[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Shire\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":61},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,37],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":16,"targetDuration":19,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":4,"overallStatus":24,"whyStopped":4,"lastUpdateSubmitDate":25,"lastUpdatePostDateStruct":26,"startDateStruct":29,"completionDateStruct":31,"leadSponsor":33,"locationsCount":36},"100308124","gaucher-disease-outcome-survey-gos-100308124",false,"NCT03291223","Gaucher Disease Outcome Survey (GOS)","Inclusion Criteria:\n\n* Patients of any age or gender with confirmed diagnosis (biochemical and\u002For genetic) of Gaucher disease\n* Signed and dated written informed consent from the patient or, for patients aged \\\u003C18 years (\\\u003C16 years in the United Kingdom \\[UK\\]), their parent and\u002For legally authorized representatives (LAR), and assent of the minor where applicable. Legally authorized representatives are also applicable for cognitively impaired patients.\n\nExclusion Criteria:\n\n\\- Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)","ALL",{"count":17,"type":18},1257,"ESTIMATED","6 Months","OBSERVATIONAL","The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician.\n\nThe objectives of the registry include to evaluate the safety and long-term effectiveness of velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher Disease-specific treatments, to gain a better understanding of the natural history of GD and to serve as a database for evidence-based management of Gaucher Disease over time in real-life clinical practice.",[23],"Gaucher Disease","RECRUITING","2026-05-11",{"date":27,"type":28},"2026-05-14","ACTUAL",{"date":30,"type":28},"2010-12-29",{"date":32,"type":18},"2026-09-30",{"name":34,"class":35},"Shire","INDUSTRY",1,{"id":38,"slug":39,"hasResults":11,"nctId":40,"briefTitle":41,"officialTitle":42,"acronym":4,"eligibilityCriteria":43,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":44,"targetDuration":4,"studyType":46,"phases":47,"briefSummary":49,"conditions":50,"keywords":4,"overallStatus":24,"whyStopped":4,"lastUpdateSubmitDate":52,"lastUpdatePostDateStruct":53,"startDateStruct":55,"completionDateStruct":57,"leadSponsor":59,"locationsCount":60},"100444472","phase-4-a-study-of-replagal-in-children-and-adults-with-fabry-disease-in-india-100444472","NCT05067868","A Study of Replagal in Children and Adults With Fabry Disease in India","A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Replagal (Agalsidase Alfa [r-DNA Origin]) in Indian Children and Adults With Fabry Disease","Inclusion Criteria:\n\n1. Male and female Replagal naïve participants (and who are not part of any other program that allows participant to get access to free enzyme replacement therapy \\[ERT\\] at the time of study enrollment and during the study period) of any age with confirmed diagnosis of Fabry disease.\n2. Participants who have documented confirmed diagnosis of Fabry disease based on proof of gene mutation: α-galactosidase A gene compatible with Fabry disease and\u002For a deficiency of α-galactosidase A (less than \\[\\\u003C\\] 4.0 nanomole per milliliter per hour (nmol\u002FmL\u002Fhour) in plasma or serum or \\\u003C8 percent (%) of average mean normal in leukocytes and sequencing of GLA gene for females).\n3. Participant must have any clinical manifestations of Fabry disease based on investigator's discretion.\n4. Participant\u002Flegal authorized representative (LAR)\u002Fguardian is able to understand and willing to give written informed consent before performing any study specific procedures and willing to adhere to protocol requirements.\n5. Female participants of childbearing potential (example, nonsterilised, premenopausal female participants) must have a documented negative pregnancy test prior to administration of the first dose of Replagal in this study. In addition, all female participants of childbearing potential must use a two medically accepted forms of contraception throughout the study, that is, either a barrier method or hormonal contraceptive with norethindrone and ethinyl estradiol or similar active components.\n6. Male participant who is nonsterilised and sexually active with a female partner of childbearing potential agrees to use barrier method of contraception (example, condom with or without spermicide) from signing of informed consent throughout the duration of the study.\n\nNote: Female participants not of childbearing potential defined as those who have been surgically sterilized (hysterectomy, bilateral oophorectomy, or tubal ligation) or who are postmenopausal (example, defined as at least 1 year since last regular menses with an appropriate clinical profile \\[that is, age appropriate, history of vasomotor symptoms\\]).\n\nExclusion Criteria:\n\n1. Participants who have received Replagal.\n2. Participants with poorly controlled hypertension as per investigator's discretion.\n3. Participants with chronic kidney disease (CKD) with estimated Glomerular Filtration rate less than 15 milliliter per minute (mL\u002Fmin) \u002F1.73 meter square (m\\^2) and who had\u002Fwill have kidney transplantation or are currently on dialysis.\n4. Participants with any serious hepatic disorder who had abnormal hepatic function test values at screening (when either alanine aminotransferase \\[ALT\\] or aspartate aminotransferase \\[AST\\] level exceeded the value three times the upper limit of normal \\[ULN\\] and total bilirubin 1.5 times as high as the ULN); and deemed as clinically significant by investigator for hematology and biochemistry. These abnormal laboratory values could be discussed with medical monitor before excluding the participant.\n5. If female, the participant is pregnant or lactating or intending to become pregnant before participating in this study, during the study; or intending to donate ova during such time period.\n6. Participant\u002FLAR\u002Fguardian is unable to understand the nature, scope, and possible consequences of the study.\n7. Participant is unable to comply with the protocol, example, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the investigator.\n8. If male, the participant intends to donate sperm during the course of this study.\n9. Participants who had participated in any other investigational drug study within the past 4 weeks prior to screening.\n10. Any participant deemed as unfit for this trial, as per investigator's clinical judgment.",{"count":45,"type":18},5,"INTERVENTIONAL",[48],"PHASE4","The main aim of this study is to learn more about the safety profile of Replagal.\n\nParticipants will receive Replagal every 2 weeks at the clinic for about 1 year.",[51],"Fabry Disease","2025-09-15",{"date":54,"type":28},"2025-09-19",{"date":56,"type":28},"2022-11-01",{"date":58,"type":18},"2026-11-30",{"name":34,"class":35},3,""]