Ignite Creation Date:
2025-12-24 @ 1:57 PM
Ignite Modification Date:
2026-01-04 @ 7:31 AM
Study NCT ID:
NCT06805695
Status:
RECRUITING
Last Update Posted:
2025-02-03
First Post:
2024-12-20
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020163', 'term': 'Ornithine Carbamoyltransferase Deficiency Disease'}, {'id': 'D056806', 'term': 'Urea Cycle Disorders, Inborn'}, {'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D022124', 'term': 'Hyperammonemia'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}], 'ancestors': [{'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 13}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-12-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-01', 'completionDateStruct': {'date': '2041-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-01-28', 'studyFirstSubmitDate': '2024-12-20', 'studyFirstSubmitQcDate': '2025-01-28', 'lastUpdatePostDateStruct': {'date': '2025-02-03', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-02-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2041-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Number of participants with antibodies to hOTC in blood.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics'}, {'measure': 'Number of participants with antibodies to M2PCSK9 in blood.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics'}, {'measure': 'Number of participants with antibodies to AAVrh79 in blood.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics'}, {'measure': 'Concentration of plasma ammonia', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics'}, {'measure': 'Concentration of plasma citrulline', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics'}, {'measure': 'Concentration of plasma glutamine', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics'}, {'measure': 'Urinary excretion of phenylacetate metabolites.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Urinary phenylactate (mcg/mL), urinary phenylactylglutamine (mcg/mL), urinary phenylacetate/ phenylglutamine ratio.'}, {'measure': 'Urinary excretion of orotic acid metabolites', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Urinary orotic acid (mmol/mol creatinine), urinary uracil (mmol/mol creatinine)'}, {'measure': 'Future DNA analysis of WBCs collected in a parent trial may be performed only in the event of an observed genomic safety signal', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Safety'}, {'measure': 'Developmental assessments as measured by age-appropriate Bayley Scale of Infant Development IV (BSID-IV)', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Total Raw Scores, Growth Score Values (GSVs), and Age Equivalent Scores will be analyzed; Raw Scores range 0-162; GSVs range 428-599; Age Equivalent Scores range 1-42 months; a higher score reflects a higher level of skill.'}, {'measure': 'Kaufman Assessment Battery for Children - Second Edition Normative Update (KABC-II NU)', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Total Raw Scores, Growth Score Values (GSVs), and Age Equivalent Scores will be analyzed; Raw Scores range 0-162; GSVs range 428-599; Age Equivalent Scores range 1-42 months; a higher score reflects a higher level of skill.'}, {'measure': 'Quality of life as measured by an age-appropriate Pediatric Quality of Life Inventory Infant Scales', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Quality of Life Outcomes'}], 'primaryOutcomes': [{'measure': 'AE and SAE (incidence, severity, seriousness, and relatedness)', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Safety'}, {'measure': 'Change from baseline over 14.5 years post infusion in length', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Length measured in cenitmeters'}, {'measure': 'Change from baseline over 14.5 years post infusion in weight', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Weight measured in kilograms'}, {'measure': 'Urinalysis (Dip Stick) Evaluations', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Urinalysis (Dip Stick) will be evaluated to monitor pathological changes to the participants urine and to monitor levels of Specific Gravity, pH, Glucose, Protein, Blood, Ketones, Bilirubin, Urobilinogen, Nitrite, Leukocyte esterase, red blood cell count, and white blood cell count as they relate to established local lab normal ranges.'}], 'secondaryOutcomes': [{'measure': 'qPCR measurement to evaluate the clearance of both vectors in blood over time.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacokinetics'}, {'measure': 'qPCR measurement to evaluate the clearance of both vectors in saliva over time.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacokinetics'}, {'measure': 'qPCR measurement to evaluate the clearance of both vectors in urine over time.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacokinetics'}, {'measure': 'qPCR measurement to evaluate the clearance of both vectors in feces over time.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacokinetics'}, {'measure': 'Percent Liver Transduction', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacokinetics'}, {'measure': 'Number of hyperammonemic crises (HAC)', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics and Efficacy'}, {'measure': 'Among participants who experience HAC with associated neurological status change and are hospitalized: Asses daily ammonia levels for duration of hospitalization for each event', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics and Safety'}, {'measure': 'Among participants who experience HAC with associated neurological status change and are hospitalized: Assess duration of hospitalization for each event.