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In this study the study team proposes to investigate the efficacy of albuterol on motor function of individuals with Late Onset Pompe Disease (LOPD) who are receiving enzyme replacement therapy, given albuterol was well-tolerated in patients with Late Onset Pompe Disease.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Albuterol | Experimental | Initially 4 mg daily for one week, then 4 mg BID per oral daily for the next 5 weeks. If the 4 mg BID per oral is well tolerated, the dose will be increased to 8 mg each morning/4 mg each evening for one week, followed by 8 mg BID per oral for the remainder of the study. |
|
| Placebo Comparator | Placebo Comparator | Initially one capsule daily for one week, then one capsule BID per oral daily for the next 5 weeks. If the one capsule BID per oral is well tolerated, the dose will be increased to two capsules each morning/one capsule each evening for one week, followed by two capsules BID per oral for the remainder of the study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Albuterol | Drug | Initially 4 mg daily for one week, 4 mg BID per oral daily for the next 5 weeks. If the 4 mg BID per oral is well tolerated, the dose will be increased to 8 mg each morning/4 mg each evening for one week, followed by 8 mg BID per oral for the remainder of the study. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events. | All participants who experienced adverse events. | 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline, Week 30, and Week 52 |
| Change in 6 Minute Walk Test |
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Inclusion Criteria:
Exclusion Criteria:
Continuous invasive ventilation (via tracheostomy or endotracheal tube).
Clinically relevant illness within two weeks of enrollment including fever > 38.2 C, vomiting more than once in 24 hours, seizure, or other symptom deemed contraindicative to new therapy.
Chronic heart disease (Myocardial infarction in the past 2 months, arrhythmia, cardiomyopathy).
History of seizure disorder.
History of diabetes.
Hypokalemia.
History of hyperthyroidism.
Pregnancy.
Patients on a non-standard schedule for enzyme replacement therapy; for example, weekly infusions as opposed to infusions every two weeks.
Anti-rhGAA antibody titer > 1:100,000
History of hypersensitivity to Beta 2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent)..
The use of the following medications:
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| Name | Affiliation | Role |
|---|---|---|
| Dwight d Koeberl, MD, PhD | Duke University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22154081 | Background | Koeberl DD, Li S, Dai J, Thurberg BL, Bali D, Kishnani PS. beta2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease. Mol Genet Metab. 2012 Feb;105(2):221-7. doi: 10.1016/j.ymgme.2011.11.005. Epub 2011 Nov 11. | |
| 31839530 | Derived | Koeberl DD, Case LE, Desai A, Smith EC, Walters C, Han SO, Thurberg BL, Young SP, Bali D, Kishnani PS. Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease. Mol Genet Metab. 2020 Feb;129(2):67-72. doi: 10.1016/j.ymgme.2019.12.008. Epub 2019 Dec 10. |
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Three enrolled participants decided to withdraw due to travel difficulties.
