NCT04808505 - A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

July 18, 2023

Primary Completion Date

April 30, 2027

Study Completion Date

April 30, 2027

Conditions

Glycogen Storage Disease Type II Infantile Onset

Interventions

BIOLOGICAL

Cipaglucosidase alfa

Sterile lyophilized powder intravenous (IV) infusion

DRUG

Miglustat

65 mg oral capsules

Trial Locations (16)

100

WITHDRAWN

National Taiwan University Hospital, Taipei

10126

NOT_YET_RECRUITING

A.O.U. Citta della Salute e della Scienza di Torino, Presidio Ospedale Infantile Regina Margherita, S.C. Neuropsichiatria Infantile U, Torino

15224

RECRUITING

UPMC Hospital of Pittsburgh, Pittsburgh

27710

RECRUITING

Duke University Early Phase Research Unit, Durham

30322

RECRUITING

The Emory Clinic, Atlanta

32610

RECRUITING

University of Florida Clinical Research Center, Gainesville

35392

RECRUITING

Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie, Giessen

48149

RECRUITING

Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1, Münster

54229

RECRUITING

Cincinnati Children's Hospital Medical Center, Cincinnati

65239

NOT_YET_RECRUITING

SphinCS GmbH, Höchheim

69120

RECRUITING

Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS), Heidelberg

80131

RECRUITING

AOU Federico II, Naples

84108

RECRUITING

University of Utah, Clinical and Translational Sciences Institute, Salt Lake City

92380

NOT_YET_RECRUITING

Hôpital Raymond Poincaré, Neurologie et réanimation pédiatriques, Garches

3015 GD

NOT_YET_RECRUITING

Erasmus MC, Sophia Kinderziekenhuis, Rotterdam

WC1N3JH

NOT_YET_RECRUITING

Great Ormond Street Hospital for Children NHS Foundation Trust, London