[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100572629":3},{"organization":4,"outcomesModule":7,"designInfo":27,"detailedDescription":11,"studyPopulation":11,"armGroups":34,"interventions":41,"overallOfficials":11,"centralContacts":47,"locations":53,"responsibleParty":86,"collaborators":11,"id":88,"slug":11,"hasResults":89,"nctId":90,"briefTitle":91,"officialTitle":92,"acronym":11,"eligibilityCriteria":93,"healthyVolunteers":89,"sex":94,"minAge":95,"maxAge":11,"enrollmentInfo":96,"targetDuration":11,"studyType":99,"phases":100,"briefSummary":103,"conditions":104,"keywords":106,"overallStatus":56,"whyStopped":11,"lastUpdateSubmitDate":109,"lastUpdatePostDateStruct":110,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":118},{"fullName":5,"class":6},"Beam Therapeutics Inc.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":11},[9],{"measure":10,"description":11,"timeFrame":12},"To evaluate the safety and tolerability of BEAM-301 in patients with GSDIa with a G6PC1 c.247C>T (p.R83C) variant by measuring incidence of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs) and dose-limiting toxicities (DL",null,"BEAM-301 administration through month 24",[14,17,19,21,23,25],{"measure":15,"description":11,"timeFrame":16},"Absolute and change from baseline in time to hypoglycemia or metabolic decompensation during a controlled fasting challenge","Baseline to month 24",{"measure":18,"description":11,"timeFrame":16},"Changes from baseline in starch supplementation dose",{"measure":20,"description":11,"timeFrame":16},"Level of serum glucose and metabolic parameters (lipid profile and uric acid) over time",{"measure":22,"description":11,"timeFrame":16},"BEAM-301 PK parameters (Cmax)",{"measure":24,"description":11,"timeFrame":16},"BEAM-301 PK parameters (AUC)",{"measure":26,"description":11,"timeFrame":16},"BEAM-301 PK parameters (half-life)",{"allocation":28,"interventionModel":29,"interventionModelDescription":30,"primaryPurpose":31,"observationalModel":11,"timePerspective":11,"maskingInfo":32},"NA","SEQUENTIAL","up to 4 planned, single-ascending-dose, dose-exploration cohorts","TREATMENT",{"masking":33,"maskingDescription":11,"whoMasked":11},"NONE",[35],{"label":36,"type":37,"description":38,"interventionNames":39},"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.",[40],"Drug: BEAM-301: Single dose of BEAM-301 administered by IV",[42],{"type":43,"name":44,"description":45,"armGroupLabels":46,"otherNames":11},"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.",[36],[48],{"name":49,"role":50,"phone":51,"phoneExt":11,"email":52},"Medical Information","CONTACT","857-327-8641","clinicalinfo@beamtx.com",[54,68,77],{"facility":55,"status":56,"city":57,"state":58,"zip":59,"country":60,"countryCode":61,"cosmosGeoPoint":62,"geoPoint":67,"contacts":11},"Clinical Study Site","RECRUITING","Orange","California","92868","United States","US",{"type":63,"coordinates":64},"Point",[65,66],-117.85311,33.78779,{"lat":66,"lon":65},{"facility":55,"status":56,"city":69,"state":70,"zip":71,"country":60,"countryCode":61,"cosmosGeoPoint":72,"geoPoint":76,"contacts":11},"Farmington","Connecticut","06030",{"type":63,"coordinates":73},[74,75],-72.83204,41.71982,{"lat":75,"lon":74},{"facility":55,"status":56,"city":78,"state":79,"zip":80,"country":60,"countryCode":61,"cosmosGeoPoint":81,"geoPoint":85,"contacts":11},"Houston","Texas","77030",{"type":63,"coordinates":82},[83,84],-95.36327,29.76328,{"lat":84,"lon":83},{"type":87,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","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":97,"type":98},36,"ESTIMATED","INTERVENTIONAL",[101,102],"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.",[105],"Glycogen Storage Disease Type Ia",[107,108],"GSDIa","GSD1a","2025-12-09",{"date":111,"type":112},"2025-12-15","ACTUAL",{"date":114,"type":112},"2024-12-06",{"date":116,"type":98},"2027-12-30",{"name":5,"class":6},3]