Dettagli per singolo studio

DEC-NET Serial number GB285

Published online	09/07/2004 16.51.00
Last updated	02/08/2005 14.24.45

Other protocol ID number	N0016131960
This trial has been approved by an ethics committee
Current trial status	Closed to recruitment of participants: follow-up continuing
First subject enrolment Target N. of subjects	11/2001 14
Major Disease (ICD9 class)	CONGENITAL HEREDITARY MUSCULAR DYSTROPHY
Experimental drug
SALBUTAMOL Treatment regimen (dosage and duration) N/A
Gender	Both
Age (range)	5-20 years
Eligibility criteria
Inclusion criteria
10 male, 4 female, ages 5-20 years. Patients diagnosed with Myotubular myopathy
Exclusion criteria
Patients receiving salbutamol for other reasons informed consent not given
Trial design/methodology
Phase	4
Kind of study	Efficacy
Design	Open pilot trial
Purpose of study
To test the hypothesis that treatment with salbutamol, which has already been successfully used in other neuromuscular diseases, might be beneficial to patients with myotubular myopathy.
Primary outcomes
If the pilot study is encouraging, a multicentre larger study will be proposed to fully evaluate the role of these compounds in Myotubular myopathy.


Summary of study design, objectives, and ongoing research findings
Open pilot trial to test the hypothesis that treatment with salbutamol, which has already been successfully used in other neuromuscular diseases, might be beneficial to patients with myotubular myopathy.