[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100572629":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":21,"locations":27,"responsibleParty":60,"collaborators":20,"id":62,"slug":63,"hasResults":64,"nctId":65,"briefTitle":66,"officialTitle":67,"acronym":20,"eligibilityCriteria":68,"healthyVolunteers":64,"sex":69,"minAge":70,"maxAge":20,"enrollmentInfo":71,"targetDuration":20,"studyType":74,"phases":75,"briefSummary":78,"conditions":79,"keywords":81,"overallStatus":30,"whyStopped":20,"lastUpdateSubmitDate":84,"lastUpdatePostDateStruct":85,"startDateStruct":88,"completionDateStruct":90,"leadSponsor":92,"locationsCount":93},{"fullName":5,"class":6},"Beam Therapeutics Inc.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"BEAM-301","EXPERIMENTAL","BEAM-301 consists of adenine base editor messenger ribonucleic acid (mRNA) and single guide ribonucleic acid (gRNA) formulated in lipid nanoparticles (LNPs) for intravenous (IV) administration.",[13],"Drug: BEAM-301: Single dose of BEAM-301 administered by IV",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"DRUG","BEAM-301: Single dose of BEAM-301 administered by IV","BEAM-301 is designed to correct the G6PC1 c.247C\\>T allele via an A:T-to-G:C base-pair substitution, resulting in restoration of G6Pase-α catalytic activity.",[9],null,[22],{"name":23,"role":24,"phone":25,"phoneExt":20,"email":26},"Medical Information","CONTACT","857-327-8641","clinicalinfo@beamtx.com",[28,42,51],{"facility":29,"status":30,"city":31,"state":32,"zip":33,"country":34,"countryCode":35,"cosmosGeoPoint":36,"geoPoint":41,"contacts":20},"Clinical Study Site","RECRUITING","Orange","California","92868","United States","US",{"type":37,"coordinates":38},"Point",[39,40],-117.85311,33.78779,{"lat":40,"lon":39},{"facility":29,"status":30,"city":43,"state":44,"zip":45,"country":34,"countryCode":35,"cosmosGeoPoint":46,"geoPoint":50,"contacts":20},"Farmington","Connecticut","06030",{"type":37,"coordinates":47},[48,49],-72.83204,41.71982,{"lat":49,"lon":48},{"facility":29,"status":30,"city":52,"state":53,"zip":54,"country":34,"countryCode":35,"cosmosGeoPoint":55,"geoPoint":59,"contacts":20},"Houston","Texas","77030",{"type":37,"coordinates":56},[57,58],-95.36327,29.76328,{"lat":58,"lon":57},{"type":61,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","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",false,"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).","ALL","18 Years",{"count":72,"type":73},36,"ESTIMATED","INTERVENTIONAL",[76,77],"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.",[80],"Glycogen Storage Disease Type Ia",[82,83],"GSDIa","GSD1a","2025-12-09",{"date":86,"type":87},"2025-12-15","ACTUAL",{"date":89,"type":87},"2024-12-06",{"date":91,"type":73},"2027-12-30",{"name":5,"class":6},3]