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drugset / Trial / NCT02185651

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

NCT02185651 ↗

Phase 1 Terminated 2 enrolled University of Florida Amicus Therapeutics · collab
Non-randomizedParallel-groupOpen-labelTreatment

Summary

Hypothesis: the effectiveness of treatment of Pompe disease with rhGAA enzyme replacement therapy (ERT) is limited at least in part because patients develop antibodies against the provided rhGAA enzyme. Treatment with Zavesca® prior to infusion may dampen or eliminate the anti-rhGAA immune response in patients receiving ERT, thereby allowing for greater ERT efficacy. Treatment with Zavesca® before a enzyme replacement therapy (ERT) may decrease the severity of, or eliminate infusion associated reactions (IAR) in people with Pompe Disease receiving ERT.

Timeline

Start
2016-10
Primary completion
2018-07
Completion
2018-07

Drugs

EvaluationDrugModalityDoseRoute
Comparator Miglustat Small molecule 100 mg Oral
Comparator Miglustat Small molecule 300 mg Oral

Indications

glycogen storage disease II hypersensitivity reaction disease