● Recruiting

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Condition: Glycogen Storage Disease Type II Infantile Onset · Sponsor: Amicus Therapeutics

PhasePhase 3

Planned participants36

Who can joinAll sexes, N/A to 17 Years

Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

For Site · 609-662-2000 · patientadvocacy@amicusrx.com

For Patient · 609-662-2000 · patientadvocacy@amicusrx.com

Always discuss trial participation with your own doctor first.

Locations (14)

University of Florida Clinical Research Center	Gainesville, Florida, United States	Recruiting
The Emory Clinic	Atlanta, Georgia, United States	Recruiting
Duke University Early Phase Research Unit	Durham, North Carolina, United States	Recruiting
Cincinnati Children's Hospital Medical Center	Cincinnati, Ohio, United States	Recruiting
UPMC Hospital of Pittsburgh	Pittsburgh, Pennsylvania, United States	Recruiting
University of Utah, Clinical and Translational Sciences Institute	Salt Lake City, Utah, United States	Recruiting
Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie	Giessen, Germany	Recruiting
Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS)	Heidelberg, Germany	Recruiting
SphinCS GmbH	Höchheim, Germany	Recruiting
Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1	Münster, Germany	Recruiting
AOU Federico II	Naples, Italy	Recruiting
Erasmus MC, Sophia Kinderziekenhuis	Rotterdam, Netherlands	Recruiting

+ 2 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT04808505