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

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

16

Participants

Timeline

Start Date

June 30, 2013

Primary Completion Date

December 16, 2016

Study Completion Date

December 16, 2016

Conditions
Pompe Disease
Interventions
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.

Trial Locations (1)

27710

Duke University Medical Center, Durham

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Duke University

OTHER