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Active, not recruitingNCT06054893BOLDUpdated Sep 3, 2026

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

A Phase 1 interventional study of Omaveloxolone in Friedreich Ataxia, sponsored by Biogen. Active, not recruiting at 1 site in United States. Open to participants aged 2 Years to 15 Years. Per ClinicalTrials.gov, last updated 2026-09-03.

Sponsored by Biogen · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
33
Allocation
Non-randomized
Ages
2 Years to 15 Years
Sex
All
01

Study summary

In this study, researchers will learn more about BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedrich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main goal of this study is to learn how BIIB141 is processed in the body of children and teens who are 2 to 15 years old.

The main question researchers want to answer in this study is:

  • How does the body process BIIB141 in children and teens?
  • How many participants have medical problems during the study?
  • Are there any changes in the participants' overall health during the study?
  • Are there any changes in the participants' heart health?
  • Are there any changes in how the participants move through puberty? Puberty is the time in someone's life when their body changes from a child to an adult.

This study will be done as follows:

  • Participants will be screened to see if they can join the study. The screening period will be up to 14 days, after which participants will check into their study research center.
  • There are 2 parts to this study. During Part 1, participants will take a single dose of BIIB141. Participants will be in 1 of 7 different groups based on their age:
  • Group A1: 12 to 15 years old, taking 150 milligrams (mg) of BIIB141
  • Group A2: 12 to 15 years old, taking a dose of BIIB141 based on the data from Group A1
  • Group B1: 7 to 11 years old, taking a dose of BIIB141 based on Group A1 data
  • Group C1: 2 to 6 years old, taking a dose of BIIB141 based on Groups A1, A2, and B1 data
  • Group A3: 12 to 15 years old, taking a dose of BIIB141 based on Groups A1, A2, and B1 data
  • Group B2: 7 to 11 years old, taking a dose of BIIB141 based on Groups A1, A2, and B1 data
  • Group C2: 2 to 6 years old, taking a dose of BIIB141 based on Group A1, A2, A3, B1, B2, and C1 data.
  • During Part 2, participants from Part 1 will take BIIB141 once in the study research center. Cohort A1 will take 150 mg of BIIB141. Dose of Cohorts A2 and B1 will be based on data from Cohort A1, dose of Cohorts C1, A3 and B2 will be based on data from Cohorts A1, A2 and B1, while Cohort C2's dose will be based on all the other groups. Participants will then take it once a day at home.
  • After leaving the study research center in Part 2, participants will return for tests at Week 4, Week 12, Week 24, and then every 24 weeks. Participants will also be contacted by telephone at Week 2, Week 8, and Week 18, and about 30 days after their last dose of the study drug.
  • Participants will be in this study for up to 302 weeks.
Read the detailed description

Recruitment will be limited to the U.S. only as participants will be able to remain on Part 2 of the study until they turn 16 and can access commercially-available drug which is FDA approved for age 16 and above. The part 1 primary objective of the study is to evaluate the pharmacokinetics (PK) of omaveloxolone following administration of a single dose in 3 age cohorts (2 to \<7 years, 7 to \<12 years, and 12 to \<16 years) and secondary objective is to evaluate safety and tolerability of drug. The part 2 primary objective is to evaluate long term safety and tolerability of omaveloxolone.

02

Conditions studied

  • Friedreich Ataxia

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Keywords

  • Friedreich's ataxia
  • FA
  • RTA 408
  • Omaveloxolone
03

In context

Friedreich Ataxia

107 studies on the registry are indexed under Friedreich Ataxia; 25 are open to participants now.

This study's enrollment of 33 is close to the median of 30 across 74 interventional studies indexed under Friedreich Ataxia.

Browse Friedreich Ataxia studies →

Lead sponsor

Biogen is the lead sponsor of 494 studies on the registry; 22 are open to participants now.

Of its 98 completed or terminated interventional studies of FDA-regulated products, 49 (50%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 15 Years
Sexes eligible
All
Accepts healthy volunteers
No

Part 1:

Inclusion criteria

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

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.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
33 participants (actual)

Study arms

  • Experimental
    Part 1 and 2: Cohort A1

    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 when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian population pharmacokinetics (popPK) analyses.

    Drug: Omaveloxolone

  • Experimental
    Part 1 and 2: Cohort A2

    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 when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses.

    Drug: Omaveloxolone

  • Experimental
    Part 1 and 2: Cohort B1

    Cohort B1 will contain participants 7 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 when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses.

    Drug: Omaveloxolone

  • Experimental
    Part 1 and 2: Cohort C1

    Cohort C1 will contain participants 2 to \<7 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 cohorts A1, A2, and B1 to select the dose in part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses.

    Drug: Omaveloxolone

  • Experimental
    Part 1 and 2: Cohort A3

    Cohort A3 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 cohorts A1, A2, and B1 to select the dose in part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses.

