Trial · NCT06953583
Registered outcomes40
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 40
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52 | Baseline, 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). |
| Primary | Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 52 | Baseline (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). |
| Primary | 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) | |
| Primary | 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 | |
| Primary | Part 2B: Change From Baseline in Height at Weeks 52 and Week 104 | Baseline (Week 52 of Part 1), Weeks 52 and 104 | |
| Primary | Part 2B: Change From Baseline in Weight at Weeks 52 and Week 104 | Baseline (Week 52 of Part 1), Weeks 52 and 104 | |
| Primary | 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 | |
| Primary | Part 2B: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) at Weeks 52 and Week 104 | Baseline (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. |
| Primary | Part 2B: Percentage of Participants at Each Tanner Stage at Weeks 52 and Week 104 | Baseline (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. |
| Primary | Part 2B: Number of Participants at Each Tanner Stage at Weeks 52 and Week 104 | Baseline (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. |
| Secondary | Part 1: Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52 | Baseline, 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). |
| Secondary | Part 1: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI) at Week 52 | Baseline, 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. |
| Secondary | Part 1: Change From Baseline in Friedreich's Ataxia-Activities of Daily Living (FA-ADL) | Baseline, Week 52 | 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. |
| Secondary | Part 1: Change From Baseline in Clinical Global Impressions-Severity (CGI-S) at Week 52 | Baseline, 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. |
| Secondary | Part 1: Change From Baseline in Patient Global Impressions-Severity (PGI-S) at Week 52 | Baseline, 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. |
| Secondary | 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) | |
| Secondary | Part 1: Number of Participants With Change From Baseline in Cardiac Function Assessed by ECHO at Week 52 | Baseline, Week 52 | |
| Secondary | Part 1: Change From Baseline in Height at Week 52 | Baseline, Week 52 | |
| Secondary | Part 1: Change From Baseline in Weight at Week 52 | Baseline, Week 52 | |
| Secondary | Part 1: Change From Baseline in Body Mass Index (BMI) at Week 52 | Baseline, Week 52 |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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