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Trial record 6 of 106 for:    Pompe Disease

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

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Pompe Disease
Interventions Drug: Albuterol
Drug: Placebo
Enrollment 16
Recruitment Details  
Pre-assignment Details Three enrolled participants decided to withdraw due to travel difficulties.
Arm/Group Title Albuterol Placebo Comparator
Hide Arm/Group Description 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. Placebo administered
Period Title: Overall Study
Started 8 5
Completed 7 5
Not Completed 1 0
Reason Not Completed
Withdrawal by Subject             1             0
Arm/Group Title Albuterol Placebo Comparator Total
Hide Arm/Group Description 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. Placebo administered Total of all reporting groups
Overall Number of Baseline Participants 8 5 13
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants 5 participants 13 participants
47.3  (9.7) 53.4  (9.9) 49.6  (9.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 5 participants 13 participants
Female
6
  75.0%
4
  80.0%
10
  76.9%
Male
2
  25.0%
1
  20.0%
3
  23.1%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 5 participants 13 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
8
 100.0%
5
 100.0%
13
 100.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 5 participants 13 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
8
 100.0%
5
 100.0%
13
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 8 participants 5 participants 13 participants
8
 100.0%
5
 100.0%
13
 100.0%
1.Primary Outcome
Title Number of Participants With Adverse Events.
Hide Description All participants who experienced adverse events.
Time Frame 52 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Albuterol Placebo Comparator
Hide Arm/Group Description:
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.
Placebo administered
Overall Number of Participants Analyzed 8 5
Measure Type: Count of Participants
Unit of Measure: Participants
5
  62.5%
5
 100.0%
2.Secondary Outcome
Title Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.
Hide Description FVC (forced vital capacity) is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible.
Time Frame Baseline, Week 30, and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Albuterol Placebo Comparator
Hide Arm/Group Description:
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.
Placebo administered
Overall Number of Participants Analyzed 7 5
Mean (Standard Deviation)
Unit of Measure: Percent of predicted FVC
Change at 30 Weeks Number Analyzed 7 participants 5 participants
-0.2  (5.7) 0.4  (7.9)
Change at 52 Weeks Number Analyzed 3 participants 2 participants
-1.3  (6.2) 3.0  (5.7)
3.Secondary Outcome
Title Change in 6 Minute Walk Test
Hide Description 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.
Time Frame Baseline, Week 6, and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Albuterol Placebo Comparator
Hide Arm/Group Description:
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.
Placebo administered
Overall Number of Participants Analyzed 3 2
Mean (Standard Deviation)
Unit of Measure: meters
Change at 6 Weeks 24.0  (10.3) 32.0  (58.1)
Change at 52 Weeks 43.6  (26.0) 13.6  (0.9)
Time Frame 52 Weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Albuterol Placebo Comparator
Hide Arm/Group Description 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. Placebo administered
All-Cause Mortality
Albuterol Placebo Comparator
Affected / at Risk (%) Affected / at Risk (%)
Total   0/8 (0.00%)   0/5 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Albuterol Placebo Comparator
Affected / at Risk (%) Affected / at Risk (%)
Total   0/8 (0.00%)   0/5 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Albuterol Placebo Comparator
Affected / at Risk (%) Affected / at Risk (%)
Total   5/8 (62.50%)   5/5 (100.00%) 
Cardiac disorders     
Ankle edema increase *  0/8 (0.00%)  1/5 (20.00%) 
Hypertension *  0/8 (0.00%)  1/5 (20.00%) 
Palpitations *  3/8 (37.50%)  2/5 (40.00%) 
Tachycardia *  4/8 (50.00%)  0/5 (0.00%) 
Ear and labyrinth disorders     
Otitis *  2/8 (25.00%)  0/5 (0.00%) 
Eye disorders     
Dry eyes *  1/8 (12.50%)  0/5 (0.00%) 
Gastrointestinal disorders     
Abdominal pain *  1/8 (12.50%)  1/5 (20.00%) 
Diarrhea *  1/8 (12.50%)  1/5 (20.00%) 
Gastritis *  1/8 (12.50%)  0/5 (0.00%) 
General disorders     
Chills *  0/8 (0.00%)  1/5 (20.00%) 
Energy decrease *  0/8 (0.00%)  1/5 (20.00%) 
Fall *  1/8 (12.50%)  4/5 (80.00%) 
Fatigue *  2/8 (25.00%)  0/5 (0.00%) 
Fever *  1/8 (12.50%)  0/5 (0.00%) 
Insomnia *  5/8 (62.50%)  1/5 (20.00%) 
Shortness of breath *  1/8 (12.50%)  0/5 (0.00%) 
Sneezing *  0/8 (0.00%)  1/5 (20.00%) 
Tremors *  2/8 (25.00%)  0/5 (0.00%) 
Weight gain *  1/8 (12.50%)  0/5 (0.00%) 
Weight loss *  1/8 (12.50%)  1/5 (20.00%) 
Musculoskeletal and connective tissue disorders     
Body aches *  0/8 (0.00%)  2/5 (40.00%) 
Body cramps *  1/8 (12.50%)  0/5 (0.00%) 
Chest tightness *  1/8 (12.50%)  0/5 (0.00%) 
Foot cramps *  3/8 (37.50%)  0/5 (0.00%) 
Hand cramps *  1/8 (12.50%)  0/5 (0.00%) 
Jaw cramps *  2/8 (25.00%)  0/5 (0.00%) 
Leg cramps *  4/8 (50.00%)  1/5 (20.00%) 
Throat cramps *  1/8 (12.50%)  0/5 (0.00%) 
Nervous system disorders     
Brain fog *  1/8 (12.50%)  0/5 (0.00%) 
Headache *  5/8 (62.50%)  1/5 (20.00%) 
Pain *  3/8 (37.50%)  5/5 (100.00%) 
Peripheral neuropathy increase *  1/8 (12.50%)  1/5 (20.00%) 
Transient global amnesia *  0/8 (0.00%)  1/5 (20.00%) 
Psychiatric disorders     
Anxiety *  0/8 (0.00%)  1/5 (20.00%) 
Renal and urinary disorders     
Urination increase *  3/8 (37.50%)  0/5 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough *  1/8 (12.50%)  0/5 (0.00%) 
Dry mucous membranes *  0/8 (0.00%)  1/5 (20.00%) 
Rhinorrhea *  1/8 (12.50%)  0/5 (0.00%) 
Sinus infection *  0/8 (0.00%)  1/5 (20.00%) 
Sore throat *  0/8 (0.00%)  2/5 (40.00%) 
Skin and subcutaneous tissue disorders     
Rash *  1/8 (12.50%)  1/5 (20.00%) 
*
Indicates events were collected by non-systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dwight Koeberl, M.D., Ph.D.
Organization: Duke University
Phone: 919-681-9919
EMail: dwight.koeberl@duke.edu
Layout table for additonal information
Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT01885936     History of Changes
Other Study ID Numbers: Pro00046020
First Submitted: June 21, 2013
First Posted: June 25, 2013
Results First Submitted: December 8, 2017
Results First Posted: February 6, 2018
Last Update Posted: July 15, 2019