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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

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Conditions

Friedreich AtaxiaFriedreich's ataxiaFARTA 408Omaveloxolone

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

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

Quick Facts

Study Start:2024-07-01
Study Completion:2030-02-21
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06054893

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 15 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Have genetically confirmed FA.
  2. * Have a left ventricular ejection fraction ≥ 40% (based on ECHO performed at Screening Visit).
  3. * 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.
  4. * 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.
  1. * Have uncontrolled diabetes (haemoglobin A1c \[HbA1c\] \>11.0%).
  2. * Have B-type natriuretic peptide (BNP) level \>200 picograms per milliliter (pg/mL) at screening.
  3. * 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.
  4. * Presence of outflow tract obstruction defined as a peak instantaneous gradient \>50 mmHg (based on ECHO performed at screening).
  5. * 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).
  6. * Have a history of CS liver disease (eg, fibrosis, cirrhosis, hepatitis), or have clinically relevant deviations in laboratory tests at screening
  7. * Plan to or have participated in any other interventional clinical study within the 30 days prior to Day 1.
  8. * Have a cognitive impairment that may preclude ability to comply with study procedures, in the opinion of the investigator.
  9. * 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.
  10. * Have previously documented mitochondrial respiratory chain disease.
  11. * Have a history of thromboembolic events within the past 5 years.
  12. * 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).
  13. * Plan to or have scheduled surgical treatment for scoliosis or foot deformity during the study.
  14. * Have had significant suicidal ideation within 30 days prior to Screening Visit, as per investigator judgment, or any history of suicide attempt.
  15. * For females, be pregnant or breastfeeding.
  16. * No discontinuation criteria have been met.
  17. * Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the Investigator.

Contacts and Locations

Study Contact

US Biogen Clinical Trial Center
CONTACT
866-633-4636
clinicaltrials@biogen.com
Global Biogen Clinical Trial Center
CONTACT
clinicaltrials@biogen.com

Principal Investigator

Medical Director
STUDY_DIRECTOR
Biogen

Study Locations (Sites)

Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States

Collaborators and Investigators

Sponsor: Biogen

  • Medical Director, STUDY_DIRECTOR, Biogen

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-07-01
Study Completion Date2030-02-21

Study Record Updates

Study Start Date2024-07-01
Study Completion Date2030-02-21

Terms related to this study

Keywords Provided by Researchers

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

Additional Relevant MeSH Terms

  • Friedreich Ataxia