threshold.Check my eligibility

What is being tested?

The trial is testing Omaveloxolone in pediatric patients with FA. It's an open-label study divided into three parts based on age groups, assessing safety, tolerability, and how the body processes the drug after a single dose.See study design

What are the potential side effects?

While specific side effects are not listed here, typical concerns may include reactions at the site of administration, gastrointestinal symptoms like nausea or diarrhea, fatigue, liver enzyme changes, and potential allergic reactions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

This trial's timeline: 3 weeks for screening, Varies for treatment, and predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Apparent clearance (CL/F) of omaveloxolone

Area under the plasma concentration-time curve from 0 extrapolated to infinity (AUC0-∞) of omaveloxolone

Area under the plasma concentration-time curve from 0 to 24 hours (AUC0-24) of omaveloxolone

+3 more

Trial Design

4Treatment groups

Experimental Treatment

Group I: Part CExperimental Treatment1 Intervention

Part C will contain patients ≥2 to <6 years of age. Subjects will receive a single oral dose of omaveloxolone at a dosage level determined by a Bayesian population pharmacokinetic (popPK) analysis using the data from Part A and Part B to select the dose.

Group II: Part BExperimental Treatment1 Intervention

Part B will contain patients ≥6 to <12 years of age and will initiate in parallel with Part A - Cohort 2. Patients will receive a single oral dose of omaveloxolone at a dosage level determined by a Bayesian population pharmacokinetic (popPK) analysis using the data from Part A - Cohort 1 to select the dose.

Group III: Part A - Cohort 2Experimental Treatment1 Intervention

Part A - Cohort 2 will contain patients ≥12 to <16 years of age. Patients will receive a single oral dose of omaveloxolone at a dosage level determined by a Bayesian population pharmacokinetic (popPK) analysis using the data from Part A - Cohort 1 to select the dose.

Group IV: Part A - Cohort 1Experimental Treatment1 Intervention

Part A - Cohort 1 will contain patients ≥12 to <16 years of age. Patients will receive a single oral dose of 150 mg omaveloxolone.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Omaveloxolone

2019

Completed Phase 1

~120

Do I qualify?Apply below to find out

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Who is running the clinical trial?

Reata Pharmaceuticals, Inc.Lead Sponsor

51 Previous Clinical Trials

6,880 Total Patients Enrolled

1 Trials studying Friedreich Ataxia

172 Patients Enrolled for Friedreich Ataxia

BiogenLead Sponsor

639 Previous Clinical Trials

467,416 Total Patients Enrolled

2 Trials studying Friedreich Ataxia

316 Patients Enrolled for Friedreich Ataxia

David Lynch, MDPrincipal InvestigatorChildren's Hospital of Philadelphia

3 Previous Clinical Trials

3,138 Total Patients Enrolled

3 Trials studying Friedreich Ataxia

3,138 Patients Enrolled for Friedreich Ataxia

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has the FDA ratified Part A - Cohort 1?

"This trial has limited data in support of safety and efficacy, so Part A - Cohort 1 received a score of 1."

Answered by AI

Do the eligibility requirements for this trial limit participants to those over 35 years of age?

"This research requires participants aged between 2 and 15 years old. Notably, there are 16 clinical trials for minors and 28 studies available for those over 65."

Answered by AI

What type of individual is most suitable to take part in this medical investigation?

"Eligibility criteria for this research trial includes possessing a diagnosable case of friedreich ataxia and being between the ages of 2 to 15. The researchers wish to recruit 20 participants in total."

Answered by AI

Are there any vacancies for potential participants in this research endeavor?

"According to clinicaltrials.gov, this particular trial is not presently recruiting participants despite having been posted on November 1st 2023 and updated as recently as September 19th 2023. Fortunately, there are 40 other studies actively seeking candidates at the current moment in time."

Answered by AI