[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"glycogen-storage-disease-type-ia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:glycogen-storage-disease-type-ia":55},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,43,74,101],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":16,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":27,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100628280","accuracy-of-home-lactate-meter-and-accu-chek-glucometer-in-patients-with-glycogen-storage-disease-100628280",false,"NCT07459582","Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease","Inclusion Criteria:\n\n1. Patients with diagnosed\u002Fconfirmed (by liver biopsy or genetic testing) Glycogen Storage Disease Type Ia\u002FIb (ICD 10 code: E74.01) or XI (74.09).\n2. For pediatric participants: Ability of child's parent\u002Flegal guardian to understand and the willingness to sign a written informed consent document\n3. For adolescents: assent to participate\n4. For adults: Ability to understand and the willingness to sign a written informed consent\n\nExclusion Criteria:\n\n1. Patients with Glycogen storage disease unspecified\n2. Patients not meeting inclusion criteria\n3. Patients unable to provide consent\n4. Patients who decline to be in the study","ALL","60 Years",{"count":18,"type":19},10,"ESTIMATED","OBSERVATIONAL","The goal of this study is to determine if the Lactate Plus meter is accurate compared to lab lactate levels, and to determine if the Accu chek guide glucometer is accurate compared to lab serum glucose levels in patients with Glycogen Storage Disease Types Ia, Ib and XI.\n\nTo determine this, patient's will have a one-time planned admission to Connecticut Children's for approximately 8 hours and receive hourly blood draws as well as finger-sticks.",[23,24,25,26],"Glycogen Storage Disease Type IA","Glycogen Storage Disease Type I","Glycogen Storage Disease Type IB","Glycogen Storage Disease Xi",[28,29],"lactate","blood glucose","RECRUITING","2026-05-29",{"date":33,"type":34},"2026-06-01","ACTUAL",{"date":36,"type":19},"2026-08",{"date":38,"type":19},"2027-12",{"name":40,"class":41},"Connecticut Children's Medical Center","OTHER",1,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":15,"minAge":50,"maxAge":4,"enrollmentInfo":51,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":53,"conditions":54,"keywords":56,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":66,"completionDateStruct":68,"leadSponsor":70,"locationsCount":73},"100564992","glycogen-storage-disease-type-ia-gsdia-disease-monitoring-program-100564992","NCT06636383","Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program","GSDIa Disease Monitoring Program","Inclusion Criteria:\n\n* Patient who had:\n\n  * DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or\n  * Prescribed DTX401(full or partial dose) administered in a post-marketing setting (Group 2)\n* Patient is willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures.\n\nExclusion Criteria:\n\n* Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator","2 Years",{"count":52,"type":19},140,"The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.",[55],"Glycogen Storage Disease Type Ia",[57,58,59,60,61,62,63],"Glycogen storage disorder Ia","AAV","Gene therapy","Von Gierke disease","Glucose metabolism disorder","GSD1","GSDIa","2026-05-28",{"date":31,"type":34},{"date":67,"type":34},"2024-11-04",{"date":69,"type":19},"2036-12",{"name":71,"class":72},"Ultragenyx Pharmaceutical Inc","INDUSTRY",19,{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":4,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":15,"minAge":81,"maxAge":16,"enrollmentInfo":82,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":83,"conditions":84,"keywords":85,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":42},"100580901","accuracy-of-lactate-meter-in-gsdia-100580901","NCT06843330","Accuracy of Lactate Meter in GSDIa","Are Portable Lactate Monitoring Devices Accurate in Patients With Glycogen Storage Disease Ia When Compared to Blood Serum Lactates?","Inclusion Criteria:\n\n* Patients with diagnosed\u002Fconfirmed (by liver biopsy or genetic testing) Glycogen Storage Disease Type Ia (ICD 10 code: E74.01).