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

Condition(s) studied

Investigational drug(s) / intervention(s)

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

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)