Clinical Trial Search

Primary: Scale for the Assessment and Rating of Ataxia(SARA) — 11.5; 9 score on a scale

Primary: Percent Change From Baseline to Post Treatment on the Scale for the Assessment and Rating of Ataxia (SARA) — -25.9; -27.8 percentage change — p=0.29

Primary: Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8 — -0.810; -1.059 score on a scale — p=0.519

Primary: Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical…

Primary: Change in Timed 25 Feet Walking Test (T25FW) — 23.7; 24.4; -1.1; -0.3 second — p=>0.05

Primary: Change in the International Cooperative Ataxia Rating Scale (ICARS) Score From Baseline — 53.0; 52.7; 43.0; 52.1 units on a scale — p=<0.001

Primary: Achievement of Independent Sitting for at Least 30 Seconds — 13 Participants — p=<0.0001

Primary: Change in Assessment and Rating of Ataxia (SARA) Score — -2.4; -0.9; -1.5; -0.2 score on a scale

Primary: Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds — 14 Participants

Primary: Laboratory Safety Data

Primary: Walking Speed in Meters Per Second as Assessed by the 10 Meter Walk Test(10MWT) — 0.13; 0.60 meters per second

Primary: Charcot Marie Tooth Neuropathy Score — 25.67; 20.22 scores on a scale

Primary: Number of Participants Who Achieved Sitting Alone for at Least 10 Seconds — 1 Participants

Primary: Number of Patients With Adverse Events — 5; 2; 5; 2 participants

Primary: Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population — -2.2; -3.4; -4.0…

Primary: Creatine Rephosphorylation Rate Post Exercise — 0.024; 0.029; 0.022; 0.029 pH per second — p=0.9133

Primary: Change in Whole Blood Frataxin Levels — -1.5 percentage of baseline frataxin level — p=0.027

Primary: Change From Baseline in the Inverse Time to Complete the 9-Hole Peg Test (9-HPT-1) — 0.00038; -0.00017; -0.00032 1/seconds — p=>0.999

Primary: FXTAS Rating Scale Score — 45.8 score on a scale

Primary: Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score — 55.0 score on a scale — p=0.336

Primary: Number of Participants With Dose Limiting Toxicities (DLT) in Part 1 - Safety Analysis Set (SAS) — 0; 0; 0; 0 Participants

Primary: Number of Participants With Treatment-emergent Adverse Events Over Time — 6; 6; 6; 2 Participants

Primary: Change From Baseline in Executive Functioning as Measured by the Behavioral Dyscontrol Scale II (BDS-II) — 17.44; 16.12; 15.66; 15.72 units on a scale

Primary: Change From Baseline to 11 Months in Maximum Consumption of Oxygen (mlO2/kg/Min) Using Cardiopulmonary Exercise Testing (CPET) — 0.05; 0.08 ml O2/min/kg — p=0.5858

Primary: Part 1: Selected Part 2 Dose of Risdiplam — 0.2 mg/kg

Primary: Incidence And Severity Of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 13; 5; 7; 7 Participants

Primary: Safety: Frequency of Adverse Events/Lab Abnormalities

Primary: Number of Participants With Tolerabilty of 200 mg Vorinostat in Niemann-Pick Disease, Type C1 — 12 Participants

Primary: Neuropsychological Score of Sustained Attention — 11 Participants

Primary: Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52 — 1.6; 1.7; 1.2; 1.1 units on a scale