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BIIB141 (Omaveloxolone) Study for Friedreich's Ataxia in Children and Teens

This study is looking into BIIB141, also known as omaveloxolone or SKYCLARYS®, for children and teens aged 2 to 15 with Friedreich's Ataxia (FA). This medication is already approved for people with FA who are 16 and older, but not yet for younger individuals. Researchers want to understand how a child's body processes BIIB141 and to learn more about its safety. They will measure how the drug moves through the body (apparent clearance, maximum concentration, and volume of distribution) at several time points after a single dose. The study aims to see if there are any medical problems or changes in overall health, including heart health, during the study. You can join if you have genetically confirmed FA and good heart function (left ventricular ejection fraction ≥ 40%).

Study design
This is an interventional study with a planned enrollment of 33 participants. It will evaluate omaveloxolone in three age groups: 2 to under 7 years, 7 to under 12 years, and 12 to under 16 years.
What's involved
Participants will receive a single dose of omaveloxolone. Blood samples will be taken at several time points up to 96 hours after the dose to measure drug levels. Females of childbearing potential must use birth control during screening, treatment, and for 28 days after the last dose.
Compensation
Not stated in the trial record.
Follow-up
Drug levels will be measured up to 96 hours after the dose. Females of childbearing potential must practice birth control until 28 days following administration of the last dose.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06054893

A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

Active, Not Recruiting
PHASE1Ages 2–15InterventionalTreatment
Biogen
~33 participants
Updated 2026-09-03 on ClinicalTrials.gov
What's tested:Omaveloxolone

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Part 1: Apparent Clearance (CL/F) of Omaveloxolone
Measured over Predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours
+19 more outcomes measured

Conditions

Friedreich Ataxia

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Friedreich Ataxia

NCT06054893

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvaniano site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Medical Director · STUDY_DIRECTOR · Biogen

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Part 1: Apparent Clearance (CL/F) of OmaveloxolonePredose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours
  • Part 1: Maximum Concentration (Cmax) of OmaveloxolonePredose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours
  • Part 1: Volume of Distribution (V/F) of OmaveloxolonePredose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours
  • Part 1: Area Under the Plasma Concentration-Time Curve From 0 Extrapolated to Infinity (AUC0-∞) of OmaveloxolonePredose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours
  • Part 1: Area Under the Plasma Concentration-Time Curve From 0 to tlast (AUC0-tlast) of OmaveloxolonePredose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours
  • Part 1: Area Under the Plasma Concentration-Time Curve From 0 to 24 Hours (AUC0-24) of OmaveloxolonePredose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours
  • Part 1: Individual Steady-State AUC0-24 (AUC0-24,ss) of OmaveloxolonePredose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours
  • Part 1: Individual Steady-State Cmax (Cmax,ss) of OmaveloxolonePredose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours
  • Part 1: Concentration at the end of a 24-Hour Dosing Interval (Ctrough,ss) of OmaveloxolonePredose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours
  • Part 2: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)From Day 1 up to the end of study (up to Week 240)

    An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory finding), symptom, or disease temporally associated with use of a medicinal (investigational) product, whether or not related to medicinal (investigational) product. SAE is any untoward medical occurrence that at any dose results in death, in the view of investigator, places the participant at immediate risk of death (life-threatening event), requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in congenital anomaly/birth defect or is medically important event.

  • Part 2: Number of Participants With Clinically Significant Abnormality in Clinical Laboratory AssessmentsFrom Day 1 up to Week 240
  • Part 2: Number of Participants With Clinically Significant Abnormality in Vital SignsFrom Day 1 up to Week 240

    Vital signs, including blood pressure (BP), heart rate (HR), and oral body temperature, will be assessed.

  • Part 2: Number of Participants With Clinically Significant Abnormality in Electrocardiograms (ECGs)From Day 1 up to Week 240
  • Part 2: Number of Participants With Change from Baseline in Echocardiogram (ECHO)From Day 1 up to Week 240
  • Part 2: Number of Participants With Change from Baseline in HeightFrom Day 1 up to Week 240
  • Part 2: Number of Participants With Change from Baseline in WeightFrom Day 1 up to Week 240
  • Part 2: Number of Participants With Change from Baseline Body Mass Index (BMI)From Day 1 up to Week 240
  • Part 2: Number of Participants With Change from Baseline in Tanner AssessmentFrom Day 1 up to Week 240
  • Part 2: Number of Participants With Change from Baseline in Paediatric Growth (Height)From Day 1 up to Week 240
  • Part 2: Number of Participants With Change from Baseline in Paediatric Growth (Weight)From Day 1 up to Week 240