NCT06805695|RecruitingDMCFDA Drug
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Sponsor study identifier
observational
13
6 countries
7
Study Start
December 23, 2024
Actual (reported)Primary Completion
July 2041
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
July 2041
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
July 2041
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
December 20, 2024
Actual (reported)December 23, 2024
Actual (reported)February 3, 2025
Actual (reported)July 2041
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
July 2041
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
AE and SAE (incidence, severity, seriousness, and relatedness)
Safety
Over 14.5 years post dosing with ECUR-506
Change from baseline over 14.5 years post infusion in length
Length measured in cenitmeters
Over 14.5 years post dosing with ECUR-506
Change from baseline over 14.5 years post infusion in weight
Weight measured in kilograms
Over 14.5 years post dosing with ECUR-506
Previously dosed in a previous iECURE study.
Enrolled in a previous iECURE study, but not dosed.
No Intervention
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.
Children's Hospital of Colorado, Anshutz Medical Campus
Aurora, Colorado, 80045, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
George Diaz, M.D., Ph.D
iECURE, Inc.
First Submitted
December 20, 2024
First Posted
February 3, 2025
Study Start
December 23, 2024
Actual (reported)Primary Completion
July 2041
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
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.
Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in blood over time.
Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in saliva over time.
Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in urine over time.
Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in feces over time.
Over 14.5 years post dosing with ECUR-506
Percent Liver Transduction
Over 14.5 years post dosing with ECUR-506
Number of participants with antibodies to hOTC in blood.
Over 14.5 years post dosing with ECUR-506
Number of participants with antibodies to M2PCSK9 in blood.
Over 14.5 years post dosing with ECUR-506
Number of participants with antibodies to AAVrh79 in blood.
Over 14.5 years post dosing with ECUR-506
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
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Great Ormond Street Hospital
London, United Kingdom
Study Completion
July 2041
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
August 31, 2026
Record last verified: 2026-08
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Source: ClinicalTrials.gov.
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