[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100495668":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":26,"locations":35,"responsibleParty":140,"collaborators":142,"id":144,"slug":145,"hasResults":146,"nctId":147,"briefTitle":148,"officialTitle":149,"acronym":150,"eligibilityCriteria":151,"healthyVolunteers":146,"sex":152,"minAge":31,"maxAge":153,"enrollmentInfo":154,"targetDuration":31,"studyType":157,"phases":158,"briefSummary":160,"conditions":161,"keywords":167,"overallStatus":38,"whyStopped":31,"lastUpdateSubmitDate":177,"lastUpdatePostDateStruct":178,"startDateStruct":181,"completionDateStruct":183,"leadSponsor":185,"locationsCount":186},{"fullName":5,"class":6},"Inozyme Pharma","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"INZ-701","EXPERIMENTAL","The first 2 study participants will receive 1 dose of 0.2 mg\u002Fkg on Day 1 and start at Dose Level A (0.2 mg\u002Fkg twice weekly) on Day 8.\n\nThe Data Review Committee (DRC) comprised of representatives of the Sponsor, the study Investigators, and a physician who is a subject matter expert not affiliated with the study or Sponsor, will review the safety data of the first study participant through Day 8. Contingent upon this review, the Sponsor will decide if additional study participants can begin receiving INZ-701.\n\nAfter the second study participant completes Day 32, the DRC will perform a cumulative review of safety and PK\u002FPD data and will make dosing recommendations, for example, modifying the dose of the ongoing study participants and\u002For changing the starting dose for future participants.\n\nDose Level A: 0.2 mg\u002Fkg twice weekly\n\nDose Level B: 0.6 mg\u002Fkg twice weekly\n\nDose Level C: 0.2 mg\u002Fkg once weekly\n\nDose Level D: 0.6 mg\u002Fkg once weekly\n\nDose Level E: 1.8 mg\u002Fkg once weekly",[13],"Drug: INZ-701",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.",[9],[20],"(rhENPP1-Fc).",[22],{"name":23,"affiliation":24,"role":25},"Medical Director, MD, MD","BioMarin Pharmaceutical","STUDY_DIRECTOR",[27,33],{"name":28,"role":29,"phone":30,"phoneExt":31,"email":32},"Trial Specialist","CONTACT","+1 800-983-4587",null,"medinfo@bmrn.com",{"name":34,"role":29,"phone":30,"phoneExt":31,"email":32},"Medical Director, MD",[36,59,70,89,108,118,129],{"facility":37,"status":38,"city":39,"state":40,"zip":41,"country":42,"countryCode":43,"cosmosGeoPoint":44,"geoPoint":49,"contacts":50},"Rady Children's Hospital","RECRUITING","San Diego","California","92123","United States","US",{"type":45,"coordinates":46},"Point",[47,48],-117.16472,32.71571,{"lat":48,"lon":47},[51,55],{"name":52,"role":29,"phone":53,"phoneExt":31,"email":54},"Nathaly Sweeney, MD","8589665818","nsweeney@rchsd.org",{"name":56,"role":29,"phone":57,"phoneExt":31,"email":58},"Sarah Lazar, MPH","8585761700","slazar@health.ucsd.edu",{"facility":60,"status":61,"city":62,"state":63,"zip":64,"country":42,"countryCode":43,"cosmosGeoPoint":65,"geoPoint":69,"contacts":31},"Boston Children's Hospital","WITHDRAWN","Boston","Massachusetts","02115",{"type":45,"coordinates":66},[67,68],-71.05977,42.35843,{"lat":68,"lon":67},{"facility":71,"status":38,"city":72,"state":73,"zip":74,"country":42,"countryCode":43,"cosmosGeoPoint":75,"geoPoint":79,"contacts":80},"Nationwide Children's Hospital","Columbus","Ohio","43205",{"type":45,"coordinates":76},[77,78],-82.99879,39.96118,{"lat":78,"lon":77},[81,85],{"name":82,"role":29,"phone":83,"phoneExt":31,"email":84},"Bimal Chaudhari, MD","614-722-3535","bimal.chaudhari@nationwidechildrens.org",{"name":86,"role":29,"phone":87,"phoneExt":31,"email":88},"Marina Artemova, PhD","614-722-2655","marina.artemova@nationwidechildrens.org",{"facility":90,"status":38,"city":91,"state":92,"zip":93,"country":42,"countryCode":43,"cosmosGeoPoint":94,"geoPoint":98,"contacts":99},"The Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"type":45,"coordinates":95},[96,97],-75.16362,39.95238,{"lat":97,"lon":96},[100,104],{"name":101,"role":29,"phone":102,"phoneExt":31,"email":103},"Olivia Lucas","267-995-0907","lucaso@chop.edu",{"name":105,"role":29,"phone":106,"phoneExt":31,"email":107},"Emily McCoy","267-251-4268","mccoye3@chop.edu",{"facility":109,"status":61,"city":110,"state":111,"zip":112,"country":42,"countryCode":43,"cosmosGeoPoint":113,"geoPoint":117,"contacts":31},"The University of Utah","Salt Lake City","Utah","84108",{"type":45,"coordinates":114},[115,116],-111.89105,40.76078,{"lat":116,"lon":115},{"facility":119,"status":120,"city":121,"state":31,"zip":31,"country":122,"countryCode":123,"cosmosGeoPoint":124,"geoPoint":128,"contacts":31},"Hospital Sant Joan de Déu","TERMINATED","Barcelona","Spain","ES",{"type":45,"coordinates":125},[126,127],2.15899,41.38879,{"lat":127,"lon":126},{"facility":130,"status":120,"city":131,"state":31,"zip":132,"country":133,"countryCode":134,"cosmosGeoPoint":135,"geoPoint":139,"contacts":31},"Royal Manchester Children's Hospital","Manchester","M13 9WL","United Kingdom","UK",{"type":45,"coordinates":136},[137,138],-2.23743,53.48095,{"lat":138,"lon":137},{"type":141,"investigatorFullName":31,"investigatorTitle":31,"investigatorAffiliation":31,"oldNameTitle":31,"oldOrganization":31},"SPONSOR",[143],{"name":24,"class":6},"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":155,"type":156},16,"ESTIMATED","INTERVENTIONAL",[159],"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.",[162,163,164,165,166],"Ectonucleotide Pyrophosphatase\u002Fphosphodiesterase1 Deficiency","Autosomal Recessive Hypophosphatemic Rickets","Generalized Arterial Calcification of Infancy","ATP-Binding Cassette Subfamily C Member 6 Deficiency","Pseudoxanthoma Elasticum",[168,169,170,164,171,172,173,165,174,175,176],"ectonucleotide pyrophosphatase\u002Fphosphodiesterase1 deficiency","hypopyrophosphatemia","ENPP1","GACI","Autosomal Recessive Hypophosphatemic Rickets Type 2","ARHR2","ABCC6","Pseudoxanthoma elasticum","PXE","2026-03-19",{"date":179,"type":180},"2026-03-24","ACTUAL",{"date":182,"type":180},"2023-06-25",{"date":184,"type":156},"2027-11-11",{"name":5,"class":6},7]