    Drug: Omaveloxolone

  • Experimental
    Part 1 and 2: Cohort B2

    Cohort B2 will contain participants 7 to \<12 years of age and will initiate in parallel with Cohort A3. Participants will receive a single oral dose of omaveloxolone, capsule, at a dosage level determined by a Bayesian popPK analysis using the data from cohorts A1, A2, and B1 to select the dose in part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses.

    Drug: Omaveloxolone

  • Experimental
    Part 1 and 2: Cohort C2

    Cohort C2 will contain participants 2 to \<7 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 Cohorts A1, A2, A3, B1, B2, and C1 to select the dose in part 1, followed by the same dose in part 2 up to when the last participant enrolled has the opportunity for their week 240. The dose in part 2 may be adjusted as per additional safety and Bayesian popPK analyses.

    Drug: Omaveloxolone

Interventions

  • DrugOmaveloxolone

    Administered as specified in the treatment arm

    Also known as: RTA 408, SKYCLARYS, BIIB141

06

What researchers measure

Primary outcomes

  1. Part 1: Apparent Clearance (CL/F) of Omaveloxolone

    Time frame: Predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

  2. Part 1: Maximum Concentration (Cmax) of Omaveloxolone

    Time frame: Predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

  3. Part 1: Volume of Distribution (V/F) of Omaveloxolone

    Time frame: Predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

  4. Part 1: Area Under the Plasma Concentration-Time Curve From 0 Extrapolated to Infinity (AUC0-∞) of Omaveloxolone

    Time frame: Predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

  5. Part 1: Area Under the Plasma Concentration-Time Curve From 0 to tlast (AUC0-tlast) of Omaveloxolone

    Time frame: Predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

  6. Part 1: Area Under the Plasma Concentration-Time Curve From 0 to 24 Hours (AUC0-24) of Omaveloxolone

    Time frame: Predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

  7. Part 1: Individual Steady-State AUC0-24 (AUC0-24,ss) of Omaveloxolone

    Time frame: Predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

  8. Part 1: Individual Steady-State Cmax (Cmax,ss) of Omaveloxolone

    Time frame: Predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

  9. Part 1: Concentration at the end of a 24-Hour Dosing Interval (Ctrough,ss) of Omaveloxolone

    Time frame: Predose (0 hour), after dose 1 hour, 2 hours, 3 hours, 4 hours, 24 hours, and 96 hours

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

    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.

    Time frame: From Day 1 up to the end of study (up to Week 240)

  11. Part 2: Number of Participants With Clinically Significant Abnormality in Clinical Laboratory Assessments

    Time frame: From Day 1 up to Week 240

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

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

    Time frame: From Day 1 up to Week 240

  13. Part 2: Number of Participants With Clinically Significant Abnormality in Electrocardiograms (ECGs)

    Time frame: From Day 1 up to Week 240

  14. Part 2: Number of Participants With Change from Baseline in Echocardiogram (ECHO)

    Time frame: From Day 1 up to Week 240

  15. Part 2: Number of Participants With Change from Baseline in Height

    Time frame: From Day 1 up to Week 240

  16. Part 2: Number of Participants With Change from Baseline in Weight

    Time frame: From Day 1 up to Week 240

  17. Part 2: Number of Participants With Change from Baseline Body Mass Index (BMI)

    Time frame: From Day 1 up to Week 240

  18. Part 2: Number of Participants With Change from Baseline in Tanner Assessment

    Time frame: From Day 1 up to Week 240

  19. Part 2: Number of Participants With Change from Baseline in Paediatric Growth (Height)

    Time frame: From Day 1 up to Week 240

  20. Part 2: Number of Participants With Change from Baseline in Paediatric Growth (Weight)

    Time frame: From Day 1 up to Week 240

Secondary outcomes

  1. Part 1: Number of Participants With AEs and SAEs

    Time frame: From Day 1 up to Day 22

  2. Part 1: Number of Participants With Abnormality in Clinical Laboratory Assessments

    Time frame: From Day 1 up to Day 22

  3. Part 1: Number of Participants With Abnormality in Vital Signs

    Time frame: From Day 1 up to Day 22

  4. Part 1: Number of Participants With Abnormality in ECGs

    Time frame: From Day 1 up to Day 22

07

Study locations

1 site
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
08

References and documents

Individual participant data

Plan to share: Yes — In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 3, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06054893
Lead sponsor
Biogen
Responsible party
Sponsor
First posted
Sep 26, 2023
Start date
Jul 1, 2024
Primary completion
Jun 4, 2030 (estimated)
Completion
Jun 4, 2030 (estimated)
Last update
Sep 3, 2026

Study contacts

Medical Director
study director · Biogen

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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