ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

PHASE3RecruitingINTERVENTIONAL
Enrollment

22

Participants

Timeline

Start Date

February 13, 2020

Primary Completion Date

June 30, 2026

Study Completion Date

June 30, 2026

Conditions
Pompe Disease (Late-onset)
Interventions
BIOLOGICAL

Cipaglucosidase Alfa

Enzyme Replacement Therapy via intravenous infusion

DRUG

Miglustat

Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221)

Trial Locations (23)

100

WITHDRAWN

National Taiwan University Hospital, Taipei

5006

RECRUITING

Women's and Children's Hospital, North Adelaide

15224

RECRUITING

UPMC Children's Hospital of Pittsburgh, Pittsburgh

20900

RECRUITING

San Gerardo Hospital, Monza

22030

RECRUITING

Lysosomal and Rare Disorders Research and Treatment Center, Inc., Fairfax

27710

RECRUITING

Duke University Medical Center, Durham

30322

NOT_YET_RECRUITING

Woodruff Memorial Research Building, Atlanta

32207

RECRUITING

Wolfson Children's Hospital, Jacksonville

32610

RECRUITING

University of Florida Clinical Research Center, Gainesville

45229

RECRUITING

Cincinnati Children's Hospital Medical Center, Cincinnati

49525

WITHDRAWN

Infusion Associates, Grand Rapids

55905

WITHDRAWN

Mayo Clinic, Rochester

63110

RECRUITING

St. Louis Children's Hospital, St Louis

65239

RECRUITING

SphinCS GmbH Clinical Science for LSD, Hochheim am Main

72202

WITHDRAWN

Arkansas Children's Hospital, Little Rock

84108

RECRUITING

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

85028

WITHDRAWN

Neuromuscular Research Center, Phoenix

94609

WITHDRAWN

UCSF Benioff Children's Hospital, Oakland

T3B 6A8

COMPLETED

University of Calgary, Calgary

594-0073

RECRUITING

Izumi City General Hospital, Osaka

371-8511

RECRUITING

Gunma University Hospital, Gunma

980-8574

RECRUITING

Tohoku University Hospital, Miyagi

162-8666

RECRUITING

Tokyo Women's Medical University, Tokyo

Sponsors
All Listed Sponsors
lead

Amicus Therapeutics

INDUSTRY

NCT03911505 - ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD | Biotech Hunter | Biotech Hunter