Phase
Condition
Pompe Disease
Liver Disorders
Treatment
Avalglucosidase alfa (GZ402666)
Alglucosidase alfa (GZ419829)
Clinical Study ID
Ages > 3 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion criteria :
- The participant has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from anytissue source and/or 2 confirmed GAA gene mutations.
- The participant must provide signed, informed consent prior to performing any studyrelated procedures. Consent of a legally authorized guardian(s) is (are) required forlegally minor participant as defined by local regulation. If the participant islegally minor, signed written consent shall be obtained from parent(s)/legal guardianand assent obtained from participants, if applicable.
Exclusion
Exclusion criteria:
- The participant is <3 years of age.
- The participant has known Pompe specific cardiac hypertrophy.
- The participant is wheelchair dependent.
- The participant is not able to ambulate 40 meters (approximately 130 feet) withoutstopping and without an assistive device.
- The participant requires invasive-ventilation (non-invasive ventilation is allowed).
- The participant is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of greater than or equal to 30% predicted andless than or equal to 85% predicted.
- The participant (and participant's legal guardian if participant is legally minor asdefined by local regulation) is (are) not able to comply with the clinical protocol.
- The participant has had previous treatment with alglucosidase alfa or anyinvestigational therapy for Pompe disease.
- The participant has prior or current use of immune tolerance induction therapy.
- The participant, if female and of childbearing potential, has a positive pregnancytest (beta-human chorionic gonadotropin) at baseline. The above information is not intended to contain all considerations relevant to aparticipant's potential participation in a clinical trial.
Study Design
Study Description
Connect with a study center
Investigational Site Number 0320001
Buenos Aires,
ArgentinaSite Not Available
Investigational Site Number 0320001
Caba, C1181ACH
ArgentinaSite Not Available
Investigational Site Number 0360001
Auchenflower, 4066
AustraliaSite Not Available
Investigational Site Number 0400001
Wien, 1090
AustriaSite Not Available
Investigational Site Number 0560003
Bruxelles, 1070
BelgiumSite Not Available
Investigational Site Number 0560001
Leuven, 3000
BelgiumSite Not Available
Investigational Site Number 0760004
Brasilia, 71625-009
BrazilSite Not Available
Investigational Site Number 0760005
Brasília, 72145-450
BrazilSite Not Available
Investigational Site Number 0760001
Sao Paulo, 04037-002
BrazilSite Not Available
Investigational Site Number 1000001
Sofia, 1431
BulgariaSite Not Available
Investigational Site Number 1240003
Hamilton, L8N 3Z5
CanadaSite Not Available
Investigational Site Number 1240002
Montreal, H3A 2B4
CanadaSite Not Available
Investigational Site Number 1240002
Montréal, H3A 2B4
CanadaSite Not Available
Investigational Site Number 1700001
Bogota,
ColombiaSite Not Available
Investigational Site Number 1700001
Bogotá,
ColombiaSite Not Available
Investigational Site Number 2030001
Praha, 12100
Czech RepublicSite Not Available
Investigational Site Number 2030001
Praha 2, 12808
CzechiaSite Not Available
Investigational Site Number 2080002
Aarhus C, 8000
DenmarkSite Not Available
Investigational Site Number 2080003
Copenhagen, 2100
DenmarkSite Not Available
Investigational Site Number 2080003
København Ø, 2100
DenmarkSite Not Available
Investigational Site Number 2500008
Angers, 49933
FranceSite Not Available
Investigational Site Number 2500007
Bordeaux,
FranceSite Not Available
Investigational Site Number 2500011
Brest Cedex 2, 29609
FranceSite Not Available
Investigational Site Number 2500004
Bron, 69677
FranceSite Not Available
Investigational Site Number 2500003
Bron Cedex, 69677
FranceSite Not Available
Investigational Site Number 2500010
Clermont Ferrand, 63003
FranceSite Not Available
Investigational Site Number 2500005
Lille, 59037
FranceSite Not Available
Investigational Site Number 2500006
Marseille, 13385
FranceSite Not Available
Investigational Site Number 2500006
Marseille Cedex 5, 13385
FranceSite Not Available
Investigational Site Number 2500002
Nice Cedex 1, 06002
FranceSite Not Available
Investigational Site Number 2500001
Paris, 75013
FranceSite Not Available
Investigational Site Number 2500001
Paris Cedex 13, 75013
FranceSite Not Available
Investigational Site Number 2760006
Bochum, 44789
GermanySite Not Available
Investigational Site Number 2760004
Hamburg, 20246
GermanySite Not Available
Investigational Site Number 2760001
Mainz, 55131
GermanySite Not Available
Investigational Site Number 2760002
Munster, 48149
GermanySite Not Available
Investigational Site Number 2760003
München, 80336
GermanySite Not Available
Investigational Site Number 2760002
Münster, 48149
GermanySite Not Available
Investigational Site Number 3480001
Budapest, 1083
HungarySite Not Available
Investigational Site Number 3800006
Brescia, 25123
ItalySite Not Available
Investigational Site Number 3800001
Messina, 98125
ItalySite Not Available
Investigational Site Number 3800002
Milano, 20122
ItalySite Not Available
Investigational Site Number 3800007
Napoli, 80131
ItalySite Not Available
Investigational Site Number 3800004
Padova, 35128
ItalySite Not Available
Investigational Site Number 3800007
Roma, 00168
ItalySite Not Available
