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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-520896-13 | Other Identifier | EU CT Number |
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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. However, it is not yet available for younger teens and children. The main goal of this study is to learn how omaveloxolone affects symptoms of FA and its safety in younger participants between the ages of 2 and 15 years old.
The main questions researchers want to answer in this study are:
Researchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) to test nerve function. The mFARS tests movement ability, balance, coordination, speech, and arm and leg functions.
They will also use a number of questionnaires to learn more about participants' quality of life, muscle strength, and ability to perform daily tasks. Researchers will also note any changes as participants go through puberty.
Finally, researchers will learn more about how the body processes omaveloxolone in children and teenagers.
This study will be done in 2 parts as follows:
The primary objective of Part 1 is to evaluate the efficacy of omaveloxolone as measured by upright stability score (USS) and the secondary objectives are to evaluate the efficacy of omaveloxolone as measured by additional secondary efficacy outcomes, safety of omaveloxolone and the plasma concentration of omaveloxolone after single and multiple dose administration.
The primary objective of Part 2A is to evaluate the efficacy of omaveloxolone and the secondary objectives are to characterize the efficacy of omaveloxolone as measured by additional secondary outcomes, evaluate the safety and tolerability of omaveloxolone and plasma concentration of omaveloxolone after single and multiple dose administration.
The primary objective for Part 2B is to evaluate the safety and tolerability of long-term omaveloxolone use and the secondary objective is to evaluate the efficacy of omaveloxolone following long-term use.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part 1: Omaveloxolone | Experimental | Participants will receive a single oral dose of omaveloxolone once a day (QD) for up to 52 weeks in Part 1 of the study. |
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| Part 1: Placebo | Placebo Comparator | Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study. |
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| Part 2A Continued Efficacy Evaluation: Omaveloxolone | Experimental | Participants will receive a single oral dose of omaveloxolone, QD for up to 104 weeks in Part 2A of the study. |
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| Part 2B Safety: Omaveloxolone | Experimental | Participants will receive a single oral dose of open-label omaveloxolone, QD for up to 104 weeks in Part 2B of the study. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Omaveloxolone | Drug | Administered as specified in the treatment arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at 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). | Baseline, Week 52 |
| Part 2A: Change From Baseline in USS Subscale E of mFARS at 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). | Baseline (Week 52 of Part 1), Week 52 |
| 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) |
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI) at Week 52 | The FA-HI is a participant reported survey that assesses overall disease burden on a 100-point scale, with 0 representing no disease burden and 100 representing the maximum level of disease burden containing 113 symptoms questions representing 18 symptomatic subscales. | Baseline, Week 52 |
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Part 1: Key inclusion criteria:
Part 1: Key exclusion criteria:
Part 2A: Eligibility criteria:
Part 2B: Eligibility criteria:
Note: Other protocol-defined Inclusion/Exclusion criteria may apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Patient Navigator | Contact | 1-877-223-3576 | 57078 | biogenBRAVE_patientnavigator@thermofisher.com |
| US Biogen Clinical Trial Center | Contact | 866-633-4636 | clinicaltrials@biogen.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA Neurology Outpatient Clinic at Westwood | Not yet recruiting | Los Angeles | California | 90095 | United States |
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| Label | URL |
|---|---|
| Related Info | View source |
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In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
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| Placebo | Drug | Administered as specified in the treatment arm. |
|
| Part 2B: Number of Participants With Change From Baseline in Cardiac Function Assessed by Echocardiogram (ECHO) at Weeks 52 and Week 104 | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2B: Change From Baseline in Height at Weeks 52 and Week 104 | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2B: Change From Baseline in Weight at Weeks 52 and Week 104 | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2B: Change From Baseline in Body Mass Index (BMI) at Weeks 52 and Week 104 | Baseline (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 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. | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2B: Percentage of Participants at Each Tanner Stage at Weeks 52 and Week 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. | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2B: Number of Participants at Each Tanner Stage at Weeks 52 and Week 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. | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 1: Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS) at 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). | Baseline, Week 52 |
