Status and phase
Conditions
Treatments
About
The goals of this study are to determine safety and efficacy with regard to motor function of oral clenbuterol in combination with ERT in subjects with LOPD
Sex
Ages
Volunteers
Inclusion criteria
Exclusion criteria
Continuous invasive ventilation (via tracheostomy or endotracheal tube)
6MWT distance >90% of expected performance (% expected)
FVC >90% of expected (upright).
Clinically relevant illness within two weeks of enrollment including fever > 38.2o C, vomiting more than once in 24 hours, seizure, or other symptom deemed contraindicative to new therapy.
Chronic heart disease (Myocardial infarction, arrythmia, cardiomyopathy)
Tachycardia
History of seizure disorder
Hyperthyroidism
Pheochromocytoma
Pregnancy
History of diabetes
History of hypersensitivity to β2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent),
Patients on a non-standard schedule for ERT; for example, weekly infusions as opposed to infusions every two weeks.
Treatment for asthma in the previous 12 months.
Renal insufficiency (elevated serum creatinine).
Having started respiratory muscle strength training in the last 6 months prior to study day 1 or having discontinued respiratory muscle strength training in the 6-month period preceding study day 1, or having started respiratory strength training greater than 6 months prior to study day 1 and unwilling to continue for the first year of study participation.
Received an investigational drug or participated in another interventional study within 90 days of Study Day 1.
Anti-rhGAA IgG with sustained titer >1:25.600 for >6 months at time of enrollment.
The use of the following concommitant meds is prohibited during the study:
Primary purpose
Allocation
Interventional model
Masking
0 participants in 2 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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