An observational study in Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease and Urea Cycle Disorders, Inborn, sponsored by iECURE, Inc.. Recruiting at 4 sites in 3 countries. Open to participants aged 7 Months to 15 Months. Per ClinicalTrials.gov, last updated 2026-11-05.
Sponsored by iECURE, Inc. · Observational
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.
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.
59 studies on the registry are indexed under Ornithine Carbamoyltransferase Deficiency Disease; 13 are open to participants now.
This study's planned enrollment of 13 is below the median of 70 across 28 observational studies indexed under Ornithine Carbamoyltransferase Deficiency Disease.
Browse Ornithine Carbamoyltransferase Deficiency Disease studies →iECURE, Inc. is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
1. 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.
Previously dosed in a previous iECURE study.
Other: No Intervention
Enrolled in a previous iECURE study, but not dosed.
Other: No Intervention
No Intervention
AE and SAE (incidence, severity, seriousness, and relatedness)
Safety
Time frame: Over 14.5 years post dosing with ECUR-506
Change from baseline over 14.5 years post infusion in length
Length measured in cenitmeters
Time frame: Over 14.5 years post dosing with ECUR-506
Change from baseline over 14.5 years post infusion in weight
Weight measured in kilograms
Time frame: Over 14.5 years post dosing with ECUR-506
Urinalysis (Dip Stick) Evaluations
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.
Time frame: Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in blood over time.
Pharmacokinetics
Time frame: Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in saliva over time.
Pharmacokinetics
Time frame: Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in urine over time.
Pharmacokinetics
Time frame: Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in feces over time.
Pharmacokinetics
Time frame: Over 14.5 years post dosing with ECUR-506
Percent Liver Transduction
Pharmacokinetics
Time frame: Over 14.5 years post dosing with ECUR-506
Number of hyperammonemic crises (HAC)
Pharmacodynamics and Efficacy
Time frame: Over 14.5 years post dosing with ECUR-506
Among participants who experience HAC with associated neurological status change and are hospitalized: Asses daily ammonia levels for duration of hospitalization for each event
Pharmacodynamics and Safety
Time frame: Over 14.5 years post dosing with ECUR-506
Among participants who experience HAC with associated neurological status change and are hospitalized: Assess duration of hospitalization for each event.
Pharmacodynamics and Safety
Time frame: Over 14.5 years post dosing with ECUR-506
Among participants who experience HAC with associated neurological status change and are hospitalized: Assess requirement for ICU care.
Pharmacodynamics and Safety
Time frame: Over 14.5 years post dosing with ECUR-506
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
Pharmacodynamics and Safety
Time frame: Over 14.5 years post dosing with ECUR-506
Scavenger drug dose per body surface area (BSA)
Efficacy
Time frame: Over 14.5 years post dosing with ECUR-506
Protein allowance g/kg
Efficacy
Time frame: Over 14.5 years post dosing with ECUR-506
Concentration of blood urea nitrogen measurements
Pharmacodynamics
Time frame: Over 14.5 years post dosing with ECUR-506
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)
Efficacy
Time frame: Over 14.5 years post dosing with ECUR-506
Transplant free survival
Efficacy
Time frame: Over 14.5 years post dosing with ECUR-506
Time to liver transplant or any cause of death from dosing to EOS.
Efficacy
Time frame: Over 14.5 years post dosing with ECUR-506
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).
Efficacy
Time frame: Over 14.5 years post dosing with ECUR-506
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).
Efficacy
Time frame: Over 14.5 years post dosing with ECUR-506
Number of participants with antibodies to hOTC in blood.
Pharmacodynamics
Time frame: Over 14.5 years post dosing with ECUR-506
Number of participants with antibodies to M2PCSK9 in blood.
Pharmacodynamics
Time frame: Over 14.5 years post dosing with ECUR-506
Number of participants with antibodies to AAVrh79 in blood.
Pharmacodynamics
Time frame: Over 14.5 years post dosing with ECUR-506
Concentration of plasma ammonia
Pharmacodynamics
Time frame: Over 14.5 years post dosing with ECUR-506
Concentration of plasma citrulline
Pharmacodynamics
Time frame: Over 14.5 years post dosing with ECUR-506
Concentration of plasma glutamine
Pharmacodynamics
Time frame: Over 14.5 years post dosing with ECUR-506
Urinary excretion of phenylacetate metabolites.
Urinary phenylactate (mcg/mL), urinary phenylactylglutamine (mcg/mL), urinary phenylacetate/ phenylglutamine ratio.
Time frame: Over 14.5 years post dosing with ECUR-506
Urinary excretion of orotic acid metabolites
Urinary orotic acid (mmol/mol creatinine), urinary uracil (mmol/mol creatinine)
Time frame: Over 14.5 years post dosing with ECUR-506
Future DNA analysis of WBCs collected in a parent trial may be performed only in the event of an observed genomic safety signal
Safety
Time frame: Over 14.5 years post dosing with ECUR-506
Developmental assessments as measured by age-appropriate Bayley Scale of Infant Development IV (BSID-IV)
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.
Time frame: Over 14.5 years post dosing with ECUR-506
Kaufman Assessment Battery for Children - Second Edition Normative Update (KABC-II NU)
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.
Time frame: Over 14.5 years post dosing with ECUR-506
Quality of life as measured by an age-appropriate Pediatric Quality of Life Inventory Infant Scales
Quality of Life Outcomes
Time frame: Over 14.5 years post dosing with ECUR-506
Plan to share: Undecided
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Ornithine Carbamoyltransferase Deficiency Disease →
iECURE, Inc.