| Part 1: Change From Baseline in Patient Global Impressions-Severity (PGI-S) at Week 52 | PGI-S will be conducted for participants 7 to < 16 years of age. These are clinically meaningful outcome measures that are participant-relevant across all age groups and disease severities for this population. PGI -S is a 1-item questionnaire where the response is recorded on a 4-point scale scored as: 1-normal, 2-mild, 3-moderate, or 4-severe. | Baseline, Week 52 |
| Part 1: Change From Baseline in Clinical Global Impressions-Severity (CGI-S) at Week 52 | The CGI-S will be conducted for all enrolled participants, 2 to < 16 years of age. The CGI-S rating evaluates the severity of individual symptoms and treatment response in participants with mental disorders. The CGI-S is a 7-point scale that requires the clinician to rate the severity of the participant's illness at the time of assessment. A rating of 1 is considered normal, or with the least severe symptoms, a rating of 7 is extremely ill, or the worst symptoms. | Baseline, Week 52 |
| Part 1: Change From Baseline in Friedreich's Ataxia-Activities of Daily Living (FA-ADL) | Participants will answer the 9 questions of the FA-ADL survey in an interview style conducted by any site staff. The FA-ADL survey assesses 9 concepts: (1) speech; (2) swallowing; (3) cutting food and handling utensils; (4) dressing; (5) personal hygiene; (6) falling; (7) walking; (8) quality of sitting position; and (9) bladder function. | Baseline, Week 52 |
| Part 1: Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE) | From first dose of study drug up to end of follow up period in Part 1 (up to Week 52) |
| Part 1: Number of Participants With Change From Baseline in Cardiac Function Assessed by ECHO at Week 52 | Baseline, Week 52 |
| Part 1: Change From Baseline in Height at Week 52 | Baseline, Week 52 |
| Part 1: Change From Baseline in Weight at Week 52 | Baseline, Week 52 |
| Part 1: Change From Baseline in Body Mass Index (BMI) at Week 52 | Baseline, Week 52 |
| Part 1: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Week 52 | 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. | Baseline, Week 52 |
| Part 1: Percentage of Participants at Each Tanner Stage at Week 52 | 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. | Baseline, Week 52 |
| Part 1: Number of Participants at Each Tanner Stage at Week 52 | 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. | Baseline, Week 52 |
| Part 1: Plasma Concentrations of Omaveloxolone | Pre-dose and post-dose on Day 1, Weeks 4, 12 and 26 |
| Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 104 | 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). | Baseline (Week 52 of Part 1), Week 104 |
| Part 2A: Change from baseline in mFARS at Weeks 52 and Week 104 | 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). | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2A: Number of Participants With TEAE and TESAE | From the first dose of the study drug in Part 2A up to the end of follow-up period in Part 2A (up to Week 104) |
| Part 2A: Number of Participants With Change From Baseline in Cardiac Function Assessed by ECHO at Weeks 52 and Week 104 | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2A: Change From Baseline in Height at Weeks 52 and Week 104 | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2A: Change From Baseline in Weight at Weeks 52 and Week 104 | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2A: Change From Baseline in BMI at Weeks 52 and Week 104 | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2A: Change From Baseline in C-SSRS at Weeks 52 and Week 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. | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2A: Percentage of Participants at Each Tanner Stage at Weeks 52 and Week 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. | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2A: Number of Participants at Each Tanner Stage at Weeks 52 and Week 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. | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Part 2A: Plasma Concentrations of Omaveloxolone | Pre-dose and post-dose on Day 1, Weeks 4, 12 and 26 |
| Part 2B: Change From Baseline in mFARS Including USS at Weeks 52 and Week 104 | 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). | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Parts 2A and 2B: Change From Baseline in FA-HI at Weeks 52 and Week 104 | The FA-HI is a participant reported survey that assesses overall disease burden on a 100-point scale, with 0 representing no disease burden and 100 representing the maximum level of disease burden containing 113 symptoms questions representing 18 symptomatic subscales. | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Parts 2A and 2B: Change From Baseline in PGI-S at Weeks 52 and Week 104 | PGI-S will be conducted for participants 7 to < 16 years of age. These are clinically meaningful outcome measures that are participant-relevant across all age groups and disease severities for this population. PGI -S is a 1-item questionnaire where the response is recorded on a 4-point scale scored as: 1-normal, 2-mild, 3-moderate, or 4-severe. | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Parts 2A and 2B: Change From Baseline in CGI-S at Weeks 52 and Week 104 | The CGI-S will be conducted for all enrolled participants, 2 to < 16 years of age. The CGI-S rating evaluates the severity of individual symptoms and treatment response in participants with mental disorders. The CGI-S is a 7-point scale that requires the clinician to rate the severity of the participant's illness at the time of assessment. A rating of 1 is considered normal, or with the least severe symptoms, a rating of 7 is extremely ill, or the worst symptoms. | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Parts 2A and 2B: Change from baseline in FA-ADL at Part 2A Weeks 52 and Week 104 | Participants will answer the 9 questions of the FA-ADL survey in an interview style conducted by any site staff. The FA-ADL survey assesses 9 concepts: (1) speech; (2) swallowing; (3) cutting food and handling utensils; (4) dressing; (5) personal hygiene; (6) falling; (7) walking; (8) quality of sitting position; and (9) bladder function. | Baseline (Week 52 of Part 1), Weeks 52 and 104 |
