[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Agios Pharmaceuticals, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":105},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,39,62,82],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":29,"startDateStruct":32,"completionDateStruct":34,"leadSponsor":36,"locationsCount":4},"100641605","phase-3-a-study-to-investigate-the-effect-of-mitapivat-on-transfusion-burden-in-subjects-with-sickle-cell-disease-scd-100641605",false,"NCT07656415","A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)","A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease","Inclusion Criteria:\n\n* Age ≥12 years.\n* Documented diagnosis of SCD (hemoglobin SS (HbSS), combined heterozygosity for hemoglobins S and C (HbSC), sickle cell hemoglobin (HbS)\u002Fβ0-thalassemia, HbS\u002Fβ+-thalassemia, or other sickle cell syndrome variants).\n* No more than 10 SCPCs in the 12 months before providing informed assent\u002Fconsent.\n* At least 1 transfusion of packed RBCs in the 12 months before informed assent\u002Fconsent.\n* Hb ≥5.5 and ≤10.5 g\u002FdL. Hb concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period.\n* Additional signs or symptoms of hemolysis, as evidenced by any laboratory assessment during the Screening Period with\n\n  * Hb \\\u003C8 g\u002FdL, or\n  * Absolute reticulocyte count \\> upper limit of normal (ULN), or\n  * Indirect bilirubin \\>ULN, or\n  * LDH \\>ULN\n* If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days prior to randomization. Discontinuation of hydroxyurea requires a 90-day washout prior to informed assent\u002Fconsent.\n* Women of childbearing potential (WOCBP) and pediatric female subjects who have attained menarche must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle or agree to use a highly effective method of contraception from the time of providing informed assent\u002Fconsent, throughout the study, and for 28 days after the last dose of study drug; if the highly effective method of contraception is hormonal contraception, then an acceptable barrier method must also be used.\n* Written informed assent\u002Fconsent (for subjects under 18 years of age, or prior to the age at which a subject is considered legally an adult per local regulations, parental permission and child assent will be obtained) must be obtained before any study-related procedures are conducted and subjects must be willing to comply with all study procedures for the duration of the study.\n\nExclusion Criteria:\n\n* Pregnant, breastfeeding, or parturient.\n* Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion); episodic transfusion in response to worsened anemia or vaso-occlusive crisis (VOC) is permitted. Additionally, a subject who requires episodic transfusion(s) may not have received a transfusion(s) within 60 days before providing informed assent\u002Fconsent or during the Screening Period.\n* Hospitalized for an SCPC and\u002For other vaso-occlusive event within 14 days prior to providing informed assent\u002Fconsent or during the Screening Period. A hospitalization is defined as an in-patient admission to a hospital that may or may not be preceded by an emergency room or outpatient clinic visit. A visit to an emergency room that does not result in an in-patient admission does not meet the definition of hospitalization.\n* Currently receiving treatment with a disease-modifying therapy for SCD (eg, voxelotor, crizanlizumab, L-glutamine), with the exception of hydroxyurea. The last dose of voxelotor, crizanlizumab, and L-glutamine must have been administered at least 90 days before randomization.\n* History of any malignancy except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ. Subjects must not have active disease or received anticancer treatment ≤5 years before providing informed assent\u002Fconsent.\n* History of active and uncontrolled cardiac or pulmonary disease within 6 months before randomization, including but not limited to:\n\n  * New York Heart Association Class III or IV heart failure or clinically significant dysrhythmia.\n  * Myocardial infarction or unstable angina pectoris; hemorrhagic, embolic, or thrombotic stroke; deep venous thrombosis; or pulmonary or arterial embolism.\n  * Heart rate-corrected QT interval using Fridericia's method of ≥470 milliseconds for female subjects and ≥450 milliseconds for male subjects, except for right or left bundle branch block.\n  * Severe pulmonary fibrosis as defined by severe hypoxia, evidence of right-sided heart failure, and radiographic pulmonary fibrosis \\>50%.\n  * Severe pulmonary hypertension as defined by severe symptoms associated with hypoxia, right heart failure, and oxygen indicated.\n* Hepatobiliary disorders including but not limited to:\n\n  * Liver disease with histopathological evidence or clinical diagnosis of cirrhosis or severe fibrosis.\n  * Clinically symptomatic cholelithiasis or cholecystitis (subjects with prior cholecystectomy are eligible).