Investigational Site Number 3800003
Torino, 10126
ItalySite Not Available
Investigational Site Number 3920003
Izumi-Shi,
JapanSite Not Available
Investigational Site Number 3920002
Kodaira-Shi,
JapanSite Not Available
Investigational Site Number 3920003
Osaka, 594-0071
JapanSite Not Available
Investigational Site Number 3920002
Tokyo, 187-8551
JapanSite Not Available
Investigational Site Number 4100004
Daejeon, 35015
Korea, Republic ofSite Not Available
Investigational Site Number 4100001
Seoul, 03080
Korea, Republic ofSite Not Available
Investigational Site Number 4100002
Seoul, 06273
Korea, Republic ofSite Not Available
Investigational Site Number 4100003
Yangsan-Si,
Korea, Republic ofSite Not Available
Investigational Site Number 4840001
Guadalajara,
MexicoSite Not Available
Investigational Site Number 4840001
Jalisco, 45030
MexicoSite Not Available
Investigational Site Number 4840001
Mexico,
MexicoSite Not Available
Investigational Site Number 5280001
Rotterdam, 3015 GE
NetherlandsSite Not Available
Investigational Site Number 6160001
Warszawa, 02-097
PolandSite Not Available
Investigational Site Number 6200001
Braga, 4710-243
PortugalSite Not Available
Investigational Site Number 6430001
Moscow, 125367
Russian FederationSite Not Available
Investigational Site Number 7240002
Barcelona, 08025
SpainSite Not Available
Investigational Site Number 7240003
Barcelona, 08950
SpainSite Not Available
Investigational Site Number 7240003
Barcelona / Esplugues De Llobregat, 08950
SpainSite Not Available
Investigational Site Number 7240004
Cadiz, 11009
SpainSite Not Available
Investigational Site Number 7240003
Esplugues De Llobregat, 08950
SpainSite Not Available
Investigational Site Number 7240001
Madrid, 28046
SpainSite Not Available
Investigational Site Number 7240005
Valencia, 46026
SpainSite Not Available
Investigational Site Number 7520001
Uppsala, 751 85
SwedenSite Not Available
Investigational Site Number 7560002
Zurich, 8091
SwitzerlandSite Not Available
Investigational Site Number 7560002
Zürich, 8091
SwitzerlandSite Not Available
Investigational Site Number 1580001
Taipai, 10043
TaiwanSite Not Available
Investigational Site Number 1580001
Taipei, 10043
TaiwanSite Not Available
Investigational Site Number 1580002
Taoyuan County, 33305
TaiwanSite Not Available
Investigational Site Number 7920001
Ankara, 06100
TurkeySite Not Available
Investigational Site Number 7920002
Istanbul, 34390
TurkeySite Not Available
Investigational Site Number 8260005
Birmingham, B15 2GW
United KingdomSite Not Available
Investigational Site Number 8260002
Cambridge, CB2 OQQ
United KingdomSite Not Available
Investigational Site Number 8260001
London, NW3 2QG
United KingdomSite Not Available
Investigational Site Number 8260004
Newcastle Upon Tyne, NE1 4LP
United KingdomSite Not Available
Investigational Site Number 8260003
Salford, M6 8HD
United KingdomSite Not Available
Investigational Site Number 8400015
Phoenix, Arizona 85013
United StatesSite Not Available
Investigational Site Number 8400020
Los Angeles, California 90095
United StatesSite Not Available
Investigational Site Number 8400011
Orange, California 92868
United StatesSite Not Available
Investigational Site Number 84000017
Palo Alto, California 94304
United StatesSite Not Available
Investigational Site Number 8400017
Stanford, California 94305
United StatesSite Not Available
Investigational Site Number 8400016
Gainesville, Florida 32610
United StatesSite Not Available
Investigational Site Number 8400016
Gainsville, Florida 32610
United StatesSite Not Available
Investigational Site Number 8400007
Decatur, Georgia 30033
United StatesSite Not Available
Investigational Site Number 8400023
Chicago, Illinois 60611
United StatesSite Not Available
Investigational Site Number 8400002
Iowa City, Iowa 52242
United StatesSite Not Available
Investigational Site Number 8400012
Kansas City, Kansas 66160-7321
United StatesSite Not Available
Investigational Site Number 8400010
Boston, Massachusetts 02114
United StatesSite Not Available
Investigational Site Number 8400001
Detroit, Michigan 48201
United StatesSite Not Available
Investigational Site Number 8400019
Minneapolis, Minnesota 55455
United StatesSite Not Available
Investigational Site Number 8400013
Saint Louis, Missouri 63110
United StatesSite Not Available
Investigational Site Number 8400026
Great Neck, New York 11020
United StatesSite Not Available
Investigational Site Number 8400021
New York, New York 10016
United StatesSite Not Available
Investigational Site Number 8400008
Valhalla, New York 10595
United StatesSite Not Available
Investigational Site Number 8400006
Durham, North Carolina 27710
United StatesSite Not Available
Investigational Site Number 8400009
Cincinnati, Ohio 45267-0542
United StatesSite Not Available
Investigational Site Number 8400014
Portland, Oregon 97239
United StatesSite Not Available
Investigational Site Number 8400004
Hershey, Pennsylvania 17033
United StatesSite Not Available
Investigational Site Number 8400025
Pittsburgh, Pennsylvania 15213
United StatesSite Not Available
Investigational Site Number 8400022
Dallas, Texas 75246
United StatesSite Not Available
Investigational Site Number 8400018
Salt Lake City, Utah 84132
United StatesSite Not Available
Investigational Site Number 8400005
Fairfax, Virginia 22030
United StatesSite Not Available
Investigational Site Number 8400024
Morgantown, West Virginia 26506
United StatesSite Not Available
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