| Norman Fixel Institute for Neurological Diseases UF Health | Recruiting | Gainesville | Florida | 32610-3010 | United States |
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| USF Health Morsani College of Medicine Department of Neurology | Recruiting | Tampa | Florida | 33612 | United States |
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| Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
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| St. Jude Children's Research Hospital - PIN | Recruiting | Memphis | Tennessee | 38105-3678 | United States |
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| CHKD's Health Center - South Campus - PIN | Recruiting | Norfolk | Virginia | 23507-1910 | United States |
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| Seattle Children's Hospital | Recruiting | Seattle | Washington | 98105-3901 | United States |
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| Sydney Children's Hospital | Not yet recruiting | Randwick | New South Wales | 2031 | Australia |
| Murdoch Childrens Research Institute (MCRI) | Recruiting | Parkville | Victoria | 3052 | Australia |
| Universitätsklinikum Innsbruck | Recruiting | Innsbruck | 6020 | Austria |
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| L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME | Recruiting | Brasília | Federal District | 70200-730 | Brazil |
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| University of Campinas (UNICAMP) School of Medical Sciences | Not yet recruiting | Campinas | São Paulo | 13083-970 | Brazil |
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| PSEG Centro de Pesquisa Clinica | Recruiting | São Paulo | São Paulo | 04024-002 | Brazil |
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| McGill University | Recruiting | Montreal | Quebec | H3H 2R9 | Canada |
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| CHU de Quebec -Universite Laval | Recruiting | Québec | Quebec | G1V 4G2 | Canada |
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| Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen | Not yet recruiting | Copenhagen | 2100 | Denmark |
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| CHU de Montpellier- Hôpital Gui De Chauliac | Recruiting | Montpellier | Hérault | 34090 | France |
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| AP-HP - Hôpital Armand Trousseau | Recruiting | Paris | 75012 | France |
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| Universitätsklinikum Aachen | Recruiting | Aachen | North Rhine-Westphalia | 52074 | Germany |
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| UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen | Recruiting | Giessen | 35392 | Germany |
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| Universitätsklinikum Hamburg Eppendorf | Recruiting | Hamburg | 20246 | Germany |
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| All India Institute of Medical Sciences (AIIMS) - New Delhi | Withdrawn | New Delhi | National Capital Territory of Delhi | 110029 | India |
| CHI at Temple Street | Recruiting | Dublin | D01 XD99 | Ireland |
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| Ospedale Pediatrico Bambino Gesù IRCCS | Not yet recruiting | Rome | Lazio | 165 | Italy |
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| IRCCS Eugenio Medea - Polo. Scientifico Veneto | Not yet recruiting | Conegliano | Veneto | 31015 | Italy |
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| Fondazione IRCCS Istituto Neurologico Carlo Besta | Recruiting | Milan | 20133 | Italy |
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| Radboud Universitair Medisch Centrum | Recruiting | Nijmegen | 6525 GA | Netherlands |
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| King Faisal Specialist Hospital & Research Centre | Withdrawn | Riyadh | Ar Riya | 12875 | Saudi Arabia |
| Hospital Sant Joan de Deu - PIN | Recruiting | Espluges de Llobregat | Barcelona | 8950 | Spain |
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| Hospital Universitario La Paz - PPDS | Recruiting | Madrid | 28046 | Spain |
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| Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi | Withdrawn | Istanbul | 34093 | Turkey (Türkiye) |
| University College Hospital - PPDS | Recruiting | London | Lincolnshire | NW1 2BU | United Kingdom |
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| John Radcliffe Hospital | Recruiting | Oxford | Oxfordshire | OX3 9DU | United Kingdom |
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| Sheffield Children's Hospital - PPDS | Recruiting | Sheffield | South Yorkshire | S10 5DD | United Kingdom |
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| ID | Term |
|---|---|
| D005621 | Friedreich Ataxia |
| ID | Term |
|---|---|
| D013132 | Spinocerebellar Degenerations |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028361 | Mitochondrial Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C000589490 | omaveloxolone |
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