AMYOTROPHIC LATERAL SCLEROSIS MedDRA version: 9.1 Level: LLT Classification code 10002026 Term: Amyotrophic lateral sclerosis
Conditions
Interventions
Product Name: Tauroursodeoxycholic acid
Pharmaceutical Form: Prolonged-release capsule, hard
INN or Proposed INN: Tauroursodeoxycholic acid
Concentration unit: mg/g milligram(s)/gram
Concentration t
Sponsors
ISTITUTO NEUROLOGICO "CARLO BESTA"
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Caucasian male or female out-patients; aged 18 to 75 years inclusive; diagnosis of ?probable? or ?definite? amyotrophic lateral sclerosis according to the El Escorial revised criteria (1); experiencing disease progression in the last 3 months; first symptoms of ALS by no more than 1.5 years; in treatment with steady regimen of riluzole and vitamin E for a minimum of 3 months before study entry, and desiring its continuation; FVC ≥ 75% of predicted; no conditions known to be contraindications to the use of TUDCA; written informed consent. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: subjects who underwent tracheostomy; subjects who underwent resection of gall bladder; subjects with signs of conduction blocks of motor nerves, sensory nerves or both on nerve conduction study; subjects with clinical signs of dementia; subjects with active peptic ulcer; subjects with active malignancy; female subjects who are pregnant or lactating subjects who have received an experimental drug or have participated in a clinical trial within 3 months prior to screening employees of the investigator or study centre with direct involvement in the proposed study or other studies under the direction of that investigator or study centre.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess if the addition of TUDCA to the conventional therapy can improve the therapeutic outcome in patients affected by ALS (ALSFRS-R). Safety will be assessed for all subjects, for the entire duration of the study.;Secondary Objective: Secondary parameters will include FVC%, the SF-36 quality of life rating scale, Time to Tracheostomy from starting of study medication dosing (if appropriate), Survival Time from starting of study medication dosing (if appropriate) and the number of patients showing an improvement ≥ 15% in ALSFRS-R slope as compared to the lead-in period (responders).;Primary end point(s): Primary evaluation parameter will be the ALSFRS-R (2) according to the consensus conference on designing and implementing clinical trials in ALS (3). Primary endpoint will be the ALSFRS-R slope during the treatment period as compared to the lead-in period | — |
Countries
Italy
Outcome results
None listed