Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: No Intervention.
- Who it may be relevant to
- Registry conditions: Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn. Basic parameters: 7 months — 15 months · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Detailed description
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.
Interventions
- Other No Intervention
No Intervention
Primary outcome measures
- AE and SAE (incidence, severity, seriousness, and relatedness) [Time frame: Over 14.5 years post dosing with ECUR-506]
- Change from baseline over 14.5 years post infusion in length [Time frame: Over 14.5 years post dosing with ECUR-506]
- Change from baseline over 14.5 years post infusion in weight [Time frame: Over 14.5 years post dosing with ECUR-506]
- Urinalysis (Dip Stick) Evaluations [Time frame: Over 14.5 years post dosing with ECUR-506]
Secondary outcome measures (12)
- qPCR measurement to evaluate the clearance of both vectors in blood over time. [Time frame: Over 14.5 years post dosing with ECUR-506]
- qPCR measurement to evaluate the clearance of both vectors in saliva over time. [Time frame: Over 14.5 years post dosing with ECUR-506]
- qPCR measurement to evaluate the clearance of both vectors in urine over time. [Time frame: Over 14.5 years post dosing with ECUR-506]
- qPCR measurement to evaluate the clearance of both vectors in feces over time. [Time frame: Over 14.5 years post dosing with ECUR-506]
- Percent Liver Transduction [Time frame: Over 14.5 years post dosing with ECUR-506]
- Number of hyperammonemic crises (HAC) [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 [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. [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. [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 [Time frame: Over 14.5 years post dosing with ECUR-506]
- Scavenger drug dose per body surface area (BSA) [Time frame: Over 14.5 years post dosing with ECUR-506]
- Protein allowance g/kg [Time frame: Over 14.5 years post dosing with ECUR-506]
Eligibility criteria
Inclusion criteria
- 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.
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Other
Study locations
United Kingdom · 1 center
- Great Ormond Street Hospital — London
Identifiers
NCT: NCT06805695 · ECUR-LTFU