\n  * History of drug-induced cholestatic hepatitis.\n  * Aspartate aminotransferase (AST) \\>2.5× ULN (unless due to hemolysis and\u002For hepatic iron deposition) and alanine aminotransferase (ALT) \\>2.5× ULN (unless due to hepatic iron deposition).\n* Renal dysfunction as defined by an estimated glomerular filtration rate \\\u003C30 milliliters per minute (mL\u002Fmin)\u002F1.73-meter square (m\\^2) by the Chronic Kidney Disease Epidemiology Collaboration creatinine equation.\n* Active uncontrolled infection requiring systemic antimicrobial therapy.\n* Positive test for hepatitis C virus (HCV) antibody (Ab) with evidence of active HCV infection, or positive test for hepatitis B surface antigen (HBsAg).\n* Positive test for human immunodeficiency virus (HIV)-1 antibody or HIV-2 antibody.\n* History of major surgery (including splenectomy) ≤16 weeks before providing informed assent\u002Fconsent and\u002For planning on undergoing a major surgical procedure during the study.\n* Current enrollment or past participation (within 90 days before randomization or a time frame equivalent to 5 half-lives of the investigational study drug, whichever is longer) in any other clinical study involving an investigational study drug or device.\n* Past enrollment in a clinical study involving mitapivat.\n* Prior exposure to gene therapy or prior bone marrow or stem cell transplantation.\n* Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered at least 90 days before randomization.\n* Receiving products that are strong inhibitors of Cytochrome3A4\u002F5 (CYP3A4\u002F5) that have not been stopped for ≥5 days or a time frame equivalent to 5 half-lives (whichever is longer), or strong inducers of Cytochrome3A4 (CYP3A4) that have not been stopped for ≥28 days or a time frame equivalent to 5 half-lives (whichever is longer), prior to randomization.\n* Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable for ≥10 weeks before randomization.\n* Known allergy to mitapivat or tablet excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, magnesium stearate, and the Opadry Blue II film-coat \\[hypromellose, titanium dioxide, lactose monohydrate, triacetin, and FD\\&C Blue #2\\]).\n* Any medical, hematological, psychological, or behavioral condition(s), including alcohol use disorder, or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and\u002For could confound the interpretation of the study data. Also excluded are:\n\n  * Subjects unable to receive RBC transfusions (eg, due to presence of allo-antibodies, lack of blood availability).\n  * Subjects deprived of liberty by court or administrative decision (eg, persons accommodated in an institution by order of an authority or court).\n  * Subjects undergoing psychiatric care without their consent.\n  * Subjects admitted to a health or social establishment for purposes other than research.\n  * Adult Subjects subject to a legal protection measure (guardian, curatorship, legal protection).\n  * Subjects unable to express their consent.\n* Receiving herbal or dietary supplements that have not been stable in dose and preparation for ≥8 weeks prior to randomization.","ALL","12 Years",{"count":19,"type":20},159,"ESTIMATED","INTERVENTIONAL",[23],"PHASE3","The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.",[26],"Sickle Cell Disease","NOT_YET_RECRUITING","2026-06-19",{"date":30,"type":31},"2026-06-23","ACTUAL",{"date":33,"type":20},"2026-08",{"date":35,"type":20},"2030-08",{"name":37,"class":38},"Agios Pharmaceuticals, Inc.","INDUSTRY",{"id":40,"slug":41,"hasResults":11,"nctId":42,"briefTitle":43,"officialTitle":44,"acronym":45,"eligibilityCriteria":46,"healthyVolunteers":11,"sex":16,"minAge":47,"maxAge":48,"enrollmentInfo":49,"targetDuration":4,"studyType":21,"phases":51,"briefSummary":52,"conditions":53,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":56,"startDateStruct":57,"completionDateStruct":59,"leadSponsor":61,"locationsCount":4},"100631913","phase-3-a-study-to-investigate-the-efficacy-pharmacokinetics-and-safety-of-mitapivat-in-pediatric-participants-with-transfusion-dependent-alpha--or-beta-thalassemia---or--tdt-100631913","NCT07506863","A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)","A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β- Transfusion-Dependent Thalassemia","ENERGIZEKids-T","Inclusion Criteria:\n\n* Written informed consent\u002Fassent from the participant (or their legally authorized representative, parent(s), or legal guardian) must be obtained before any study-related procedures are conducted and participants must be willing to comply with all study procedures for the duration of the study.\n* Aged 1 to \\\u003C18 years and weighing at least 7 kilograms (kg) at the time of providing informed consent\u002Fassent.