Clinical Trial Search

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: Change From Baseline in Modified International Cooperative Ataxia Rating Scale (mICARS) — 0.8; 1.0; 2.3 score on a scale — p=0.0847

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

Primary: Rescored Modified International Cooperative Ataxia Rating Scale (RmICARS) — 0.94; 2.24 RmICARS score (0-29 points)

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 With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs — 78; 61; 4; 0 participants

Primary: Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score — -0.6; -1.0 units on a scale — p=0.5442

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

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

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

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: Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs — 21; 7; 0; 0 participants

Primary: 2-yr Overall Survival — 0.70 probability

Primary: Number of Participants With Response to Rabbit Antithymocyte Globulin (Thymoglobuline) — 1; 11; 18; 3 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: Number (Percentage) of Patients at Each Dosing Cohort Who Experience no Toxicity and in Whom Graft Versus Host Disease (GVHD) is Stable or Improved — 6; 2; 2 Participants

Primary: Change From Baseline in the Average Number of Time Constant 1 (T1) Contrast-enhanced Lesions (CELs) Over On-treatment Scans — 7.4; -2.4 lesions

Primary: Participants no Longer Meeting Criteria for Severe Aplastic Anemia. — 18; 17; 9; 9 participants

Primary: Change in mFARS (Modified Friedreich's Ataxia Rating Scale) Score — 2.9; 4.9; 7.7; 2.2 units on a scale

Primary: Change in Lesion Area of Treated Telangiectasia. — -6; -8 mm^2

Primary: Adverse Events Possibly, Probably or Definitely Related to Study Drug — 2; 2; 3; 2 participants

Primary: Number of Patients With Donor Cell Engraftment — 26 Participants

Primary: Overall Response Rate — 52 percentage of participants

Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…

Primary: California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score — 34.0; 33.5 score on a scale

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

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