[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Beam Therapeutics Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":119},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,42,67,95],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100546049","phase-1-a-study-to-evaluate-the-safety-and-efficacy-of-beam-302-in-adult-patients-with-alpha-1-antitrypsin-deficiency-aatd-100546049",false,"NCT06389877","A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)","A Phase 1\u002F2 Dose-exploration and Dose-expansion Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and\u002For Liver Disease","Part A:\n\nInclusion Criteria:\n\n* Males or females 18 - 70 years of age inclusive at the time of consent.\n* Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).\n* Blood total AAT level \\\u003C11 μM or equivalent protein in mg\u002FdL.\n* Patients receiving augmentation therapy in regions where augmentation is not SoC must be willing to washout augmentation therapy for at least 6 weeks prior to signing the ICF and for the length of the study (unless clinically indicated)\n* A postbronchodilator FEV1 ≥40% of predicted and an FEV1\u002FFVC \\\u003C70% at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)\n* Evidence of emphysema on a historic CT scan or a DLCO ≤70% of the predicted value (corrected for hemoglobin) at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)\n\nExclusion Criteria:\n\n* Body mass index \\>30\n* Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.\n* Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \\[\\>4x\u002Fyear\\]).\n* Liver disease with any of the following:\n\n  * FibroScan liver stiffness measurement ≥7.5 kilopascals (kPa). (For sites without access to FibroScan, APRI \\>0.5 can be used as a surrogate exclusion criterion \\[Yilmaz, 2011\\].\n  * Known history of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).\n  * Presence of ≥F2 liver fibrosis if a patient has previously had a liver biopsy.\n  * Have ALT or AST \\> upper limit of normal (ULN).\n  * Total bilirubin levels \\> ULN; if documented Gilbert's Syndrome, total bilirubin \\>2 × ULN.\n  * INR ≥1.2 at screening. If deemed appropriate by the investigator and\u002For prescribing physician, the patient may stop taking anticoagulants for an appropriate washout period or reversal with vitamin K and if indicated, a repeat INR within \\\u003C1.2 would be acceptable.\n  * Seropositive for hepatitis B (positive surface Ag).\n  * Active hepatitis C by hepatitis C virus (HCV) antibody. If HCV antibody positive, must be HCV RNA polymerase chain reaction (PCR) negative.\n\nPart B:\n\nInclusion Criteria:\n\n* Males or females 18 - 70 years of age inclusive at the time of consent.\n* Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing).\n* Evidence of METAVIR F1, F2, or F3 liver fibrosis based on a central read of a baseline liver biopsy during the screening period or a histological diagnosis made no more than 6 months before enrollment and stage confirmed by central read.\n* A postbronchodilator FEV1 ≥40% of predicted at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.)\n\nExclusion Criteria:\n\n* Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery.\n* Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \\[\\>4x\u002Fyear\\])\n* Previous diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).","ALL","18 Years","70 Years",{"count":20,"type":21},106,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This is a Phase 1\u002F2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK\u002FPD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and\u002For liver disease and to determine the optimal biological dose (OBD).",[28],"Alpha 1-Antitrypsin Deficiency","RECRUITING","2026-03-18",{"date":32,"type":33},"2026-03-20","ACTUAL",{"date":35,"type":33},"2024-06-19",{"date":37,"type":21},"2030-05",{"name":39,"class":40},"Beam Therapeutics Inc.","INDUSTRY",11,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":4,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":16,"minAge":49,"maxAge":50,"enrollmentInfo":51,"targetDuration":4,"studyType":53,"phases":4,"briefSummary":54,"conditions":55,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":66},"100621670","a-long-term-follow-up-study-in-patients-who-received-beam-101-100621670","NCT07373639","A Long-term Follow-up Study in Patients Who Received BEAM-101","A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells","Inclusion Criteria:\n\n1. Have received BEAM-101 in Study BTX-AUT-001 and are in the process of completing that study's end-of-study (EOS) visit.\n2. Provide signed, written informed consent according to local institutional review board (IRB)\u002Findependent ethics committee (IEC) and institutional requirements.