Motor neuron disease characterized by atrophy and muscle weakness in the spinal and bulbar region MedDRA version: 20.1 Level: PT Classification code 10068597 Term: Bulbospinal muscular atrophy congenital System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: MONORES 20 mcg 30 compresse
Product Name: MONORES 20 mcg 30 compresse
Product Code: [024217034]
Pharmaceutical Form: Tablet
INN or Proposed INN: CLENBUTEROLO
Current Sponsor code: nessuno
Sponsors
AZIENDA OSPEDALIERA DI PADOVA
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: For inclusion in the study, subjects must fulfill ALL of the following criteria: 1) males who have received a genetically confirmed diagnosis of SBMA (AR CAG repeat number > 38); 2) aged between 18 and 75 (+364 days) years; 3) displaying one or more of the following clinical symptoms: muscle atrophy, limb weakness, bulbar palsy; 4) able to walk independently with or without a cane or other supporting device (all supporting devices are acceptable except on wheelchair); 5) providing a 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 60 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 30
Exclusion criteria
Exclusion criteria: The subject may not enter or continue in the study if ANY of the following apply at any study visit prior to randomization: 1) a documented cardiovascular disease precluding the use of beta2 agonists; 2) glaucoma, severe prostatic hypertrophy, hyperthyroidism, pheochromocytoma, and other medical conditions that, in the judgment of the investigators, would expose the patient to undue risk of harm or prevent the patient from completing the study; 3) concomitant treatment with either beta-blockers or sympathomimetic drugs; 4) inability to walk or walking only with the support of a caregiver; 5) use of beta2 agonists in the preceding 6 months; 6) participation to a interventional trial in the preceding 3 months. 7) neuromuscular disease other than SBMA
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluate the effect of Beta2-agonist stimulation on muscle as a therapeutic strategy for SBMA;Secondary Objective: The secondary objectives include the assessment of the persistence of the effect (if any) after prolonged treatment and the tolerability of the treatment in terms of adverse effects emerging from the treatment;Primary end point(s): The primary end point is calculated as a 15% increase in the distance traveled in six minutes (6MWT test) at 12 months.;Timepoint(s) of evaluation of this end point: 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1547 / 5000 Risultati della traduzione 1. mean change in 6MWT test score over 12 months of treatment (V1-V7) in the two therapeutic arms; 2. mean change in SBMA-FRS total score during 12 months of treatment (V1-V7) in the two therapeutic arms; 3. mean change in the total score on the AMAT scale during the 12 months of treatment (V1-V7) in the two therapeutic arms; 4. mean change in FVC over 12 months of treatment (V1-V7) in the two therapeutic arms; 5. mean change in the 6K total score during 12 months of treatment (V1-V7) in the two therapeutic arms; 6. mean change in serum creatinine levels over 12 months of treatment (V1-V7) in the two therapeutic arms; 7. mean change in ALSAQ-40 total score during 12 months of treatment (V1-V7) in the two therapeutic arms; 8. mean change in total score on the INQOL scale over 12 months of treatment (V1-V7) in the two therapeutic arms; 9. Percentage of patients experiencing adverse events and serious adverse events in the two therapeutic arms at V7; 10. percentage of patients experiencing side effects involving the cardiovascular system in the two therapeutic arms at V7; 11. percentage of patients with edema (identified by muscle MRI) in the two therapeutic arms at V7; 12. degree of atrophy (measured by muscle MRI) in the two therapeutic arms at V7; 13. degree of substitution of adipose fiber (measured by muscle MRI) in the two therapeutic arms at V7.;Timepoint(s) of evaluation of this end point: 12 months | — |
Countries
Italy
Contacts
Public ContactNucleo Ricerca Clinica
Azienda Ospedale Università di Padova
Outcome results
None listed