MedTrace Logo

ERT in Pompe Disease: Elucidation of Molecular Structures Contributing to Enzyme Uptake and Immunoreactivity

NCT05448131 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 50

Last updated 2023-02-21

No results posted yet for this study

Summary

In the first part of this study, the aim is to characterize the molecular structure of wildtype GAA and, in particular, of mutated GAA variants with and without HSAT, in order to learn how mutation impairs uptake of GAA into the cell via the M6P receptor. In the second part of the study the aim is to learn to which epitopes antibodies bind and to which not. To accomplish this the investigators will synthesize and chemically modify the epitope peptides, in order to block effectively antibodies directed against the therapeutic enzyme.

Conditions

  • Pompe-Disease

Sponsors & Collaborators

  • University of Giessen

    collaborator OTHER

  • Centre for Analytical Biochemistry and Biomedical Mass Spectrometry

    lead OTHER

Principal Investigators

  • Michael Przybylski, PhD · Centre for Analytical Biochemistry, 65428 Ruesselsheim am Main, Germany

Eligibility

Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2023-02-01
Primary Completion
2024-08-06
Completion
2024-10-07

Countries

  • Germany

Study Locations

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 NCT05448131 on ClinicalTrials.gov