\n* Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)\u002Fβ-thalassemia, or α-thalassemia\u002Fhemoglobin H (HbH) disease) based on Hemoglobin (Hb) electrophoresis, Hb high-performance liquid chromatography, and\u002For DNA analysis from the participant's medical record. If this information is not available from the participant's medical record, the test(s) can be performed by a local laboratory during the Screening Period. If a local laboratory is unable to perform the test(s), results from the comprehensive α- and β-globin genotyping performed by the study central laboratory can be used.\n* Transfusion dependent, defined as 6 to 20 transfusion episodes (also referred to as \"transfusion events\") and a ≤6-week transfusion-free period during the 24-week period before randomization.\n* If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization.\n* Female participants who have attained menarche must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of informed consent\u002Fassent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can include an acceptable barrier method.\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding.\n* Documented history of homozygous or heterozygous hemoglobin S (HbS) or hemoglobin C (HbC).\n* Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any prior exposure to myeloablative chemotherapy.\n* Any conditions other than thalassemia expected to affect sexual maturation.\n* Currently receiving treatment with luspatercept; the last dose must have been administered ≥36 weeks before randomization.\n* Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥36 weeks before randomization.\n* History of malignancy (active or treated) ≤5 years before providing informed consent\u002Fassent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ.\n* History of active and\u002For uncontrolled cardiac or pulmonary disease or clinically relevant QT prolongation within 6 months before providing informed consent\u002Fassent.\n* Hepatobiliary disorders, including but not limited to:\n\n  * Liver disease with histopathological evidence or clinical diagnosis of cirrhosis or severe fibrosis.\n  * Clinically symptomatic cholelithiasis or cholecystitis (prior cholecystectomy is not exclusionary).\n  * History of drug-induced cholestatic hepatitis.\n  * Aspartate Aminotransferase (AST) \\>2.5\\*Upper Limit of Normal (ULN) (unless due to hemolysis and hepatic iron deposition) and Alanine Transaminase (ALT) \\>2.5\\*ULN (unless due to hepatic iron deposition).\n* Renal dysfunction as defined by an estimated glomerular filtration rate \\\u003C60 milliliters per minute (mL\u002Fmin)\u002F1.73-meter square (m\\^2).\n* Nonfasting triglycerides \\>215 milligrams per deciliter (mg\u002FdL) \\[5 millimole per liter (mmol\u002FL)\\].\n* Active infection requiring systemic antimicrobial therapy at the time of providing informed consent\u002Fassent. If antimicrobial therapy is required during the Screening Period, screening procedures should not be performed while antimicrobial therapy is being administered, and the last dose of antimicrobial therapy must be administered ≥7 days before randomization.\n* Participants with known active hepatitis B or hepatitis C virus infection.\n* Participants with known human immunodeficiency virus (HIV) infection.\n* History of major surgery (including splenectomy) ≤ 6 months before providing informed consent\u002Fassent and\u002For a major surgical procedure planned during the study.\n* Current enrollment or past participation (within ≤12 weeks or a timeframe equivalent to 5 half-lives of the investigational study drug before administration of the first dose of study drug, whichever is longer) in any other clinical study involving an investigational treatment or device.\n* Receiving strong Cytochrome3A4\u002F5 (CYP3A4\u002F5) inhibitors that have not been stopped for ≥5 days or a timeframe equivalent to 5 half-lives (whichever is longer), or strong CYP3A4\u002F5 inducers that have not been stopped for ≥4 weeks or a timeframe equivalent to 5 half-lives (whichever is longer), before randomization.\n* Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable for ≥12 weeks before randomization.\n* Known allergy, or other contraindication, to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, magnesium stearate, and the Opadry filmcoat \\[hypromellose, titanium dioxide, lactose monohydrate triacetin, and FD\\&C Blue #2\\]).\n* Any medical, hematological, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and\u002For could confound the interpretation of the study data. Also excluded are:\n\n  * Participants who are institutionalized by regulatory or court order.