[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100641521":3},{"organization":4,"armGroups":7,"interventions":24,"overallOfficials":39,"centralContacts":43,"locations":52,"responsibleParty":67,"collaborators":30,"id":70,"slug":30,"hasResults":71,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":30,"eligibilityCriteria":75,"healthyVolunteers":71,"sex":76,"minAge":77,"maxAge":78,"enrollmentInfo":79,"targetDuration":30,"studyType":82,"phases":83,"briefSummary":85,"conditions":86,"keywords":30,"overallStatus":54,"whyStopped":30,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":92,"completionDateStruct":94,"leadSponsor":96,"locationsCount":97},{"fullName":5,"class":6},"Mayo Clinic","OTHER",[8,14,19],{"label":9,"type":10,"description":11,"interventionNames":12},"Gene Therapy First Cohort (3 patients)","EXPERIMENTAL","AAVrh10-PCCA, single dose of 2 x 10\\^12 vg per kilogram of body weight (first three patients), IV administration",[13],"Drug: AAVrh10-PCCA low dose",{"label":15,"type":10,"description":16,"interventionNames":17},"Gene Therapy Second Cohort (3 patients)","AAVrh10-PCCA, single dose of 8 x 10\\^12 vg per kilogram of body weight (middle three patients), IV administration",[18],"Drug: AAVrh10-PCCA middle dose",{"label":20,"type":10,"description":21,"interventionNames":22},"Gene Therapy Third Cohort (3 patients)","AAVrh10-PCCA, single dose of 3.2 x 10\\^13 vg per kilogram of body weight (last three patients), IV administration",[23],"Drug: AAVrh10-PCCA high dose",[25,31,35],{"type":26,"name":27,"description":28,"armGroupLabels":29,"otherNames":30},"DRUG","AAVrh10-PCCA low dose","AAVrh10-PCCA (Dose of 2 x 10\\^12 vg per kg body weight) is an adeno-associated viral vector containing the adeno-associated virus terminal repeat sequences flanking a transgene cassette harboring the cytomegalovirus (CMV) immediate-early enhancer and beta actin promoter, the human PCCA cDNA, and the bovine growth hormone polyadenylation sequence.",[9],null,{"type":26,"name":32,"description":33,"armGroupLabels":34,"otherNames":30},"AAVrh10-PCCA middle dose","AAVrh10-PCCA (Dose of 8 x 10\\^12 vg per kg body weight) is an adeno-associated viral vector containing the adeno-associated virus terminal repeat sequences flanking a transgene cassette harboring the cytomegalovirus (CMV) immediate-early enhancer and beta actin promoter, the human PCCA cDNA, and the bovine growth hormone polyadenylation sequence.",[15],{"type":26,"name":36,"description":37,"armGroupLabels":38,"otherNames":30},"AAVrh10-PCCA high dose","AAVrh10-PCCA (Dose of 3.2 x 10\\^13 vg per kg body weight) is an adeno-associated viral vector containing the adeno-associated virus terminal repeat sequences flanking a transgene cassette harboring the cytomegalovirus (CMV) immediate-early enhancer and beta actin promoter, the human PCCA cDNA, and the bovine growth hormone polyadenylation sequence.",[20],[40],{"name":41,"affiliation":5,"role":42},"David R. Deyle, MD","PRINCIPAL_INVESTIGATOR",[44,49],{"name":45,"role":46,"phone":47,"phoneExt":30,"email":48},"Clinical Genomics Clinical Research Team","CONTACT","507-538-6151","rstcgresearch@mayo.edu",{"name":50,"role":46,"phone":30,"phoneExt":30,"email":51},"Wyatt Anians, M.S., CCRP","anians.wyatt@mayo.edu",[53],{"facility":5,"status":54,"city":55,"state":56,"zip":57,"country":58,"cosmosGeoPoint":59,"geoPoint":64,"contacts":65},"RECRUITING","Rochester","Minnesota","55905","United States",{"type":60,"coordinates":61},"Point",[62,63],-92.4699,44.02163,{"lat":63,"lon":62},[66],{"name":45,"role":46,"phone":30,"phoneExt":30,"email":48},{"type":42,"investigatorFullName":68,"investigatorTitle":69,"investigatorAffiliation":5,"oldNameTitle":30,"oldOrganization":30},"David R. Deyle","Principal Investigator","100641521",false,"NCT07643844","AAVrh10-PCCA Gene Therapy for Propionic Acidemia","Phase 1 Study of Intravenous Administration of a Serotype rh.10 Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human Propionyl-CoA Carboxylase cDNA (AAVrh10-PCCA) to Individuals With Propionic Acidemia","Inclusion Criteria:\n\n* Age six months to 2 years of age at day of vector infusion. For those \\\u003C1 year of age they must have been ≥37 weeks gestational age at the time of birth and without other conditions\u002Fcomorbidities that in the opinion of the Investigator may interfere with the interpretation of study results.\n* Confirmed diagnosis of propionic acidemia with biallelic PCCA gene mutations based on molecular genetic testing.\n* Study participants must have a diagnosis of neonatal-onset propionic acidemia with a documented episode of decompensation that can include any of the following findings: lethargy, poor feeding, irritability, vomiting, encephalopathy, respiratory failure, seizures, coma, metabolic acidosis, lactic acidosis, ketonuria, hypoglycemia, hyperammonemia, and cytopenias or history of recurrent hospitalizations.\n* Parents or legal guardians of study participants must agree to comply in good faith with the conditions of the study, including attending all of the required baseline and follow-up assessments, and parents or legal guardians must give consent for their child's participation.\n\nExclusion Criteria:\n\n* Hemoglobin \\\u003C10 g\u002Fdl\n* Platelet count \\\u003C 100,000 per mm3\n* Liver Enzyme ALT\u002FAST \\>2.5 ULN\n* Direct Bilirubin \\> 1.5\n* Active viral infection (includes HIV or serology positive for hepatitis B or C).\n* Previous liver transplant\n* Subjects with active decompensation as demonstrated by a pH \\\u003C 7.3, bicarbonate \\\u003C 15 mmol\u002FL, NH3 \\> 75 mcmol\u002FL, lactate \\> 2.5 mmol\u002FL, urine ketones\n* Previously received gene therapy or messenger ribonucleic acid (mRNA) treatments for PA.\n* Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.\n* Family does not want to disclose patient's study participation with primary care physician and other medical providers.","ALL","6 Months","2 Years",{"count":80,"type":81},9,"ESTIMATED","INTERVENTIONAL",[84],"PHASE1","Propionic acidemia is a genetic metabolic disorder characterized by metabolic acidosis, ketosis, vomiting, lethargy, cognitive impairment, and risk of death. It results from loss of function of the mitochondrial enzyme propionyl-CoA carboxylase and can be due to disease-causing variants in the PCCA gene, leading to accumulation of propionyl-CoA and its toxic metabolites. The purpose of this trial is to evaluate the safety and potential therapeutic benefit of an AAV-based gene therapy for propionic acidemia in patients with genetically confirmed biallelic variants in PCCA.",[87],"Propionic Acidemia","2026-06-18",{"date":90,"type":91},"2026-06-23","ACTUAL",{"date":93,"type":81},"2026-06",{"date":95,"type":81},"2033-12",{"name":5,"class":6},1]