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics and Safety'}, {'measure': 'Among participants who experience HAC with associated neurological status change and are hospitalized: Assess requirement for ICU care.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics and Safety'}, {'measure': 'Number of HAC with the following severities: a. Mild: adjustment of dietary protein intake and oral scavenger medication b. Moderate: cessation of dietary protein intake and initiation of IV scavenger therapy c. Severe: requirement for hemodialysis', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics and Safety'}, {'measure': 'Scavenger drug dose per body surface area (BSA)', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Efficacy'}, {'measure': 'Protein allowance g/kg', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Efficacy'}, {'measure': 'Concentration of blood urea nitrogen measurements', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Pharmacodynamics'}, {'measure': 'Time to liver transplant from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol)', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Efficacy'}, {'measure': 'Transplant free survival', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Efficacy'}, {'measure': 'Time to liver transplant or any cause of death from dosing to EOS.', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Efficacy'}, {'measure': 'Overall Survival: Survival measured from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol).', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Efficacy'}, {'measure': 'Time to any-cause death from dosing to EOS. Survival measured from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol).', 'timeFrame': 'Over 14.5 years post dosing with ECUR-506', 'description': 'Efficacy'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Amino Acid Metabolism, Inborn Errors', 'Ammonia', 'Brain Diseases', 'Brain Diseases, Metabolic', 'Brain Diseases, Metabolic, Inborn', 'Central Nervous System Diseases', 'Genetic Diseases, Inborn', 'Genetic Diseases, X-Linked', 'High Ammonia', 'Hyperammonemia', 'Inborn', 'Inborn Errors', 'Liver Disease', 'Liver Transplant', 'Metabolism', 'Metabolic Diseases', 'Metabolism, Inborn Errors', 'Neonatal', 'Nervous System Diseases', 'NH4', 'Ornithine', 'Ornithine Transcarbamylase Deficiency', 'OTC', 'OTC Deficiency', 'OTCD', 'Transcarbamylase', 'UCD', 'Urea Cycle Disorders', 'Long Term Follow Up', 'Long-Term Follow-Up', 'LTFU'], 'conditions': ['Ornithine Transcarbamylase Deficiency', 'Ornithine Transcarbamylase Deficiency Disease', 'Urea Cycle Disorders, Inborn']}, 'descriptionModule': {'briefSummary': 'This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol.', 'detailedDescription': 'This is a 14.5 year LTFU protocol to assess long term safety and clinical response to IP as well as to capture natural disease history of participants not dosed with IP in a parent protocol. No IP is provided as part of this protocol. Participants will remain on standard of care medication as indicated and prescribed by their physicians.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '15 Months', 'minimumAge': '7 Months', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'All participants from an iECURE parent treatment protocol, whether they received IP or not, will be consented to enter this LTFU study. Enrollment into the LTFU will begin upon either premature discontinuation from, or completion of, the parent treatment protocol.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol\n2. Participant parent(s)/LAR is willing and able to adhere to the protocol requirements.\n3. Consent was obtained by the participants parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected.\n\nExclusion Criteria:\n\n1\\. Participants who enroll into an interventional drug or gene therapy clinical trial utilizing an IP other than the IP provided in the parent protocol will be excluded from this protocol.'}, 'identificationModule': {'nctId': 'NCT06805695', 'briefTitle': 'Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)', 'organization': {'class': 'INDUSTRY', 'fullName': 'iECURE, Inc.'}, 'officialTitle': 'Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)', 'orgStudyIdInfo': {'id': 'ECUR-LTFU'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Enrolled and Dosed', 'description': 'Previously dosed in a previous iECURE study.', 'interventionNames': ['Other: No Intervention']}, {'label': 'Enrolled but Not Dosed', 'description': 'Enrolled in a previous iECURE study, but not dosed.', 'interventionNames': ['Other: No Intervention']}], 'interventions': [{'name': 'No Intervention', 'type': 'OTHER', 'description': 'No Intervention', 'armGroupLabels': ['Enrolled and Dosed', 'Enrolled but Not Dosed']}]}, 'contactsLocationsModule': {'locations': [{'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Christopher Jackson', 'role': 'CONTACT', 'email': 'christopher.jackson@gosh.nhs.uk'}], 'facility': 'Great Ormond Street Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'centralContacts': [{'name': 'George Diaz, M.D., Ph.D.', 'role': 'CONTACT', 'email': 'medinfo@iecure.com', 'phone': '1-877-694-3558'}], 'overallOfficials': [{'name': 'George Diaz, M.D., Ph.D', 'role': 'STUDY_DIRECTOR', 'affiliation': 'iECURE, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'iECURE, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}