RecruitingPhase 1

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

United States35 participantsStarted 2024-07-01

Plain-language 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 objective 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. * Participants will be in this study for up to 240 weeks.

Who can participate

Age range2 Years – 15 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 cl…

What they're measuring

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

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

Part 1: Maximum Concentration (Cmax) of Omaveloxolone

Timeframe: Predose (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 Omaveloxolone

Timeframe: Predose (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 Omaveloxolone

Timeframe: Predose (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 Omaveloxolone

Timeframe: Predose (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 Omaveloxolone

Timeframe: Predose (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 Omaveloxolone

Trial details

NCT IDNCT06054893

SponsorBiogen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2030-02-21

Contact for this trial

US Biogen Clinical Trial Center

866-633-4636 clinicaltrials@biogen.com

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