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A Phase 1/2/3 First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less Than 9 Months of Age With Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency
Sponsor study identifier
Secondary identifiers
OTHER · iECURE, Inc.
interventional
20
5 countries
12
Study Start
April 8, 2024
Actual (reported)Primary Completion
December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxifying of ammonia. Individuals with OTC deficiency can develop elevated levels of ammonia in the blood, potentially resulting in severe consequences, including cumulative and irreversible neurological damage, coma, and death. The most severe form presents shortly after birth and occurs more commonly in boys than girls. This is a Phase 1/2/3, open-label, multicenter study evaluating the safety, efficacy, and dose of ECUR-506 in male babies with neonatal-onset OTC deficiency. The primary objective is to evaluate the safety, tolerability, and efficacy of up to three dose levels of ECUR-506 following intravenous (IV) administration of a single dose.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
December 19, 2023
Actual (reported)February 13, 2024
Actual (reported)April 8, 2024
Actual (reported)December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Treatment-emergent adverse events (incidence, severity, seriousness, and relatedness)
Toxicity is graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. Adverse events (AEs) are reported based on clinical laboratory tests, vital signs, physical examinations, Pediatric Neurologist exam parameters, electrocardiograms, and any other medically indicated assessments from the time informed consent is signed through the end of the safety follow-up period. AEs are considered to be treatment emergent (TEAE) if they occur or worsen in severity following the administration of the study treatment. TEAEs are considered treatment-related if relationship to study drug is possibly related, probably related, or definitely related.
Over 24 weeks post infusion
Physical exam parameters
Safety- (Includes PI assessment of General Appearance, Dermatological, HEENT, Lymphatic, Respiratory, Cardiovascular, Gastrointestinal, Musculoskeletal, Neurological (Cranial Nerve Function, Motor System Function, Sensory System Function, Primitive Reflexes))
Assessed as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Participants will receive the Low Dose of ECUR-506 delivered one time via IV Infusion.
Participants will receive an intermediate dose of ECUR-506 delivered on time via IV infusion
Participants will receive a higher dose of ECUR-506 delivered one time via IV infusion.
ECUR-506 is a gene editing treatment delivering a gene encoding the editing enzyme and an OTC gene.
UCLA Mattel Children's Hospital
Los Angeles, California, 90095, United States
Children's Hospital of Colorado, Anshutz Medical Campus
Aurora, Colorado, 80045, United States
Emory University School of Medicine
Atlanta, Georgia, 30322, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
The Children's Hospital at Westmead
George Diaz, M.D., Ph.D
iECURE, Inc.
First Submitted
December 19, 2023
First Posted
February 13, 2024
Study Start
April 8, 2024
Actual (reported)Primary Completion
Vital sign parameters
Safety- (Includes PI assessment of Systolic Blood Pressure, Diastolic Blood Pressure, Pulse Rate, Respiratory Rate, Temperature)
Assessed as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Pediatric neurologist exam parameters
Safety- (Includes Pediatric Neurologist assessment of Neurological status by review of Cranial Nerve Function, Motor System Function, Sensory System Function, Primitive Reflexes.)
Assessed as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Blood safety tests including hematology, serum chemistry, liver function tests, coagulation tests
Safety- (Blood Safety tests to be reviewed in relation to established normal ranges for each assessment for the applicable age group)
as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Urinalysis evaluations
Toxicity is graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. Adverse events (AEs) are reported based on clinical laboratory tests, vital signs, physical examinations, electrocardiograms, and any other medically indicated assessments from the time informed consent is signed through the end of the safety follow-up period. AEs are considered to be treatment emergent (TEAE) if they occur or worsen in severity following the administration of the study treatment. TEAEs are considered treatment-related if relationship to study drug is possibly related, probably related, or definitely related.
Assessed as change from baseline at pre-specified timepoints through Week 24 post infusion.
12 lead ECG parameters
Safety- (Includes PI review of Heart Rate, PR Interval, RR Interval, QRS Duration, QT Interval, QTcF Interval, Overall Interpretation)
as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Complete clinical response
Discontinuation of scavenger medication for a minimum duration of 28 days without reductions in prescribed daily protein intake during this time period by end of study.
Over 24 weeks post infusion
Number of HAEs/person-year
Day 1 post dose through Week 24
qPCR measurement to evaluate the clearance of both vectors in body fluids over time
Over 24 weeks post infusion
Incidence of hyperammonemic episode (HAE)
Over 24 weeks post infusion
Incidence and number of hyperammonemic episodes (HAE/HAC) resulting in hospitalization
Over 24 weeks post infusion
Overall and by hospitalization severity (Mild: adjustment of dietary protein intake and oral scavenger medication / Moderate: cessation of dietary protein intake and initiation of IV scavenger therapy / Severe: requirement for hemodialysis)
Will be assessed Day 1 post dose through Wk 24 on all enrolled and dosed participants
Clinical Response (CR) defined as reduction in baseline standard of care therapy
Assessed at Week 24
Serum Neurofilament Light Chain
Assessed as change from baseline at pre-specified timepoints through Week 24 post infusion.
Number of participants with antibodies to AAVrh79 capsid in blood
Assessed as change from baseline at pre-specified timepoints through Week 24 post infusion.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Contact the study team to confirm eligibility.
Sydney, New South Wales, Australia
The Royal Children's Hospital
Melbourne, Victoria, 3052, Australia
Hopsital Sant Joan de Deu
Barcelona, 08950, Spain
Hospital Universitario 12 de Octubre
Madrid, 28041, Spain
Great Ormond Street Hospital
London, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust- Great North Children's Hospital
Newcastle upon Tyne, United Kingdom
December 2027
Planned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
December 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
September 30, 2026
Record last verified: 2026-09
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