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Clinical Trials in the UK / NCT06805695
Recruiting Observational

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

NCT06805695 · tracked via the Priya Life Science UK tracker
Sponsor
iECURE, Inc
Phase
Observational
Started
2024-12-23
Last updated
2026-08-31

Condition(s) studied

Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency DiseaseUrea Cycle Disorders, Inborn

Investigational drug(s) / intervention(s)

No Intervention

No Intervention: No Intervention

Study summary

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.

Eligibility

Sex
ALL
Min age
7 Months
Max age
15 Months
Healthy volunteers
No
Inclusion Criteria: 1. Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol 2. Participant parent(s)/LAR is willing and able to adhere to the protocol requirements. 3. 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.

Primary outcome measure(s)

  • AE and SAE (incidence, severity, seriousness, and relatedness) — Over 14.5 years post dosing with ECUR-506
    Safety
  • Change from baseline over 14.5 years post infusion in length — Over 14.5 years post dosing with ECUR-506
    Length measured in cenitmeters
  • Change from baseline over 14.5 years post infusion in weight — Over 14.5 years post dosing with ECUR-506
    Weight measured in kilograms
  • Urinalysis (Dip Stick) Evaluations — Over 14.5 years post dosing with ECUR-506
    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.

Trial sites (3)

FacilityCityRegionStatus
Children's Hospital of Colorado, Anshutz Medical Campus Aurora Colorado Recruiting
Icahn School of Medicine at Mount Sinai New York New York Recruiting
Great Ormond Street Hospital London United Kingdom Recruiting

More iECURE, Inc. trials in the UK

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency Active, not recruiting

Other trials for the same condition

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06805695 on ClinicalTrials.gov ↗ ← All trials in the UK