Trial · NCT01885936

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

NCT01885936, Phase 1, Phase 2, Completed, Duke University; results first posted 2018-02-06

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease. Trials, record NCT01885936. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01885936

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
NCT number: NCT01885936
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 16 participants, actual
Lead sponsor: Duke University
Conditions: Pompe Disease
Interventions: Albuterol, Placebo
First posted: 2013-06-25
Last updated: 2019-07-15
Results first posted: 2018-02-06

Source ClinicalTrials.gov

Registered outcomes3

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Adverse Events.	52 weeks	All participants who experienced adverse events.
Secondary	Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.	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.
Secondary	Change in 6 Minute Walk Test	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.

Source ClinicalTrials.gov

Outcomes3

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Adverse Events.	52 weeks		Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.	Baseline, Week 30, and Week 52	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.	Percent of predicted FVC	MEAN
SECONDARY	Change in 6 Minute Walk Test	Baseline, Week 6, and Week 52	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.	meters	MEAN

Source ClinicalTrials.gov

Results by group10

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Number of Participants With Adverse Events.	Albuterol		5		Participants
Number of Participants With Adverse Events.	Placebo Comparator		5		Participants
Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.	Albuterol	Change at 30 Weeks	-0.2	Standard Deviation 5.7	Percent of predicted FVC
Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.	Placebo Comparator	Change at 30 Weeks	0.4	Standard Deviation 7.9	Percent of predicted FVC
Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.	Albuterol	Change at 52 Weeks	-1.3	Standard Deviation 6.2	Percent of predicted FVC
Change in Forced Vital Capacity From Pulmonary Function Tests at 30 Weeks and 52 Weeks.	Placebo Comparator	Change at 52 Weeks	3.0	Standard Deviation 5.7	Percent of predicted FVC
Change in 6 Minute Walk Test	Albuterol	Change at 6 Weeks	24.0	Standard Deviation 10.3	meters
Change in 6 Minute Walk Test	Placebo Comparator	Change at 6 Weeks	32.0	Standard Deviation 58.1	meters
Change in 6 Minute Walk Test	Albuterol	Change at 52 Weeks	43.6	Standard Deviation 26.0	meters
Change in 6 Minute Walk Test	Placebo Comparator	Change at 52 Weeks	13.6	Standard Deviation 0.9	meters

Source ClinicalTrials.gov

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Albuterol	8
Overall Study	STARTED	Placebo Comparator	5
Overall Study	COMPLETED	Albuterol	7
Overall Study	COMPLETED	Placebo Comparator	5
Overall Study	NOT COMPLETED	Albuterol	1
Overall Study	NOT COMPLETED	Placebo Comparator	0

Source ClinicalTrials.gov

Adverse events82

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Abdominal pain	Gastrointestinal disorders	Albuterol	1	8
other	Abdominal pain	Gastrointestinal disorders	Placebo Comparator	1	5
other	Ankle edema increase	Cardiac disorders	Albuterol	0	8
other	Ankle edema increase	Cardiac disorders	Placebo Comparator	1	5
other	Anxiety	Psychiatric disorders	Albuterol	0	8
other	Anxiety	Psychiatric disorders	Placebo Comparator	1	5
other	Body aches	Musculoskeletal and connective tissue disorders	Albuterol	0	8
other	Body aches	Musculoskeletal and connective tissue disorders	Placebo Comparator	2	5
other	Body cramps	Musculoskeletal and connective tissue disorders	Albuterol	1	8
other	Body cramps	Musculoskeletal and connective tissue disorders	Placebo Comparator	0	5

1 to 10 of 82Source ClinicalTrials.gov

See all 82 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Albuterol	0	8
Total, all-cause mortality	Placebo Comparator	0	5
Total, other adverse events	Albuterol	5	8
Total, other adverse events	Placebo Comparator	5	5
Total, serious adverse events	Albuterol	0	8
Total, serious adverse events	Placebo Comparator	0	5

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
22154081	BACKGROUND	PubMed
31839530	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons3

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Albuterol vs Placebo Comparator: Number of Participants With Adverse Events.	Trial comparisons
Albuterol vs Placebo Comparator: deaths from any cause	Trial comparisons
Albuterol vs Placebo Comparator: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01885936

GET https://conductscience.org/api/trials/v1/NCT01885936/events?page=2

API reference For AI assistants