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Friedreich AtaxiaPhase 1BOLDNCT06054893

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 study testing Omaveloxolone in people with Friedreich Ataxia, run by Biogen at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 2 to 15 years
Conditions the study is for
Friedreich Ataxia
What is being tested?
Omaveloxolone
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
33 participants
Where
1 siteUnited States
Study dates
1 Jul 2024to 4 Jun 2030
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 15 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.

Who cannot join

  • 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
Show all 20Show fewer
  • 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.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official title: 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

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:

Read more
  • 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.

Being tested
Omaveloxolone
Keywords
Friedreich's ataxia, FA, RTA 408, Omaveloxolone
Medical subject headings
Friedreich Ataxia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • OmaveloxoloneDrug

    Also called RTA 408, SKYCLARYS, BIIB141

    Administered as specified in the treatment arm

Study groups in NCT06054893
Study groupRoleWhat they receive
Part 1 and 2: Cohort A1ExperimentalCohort 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.
Receives: Drug: Omaveloxolone
Part 1 and 2: Cohort A2ExperimentalCohort 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.
Receives: Drug: Omaveloxolone
Part 1 and 2: Cohort B1ExperimentalCohort 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.
Receives: Drug: Omaveloxolone
Part 1 and 2: Cohort C1ExperimentalCohort 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.
Receives: Drug: Omaveloxolone
Part 1 and 2: Cohort A3ExperimentalCohort 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.
Receives: Drug: Omaveloxolone
Part 1 and 2: Cohort B2ExperimentalCohort 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.
Receives: Drug: Omaveloxolone
Part 1 and 2: Cohort C2ExperimentalCohort 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.
Receives: Drug: Omaveloxolone

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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

    Measured: 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.

    Measured: 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

    Measured: 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.

    Measured: From Day 1 up to Week 240

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

    Measured: From Day 1 up to Week 240

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

    Measured: From Day 1 up to Week 240

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

    Measured: From Day 1 up to Week 240

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

    Measured: From Day 1 up to Week 240

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

    Measured: From Day 1 up to Week 240

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

    Measured: From Day 1 up to Week 240

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

    Measured: From Day 1 up to Week 240

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

    Measured: From Day 1 up to Week 240

Secondary outcomes (4)

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

    Measured: From Day 1 up to Day 22

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

    Measured: From Day 1 up to Day 22

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

    Measured: From Day 1 up to Day 22

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

    Measured: From Day 1 up to Day 22

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Sep 2023
  2. Study start1 Jul 2024
  3. Primary completion4 Jun 2030 (estimated)
  4. Study completion4 Jun 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
33 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06054893
Sponsor's protocol ID
408-C-2001
Acronym
BOLD
Status last verified
Sep 2026
First submitted
13 Sep 2023
Last updated
3 Sep 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Omaveloxolone in people with Friedreich Ataxia, run by Biogen at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). The study lists 20 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06054893.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2026.

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