[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Inozyme Pharma\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":134},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,55,80,109],{"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,{"id":81,"slug":82,"hasResults":11,"nctId":83,"briefTitle":84,"officialTitle":85,"acronym":4,"eligibilityCriteria":86,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":87,"targetDuration":89,"studyType":90,"phases":4,"briefSummary":91,"conditions":92,"keywords":94,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":100,"lastUpdatePostDateStruct":101,"startDateStruct":103,"completionDateStruct":105,"leadSponsor":107,"locationsCount":108},"100539330","propel---a-prospective-observational-patient-registry-to-evaluate-enpp1-and-abcc6-deficiency-100539330","NCT06302439","PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency","A Prospective Observational Patient Registry to Evaluate Disease Progression in Patients With ENPP1 Deficiency and Infantile-Onset ABCC6 Deficiency (GACI Type 2)","Inclusion Criteria:\n\nIndividuals eligible to participate must meet all the following inclusion criteria:\n\n1. Must provide written or electronic consent after the nature of the registry has been explained, and prior to any research-related procedures, per International Council for Harmonisation (ICH) Good Clinical Practice (GCP)\n2. Agree to provide access to relevant medical records\n3. One of the following genetic or clinical criteria\n\n   1. A confirmed prenatal or postnatal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists\u002FClinical Laboratory Improvement Amendments (CAP\u002FCLIA) certified laboratory or regional equivalent\n\n      OR\n   2. Monoallelic ENPP1 mutation confirmed by a certified CAP\u002FCLIA laboratory or regional equivalent and any of the following clinical symptoms:\n\n   i. ≥ 1 traumatic vertebral fracture\n\nii. ≥ 2 fractures as an adult (eg, long-bones, digits, vertebrae)\n\niii. Low bone mineral density (dual-energy X-ray absorptiometry \\[DXA\\] Z-score \\\u003C1.5) and \\\u003C55 years of age\n\niv. Bone or joint pain interfering with movement or daily activities\n\nv. History of myocardial infarction (MI), unstable angina, transient ischemic attack (TIA) or low cardiac output before the age of 40 yrs.\n\nvi. History of rickets or bone deformity\n\nvii. Diagnosis of ossification of the posterior longitudinal ligament (OPLL)\n\nviii. Other clinical symptoms, with approval by Inozyme\n\nOR\n\nc. A confirmed prenatal or postnatal molecular genetic diagnosis of ABCC6 Deficiency with biallelic mutations confirmed by a certified CAP\u002FCLIA laboratory or regional equivalent, and \\\u003C18 years of age\n\nExclusion Criteria:\n\nIndividuals who meet the following exclusion criteria will not be eligible to participate:\n\n1. Participant or their legally designated representative does not have the cognitive capacity to provide informed consent\n2. Patients who are currently participating in an INZ-701 interventional clinical study, with the exception of expanded access programs and long-term safety follow-up studies\n\n   1. Participants in interventional studies may be approached for inclusion in the registry once their involvement in the treatment period of the clinical study has been completed",{"count":88,"type":21},1000,"10 Years","OBSERVATIONAL","The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase\u002Fphosphodiesterase1(ENPP1) Deficiency and the infantile-onset form of adenosine triphosphate (ATP) binding cassette transporter protein subfamily C member 6 (ABCC6) Deficiency longitudinally. The registry will prospectively gather information about the genetic, biochemical, physiological, anatomic, radiographic, and functional manifestations (including patient reported outcomes \\[PROs\\]) of each disease during routine, standard-of-care visits, with the aim of developing a comprehensive understanding of the burden of illness and progressive nature of the disease.",[93,30],"Ectonucleotide Pyrophosphatase\u002FPhosphodiesterase 1 Deficiency",[95,35,29,36,37,38,96,97,30,39,98,99],"Ectonucleotide pyrophosphatase","Observational","Registry","PROPEL","Phosphodiesterase 1","2025-12-15",{"date":102,"type":46},"2025-12-22",{"date":104,"type":46},"2024-07-25",{"date":106,"type":21},"2034-05",{"name":52,"class":53},14,{"id":110,"slug":111,"hasResults":11,"nctId":112,"briefTitle":113,"officialTitle":114,"acronym":4,"eligibilityCriteria":115,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":116,"targetDuration":4,"studyType":22,"phases":118,"briefSummary":120,"conditions":121,"keywords":124,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":125,"lastUpdatePostDateStruct":126,"startDateStruct":128,"completionDateStruct":130,"leadSponsor":132,"locationsCount":133},"100551627","phase-2-adapt-study-long-term-safety-study-of-inz-701-in-patients-with-enpp1-deficiency-and-abcc6-deficiency-100551627","NCT06462547","ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency","The ADAPT Study: An Open-label, Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency","Individuals eligible to participate must meet all of the following inclusion criteria:\n\n1. Provide written or electronic informed consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP)\n2. Provide assent in accordance with local regulations, if \\\u003C18 years of age\n3. Male or female, greater than 1 year of age\n4. Must have completed the protocol-required safety and PK\u002FPD and\u002For efficacy period(s) of a previous INZ-701 clinical study in ENPP1 or ABCC6 Deficiency, as confirmed by the Sponsor\n5. Female participants of childbearing potential who are sexually active must be using or agree to use 1 highly effective form of contraception (per CTFG 2020) from at least 1 month before the first dose of INZ-701 through 30 days after last dose of INZ-701 (greater than 5 half-lives of INZ-701); participants must agree to not donate ova from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701\n6. Male participants who are sexually active must agree to use condoms from the period following the first dose of INZ-701 through 30 days after the last dose of INZ-701; participants must agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701\n7. In the opinion of the Investigator, able to complete all aspects of the study\n\nIndividuals who meet any of the following exclusion criteria will not be eligible to participate:\n\n1. In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality not associated with ENPP1 Deficiency or ABCC6 Deficiency, that will preclude study participation and\u002For may confound interpretation of study results\n2. Known intolerance to INZ-701 or any of its excipients\n3. Concurrent participation in another interventional clinical study and\u002For has received an investigational drug other than INZ-701 within 5 half-lives or within 4 weeks prior to the first dose of INZ-701 in this study, whichever is longer, or use of an investigational device\n4. Pregnant, trying to become pregnant, or breastfeeding\n5. Male participants trying to father a child",{"count":117,"type":21},200,[119],"PHASE2","The purpose of this study (Study INZ701-304 \\[ADAPT\\]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to continue dosing for the potential treatment of their condition.",[122,31,123,27,37],"Gene Mutations","Arterial Calcification",[39,30,41,40,36,29,38,37,35,33,34],"2024-11-26",{"date":127,"type":46},"2024-11-29",{"date":129,"type":46},"2024-06-19",{"date":131,"type":21},"2030-12",{"name":52,"class":53},5,""]