Trial Outcomes & Findings for Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease (NCT NCT01885936)
NCT ID: NCT01885936
Last Updated: 2019-07-15
Results Overview
All participants who experienced adverse events.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
16 participants
Primary outcome timeframe
52 weeks
Results posted on
2019-07-15
Participant Flow
Three enrolled participants decided to withdraw due to travel difficulties.
Participant milestones
| Measure |
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.
|
Placebo Comparator
Placebo administered
|
|---|---|---|
|
Overall Study
STARTED
|
8
|
5
|
|
Overall Study
COMPLETED
|
7
|
5
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
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.
|
Placebo Comparator
Placebo administered
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
Baseline characteristics by cohort
| Measure |
Albuterol
n=8 Participants
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 Comparator
n=5 Participants
Placebo administered
|
Total
n=13 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
47.3 years
STANDARD_DEVIATION 9.7 • n=5 Participants
|
53.4 years
STANDARD_DEVIATION 9.9 • n=7 Participants
|
49.6 years
STANDARD_DEVIATION 9.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 52 weeksAll participants who experienced adverse events.
Outcome measures
| Measure |
Albuterol
n=8 Participants
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 Comparator
n=5 Participants
Placebo administered
|
|---|---|---|
|
Number of Participants With Adverse Events.
|
5 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 30, and Week 52Population: 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.
FVC (forced vital capacity) is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible.
Outcome measures
| Measure |
Albuterol
n=7 Participants
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 Comparator
n=5 Participants
Placebo administered
|
|---|---|---|
|
Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.
Change at 30 Weeks
|
-0.2 Percent of predicted FVC
Standard Deviation 5.7
|
0.4 Percent of predicted FVC
Standard Deviation 7.9
|
|
Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.
Change at 52 Weeks
|
-1.3 Percent of predicted FVC
Standard Deviation 6.2
|
3.0 Percent of predicted FVC
Standard Deviation 5.7
|
SECONDARY outcome
Timeframe: Baseline, Week 6, and Week 52Population: 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.
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.
Outcome measures
| Measure |
Albuterol
n=3 Participants
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 Comparator
n=2 Participants
Placebo administered
|
|---|---|---|
|
Change in 6 Minute Walk Test
Change at 6 Weeks
|
24.0 meters
Standard Deviation 10.3
|
32.0 meters
Standard Deviation 58.1
|
|
Change in 6 Minute Walk Test
Change at 52 Weeks
|
43.6 meters
Standard Deviation 26.0
|
13.6 meters
Standard Deviation 0.9
|
Adverse Events
Albuterol
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Placebo Comparator
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Albuterol
n=8 participants at risk
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 Comparator
n=5 participants at risk
Placebo administered
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
12.5%
1/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Cardiac disorders
Ankle edema increase
|
0.00%
0/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Musculoskeletal and connective tissue disorders
Body aches
|
0.00%
0/8 • 52 Weeks
|
40.0%
2/5 • 52 Weeks
|
|
Musculoskeletal and connective tissue disorders
Body cramps
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Nervous system disorders
Brain fog
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Musculoskeletal and connective tissue disorders
Chest tightness
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
General disorders
Chills
|
0.00%
0/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Gastrointestinal disorders
Diarrhea
|
12.5%
1/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Eye disorders
Dry eyes
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dry mucous membranes
|
0.00%
0/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
General disorders
Energy decrease
|
0.00%
0/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
General disorders
Fall
|
12.5%
1/8 • 52 Weeks
|
80.0%
4/5 • 52 Weeks
|
|
General disorders
Fatigue
|
25.0%
2/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
General disorders
Fever
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Musculoskeletal and connective tissue disorders
Foot cramps
|
37.5%
3/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Gastrointestinal disorders
Gastritis
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Musculoskeletal and connective tissue disorders
Hand cramps
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Nervous system disorders
Headache
|
62.5%
5/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Cardiac disorders
Hypertension
|
0.00%
0/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
General disorders
Insomnia
|
62.5%
5/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Musculoskeletal and connective tissue disorders
Jaw cramps
|
25.0%
2/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Musculoskeletal and connective tissue disorders
Leg cramps
|
50.0%
4/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Ear and labyrinth disorders
Otitis
|
25.0%
2/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Nervous system disorders
Pain
|
37.5%
3/8 • 52 Weeks
|
100.0%
5/5 • 52 Weeks
|
|
Cardiac disorders
Palpitations
|
37.5%
3/8 • 52 Weeks
|
40.0%
2/5 • 52 Weeks
|
|
Nervous system disorders
Peripheral neuropathy increase
|
12.5%
1/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
12.5%
1/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
General disorders
Shortness of breath
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sinus infection
|
0.00%
0/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
General disorders
Sneezing
|
0.00%
0/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
0.00%
0/8 • 52 Weeks
|
40.0%
2/5 • 52 Weeks
|
|
Cardiac disorders
Tachycardia
|
50.0%
4/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Musculoskeletal and connective tissue disorders
Throat cramps
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Nervous system disorders
Transient global amnesia
|
0.00%
0/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
|
General disorders
Tremors
|
25.0%
2/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
Renal and urinary disorders
Urination increase
|
37.5%
3/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
General disorders
Weight gain
|
12.5%
1/8 • 52 Weeks
|
0.00%
0/5 • 52 Weeks
|
|
General disorders
Weight loss
|
12.5%
1/8 • 52 Weeks
|
20.0%
1/5 • 52 Weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place