NCT02185651 - A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

October 31, 2016

Primary Completion Date

July 31, 2018

Study Completion Date

July 31, 2018

Conditions

Pompe DiseaseHypersensitivity Reaction

Interventions

DRUG

Zavesca® Prescription

"Following baseline evaluation, Zavesca® prescription is given.~Week 2, 4, and 6 ERT infusion with pre-medication are completed at local/home infusion center. Travel to site for week 7 study visit includes physical exam, blood collection and punch muscle biopsy. Health survey is completed."