idebenone and Friedreich Disease

idebenone has been researched along with Friedreich Disease in 65 studies

Research

Studies (65)

Authors

Clinical Trials (8)

Trial Overview

Trial Outcomes

Absolute Change From Baseline to Week 24 in Left Ventricular Mass Index (LVMI)

To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography (NCT00537680)
Timeframe: baseline and 6 months

Absolute Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle

To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography (NCT00537680)
Timeframe: baseline and 6 months

Absolute Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)

To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography (NCT00537680)
Timeframe: baseline and 6 months

Absolute Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle

To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography (NCT00537680)
Timeframe: baseline and 6 months

Activities of Daily Living (ADL) of Friedreich's Ataxia Rating Scale (FARS), Change in ADL (Total Score ) From Baseline to Week 24

"To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on Activities of Daily Living (ADL) assessed by the ADL scale of the Friedreich's Ataxia Rating Scale (FARS)~score 0 to 36, higher score indicates greater impairment" (NCT00537680)
Timeframe: baseline and 6 months

Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline to Week 24

"To compare the efficacy of 6 months' treatment with 2 different doses of idebenone with that of placebo on neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS)~FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population.~The FARS exam scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability.~Calculation details FARS score at week 24 minus FARS score baseline" (NCT00537680)
Timeframe: baseline and 6 months

Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 12

"To compare the efficacy of 3 months' treatment with 2 different doses of idebenone with that of placebo on neurological impairment as assessed by the International Cooperative Ataxia Rating Scale (ICARS)~International Cooperative Ataxia Rating Scale (ICARS):~ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability.~Minimum score: 0, maximum score 100, higher score indicates greater impairment~Calculation details: ICARS score at week 12 minus ICARS score baseline" (NCT00537680)
Timeframe: baseline and 12 weeks

Change in the International Cooperative Ataxia Rating Scale (ICARS) From Baseline Assessment to Week 24

"International Cooperative Ataxia Rating Scale (ICARS):~ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability.~Minimum score: 0, maximum score 100, higher score indicates greater impairment~Calculation details: ICARS score at week 24 minus ICARS score baseline" (NCT00537680)
Timeframe: baseline and 6 months

Percent Change From Baseline to Week 24 in Left Ventricular Mass Index

To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography (NCT00537680)
Timeframe: baseline and 6 months

Percent Change From Baseline to Week 24 in the Relative Wall Thickness (RWT) of the Left Ventricle

To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography (NCT00537680)
Timeframe: baseline and 6 months

Percent Change From Baseline to Week 24 in the Thickness of the Interventricular Septum (IVS)

To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography (NCT00537680)
Timeframe: baseline and 24 weeks

Percent Change From Baseline to Week 24 in the Thickness of the Posterior Wall (PW) of the Left Ventricle

To compare the effects of idebenone on cardiac anatomy and function in patients with Friedreich's ataxia (FRDA) as assessed by echocardiography (NCT00537680)
Timeframe: baseline and 6 months

Change in ICARS

"International Cooperative Ataxia Rating Scale (ICARS):~ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability.~A total score of 0 points represents the best possible score, and a value of 100 points is the worst possible score. Therefore, a negative change in ICARS score represents an improvement. A higher score also indicates more disability." (NCT00697073)
Timeframe: baseline and 12 months

FARS (Friedreich's Ataxia Rating Scale)

FARS consists of a 25 manoeuvre exam along with 4 quantitative performance measures. The neurological exam covers bulbar function, upper limb coordination, lower limb coordination, peripheral nervous system function, deep tendon reflexes, stability and gait. The use of FARS has been recently validated as a sensitive scale for this population. The FARS consists of three subscales, comprising a general score for ataxia, a score for activities of daily living (ADL) and a neurological examination. The scores can be added to make a total score ranging from 0 to 159. A higher score indicates a greater level of disability. (NCT00697073)
Timeframe: baseline and 12 Months

Frequency/Number of Mild, Moderate, and Severe Adverse Events

(NCT00697073)
Timeframe: 12 months

Nature of Adverse Events

(NCT00697073)
Timeframe: 12 Months

Area Under the Curve (AUC) of the Combinatorial Weight-Adjusted Disability Score (CombiWISE)

