[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"autosomal-recessive-hypophosphatemic-rickets\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:autosomal-recessive-hypophosphatemic-rickets":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,55],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":32,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":43,"lastUpdatePostDateStruct":44,"startDateStruct":47,"completionDateStruct":49,"leadSponsor":51,"locationsCount":54},"100495668","phase-1-the-energy-study-evaluation-of-safety-and-tolerability-of-inz-701-in-infants-with-enpp1-deficiency-or-abcc6-deficiency-100495668",false,"NCT05734196","The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency","The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants With Ectonucleotide Pyrophosphatase\u002F Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency","ENERGY","Inclusion Criteria:\n\n1. Infant aged ≤ 1 year at the time of enrollment\n2. Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency\n3. Study participants must have clinical manifestations of generalized arterial calcification of infancy (GACI) or GACI-2, which must include at least one of the following: ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly.\n4. Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study\n5. Written informed consent provided by a parent or legal guardian\n\nExclusion Criteria:\n\n1. In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality that precludes study participation or may confound interpretation of study result\n2. Receiving end of life or hospice care\n3. Known malignancy\n4. Concurrent participation in another non-Inozyme interventional study\n5. Treatment with any non-Inozyme product or investigational device during study participation","ALL","1 Year",{"count":20,"type":21},16,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.",[27,28,29,30,31],"Ectonucleotide Pyrophosphatase\u002Fphosphodiesterase1 Deficiency","Autosomal Recessive Hypophosphatemic Rickets","Generalized Arterial Calcification of Infancy","ATP-Binding Cassette Subfamily C Member 6 Deficiency","Pseudoxanthoma Elasticum",[33,34,35,29,36,37,38,30,39,40,41],"ectonucleotide pyrophosphatase\u002Fphosphodiesterase1 deficiency","hypopyrophosphatemia","ENPP1","GACI","Autosomal Recessive Hypophosphatemic Rickets Type 2","ARHR2","ABCC6","Pseudoxanthoma elasticum","PXE","RECRUITING","2026-03-19",{"date":45,"type":46},"2026-03-24","ACTUAL",{"date":48,"type":46},"2023-06-25",{"date":50,"type":21},"2027-11-11",{"name":52,"class":53},"Inozyme Pharma","INDUSTRY",7,{"id":56,"slug":57,"hasResults":11,"nctId":58,"briefTitle":59,"officialTitle":60,"acronym":4,"eligibilityCriteria":61,"healthyVolunteers":11,"sex":17,"minAge":62,"maxAge":18,"enrollmentInfo":63,"targetDuration":4,"studyType":22,"phases":65,"briefSummary":67,"conditions":68,"keywords":70,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":71,"lastUpdatePostDateStruct":72,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":79},"100629384","phase-3-energy-2-evaluation-of-the-efficacy-and-safety-of-inz-701-in-infants-with-enpp1-deficiency-100629384","NCT07473973","ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency","The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants With Ectonucleotide Pyrophosphatase\u002FPhosphodiesterase 1 (ENPP1) Deficiency","Participants must meet all of the following:\n\nInclusion Criteria:\n\n1. Infant aged ≤ 1 year at the time of enrollment.\n2. Confirmed diagnosis of ENPP1 deficiency, based on genetic testing.\n3. Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcification or cardiac involvement).\n4. Medically stable to participate in a 52-week treatment study.\n5. Written informed consent provided by a parent or legal guardian.\n\nExclusion Criteria\n\nParticipants will not be eligible if any of the following apply:\n\n1. Receiving end-of-life or hospice care.\n2. Prior treatment with INZ-701, unless received through an approved expanded access program.\n3. Concurrent participation in another interventional clinical trial.\n4. Planned major surgery during the study period that would interfere with study participation.","0 Years",{"count":64,"type":21},12,[66],"PHASE3","The primary purpose of ENERGY 2 (Study INZ701-105) is to assess the efficacy and safety of INZ-701 in infants with ENPP1 Deficiency.",[27,28,69],"Generalized Arterial Calcification of Infancy 1",[29,36,37,38],"2026-03-16",{"date":73,"type":46},"2026-03-18",{"date":75,"type":46},"2025-03-26",{"date":77,"type":21},"2028-11-07",{"name":52,"class":53},8]