N/A
N=108
Neurology Measures in FA Children
Friedreich Ataxia
Bottom Line
View on ClinicalTrials.gov: NCT03418740 ↗Enrolled (actual)
108
Serious AEs
—
Results posted
Jan 2025
Primary outcome: Primary: Change in mFARS (Modified Friedreich's Ataxia Rating Scale) Score — 2.9; 4.9; 7.7; 2.2 units on a scale
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- —
- Age
- Pediatric, Adult · 2+ yrs
- Sex
- All
- Sponsor
- Children's Hospital of Philadelphia
- Primary completion
- Feb 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in mFARS (Modified Friedreich's Ataxia Rating Scale) Score |
2.9; 4.9; 7.7; 2.2; 3.7; 5.5 | — |
| SECONDARY Change in Timed 25-Foot Walk (T25FW) Test |
-0.2; -0.4; -0.5 | — |
| SECONDARY Change in 9-Hole Peg Test (9HPT) |
-0.1; -0.2; -0.3 | — |
| SECONDARY Change in Timed Up and Go (TUG) Test |
-1.4; -3.6; -4.9 | — |
| SECONDARY Change in Berg Balance Scale (Full Length) (BBS) Score |
-6.5; -10.4; -15 | — |
| SECONDARY Change in FA-Activities of Daily Living Scale (ADL) Score |
0.1; 1.9; 3 | — |
| SECONDARY Change in 1-minute Walk (1MW) and 6-minute Walk (6MW) Tests |
-11; -15.7; -23.1; -13.1; -65.8; -67.4 | — |
Summary
The purpose of this study is to identify ways to follow progression of Friedreich's Ataxia (FA) and be able to measure changes over time in children with FA. Participants will have biannual visits to observe how the disease progresses over time and determine the rate of progression.
Funding Source- Food and Drug Administration Office of Orphan Products Development (FDA OOPD).
Eligibility Criteria
Inclusion Criteria
- Males or females age 2 to 18 years.
- Genetically confirmed diagnosis of Friedreich's Ataxia (FA) or clinically confirmed diagnosis of FA, pending confirmatory genetic testing through a commercial or research laboratory
- Parental/guardian permission (informed consent) and if appropriate, child assent.
Exclusion Criteria
- Inability to complete study evaluations
Data sourced from ClinicalTrials.gov (NCT03418740). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.