"CombiWISE is a composite scale derived from Expanded Disability Status Scale (EDSS) , Scripps Neurological Disability Scale (SNRS), times 25 foot walk (25FW), and non-dominant hand of 9 hole peg test (9HPT) with a minimum value of 0 (no disability) and maximum value of 100 (maximum disability).~The AUC values were calculated for both study groups (Active treatment group in the 09-I-0197 trial and Placebo group in the 09-I-0197) as follows:~pre-treatment baseline during the 09-I-0197 trial (from Months -12, -6, and 0)~double-blind phase during the 09-I-0197 trial (from Months 0, 6, 12, 18, and 24)~extension phase during the 13-I-0088 trial (from Months 24, 30, and 36)~Because the follow-up times varied from patient to patient, the AUC values were made comparable by scaling them by dividing the AUC value by the square of the actual duration (in years) of each of the phases." (NCT01854359)
Timeframe: 1-year pre-treatment baseline vs 2-year randomized double-blind phased vs 1-year treatment period

Change in Slopes of Scripps Neurological Rating Scale (SNRS) Score

"Slopes of measured SNRS scores during the 3-year 09-I-0197 trial (one year of pre-treatment baseline and two years of double-blind randomized treatment) and during one year extension phase of the 13-I-0088 trial. The slopes were measured separately for placebo and active treatment arm as randomized in the the 09-I-0197 trial.~SNRS scale combines various elements of a neurological exam into a single number. The scale ranges from 100 to 0, where 100 marks no disability and 0 marks maximum disability.~SNRS was assessed every 6 months." (NCT01854359)
Timeframe: 3-years double-blind phase and 1-year extension phase

Slopes of 25 Foot Walk (25FW) Time

"slopes of measured times of 25FW during the 3-year 09-I-0197 trial (one year of pre-treatment baseline and two years of double-blind randomized treatment) and during one year extension phase of the 13-I-0088 trial. The slopes were measured separately for placebo and active treatment arm as randomized in the the 09-I-0197 trial.~Lower extremity disability was measured by an average of two trials of timed 25 foot walk assessed every 6 month.~The maximum time assigned for a trial is 180s. Patients unable to complete the 25 foot trial within this time limit are coded as 179.9" (NCT01854359)
Timeframe: 3-years double-blind phase and 1-year extension phase

Slopes of 9 Hole Peg Test (9HPT) Time

"Slopes of measured times of 9HPT during the 3-year 09-I-0197 trial (one year of pre-treatment baseline and two years of double-blind randomized treatment) and during one year extension phase of the 13-I-0088 trial. The slopes were measured separately for placebo and active treatment arm as randomized in the the 09-I-0197 trial.~Upper extremity/fine motor movements disability was measured as an average of left and right hand time, with each hand assessed as an average of two trials with upper limit of 5 (300s) per trial. Patients unable to complete the task within this time are coded as 777 The outcome was assessed every 6 months." (NCT01854359)
Timeframe: 3-years double-blind phase and 1-year extension phase

Slopes of Expanded Disability Status Scale (EDSS) Score

"Slopes of measured EDSS scores during the 3-year 09-I-0197 trial (one year of pre-treatment baseline and two years of double-blind randomized treatment) and during one year extension phase of the 13-I-0088 trial. The slopes were measured separately for placebo and active treatment arm as randomized in the the 09-I-0197 trial.~EDSS scale combines various elements of neurological exam. EDSS is a discrete scale ranging from 0 to 10 with 0.5 point increments. EDSS of 0 means no neurological disability, while EDSS of 10 marks death due to Multiple Sclerosis (MS). EDSS was assessed every 6 months." (NCT01854359)
Timeframe: 3-years double-blind phase and 1-year extension phase

Change in Slopes of 25FW Time From Baseline to Treatment Phase

"Lower extremity disability was measured by an average of two trials of timed 25 foot walk assessed at month -12, -6, and 0 for the baseline phase and at month 0, 6, 12, 18, and 24 for the treatment phase. The progression rate was calculated as a difference between baseline and treatment slopes using a piecewise linear mixed-effect model with breaking point at month 0.~The maximum time assigned for a trial is 180s. Patients unable to complete the 25 foot trial within this time limit are coded as 179.9" (NCT00950248)
Timeframe: 1-year pre-treatment baseline vs 2-year treatment period

