NCT02185651View on ClinicalTrials.gov

A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction

PHASE1TerminatedINTERVENTIONAL
Enrollment

2

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."

Sponsors

Collaborators (1)

Amicus Therapeutics
All Listed Sponsors
collaborator

Amicus Therapeutics

INDUSTRY

lead

University of Florida

OTHER