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Tests treatment safety and results for Friedreich Ataxia
Official title A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
ClinicalTrials.gov ID: NCT06953583
What this study is testing
What is Omaveloxolone?
Omaveloxolone is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for friedreich ataxia.
Also referred to as BIIB141, SKYCLARYS, RTA-408.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich's Ataxia (FA) who are 16 years of age or older.
- Phase 3: a large, late-stage study
- Time commitment: about 3 years
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 15
You may be able to join if
- Part 1: Key
- Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion in intron-1 of the...
- Symptomatic for FA as confirmed by clinician assessment. a. Children 7 to \< 16 years must also have an upright stability score (USS) score of 10 to...
You likely can't join if
- Glycosylated hemoglobin A1C (HbA1c) \> 11%
- B-type natriuretic peptide (BNP) \> 200 picograms per milliliter (pg/mL) at screening
- Ejection fraction (EF) \< 40% [based on echocardiogram (ECHO) performed at screening visit]
- Clinically significant cardiac disease except mild to moderate cardiomyopathy Part 2A: Eligibility criteria:
- They have completed Part 1 of the study and no discontinuation criteria have been met.
- Safety and side effects data from Part 1 are supportive of continuation in the judgement of the investigator. Part 2B: Eligibility criteria:
See the full eligibility criteria
- Part 1: Key
- Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion in intron-1 of the frataxin gene, or GAA repeat expansion in 1 allele and with point...
- Symptomatic for FA as confirmed by clinician assessment. a. Children 7 to \< 16 years must also have an upright stability score (USS) score of 10 to ≤ 34 at baseline Part 1: Key
- Glycosylated hemoglobin A1C (HbA1c) \> 11%
- B-type natriuretic peptide (BNP) \> 200 picograms per milliliter (pg/mL) at screening
- Ejection fraction (EF) \< 40% [based on echocardiogram (ECHO) performed at screening visit]
- Clinically significant cardiac disease except mild to moderate cardiomyopathy Part 2A: Eligibility criteria:
- They have completed Part 1 of the study and no discontinuation criteria have been met.
- Safety and side effects data from Part 1 are supportive of continuation in the judgement of the investigator. Part 2B: Eligibility criteria:
- Participants have completed Part 1 of the study and no discontinuation criteria have been met.
- Safety and side effects data from Part 1 are supportive of continuation in the judgement of the Investigator. Note: Other protocol-defined Inclusion/Exclusion criteria may apply.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Gainesville, Florida, United States
- Tampa, Florida, United States
- Philadelphia, Pennsylvania, United States
- Memphis, Tennessee, United States
- Norfolk, Virginia, United States
- Seattle, Washington, United States
- Randwick, New South Wales, Australia
- Parkville, Victoria, Australia
- Innsbruck, Austria
- Brasília, Federal District, Brazil
- Campinas, São Paulo, Brazil
- São Paulo, São Paulo, Brazil
- Montreal, Quebec, Canada
- Québec, Quebec, Canada
- Copenhagen, Denmark
- Montpellier, Hérault, France
- Paris, France
- Aachen, North Rhine-Westphalia, Germany
- Giessen, Germany
+ 14 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 3 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 15 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Gainesville, Florida, United States; Tampa, Florida, United States; Philadelphia, Pennsylvania, United States; Memphis, Tennessee, United States; Norfolk, Virginia, United States and 28 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.