Patients affected by SMA MedDRA version: 9.1 Level: LLT Classification code 10041582 Term: Spinal muscular atrophy
Conditions
Interventions
Trade Name: VOLMAX*30CPR 4MG R.P.
Pharmaceutical Form: Modified-release tablet
INN or Proposed INN: Salbutamol
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 4-
Ph
Sponsors
ISTITUTO NEUROLOGICO "CARLO BESTA"
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: male or female out-patients; clinical diagnosis of SMA III, confirmed by molecular analysis; range of age between 18-50 years; females should be either of non-childbearing potential (surgical sterilization or postmenopausal) or of childbearing potential. Females of childbearing potential must agree to be astinent or to use at least one medically acceptable method of contraception; patients able to understand and give the informed consent and to perform evaluation tests; absence of contra-indications to the use of salbutamol; 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) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: severe respiratory failure, asthma, cardiovascular disorders (especially coronary insufficiency, hypertension, and cardiac arrhythmia) convulsive disorders, hyperthyroidism, diabetes mellitus, hepatic or renal disorders; concomitant treatment with thiazide diuretics, monoamine oxidase inhibitors or tricyclic antidepressants, or within 2 weeks of discontinuation of such agents; use of beta2-stimulants or other drugs known to induce SMN2 up-regulation in vitro or in pilot open label study within 6 months before the study; pregnant or lactating women; 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: the primary objective of this study is to evaluate the effect on SMN2 transcripts of salbutamol administered at the daily oral dose of 12 mg for one year, as add on therapy, in patients affected by SMA type III, often referred to as Kugelberg-Welander or Juvenile Spinal Muscular Atrophy, in comparison with placebo under double blind conditions.Safety will be assessed for all subjects, for the entire duration of the study.;Secondary Objective: Secondary objective will be to evaluate the therapeutic efficacy of salbutamol.;Primary end point(s): The primary efficacy endpoint will be the number of responding patients (responders), defined as patients which after one year of treatment show an increment of SMN2 transcripts of at least 90%. | — |
Countries
Italy
Outcome results
None listed