Understanding how omaveloxolone works in children with Friedreich's Ataxia

An Open-Label, Phase 1, Study to Evaluate the Pharmacokinetics, Safety, Tolerability, Pharmacodynamics, and Efficacy of Omaveloxolone in Participants ≥2 to <16 Years of Age With Friedreich's Ataxia

This study is testing how a new drug called omaveloxolone works and is tolerated in children and teens with Friedreich's Ataxia to see if it helps improve their health.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 35 (estimated)
• Ages: 2 Years to 15 Years
• Sex: All
• Sponsor: Biogen (Industry-sponsored)
• Locations: 1 site (Philadelphia, Pennsylvania)
• Trial ID: NCT06054893 on ClinicalTrials.gov

What this trial studies

This study investigates the pharmacokinetics and safety of omaveloxolone in children and teens aged 2 to 15 years with Friedreich's Ataxia. It aims to determine how the drug is processed in their bodies and monitor any medical issues that arise during the study. Participants will be divided into three age cohorts to evaluate the drug's effects and tolerability. The study will also assess changes in overall health, heart health, and puberty progression. The research is limited to the U.S. and will allow participants to continue until they reach the age of 16.

Who should consider this trial

Good fit: Ideal candidates are children and teens aged 2 to 15 years with genetically confirmed Friedreich's Ataxia.

Not a fit: Patients with uncontrolled diabetes or significant heart disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide critical safety and efficacy data for using omaveloxolone in younger patients with Friedreich's Ataxia.

How similar studies have performed: While omaveloxolone has been studied in older populations, this specific investigation in younger patients is novel.

Eligibility criteria

Part 1:

Inclusion Criteria:

* Have genetically confirmed FA.
* Have a left ventricular ejection fraction ≥ 40% (based on ECHO performed at Screening Visit).
* During screening, during the treatment period, and until 28 days following administration of the last dose of omaveloxolone, females of childbearing potential must practice at least 1 of the acceptable methods of birth control.
* During screening, during the treatment period, and until 28 days after the last dose of omaveloxolone, fertile males who have female partners of childbearing potential must practice one of the acceptable methods of birth control.

Exclusion Criteria:

* Have uncontrolled diabetes (haemoglobin A1c \[HbA1c\] \>11.0%).
* Have B-type natriuretic peptide (BNP) level \>200 picograms per milliliter (pg/mL) at screening.
* Have a history of clinically significant (CS) left-sided heart disease and/or CS cardiac disease, with the exception of mild to moderate cardiomyopathy associated with FA.
* Presence of outflow tract obstruction defined as a peak instantaneous gradient \>50 mmHg (based on ECHO performed at screening).
* Have taken any moderate or strong inhibitors and/or inducers of cytochrome P450 3A4 within the 7 days prior to Day 1 or plan to take during study participation (eg, itraconazole, carbamazepine, phenytoin, ciprofloxacin, grapefruit juice, cannabidiol, fluconazole, fluvoxamine, verapamil, diltiazem).
* Have a history of CS liver disease (eg, fibrosis, cirrhosis, hepatitis), or have clinically relevant deviations in laboratory tests at screening
* Plan to or have participated in any other interventional clinical study within the 30 days prior to Day 1.
* Have a cognitive impairment that may preclude ability to comply with study procedures, in the opinion of the investigator.
* Be unable to comply with the requirements of the study protocol or be unsuitable for the study for any reason, in the opinion of the investigator.
* Have previously documented mitochondrial respiratory chain disease.
* Have a history of thromboembolic events within the past 5 years.
* Plan to or have taken anticoagulant therapy within 30 days prior to Day 1 with the exception of a daily low dose aspirin (up to 81 mg).
* Plan to or have scheduled surgical treatment for scoliosis or foot deformity during the study.
* Have had significant suicidal ideation within 30 days prior to Screening Visit, as per investigator judgment, or any history of suicide attempt.
* For females, be pregnant or breastfeeding.

Part 2:

* No discontinuation criteria have been met.
* Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the Investigator.

In the event of intercurrent illness or other change in health status of the participant, additional Part 1 Screening assessments may be repeated prior to initiation of Part 2, based on the judgement of the Investigator in consultation with the Medical Monitor.

NOTE: Other protocol- defined Inclusion/Exclusion criteria may apply.

Where this trial is running

Philadelphia, Pennsylvania

• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)

Study contacts

• Study coordinator: US Biogen Clinical Trial Center

Conditions Friedreich Ataxia; Friedreich's ataxia; FA; RTA 408; Omaveloxolone