Dettagli per singolo studio

DEC-NET Serial number GB286

Published online	09/07/2004 17.00.00
Last updated	02/08/2005 14.26.05

Other protocol ID number	N0016131958
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 15
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
7 males, 8 females, ages 5-20 years. patients with a diagnosis of nemaline 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 patinets with nemaline myopathy.
Primary outcomes
If the results are encouraging, a multicentre larger study will be proposed to fully evaluate the role of these compounds in Nemaline 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 nemaline myopathy.