MedTrace Logo

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

NCT02185651 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2018-12-05

No results posted yet for this study

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.

Conditions

  • Pompe Disease
  • Hypersensitivity 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

  • Amicus Therapeutics

    collaborator INDUSTRY

  • University of Florida

    lead OTHER

Principal Investigators

  • Barry J. Byrne, MD, PhD · University of Florida

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-10-31
Primary Completion
2018-07-31
Completion
2018-07-31

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02185651 on ClinicalTrials.gov