This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
36
Sterile lyophilized powder intravenous (IV) infusion
65 mg oral capsules
University of Florida Clinical Research Center
Gainesville, Florida, United States
RECRUITINGThe Emory Clinic
Proportion of subjects with infusion-associated reactions (IARs)
Time frame: 104 weeks
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Atlanta, Georgia, United States
Duke University Early Phase Research Unit
Durham, North Carolina, United States
RECRUITINGCincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
RECRUITINGUPMC Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
RECRUITINGUniversity of Utah, Clinical and Translational Sciences Institute
Salt Lake City, Utah, United States
RECRUITINGUniversitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie
Giessen, Germany
RECRUITINGUniversitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS)
Heidelberg, Germany
RECRUITINGSphinCS GmbH
Höchheim, Germany
RECRUITINGUniversitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1
Münster, Germany
RECRUITING...and 4 more locations