\n* Connecticut Children's Emergency Department visit and\u002For admission to Connecticut Children's during time study is active\n* For pediatric participants: Ability of child's parent\u002Flegal guardian to understand and the willingness to sign a written informed consent document\n* For adults: Ability to understand and the willingness to sign a written informed consent\n\nExclusion Criteria:\n\n* Patients with Glycogen storage disease unspecified 74.00, or Ib\n* Patients not meeting inclusion criteria","0 Years",{"count":18,"type":19},"The goal of this observational study is to determine if home lactate meters (both capillary and serum sample) are accurate, compared to lab serum lactate in a population of patients with glycogen storage disease type 1a and to determine if the Accu Chek Guide glucometer (capillary sample) is accurate, in a population of patients with glycogen storage disease type 1a.",[55],[86,87,88,89,63,90,91,92],"Glycogen Storage Disease type Ia","Blood lactate","Glycogen Storage Disease Ia blood glucose","GSD I","Blood glucose","Accu chek glucometer","Lactate plus","2026-03-05",{"date":95,"type":34},"2026-03-06",{"date":97,"type":34},"2025-04-14",{"date":99,"type":19},"2027-12-10",{"name":40,"class":41},{"id":102,"slug":103,"hasResults":11,"nctId":104,"briefTitle":105,"officialTitle":106,"acronym":4,"eligibilityCriteria":107,"healthyVolunteers":11,"sex":15,"minAge":108,"maxAge":4,"enrollmentInfo":109,"targetDuration":4,"studyType":111,"phases":112,"briefSummary":115,"conditions":116,"keywords":117,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":119,"lastUpdatePostDateStruct":120,"startDateStruct":122,"completionDateStruct":124,"leadSponsor":126,"locationsCount":128},"100572629","phase-1-a-phase-12-dose-exploration-study-to-evaluate-the-safety-and-efficacy-of-beam-301-in-patients-with-glycogen-storage-disease-type-ia-gsdia-100572629","NCT06735755","A Phase 1\u002F2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)","A Phase 1\u002F2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) Homozygous or Compound Heterozygous for the G6PC1 c.247C>T (p.R83C) Variant","Inclusion Criteria:\n\n1. Males or females age ≥18 years of age at the time of consent.\n2. Diagnosis of GSDIa and homozygous or compound heterozygous for the G6PC1 c.247C\\>T (p.R83C) variant (confirmed by genetic testing).\n3. History of at least 1 episode of hypoglycemia \\\u003C60 mg\u002FdL within the 2 years prior to signing the ICF.\n\nExclusion Criteria:\n\n1. Liver transplant recipient\u002Fon waiting list for liver transplant or known history of liver cirrhosis.\n2. Presence of liver adenoma \\>5 cm in size based on liver MRI or liver ultrasound performed at screening or within 6 months prior to screening.\n3. Presence of liver adenoma \\>3 cm and ≤5 cm with a documented annual growth rate of ≥0.5 cm per year.\n4. Have aspartate transaminase or alanine transaminase \\>upper limit of normal (ULN).\n5. Total bilirubin levels \\>ULN; if documented Gilbert's Syndrome, total bilirubin \\>2 × ULN.\n6. Previous treatment with any cell-, gene-, or viral vector-derived therapy Hospitalization for management of hypoglycemia within 4 weeks prior to signing the ICF.\n7. Have triglycerides \\>1000 mg\u002FdL or a history of pancreatitis or a history of acute pancreatitis within 5 years that does not have a known and corrected etiology (eg, cholecystectomy for gallstone pancreatitis).","18 Years",{"count":110,"type":19},36,"INTERVENTIONAL",[113,114],"PHASE1","PHASE2","This is a Phase 1\u002F2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygous for the G6PC1 c.247C\\>T (p.R83C) variant and to determine the optimal biological dose.",[55],[63,118],"GSD1a","2025-12-09",{"date":121,"type":34},"2025-12-15",{"date":123,"type":34},"2024-12-06",{"date":125,"type":19},"2027-12-30",{"name":127,"class":72},"Beam Therapeutics Inc.",3]