\n  * Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor).","1 Year","17 Years",{"count":50,"type":20},54,[23],"The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.",[54,55],"Transfusion-dependent Alpha-Thalassemia","Transfusion-dependent Beta-Thalassemia",{"date":30,"type":31},{"date":58,"type":20},"2026-09",{"date":60,"type":20},"2032-06",{"name":37,"class":38},{"id":63,"slug":64,"hasResults":11,"nctId":65,"briefTitle":66,"officialTitle":67,"acronym":68,"eligibilityCriteria":69,"healthyVolunteers":11,"sex":16,"minAge":47,"maxAge":48,"enrollmentInfo":70,"targetDuration":4,"studyType":21,"phases":72,"briefSummary":73,"conditions":74,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":77,"startDateStruct":78,"completionDateStruct":79,"leadSponsor":81,"locationsCount":4},"100632703","phase-3-a-study-to-investigate-the-efficacy-pharmacokinetics-and-safety-of-mitapivat-in-pediatric-participants-with---or--non-transfusion-dependent-thalassemia-100632703","NCT07517133","A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia","A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β-Non-Transfusion-Dependent Thalassemia","ENERGIZEKids","Inclusion Criteria:\n\n* Written informed consent\u002Fassent from the participant (or their legally authorized representative, parent(s), or legal guardian) must be obtained before any study-related procedures are conducted and participants must be willing to comply with all study procedures for the duration of the study.\n* Aged 1 to \\\u003C18 years and weighing at least 7 kilograms (kg) at the time of providing informed consent\u002Fassent.\n* Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)\u002Fβ-thalassemia, or α-thalassemia\u002Fhemoglobin H (HbH) disease) based on Hemoglobin (Hb) electrophoresis, Hb high-performance liquid chromatography, and\u002For DNA analysis from the participant's medical record. If this information is not available from the participant's medical record, the test(s) can be performed by a local laboratory during the Screening Period. If a local laboratory is unable to perform the test(s), results from the comprehensive α- and β-globin genotyping performed by the study central laboratory can be used.\n* Hb concentration ≤10.0 grams per deciliter (g\u002FdL) \\[100.0 grams per liter (g\u002FL)\\], based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period.\n* Non-transfusion dependent, defined as ≤5 transfusion episodes (also referred to as \"transfusion events\") during the 24-week period before randomization and no red blood cells (RBC) transfusions ≤8 weeks before providing informed consent\u002Fassent and no RBC transfusions during the Screening Period.\n* If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization.\n* Female participants who have attained menarche must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of informed consent\u002Fassent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can include an acceptable barrier method.\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding.\n* Documented history of homozygous or heterozygous hemoglobin S (HbS) or hemoglobin C (HbC).\n* Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any prior exposure to myeloablative chemotherapy.\n* Any conditions other than thalassemia expected to affect sexual maturation.\n* Currently receiving treatment with luspatercept; the last dose must have been administered ≥18 weeks before randomization.\n* Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥18 weeks before randomization.\n* History of malignancy (active or treated) ≤5 years before providing informed consent\u002Fassent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ.\n* History of active and\u002For uncontrolled cardiac or pulmonary disease or clinically relevant QT prolongation within 6 months before providing informed consent\u002Fassent.\n* Hepatobiliary disorders, including but not limited to:\n\n  * Liver disease with histopathological evidence or clinical diagnosis of cirrhosis or severe fibrosis.\n  * Clinically symptomatic cholelithiasis or cholecystitis (prior cholecystectomy is not exclusionary).\n  * History of drug-induced cholestatic hepatitis.\n  * Aspartate Aminotransferase (AST) \\>2.5\\*Upper Limit of Normal (ULN) (unless due to hemolysis and hepatic iron deposition) and Alanine Transaminase (ALT) \\>2.5\\*ULN (unless due to hepatic iron deposition).\n* Renal dysfunction as defined by an estimated glomerular filtration rate \\\u003C60 milliliters per minute (mL\u002Fmin)\u002F1.73-meter square (m\\^2).\n* Nonfasting triglycerides \\>215 milligrams per deciliter (mg\u002FdL) \\[5 millimole per liter (mmol\u002FL)\\].\n* Active infection requiring systemic antimicrobial therapy at the time of providing informed consent\u002Fassent. If antimicrobial therapy is required during the Screening Period, screening procedures should not be performed while antimicrobial therapy is being administered, and the last dose of antimicrobial therapy must be administered ≥7 days before randomization.\n* Participants with known active hepatitis B or hepatitis C virus infection.\n* Participants with known human immunodeficiency virus (HIV) infection.\n* History of major surgery (including splenectomy) ≤16 weeks before providing informed consent\u002Fassent and\u002For a major surgical procedure planned during the study.