Omaveloxolone for Children and Teens with Friedreich's Ataxia
This study is looking into omaveloxolone (also known as BIIB141 or SKYCLARYS®) for children and teens aged 2 to 15 years old who have Friedreich's Ataxia (FA). Omaveloxolone is already approved for adults with FA, but not for younger individuals. Researchers want to understand how omaveloxolone affects FA symptoms and its safety in this younger age group. They will measure changes in balance (upright stability) and track any side effects. To join, you must have a confirmed diagnosis of FA, including specific genetic markers (GAA repeat expansion or other mutations in the frataxin gene). The study aims to enroll 255 participants, but its current status is unclear.
- Study design
- This is an interventional study planning to enroll 255 participants. It will compare omaveloxolone to a placebo (an inactive substance).
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants in Part 2B will be followed for safety up to Week 104 after their first dose.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
At a glance
Conditions
NCT06953583
Where you'd take part
This study runs at 34 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
AP-HP - Hôpital Armand Trousseau
Paris, Francestudy coordinator listed
Recruiting
CHI at Temple Street
Dublin, Irelandstudy coordinator listed
Recruiting
Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN
Philadelphia, Pennsylvaniastudy coordinator listed
Recruiting
CHKD's Health Center - South Campus - PIN
Norfolk, Virginiastudy coordinator listed
Recruiting
CHU de Montpellier- Hôpital Gui De Chauliac
Montpellier, Hérault, Francestudy coordinator listed
Recruiting
CHU de Quebec -Universite Laval
Québec, Quebec, Canadastudy coordinator listed
Recruiting
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italystudy coordinator listed
Recruiting
Hospital Sant Joan de Deu - PIN
Espluges de Llobregat, Barcelona, Spainstudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Medical Director · STUDY_DIRECTOR · Biogen
Who to contact
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Inclusion
Exclusion
What this trial measures
- Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52Baseline, Week 52
The mFARS is a validated and sensitive rating scale that was developed to quantitatively assess the severity of the neurologic features of FA in adults and adolescents. Scores on the mFARS range from 0 to 93, with lower scores indicating better neurological function. The subscales of the mFARS assessment and maximum score for each subscale are: bulbar function (Subscale A; 2 assessments of speech and cough; maximum score = 5), upper limb coordination (Subscale B; 5 assessments of coordination of movement and function in arms and hands with each limb scored individually; maximum score = 36), lower limb coordination (Subscale C; 2 assessments of coordination of movement and function of lower limbs with each limb scored individually; maximum score = 16), and upright stability (USS, Subscale E; 9 assessments of sitting posture, stance, tandem walk, and gait assessments; maximum score = 36).
- Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 52Baseline (Week 52 of Part 1), Week 52
The mFARS is a validated and sensitive rating scale that was developed to quantitatively assess the severity of the neurologic features of FA in adults and adolescents. Scores on the mFARS range from 0 to 93, with lower scores indicating better neurological function. The subscales of the mFARS assessment and maximum score for each subscale are: bulbar function (Subscale A; 2 assessments of speech and cough; maximum score = 5), upper limb coordination (Subscale B; 5 assessments of coordination of movement and function in arms and hands with each limb scored individually; maximum score = 36), lower limb coordination (Subscale C; 2 assessments of coordination of movement and function of lower limbs with each limb scored individually; maximum score = 16), and upright stability (USS, Subscale E; 9 assessments of sitting posture, stance, tandem walk, and gait assessments; maximum score = 36).
- Part 2B: Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE)From the first dose of the study drug in Part 2B up to the end of follow-up period in Part 2B (up to Week 104)
- Part 2B: Number of Participants With Change From Baseline in Cardiac Function Assessed by Echocardiogram (ECHO) at Weeks 52 and Week 104Baseline (Week 52 of Part 1), Weeks 52 and 104
- Part 2B: Change From Baseline in Height at Weeks 52 and Week 104Baseline (Week 52 of Part 1), Weeks 52 and 104
- Part 2B: Change From Baseline in Weight at Weeks 52 and Week 104Baseline (Week 52 of Part 1), Weeks 52 and 104
- Part 2B: Change From Baseline in Body Mass Index (BMI) at Weeks 52 and Week 104Baseline (Week 52 of Part 1), Weeks 52 and 104
- Part 2B: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Weeks 52 and Week 104Baseline (Week 52 of Part 1), Weeks 52 and 104
The C-SSRS is a low-burden measure of the spectrum of suicidal ideation and behavior that was developed to assess severity and track suicidal events through any treatment of individuals ≥ 6 years of age. The C-SSRS is a clinical interview providing a summary of both ideation and behavior that can be administered by the clinician during any evaluation or risk assessment to identify the level and type of suicidality present. The assessment includes "yes" or "no" responses for 5 questions each, related to suicidal ideation (wish to be dead, non-specific active suicidal thoughts, active suicidal ideation with any methods, active suicidal ideation with some intent, active suicidal ideation with specific plan) and suicidal behavior (preparatory acts or behavior, aborted attempt, interrupted attempt, actual attempt, suicide). Numeric ratings are provided for severity of ideation, from 1 to 5, with 5 being the most severe.
- Part 2B: Percentage of Participants at Each Tanner Stage at Weeks 52 and Week 104Baseline (Week 52 of Part 1), Weeks 52 and 104
Assessment of Tanner stages (a scale of physical development) will be performed by a medical doctor experienced with this assessment. Tanner score ranges from Stage 1 (childhood) to Stage 5 (full physical maturity). Information regarding Tanner staging will be collected at baseline for all participants and will be stopped once the participant reaches Tanner Stage 5 in all gender-appropriate scales.
- Part 2B: Number of Participants at Each Tanner Stage at Weeks 52 and Week 104Baseline (Week 52 of Part 1), Weeks 52 and 104
Assessment of Tanner stages (a scale of physical development) will be performed by a medical doctor experienced with this assessment. Tanner score ranges from Stage 1 (childhood) to Stage 5 (full physical maturity). Information regarding Tanner staging will be collected at baseline for all participants and will be stopped once the participant reaches Tanner Stage 5 in all gender-appropriate scales.