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Trial record 68 of 858 for:    ALBUTEROL

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01885936
Recruitment Status : Completed
First Posted : June 25, 2013
Results First Posted : February 6, 2018
Last Update Posted : July 15, 2019
Sponsor:
Information provided by (Responsible Party):
Duke University

Tracking Information
First Submitted Date  ICMJE June 21, 2013
First Posted Date  ICMJE June 25, 2013
Results First Submitted Date  ICMJE December 8, 2017
Results First Posted Date  ICMJE February 6, 2018
Last Update Posted Date July 15, 2019
Study Start Date  ICMJE June 2013
Actual Primary Completion Date December 16, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 10, 2018)
Number of Participants With Adverse Events. [ Time Frame: 52 weeks ]
All participants who experienced adverse events.
Original Primary Outcome Measures  ICMJE
 (submitted: June 21, 2013)
Number of Participants With Adverse Events. [ Time Frame: 52 weeks ]
Change History Complete list of historical versions of study NCT01885936 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 10, 2018)
  • Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks. [ Time Frame: Baseline, Week 30, and Week 52 ]
    FVC (forced vital capacity) is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible.
  • Change in 6 Minute Walk Test [ Time Frame: Baseline, Week 6, and Week 52 ]
    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.
Original Secondary Outcome Measures  ICMJE
 (submitted: June 21, 2013)
  • Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks. [ Time Frame: Baseline and weeks 30 and 52. ]
  • Change in 6 Minute Walk Test [ Time Frame: Weeks 6 and 52. ]
    Assessed by physical therapist
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
Official Title  ICMJE A Phase 1/2 Double-Blind Study of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement Therapy
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Pompe Disease
Intervention  ICMJE
  • Drug: 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.
  • Drug: Placebo
    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.
Study Arms  ICMJE
  • Experimental: Albuterol
    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.
    Intervention: Drug: Albuterol
  • 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.
    Intervention: Drug: Placebo
Publications * Koeberl DD, Li S, Dai J, Thurberg BL, Bali D, Kishnani PS. β2 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 22, 2016)
16
Original Estimated Enrollment  ICMJE
 (submitted: June 21, 2013)
20
Actual Study Completion Date  ICMJE December 16, 2016
Actual Primary Completion Date December 16, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Diagnosis of Pompe disease by blood acid alpha-glucosidase assay and acid alpha-glucosidase gene sequencing,
  2. Age: 18+ years at enrollment.
  3. Receiving enzyme replacement therapy at standard dose (20 mg/kg every 2 weeks) for at least 52 weeks.
  4. Subjects are capable of giving written consent.

Exclusion Criteria:

  1. Continuous invasive ventilation (via tracheostomy or endotracheal tube).
  2. 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.
  3. Chronic heart disease (Myocardial infarction in the past 2 months, arrhythmia, cardiomyopathy).
  4. History of seizure disorder.
  5. History of diabetes.
  6. Hypokalemia.
  7. History of hyperthyroidism.
  8. Pregnancy.
  9. Patients on a non-standard schedule for enzyme replacement therapy; for example, weekly infusions as opposed to infusions every two weeks.
  10. Anti-rhGAA antibody titer > 1:100,000
  11. History of hypersensitivity to Beta 2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent)..
  12. The use of the following medications:

    • diuretics (water pill);
    • digoxin (digitalis, Lanoxin);
    • beta-blockers such as atenolol (Tenormin), metoprolol (Lopressor), and propranolol (Inderal);
    • tricyclic antidepressants such as amitriptyline (Elavil, Etrafon), doxepin (Sinequan), imipramine (Janimine, Tofranil), and nortriptyline (Pamelor);
    • Monoamine oxidase inhibitors such as isocarboxazid (Marplan), phenelzine (Nardil), rasagiline (Azilect), selegiline (Eldepryl, Emsam), or tranylcypromine (Parnate); or
    • bronchodilators such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline (Brethine, Bricanyl), salmeterol (Serevent), isoetharine (Bronkometer), metaproterenol (Alupent, Metaprel), or isoproterenol (Isuprel Mistometer) within 12 weeks prior to enrollment.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01885936
Other Study ID Numbers  ICMJE Pro00046020
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Duke University
Study Sponsor  ICMJE Duke University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Dwight d Koeberl, MD, PhD Duke University
PRS Account Duke University
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP