[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Genzyme, a Sanofi Company\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":148},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,45,73,98,123],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":20,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":26,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100075056","pompe-disease-registry-protocol-100075056",false,"NCT00231400","Pompe Disease Registry Protocol","Pompe Disease Registry","Inclusion Criteria:\n\nAll patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and\u002For documentation of 2 GAA gene mutations.\n\nExclusion Criteria:\n\nThere are no exclusion criteria in this Registry","ALL",{"count":18,"type":19},2000,"ESTIMATED","5 Years","OBSERVATIONAL","The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development\u002Freimbursement, and for other research and non-research related purposes.\n\nThe objectives of the Registry are:\n\n* To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention.\n* To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care.\n* To characterize the Pompe disease population.\n* To evaluate the long-term effectiveness of alglucosidase alfa.",[24,25],"Glycogen Storage Disease Type II","Pompe Disease",[27,28,25,29,30,31],"Glycogen Storage Disease Type II (GSD-II)","GSD-II","Pompe Disease (late-onset)","Acid Maltase Deficiency Disease","Glycogenosis II","RECRUITING","2026-06-19",{"date":35,"type":36},"2026-06-23","ACTUAL",{"date":38,"type":36},"2004-09-15",{"date":40,"type":19},"2034-01-31",{"name":42,"class":43},"Genzyme, a Sanofi Company","INDUSTRY",272,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":52,"targetDuration":54,"studyType":21,"phases":4,"briefSummary":55,"conditions":56,"keywords":58,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":67,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":71,"locationsCount":72},"100072469","fabry-disease-registry--pregnancy-sub-registry-100072469","NCT00196742","Fabry Disease Registry & Pregnancy Sub-registry","Fabry Disease Registry Protocol","Inclusion Criteria\n\n* Fabry Registry: All patients with a confirmed diagnosis of Fabry disease who have signed the informed consent and patient authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as a documented deficiency in plasma or leukocyte αGAL (alpha-galactosidase) enzyme activity and\u002For mutation(s) in the gene coding for αGAL.\n* Fabry Pregnancy Sub-registry:\n\n  * Eligible women must:\n\n    * be enrolled in the Fabry Registry.\n    * be pregnant, or have been pregnant with appropriate medical documentation available.\n    * provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.\n\nExclusion Criteria Fabry Registry: There are no exclusion criteria. Fabry Pregnancy Sub-registry: There are no exclusion criteria.",{"count":53,"type":19},9000,"52 Weeks","The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.\n\nThe primary objectives of the Registry are:\n\n* To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease;\n* To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;\n* To characterize and describe the Fabry population as a whole;\n* To evaluate the long-term safety and effectiveness of Fabrazyme®\n\nFabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development\u002Freimbursement, and for other research and non-research-related purposes. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.",[57],"Fabry Disease",[59,60,61,62,63,64,65,66],"alpha Galactosidase A","aGAL (alpha-galactosidase)","Fabry","GL3 (globotriaosylceramide)","Anderson-Fabry Disease","angiokeratomas","GLA deficiency (gene deficiency)","errors in metabolism",{"date":35,"type":36},{"date":69,"type":36},"2001-07-31",{"date":40,"type":19},{"name":42,"class":43},284,{"id":74,"slug":75,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":78,"acronym":4,"eligibilityCriteria":79,"healthyVolunteers":11,"sex":80,"minAge":4,"maxAge":4,"enrollmentInfo":81,"targetDuration":83,"studyType":21,"phases":4,"briefSummary":84,"conditions":85,"keywords":88,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":90,"lastUpdatePostDateStruct":91,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":96,"locationsCount":97},"100100246","pompe-pregnancy-sub-registry-100100246","NCT00567073","Pompe Pregnancy Sub-Registry","A Sub-registry to Observe the Effect of Alglucosidase Alfa or Avalglucosidase Alfa Treatment on Pregnancy and Infant Growth in Women With Pompe Disease","Inclusion Criteria:\n\nEligible women must:\n\n* be enrolled in the Pompe registry (NCT00231400)\n* be pregnant, or have been pregnant with appropriate medical documentation available.\n* provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.\n\nNote: It is recommended that pregnancy data be collected on eligible women regardless of infant enrollment. In the event of patients having multiple pregnancies, participation in this Sub-Registry is encouraged for each individual pregnancy.\n\nExclusion Criteria:\n\nThere are no exclusion criteria for this Sub-Registry","FEMALE",{"count":82,"type":19},20,"4 Years","This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglucosidase alfa or avalglucosidase alfa) and irrespective of the commercial product with which she may be treated. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.\n\nThe primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Pompe disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with alglucosidase alfa or avalglucosidase alfa.",[27,86,87],"Pompe Disease (Late-onset)","Glycogenesis 2 Acid Maltase Deficiency",[27,28,25,89,30,31],"Pompe Disease (Late-Onset)","2026-04-15",{"date":92,"type":36},"2026-04-16",{"date":94,"type":36},"2007-06-18",{"date":40,"type":19},{"name":42,"class":43},35,{"id":99,"slug":100,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":4,"eligibilityCriteria":104,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":105,"targetDuration":107,"studyType":21,"phases":4,"briefSummary":108,"conditions":109,"keywords":114,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":115,"lastUpdatePostDateStruct":116,"startDateStruct":118,"completionDateStruct":120,"leadSponsor":121,"locationsCount":122},"100084590","international-collaborative-gaucher-group-icgg-gaucher-disease-registry--pregnancy-sub-registry-100084590","NCT00358943","International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry","Gaucher Disease Registry Protocol","Inclusion Criteria:\n\nICGG Gaucher Registry\n\n* All patients with a confirmed diagnosis of Gaucher disease are eligible for inclusion in the Registry. Confirmed diagnosis is defined as a documented β-glucocerebrosidase deficiency and\u002For mutation in the β-glucocerebrosidase gene.\n* For all patients, appropriate patient authorization will be obtained.\n\nGaucher Pregnancy Sub-registry:\n\n* be enrolled in the ICGG Gaucher Registry.\n* be pregnant, or have been pregnant with appropriate medical documentation available.\n* provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.\n\nExclusion Criteria:\n\n\\- No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.",{"count":106,"type":19},12000,"12 Months","The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.\n\nThe objectives of the Registry are:\n\n* To enhance understanding of the variability, progression, identification, and natural history of Gaucher disease, with the ultimate goal of better guiding and assessing therapeutic intervention.\n* To assist the Gaucher medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care.\n* To characterize the Gaucher disease population.\n* To evaluate the long-term effectiveness of imiglucerase and of eliglustat.\n\nGaucher Pregnancy Sub-registry: The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.",[110,111,112,113],"Gaucher Disease","Cerebroside Lipidosis Syndrome","Glucocerebrosidase Deficiency Disease","Glucosylceramide Beta-Glucosidase Deficiency Disease",[110,112],"2026-04-13",{"date":117,"type":36},"2026-04-14",{"date":119,"type":36},"1991-04-01",{"date":40,"type":19},{"name":42,"class":43},318,{"id":124,"slug":125,"hasResults":11,"nctId":126,"briefTitle":127,"officialTitle":128,"acronym":4,"eligibilityCriteria":129,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":130,"enrollmentInfo":131,"targetDuration":4,"studyType":133,"phases":134,"briefSummary":136,"conditions":137,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":139,"lastUpdatePostDateStruct":140,"startDateStruct":142,"completionDateStruct":144,"leadSponsor":146,"locationsCount":147},"100567301","phase-4-china-post-approval-commitment-pac-study-of-avalglucosidase-alfa-in-participants-with-iopd-100567301","NCT06666413","China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD","A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)","Inclusion Criteria:\n\n* Participant must be \\\u003C18 years of age, at the time of signing the informed consent.\n* Participants have documented onset of Pompe disease symptoms before 12 months of age (corrected for gestation if born before 40 weeks); and diagnosis of IOPD is confirmed by GAA enzyme deficiency from any tissue source and GAA gene pathogenic mutations.\n* Participants must have documented cardiomyopathy at the time of diagnosis.\n* Contraceptive use should be consistent with local regulations Participant's parent\u002Flegally authorized representative (LAR) must be capable of giving signed informed consent.\n\nExclusion Criteria: Participants are excluded from the study if any of the following criteria apply:\n\n* Participants with major congenital abnormality that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.\n* Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).\n* Participants who have received an ERT other than alglucosidase alfa or avalglucosidase alfa, or any other treatment for Pompe disease, including gene therapy prior to the enrollment.\n* Participants who have received alglucosidase alfa or avalglucosidase alfa less than 1 week prior to the first dose of avalglucosidase alfa given as IMP Participants who are anticipated to take prohibited therapy (ie, any other treatment for Pompe disease) during this study.\n* Participants who have taken other investigational drugs (not Pompe disease specific) within 30 days or 5 elimination half-lives in blood of that drug before enrollment, whichever is longer, or are anticipated to take any other concurrent investigational treatments.\n* Participants not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.\n\nThe above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.","17 Years",{"count":132,"type":19},13,"INTERVENTIONAL",[135],"PHASE4","This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT.\n\nStudy details include:\n\n* The study duration: total study duration is approximately 64 weeks.\n\n  * Screening period of up to 8 weeks\n  * Treatment period of 52 weeks\n  * Follow-up period of 4 weeks. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks)\n* The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.",[24,138],"Pompe's Disease","2026-03-19",{"date":141,"type":36},"2026-03-23",{"date":143,"type":36},"2025-05-07",{"date":145,"type":19},"2028-05-02",{"name":42,"class":43},1,""]