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.
Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol
Participant parent(s)/LAR is willing and able to adhere to the protocol requirements.
Consent was obtained by the participants parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected.
None
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.
Other
Prospective
No Intervention
Other interventionNo Intervention
AE and SAE (incidence, severity, seriousness, and relatedness)
Safety
Change from baseline over 14.5 years post infusion in length
Length measured in cenitmeters
Change from baseline over 14.5 years post infusion in weight
Weight measured in kilograms
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.
qPCR measurement to evaluate the clearance of both vectors in blood over time.
Pharmacokinetics
qPCR measurement to evaluate the clearance of both vectors in saliva over time.
Pharmacokinetics
qPCR measurement to evaluate the clearance of both vectors in urine over time.
Pharmacokinetics
qPCR measurement to evaluate the clearance of both vectors in feces over time.
Pharmacokinetics
Number of participants with antibodies to hOTC in blood.
Pharmacodynamics
Number of participants with antibodies to M2PCSK9 in blood.
Pharmacodynamics
Number of participants with antibodies to AAVrh79 in blood.
Pharmacodynamics
Concentration of plasma ammonia
Pharmacodynamics
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