\n\nExclusion Criteria:\n\n* There are no exclusion criteria for this study.","14 Years","37 Years",{"count":52,"type":21},50,"OBSERVATIONAL","This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing Study BTX-AUT-001. Patients who enter into this study will be followed for 13 years (a total of 15 years after receiving BEAM-101).",[56,57],"Hemoglobinopathy","Sickle Cell Disease","2026-01-23",{"date":60,"type":33},"2026-01-28",{"date":62,"type":33},"2025-12-15",{"date":64,"type":21},"2043-02-12",{"name":39,"class":40},17,{"id":68,"slug":69,"hasResults":11,"nctId":70,"briefTitle":71,"officialTitle":72,"acronym":4,"eligibilityCriteria":73,"healthyVolunteers":74,"sex":16,"minAge":17,"maxAge":75,"enrollmentInfo":76,"targetDuration":4,"studyType":22,"phases":78,"briefSummary":79,"conditions":80,"keywords":83,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":86,"lastUpdatePostDateStruct":87,"startDateStruct":89,"completionDateStruct":91,"leadSponsor":93,"locationsCount":94},"100616375","phase-1-this-is-a-phase-1-randomized-single-blind-placebo-controlled-study-to-assess-the-safety-pharmacokinetics-pk-and-pharmacodynamics-pd-of-beam-103-in-healthy-subjects-100616375","NCT07304791","This is a Phase 1, Randomized, Single-blind, Placebo-controlled Study to Assess the Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of BEAM-103 in Healthy Subjects","A Randomized, Single-Blind, Placebo-Controlled, Phase 1 Single Ascending Dose Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of BEAM-103 in Healthy Subjects","Inclusion Criteria:\n\n* Ages 18 to ≤55 years\n* Body mass index (BMI) of 18.5 to 25 kg\u002Fm2\n* Morning neutrophil count ≥2.6 × 109 cells\u002FL for Black\u002FAfrican American subjects or ≥3.5 ×109 cells\u002FL for Caucasian\u002FOther subjects\n* Baseline hemoglobin value of ≥14 g\u002FdL for males and ≥12 g\u002FdL for females\n* Baseline platelet count of \\>150 × 109\u002FL.\n* Subjects must be in general good health without significant medical conditions (based on physical exam, electrocardiogram (ECG), and laboratory test results with no clinically significant deviations), per the investigator's assessment\n\nExclusion Criteria:\n\n* Known hypersensitivity to any component of the investigational medicinal product (IMP).\n* Participation in another clinical trial involving treatment with an investigational agent within 90 days of informed consent is prohibited.\n* Positive serum pregnancy test or breastfeeding at screening (female participants).\n* Live virus vaccination within 4 weeks prior to signing informed consent.\n* Any severe or uncontrolled medical condition (eg. severe asthma or severe peanut allergy) that in the opinion of the investigator would put the subject at undue medical risk or impair the ability to interpret study results.",true,"55 Years",{"count":77,"type":21},30,[24],"The purpose of this Phase I single ascending dose study is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of BEAM-103 in healthy subjects between the ages of 18 and 55. The main questions the study aims to answer are:\n\n* Safety and tolerability of BEAM-103\n* The pharmacokinetic (PK) profile and pharmacodynamic (PD) characteristics of EAM-103\n* The effect of BEAM-103 on hematologic parameters\n* To assess the immunogenicity of BEAM-103\n\nResearchers will determine and establish the therapeutic window of the antibody and select optimal doses for future trials in patients.\n\nSubjects will:\n\n* Be asked to participate in the study for a duration of 4-5 months total\n* Be asked to sign informed consent\n* Be assessed for eligibility\n* Provide medical and medication history\n* Receive a single intravenous infusion of BEAM-103 or placebo on study Day 1 according to their assigned cohort\n* Be followed up to 4 months after infusion",[81,82],"Healthy Subjects","Healthy Participant Study",[84,85],"ESCAPE","BEAM-103","2025-12-12",{"date":88,"type":33},"2025-12-26",{"date":90,"type":33},"2025-10-27",{"date":92,"type":21},"2026-05-29",{"name":39,"class":40},1,{"id":96,"slug":97,"hasResults":11,"nctId":98,"briefTitle":99,"officialTitle":100,"acronym":4,"eligibilityCriteria":101,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":102,"targetDuration":4,"studyType":22,"phases":104,"briefSummary":105,"conditions":106,"keywords":108,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":111,"lastUpdatePostDateStruct":112,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":118},"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).",{"count":103,"type":21},36,[24,25],"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.",[107],"Glycogen Storage Disease Type Ia",[109,110],"GSDIa","GSD1a","2025-12-09",{"date":62,"type":33},{"date":114,"type":33},"2024-12-06",{"date":116,"type":21},"2027-12-30",{"name":39,"class":40},3,""]