Change in Slopes of 9HPT Time From Baseline to Treatment Phase

"Upper extremity/fine motor movements disability was measured as an average of left and right hand time, with each hand assessed as an average of two trials with upper limit of 5 (300s) per trial. Patients unable to complete the task within this time are coded as 777 The outcome was assessed at month -12, -6, and 0 for the baseline phase and at month 0, 6, 12, 18, and 24 for the treatment phase. The progression rate was calculated as a difference between baseline and treatment slopes using a piecewise linear mixed-effect model with breaking point at month 0." (NCT00950248)
Timeframe: 1-year pre-treatment baseline vs 2-year treatment period

Change in Slopes of EDSS From Baseline to Treatment Phase

EDSS scale combines various elements of neurological exam. EDSS is a discrete scale ranging from 0 to 10 with 0.5 point increments. EDSS of 0 means no neurological disability, while EDSS of 10 marks death due to MS. EDSS was assessed at month -12, -6, and 0 for the baseline phase and at month 0, 6, 12, 18, and 24 for the treatment phase.The progression rate was calculated as a difference between baseline and treatment slopes using a piecewise linear mixed-effect model with breaking point at month 0. (NCT00950248)
Timeframe: 1-year pre-treatment baseline vs 2-year treatment period

Change in Slopes of SNRS From Baseline to Treatment Phase on

SNRS scale combines various elements of a neurological exam into a single number. The scale ranges from 100 to 0, where 100 marks no disability and 0 marks maximum disability. SNRS was assessed at month -12, -6, and 0 for the baseline phase and at month 0, 6, 12, 18, and 24 for the treatment phase. The progression rate was calculated as a difference between baseline and treatment slopes using a piecewise linear mixed-effect model with breaking point at month 0. (NCT00950248)
Timeframe: 1-year pre-treatment baseline vs 2-year treatment period

Change in the Area Under the Curve (AUC) of the Combinatorial Weight-Adjusted Disability Score (CombiWISE) From Baseline to Treatment Phase

"The AUCs of the CombiWISE scores during the 2-year treatment period was analyzed using an Analysis of Covariance (ANCOVA) model with the AUC of the pre-treatment CombiWISE scores, Baseline (Month 0) CombiWISE score and Baseline age as covariates.~CombiWISE is a composite scale derived from Expanded Disability Status Scale (EDSS) , Scripps Neurological Disability Scale (SNRS), times 25 foot walk (25FW), and non-dominant hand of 9 hole peg test (9HPT) with a minimum value of 0 (no disability) and maximum value of 100 (maximum disability).~The AUC values were calculated for both the pre-treatment baseline phase (from Months -12, -6, and 0) and for the double-blind phase (from Months 0, 6, 12, 18, and 24).~Because the follow-up times varied from patient to patient, the AUC values were made comparable by scaling them by dividing the AUC value by the square of the actual duration (in years) of each of the phases." (NCT00950248)
Timeframe: 1-year pre-treatment baseline vs 2-year treatment period

Change in the AUC of Individualized Rates of Enlargement of Ventricular Volume From Baseline to Treatment Phase

"The AUCs of the Ventricular volume scores (individualized rates of enlargement of segmented volume of lateral and 3rd ventricles) during the baseline and the 2-year treatment period were assessed using an ANCOVA model with the AUC of the pre-treatment Volumetric score, Baseline (Month 0) Volumetric score, and group as covariates.~The AUC values were calculated for both the pre-treatment baseline phase (from Months -12, -6, and 0) and for the double-blind phase (from Months 0, 6, 12, 18 and 24).~Because the follow-up times varied from patient to patient, the AUC values were made comparable by scaling them by dividing the AUC value by the square of the actual duration (in years) of each of the phases." (NCT00950248)
Timeframe: 1-year pre-treatment baseline vs 2-year treatment period

Disability Progression Measured by EDSS-plus

"Categorical time-to-event endpoints (EDSS-plus) were analyzed using Cox Proportional hazards models, with treatment group as a covariate. The EDSS-plus event was defined as disability progression on at least 1 of 3 components [EDSS, 25FW, and/or non-dominant hand 9HPT]) confirmed 6 months apart and with a ≥ 20% minimum threshold change for 25FW and non-dominant hand 9HPT).~The patients who did not have an event during the study were censored at the time of the last assessment of EDSS-plus. The number of months from the date of first dose to date of event or censoring were used as endpoint. The measure is time to disease progression and unit of this measure is months." (NCT00950248)
Timeframe: 2-year treatment period

Reviews

15 reviews available for idebenone and Friedreich Disease

Trials

19 trials available for idebenone and Friedreich Disease

Other Studies

31 other studies available for idebenone and Friedreich Disease