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| ID | Title | Description |
|---|---|---|
| FG000 | Albuterol | Initially 4 mg daily for one week, 4 mg BID (twice a day) per oral for the next 5 weeks. If the 4 mg BID per oral is well tolerated, the dose will be increased to 8 mg each morning/4 mg each evening for one week, followed by 8 mg BID per oral for the remainder of the study. |
| FG001 | Placebo Comparator | Placebo administered |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Albuterol | Initially 4 mg daily for one week, 4 mg BID per oral daily for the next 5 weeks. If the 4 mg BID per oral is well tolerated, the dose will be increased to 8 mg each morning/4 mg each evening for one week, followed by 8 mg BID per oral for the remainder of the study. |
| BG001 | Placebo Comparator |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Adverse Events. | All participants who experienced adverse events. | Posted | Count of Participants | Participants | 52 weeks |
|
52 Weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Albuterol | Initially 4 mg daily for one week, 4 mg BID per oral daily for the next 5 weeks. If the 4 mg BID per oral is well tolerated, the dose will be increased to 8 mg each morning/4 mg each evening for one week, followed by 8 mg BID per oral for the remainder of the study. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dwight Koeberl, M.D., Ph.D. | Duke University | 919-681-9919 | dwight.koeberl@duke.edu |
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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 |
|---|---|
| D000420 | Albuterol |
| ID | Term |
|---|---|
| D004983 | Ethanolamines |
| D000605 | Amino Alcohols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
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|
| Placebo | Drug | Initially one capsule daily for one week, then one capsule BID per oral daily for the next 5 weeks. If the one capsule BID per oral is well tolerated, the dose will be increased to two capsules each morning/one capsule each evening for one week, followed by two capsules BID per oral for the remainder of the study. |
|
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. |
| Baseline, Week 6, and Week 52 |
Placebo administered |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| Units | Counts |
|---|
| Participants |
|
|
| Secondary | 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. | One subject dropped out from the albuterol group. Later participants were unblinded and switched to drug before Week 52 under an IRB (Institutional Review Board) approved amendment, and 4 late-enrolled drug and 3 late-enrolled placebo subjects could not be included in the analysis at Week 52. | Posted | Mean | Standard Deviation | Percent of predicted FVC | Baseline, Week 30, and Week 52 |
|
|
|
| Secondary | 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. | Early participants were not randomized until 6 weeks; 3 drug and 2 placebo subjects could not be included in the analysis. One drug subject missed the Week 6 visit, and one placebo subject could not perform the 6 minute walk test. Later participants were unblinded before Week 52. Four drug and 3 placebo subjects could not be included at Week 52. | Posted | Mean | Standard Deviation | meters | Baseline, Week 6, and Week 52 |
|
|
|
| 0 |
| 8 |
| 0 |
| 8 |
| 5 |
| 8 |
| EG001 | Placebo Comparator | Placebo administered | 0 | 5 | 0 | 5 | 5 | 5 |
| Ankle edema increase | Cardiac disorders | Non-systematic Assessment |
|
| Anxiety | Psychiatric disorders | Non-systematic Assessment |
|
| Body aches | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Body cramps | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Brain fog | Nervous system disorders | Non-systematic Assessment |
|
| Chest tightness | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Chills | General disorders | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | Non-systematic Assessment |
|
| Dry eyes | Eye disorders | Non-systematic Assessment |
|
| Dry mucous membranes | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Energy decrease | General disorders | Non-systematic Assessment |
|
| Fall | General disorders | Non-systematic Assessment |
|
| Fatigue | General disorders | Non-systematic Assessment |
|
| Fever | General disorders | Non-systematic Assessment |
|
| Foot cramps | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Gastritis | Gastrointestinal disorders | Non-systematic Assessment |
|
| Hand cramps | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Headache | Nervous system disorders | Non-systematic Assessment |
|
| Hypertension | Cardiac disorders | Non-systematic Assessment |
|
| Insomnia | General disorders | Non-systematic Assessment |
|
| Jaw cramps | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Leg cramps | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Otitis | Ear and labyrinth disorders | Non-systematic Assessment |
|
| Pain | Nervous system disorders | Non-systematic Assessment |
|
| Palpitations | Cardiac disorders | Non-systematic Assessment |
|
| Peripheral neuropathy increase | Nervous system disorders | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Shortness of breath | General disorders | Non-systematic Assessment |
|
| Sinus infection | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Sneezing | General disorders | Non-systematic Assessment |
|
| Sore throat | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Tachycardia | Cardiac disorders | Non-systematic Assessment |
|
| Throat cramps | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Transient global amnesia | Nervous system disorders | Non-systematic Assessment |
|
| Tremors | General disorders | Non-systematic Assessment |
|
| Urination increase | Renal and urinary disorders | Non-systematic Assessment |
|
| Weight gain | General disorders | Non-systematic Assessment |
|
| Weight loss | General disorders | Non-systematic Assessment |
|
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| 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 |
| D010627 | Phenethylamines |
| D005021 | Ethylamines |
| Change at 52 Weeks |
|
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