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Omaveloxolone for Friedreich's Ataxia (BOLD Trial)

Phase 1

Recruiting

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 from day 1 up to week 240

Awards & highlights

No Placebo-Only Group

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.

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.

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 ~ from day 1 up to week 240

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from day 1 up to week 240

This trial's timeline: 3 weeks for screening, Varies for treatment, and from day 1 up to week 240 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Part 2: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Part 2: Number of Participants With Clinically Significant Abnormality in Vital Signs

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups

Experimental Treatment

Group I: Part 1 and 2: Cohort CExperimental Treatment1 Intervention

Cohort C will contain participants ≥2 to \<6 years of age. Participants will receive a single oral dose of omaveloxolone at a dosage level determined by a Bayesian popPK analysis using the data from cohorts A1, A2, and B to select the dose in part 1 followed by the same dose in part 2 up to week 240 or commercial availability whichever comes first. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses.

Group II: Part 1 and 2: Cohort BExperimental Treatment1 Intervention

Cohort B will contain participants ≥6 to \<12 years of age and will initiate in parallel with Cohort A2. Participants will receive a single oral dose of omaveloxolone, capsule, at a dosage level determined by a Bayesian popPK analysis using the data from Cohort A1 to select the dose in part 1 followed by the same dose in part 2 up to week 240 or commercial availability whichever comes first. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses.

Group III: Part 1 and 2: Cohort A2Experimental Treatment1 Intervention

Cohort A2 will contain participants ≥12 to \<16 years of age. Participants will receive a single oral dose of omaveloxolone, capsule, at a dosage level determined by a Bayesian popPK analysis using the data from Cohort A1 to select the dose in part 1 followed by the same dose in part 2 up to week 240 or commercial availability whichever comes first. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses.

Group IV: Part 1 and 2: Cohort A1Experimental Treatment1 Intervention

Cohort A1 will contain participants ≥12 to \<16 years of age. Participants will receive a single oral dose of omaveloxolone 150 milligrams (mg), capsule, on Day 1 of the treatment period of part 1 followed by the same dose in part 2 up to week 240 or commercial availability whichever comes first. The dose in part 2 may be adjusted as per additional safety and Bayesian population pharmacokinetics (popPK) analyses.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Omaveloxolone

2019

Completed Phase 1

~120

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