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hasn't got the funding
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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| clenbuterol | Experimental | The initial dose of clenbuterol will be 40 mcg per oral each morning for one week, followed by 40 mcg twice per day (BID) for the next 5 weeks until Week 6. If the 40 mcg BID per oral is well tolerated, the dose will be increased to 80 mcg each morning/40 mcg each evening for one week, followed by 80 mcg BID for the next 5 weeks until the Week 12 visit. If 80 mcg BID is tolerated at Week 12, the subject will continue on that dose until Week 52. |
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| placebo | Placebo Comparator | Initially, one capsule each morning for one week, followed by one capsule BID for the next 5 weeks until Week 6. If tolerated, the dose will be increased to two capsules each morning and 1 capsule each evening for one week, followed by two capsules BID for the next 5 weeks until the Week 12 visit. If two capsules BID is tolerated at Week 12, the subject will continue on that dose until Week 52. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Clenbuterol | Drug | 20 mcg spiropent tablets will be overencapsulated (two 20 mcg tablets per capsule) |
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| Measure | Description | Time Frame |
|---|---|---|
| Changes in 6 minute walk test (MWT) distance | Baseline (week 0) through 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Changes in pulmonary function tests: forced expiratory volume in 1 second (FEV1) | Baseline (week 0) through 52 weeks | |
| Changes in pulmonary function tests: forced vital capacity (FVC) | Baseline (week 0) through 52 weeks |
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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:
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| Name | Affiliation | Role |
|---|---|---|
| Dwight Koeberl, MD, PhD | Duke University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
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| ID | Term |
|---|---|
| D006009 | Glycogen Storage Disease Type II |
| ID | Term |
|---|---|
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
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| ID | Term |
|---|---|
| D002976 | Clenbuterol |
| ID | Term |
|---|---|
| D004983 | Ethanolamines |
| D000605 | Amino Alcohols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
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| Placebos | Drug | dextrose-filled capsules |
|
| Changes in pulmonary function tests: maximum expiratory pressure (MEP) | Baseline (week 0) through 52 weeks |
| Changes in pulmonary function tests: Maximum inspiratory pressure (MIP) | Baseline (week 0) through 52 weeks |
| Changes in graded functional test: Gait, Stairs, Gower, Chair (GSCS) | Baseline (week 0) through 52 weeks |
| Changes in graded functional test: Quick Motor Function Test (QMFT) | Baseline (week 0) through 52 weeks |
| Changes in the concentration of alanine transaminase (ALT) in serum | Avoidance of liver toxicity as defined by a a persistent (sustained >2 weeks) >3x increase in aspartate aminotransferase (AST) or alanine aminotransferase (ALT) from the respective baseline values and/or an increase in direct, indirect or total bilirubin of >3x the upper limit of normal (no liver toxicity has been reported in association with clenbuterol administration and therefore repeat testing should be acceptable) | Baseline (week 0) through 52 weeks |
| Changes in the concentration of creatine kinase (CK) in serum | Avoidance of Worsening muscle involvement (i.e. muscle weakness, cramping, or fatigue) accompanied by a persistent (sustained >2 weeks) >3x increase in CK from baseline that is >2x the upper limit of normal (elevated CK is associated with LOPD and minor elevations of CK have been reported in association with clenbuterol administration, therefore repeat testing should be acceptable). | Baseline (week 0) through 52 weeks |
| Changes in the concentration of urinary glucose tetramer (Glc4) | Baseline (week 0) through 52 weeks |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000588 |
| Amines |