clenbuterol and Glycogen Storage Disease Type II

clenbuterol has been researched along with Glycogen Storage Disease Type II in 6 studies

Clenbuterol: A substituted phenylaminoethanol that has beta-2 adrenomimetic properties at very low doses. It is used as a bronchodilator in asthma.
clenbuterol : A substituted aniline that is 2,6-dichloroaniline in which the hydrogen at position 4 has been replaced by a 2-(tert-butylamino)-1-hydroxyethyl group.

Glycogen Storage Disease Type II: An autosomal recessively inherited glycogen storage disease caused by GLUCAN 1,4-ALPHA-GLUCOSIDASE deficiency. Large amounts of GLYCOGEN accumulate in the LYSOSOMES of skeletal muscle (MUSCLE, SKELETAL); HEART; LIVER; SPINAL CORD; and BRAIN. Three forms have been described: infantile, childhood, and adult. The infantile form is fatal in infancy and presents with hypotonia and a hypertrophic cardiomyopathy (CARDIOMYOPATHY, HYPERTROPHIC). The childhood form usually presents in the second year of life with proximal weakness and respiratory symptoms. The adult form consists of a slowly progressive proximal myopathy. (From Muscle Nerve 1995;3:S61-9; Menkes, Textbook of Child Neurology, 5th ed, pp73-4)

Research

Studies (6)

Authors

Clinical Trials (3)

Trial Overview

Trial Outcomes

Change in 6 Minute Walk Test

Assess exercise tolerance in study patients; test administered by physical therapist. Subjects were asked to walk for 6 minutes, unassisted. The distance walked was recorded in meters. (NCT01942590)
Timeframe: Baseline, week 18

Change in 6 Minute Walk Test

Assess exercise tolerance in study patients; test administered by physical therapist. Subjects were asked to walk for 6 minutes, unassisted. The distance walked was recorded in meters. (NCT01942590)
Timeframe: Baseline, week 52

Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing

Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test. (NCT01942590)
Timeframe: Baseline, Week 18

Change in Forced Vital Capacity (FVC) in Pulmonary Function Testing

Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test. (NCT01942590)
Timeframe: Baseline, Week 52

Change in Urinary Glc4 Biomarker

(NCT01942590)
Timeframe: Baseline, Week 52

Change in Urinary Glc4 Biomarker

The Glc4 biomarker is measured in urine and correlates with muscle glycogen content. It is a noninvasive measurement that serves as a biomarker for Pompe disease. (NCT01942590)
Timeframe: Baseline, Week 18

Number of Participants With a Change in Aspartate Aminotransferase (AST), Alanine Transaminase (ALT), and Bilirubin Representing Liver Toxicity

Liver toxicity, as defined by a >3x increase in AST or ALT from the respective baseline values and/or an increase in direct, indirect or total bilirubin of >3x the upper limit of normal (NCT01942590)
Timeframe: Any point up to week 52

Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement

Worsening muscle involvement, as defined by >3x increase in CK from baseline that is >2x the upper limit of normal (NCT01942590)
Timeframe: Any point up to week 52

GSGC (Gait, Stairs, Gowers, Arising From a Chair.)

The GSGC is a criterion referenced assessment designed to measure functional status and change in gross motor function over time and, in particular, to measure clinically relevant change. Consists of 4 components: Gait, Climbing Stairs, Gower's Manuever, Arising From a Chair. Lowest score 4 = normal muscle function, highest score 27 = unable to perform motor function tests. (NCT01942590)
Timeframe: Baseline, Week 18, and Week 52

Late-Life Function and Disability Instrument (LLFDI)

The Late-Life Function & Disability Instrument (Late-Life FDI) is an evaluative outcome instrument for community-dwelling older adults. Highest score 240 = normal function and no disability, lowest score 0 = low levels of frequency of participating in life tasks. (NCT01942590)
Timeframe: Baseline, Week 18, Week 52

Maximum Expiratory Pressure (MEP)

MEP reflects the strength of the abdominal muscles and other expiratory muscles. (NCT01942590)
Timeframe: Baseline, Week 18, and Week 52

Predicted Maximum Inspiration Pressure (MIP)

MIP is a measurement of inspiratory muscle weakness, including weakness of the diaphragm. MIP is decreased in Pompe disease and reflects weakness of respiratory muscles. (NCT01942590)
Timeframe: Baseline, Week 18, and Week 52

Quick Motor Function Test (QMFT)

The QMFT is a criterion referenced assessment designed to measure functional status and change in gross motor function over time and, in particular, to measure clinically relevant change. Consists of 16 motor function tests. Lowest score 0 = unable to perform motor function tests, highest score 64 = normal muscle function. (NCT01942590)
Timeframe: Baseline, Week 18, and Week 52

Number of Participants With Adverse Events.

All participants who experienced adverse events. (NCT01885936)
Timeframe: 52 weeks

Change in 6 Minute Walk Test

The distance covered over a time of 6 minutes is used as the outcome by which to compare changes in performance capacity. Assessed by physical therapist. (NCT01885936)
Timeframe: Baseline, Week 6, and Week 52

Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.

FVC (forced vital capacity) is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. (NCT01885936)
Timeframe: Baseline, Week 30, and Week 52

Trials

1 trial available for clenbuterol and Glycogen Storage Disease Type II

Other Studies

5 other studies available for clenbuterol and Glycogen Storage Disease Type II