sorbitol and Atrophy, Muscular, Peroneal

sorbitol has been researched along with Atrophy, Muscular, Peroneal in 7 studies

D-glucitol : The D-enantiomer of glucitol (also known as D-sorbitol).

Research

Studies (7)

Authors

Clinical Trials (2)

Trial Overview

Trial Outcomes

Number of Participants With ONLS Therapy Response 1

ONLS Therapy Response 1 was defined as the number of participants (responders) with an improvement on final ONLS Total Score of at least one point. A higher response rate indicate a better clinical condition. (NCT02579759)
Timeframe: From Baseline to Month 15

Number of Participants With ONLS Therapy Response 2

"ONLS Therapy Response 2 was defined as the number of participants with no deterioration (responders) on final ONLS Total Score.~A higher response rate indicates a better clinical condition." (NCT02579759)
Timeframe: From Baseline to Month 15

Incidence of AE Leading to Withdrawal of Study Drug

Safety and tolerability of PXT3003 were compared to placebo on the incidence of TEAEs leading to withdrawal of study drug. (NCT02579759)
Timeframe: The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months)

Incidence of SAEs

Safety and tolerability of PXT3003 were compared to placebo on the incidence of serious adverse events (SAEs). (NCT02579759)
Timeframe: The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months).

Mean of Ten Meter Walking Test (10MWT)

"This outcome measure is the mean of the available 10MWT values at month 12 and month 15.~The 10MWT is a simple to administer, standardized, reliable and valid evaluation of functional exercise capacity and gait that has been used to evaluate neurologic disorders and CMT patients.~Lower Time to Walk 10 Meters values indicate a better clinical condition.~Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin)." (NCT02579759)
Timeframe: From Baseline to Month 15

Mean of the CMTNS-v2 Examination Score (CMTES-v2)

"This outcome measure is the mean of the available CMTNS-v2 Examination Score values at month 12 and month 15.~The CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.~The CMTES-v2 is summed of item 1 to 7 of the CMTNS-v2 (limited to impairment items and excluding electrophysiological items). It is a 28-point score: 0 (no impairment) to 28 (maximum impairment).~Lower CMTES-v2 values indicate a better clinical condition.~Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin)." (NCT02579759)
Timeframe: From Baseline to Month 15

Mean of the CMTNS-v2 Sensory Score

"This outcome measure is the mean of the available CMTNS-v2 Sensory Score values at month 12 and month 15.~The CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.~The CMTNS-v2 Sensory score is summed of items 1+4+5 of CMTNS-v2 (Sensory symptoms, Pinprick sensibility and Vibration). It is a 12-point score: 0 (no impairment) to 12 (maximum impairment).~Lower CMTNS-v2 Sensory Score values indicate a better clinical condition.~Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin)." (NCT02579759)
Timeframe: From Baseline to Month 15

Mean of the CMTNS-v2 Sensory Symptoms

"This outcome measure is the mean of the available CMTNS-v2 Sensory Symptoms values at month 12 and month 15.~The CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.~The CMTNS-v2 Sensory Symptoms is the first item of the CMTNS-v2. It is a 4-point score: 0 (no impairment) to 4 (maximum impairment).~Lower CMTNS-v2 Sensory Symptoms values indicate a better clinical condition.~Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin)." (NCT02579759)
Timeframe: From Baseline to Month 15

Mean of the Results at the Nine-Hole Peg Test (9-HPT)

"This outcome measure is the mean of the available 9-HPT values at month 12 and month 15.~The Nine-Hole Peg Test (9HPT) is a simple timed test of fine motor coordination of extremitied in the upper limbs. It measures the time needed by the patient to insert 9 pegs in nine holes and to remove them (normal required time 18 seconds).~Lower 9HPT values indicate a better clinical condition.~Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin)." (NCT02579759)
Timeframe: From Baseline to Month 15

Number of Subjects With at Least One TEAE

"Safety selection was to include all randomized patients that have received at least one dose of study treatment.~Safety and tolerability of PXT3003 were compared to placebo on the incidence of treatment-emergent adverse events (TEAEs); they were evaluated by type/nature, severity/intensity, seriousness, and relationship to study drug." (NCT02579759)
Timeframe: The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months)

Overall Neuropathy Limitation Scale (ONLS) Total Score

"The primary efficacy variable used in the main analysis is the mean of the available ONLS values at month 12 and month 15.~The ONLS is a disability scale that was derived and improved from the Overall Disability Sum Score (ODSS) to measure limitations in the everyday activities of the upper limbs (rated on 5 points) and the lower limbs (rated on 7 points). The total score is a 12-point scale: 0 (no disability) to 12 (maximum disability). Lower values in the ONLS indicate a better clinical condition.~Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin)." (NCT02579759)
Timeframe: From Baseline to Month 15

Plasma Concentrations of 6β-naltrexol at Trough and at 90 Min After Drug Intake

"Plasma concentration of PXT3003 components were measured at trough (prior to dose) and peak (90 minutes post dose).~The mean plasma values of the baseline correspond to half of the administered dose." (NCT02579759)
Timeframe: At Month 12 and Month 15

Plasma Concentrations of Baclofen at Trough and at 90 Min After Drug Intake

"Plasma concentration of PXT3003 components were measured at trough (prior to dose) and 90 minutes after drug intake.~The mean plasma values of the baseline correspond to half of the administered dose." (NCT02579759)
Timeframe: At Month 12 and Month 15

Plasma Concentrations of Naltrexone at Trough and at 90 Min After Drug Intake

"Plasma concentration of PXT3003 components were measured at trough (prior to dose) and 90 minutes after drug intake.~The mean plasma values of the baseline correspond to half of the administered dose." (NCT02579759)
Timeframe: At Month 12 and month 15

Safety and Tolerability of PXT3003

"The Primary Objective is to assess the clinical and laboratory safety and tolerability of three doses of PXT3003 administered orally for 12 months to CMT1A patients versus placebo.~Number of participants with adverse events in each arm." (NCT01401257)
Timeframe: Screening, randomization, 1-, 3-, 6-, 9-, 12-month treatment and 1-month follow-up

Trials

2 trials available for sorbitol and Atrophy, Muscular, Peroneal

Other Studies

5 other studies available for sorbitol and Atrophy, Muscular, Peroneal