\n* Current enrollment or past participation (within ≤12 weeks or a timeframe equivalent to 5 half-lives of the investigational study drug before administration of the first dose of study drug, whichever is longer) in any other clinical study involving an investigational treatment or device.\n* Receiving strong Cytochrome3A4\u002F5 (CYP3A4\u002F5) inhibitors that have not been stopped for ≥5 days or a timeframe equivalent to 5 half-lives (whichever is longer), or strong CYP3A4\u002F5 inducers that have not been stopped for ≥4 weeks or a timeframe equivalent to 5 half-lives (whichever is longer), before randomization.\n* Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable for ≥12 weeks before randomization.\n* Known allergy, or other contraindication, to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, magnesium stearate, and the Opadry filmcoat \\[hypromellose, titanium dioxide, lactose monohydrate triacetin, and FD\\&C Blue #2\\]).\n* Any medical, hematological, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and\u002For could confound the interpretation of the study data. Also excluded are:\n\n  * Participants who are institutionalized by regulatory or court order.\n  * Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor).",{"count":71,"type":20},45,[23],"The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.",[75,76],"Non-Transfusion-dependent Alpha-Thalassemia","Non-Transfusion-dependent Beta-Thalassemia",{"date":30,"type":31},{"date":58,"type":20},{"date":80,"type":20},"2032-03",{"name":37,"class":38},{"id":83,"slug":84,"hasResults":11,"nctId":85,"briefTitle":86,"officialTitle":87,"acronym":4,"eligibilityCriteria":88,"healthyVolunteers":11,"sex":16,"minAge":89,"maxAge":90,"enrollmentInfo":91,"targetDuration":4,"studyType":21,"phases":93,"briefSummary":95,"conditions":96,"keywords":4,"overallStatus":98,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":99,"startDateStruct":100,"completionDateStruct":102,"leadSponsor":104,"locationsCount":5},"100611488","phase-1-a-study-to-evaluate-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-ag-181-in-subjects-with-phenylketonuria-100611488","NCT07241234","A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria","A Phase 1b, Open-label, Multicenter, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of AG-181 in Subjects With Phenylketonuria","Key Inclusion Criteria:\n\n* Diagnosis of PKU, defined as documented presence of 2 mutant alleles in the phenylalanine hydroxylase (PAH) gene, of which at least 1 is the R408W mutation, as determined during Screening per the genotyping performed by the study central genotyping laboratory.\n* At least 1 plasma Phe concentration greater than (\\>) 600 micromoles per liter (μmol\u002FL) in the 52 weeks before providing informed consent.\n* Average concentration of plasma Phe \\> 600 μmol\u002FL in Phe samples taken during Screening, with no individual assessment below 360 μmol\u002FL. Any Phe samples taken after Day -20 will not be included.\n* Body mass index (BMI) greater than or equal to (≥) 18.0 kilograms per meter square (kg\u002Fm\\^2) to lesser than or equal to (≤) 35.0 kg\u002Fm\\^2 and weight ≥ 50 kilograms (kg) at any time during the Screening Period.\n* Documented approval from a dietitian confirming that the subject can maintain their diet consistent in protein and Phe intake throughout the study as outlined in the Diet Manual.\n\nKey Exclusion Criteria:\n\n* Prior exposure to AG-181.\n* Receiving inhibitors of P-glycoprotein (P-gp) that have not been stopped for ≥ 5 days or a timeframe equivalent to 5 half-lives (whichever is longer) before administration of the first dose of study drug.\n* Receiving products that are strong inhibitors or strong inducers of cytochrome P450 CYP1A2, CYP2C8, or CYP3A that have not been stopped for ≥ 28 days before administration of the first dose of study drug.\n* Receiving treatment with an acid-reducing agent, including but not limited to proton pump inhibitors and H2 blockers. Short-acting acid-reducing agents such as calcium carbonate are permitted.\n* Any preexisting condition that could (in the opinion of the Investigator) interfere with gastrointestinal anatomy or motility that may disrupt the absorption, metabolism, and\u002For excretion of the study drug.\n* Any preexisting condition that could (in the opinion of the Investigator) interfere with hepatic or renal function that may disrupt the absorption, metabolism, and\u002For excretion of the study drug.\n* Inability to tolerate oral medication.\n* Unwillingness to washout from tetrahydrobiopterin (BH4) supplementation (eg, sapropterin dihydrochloride, Kuvan), pegvaliase-pqpz (Palynziq), or any other PKU therapy by Day -30 during Screening.","18 Years","69 Years",{"count":92,"type":20},20,[94],"PHASE1","The primary purpose of this study is to assess the safety and tolerability of AG-181 in subjects with Phenylketonuria (PKU).",[97],"Phenylketonuria","RECRUITING",{"date":30,"type":31},{"date":101,"type":31},"2026-04-17",{"date":103,"type":20},"2028-01-17",{"name":37,"class":38},""]