ECUR-506 for Neonatal-Onset OTC Deficiency in Male Babies
This study is testing a new gene editing treatment called ECUR-506 for male babies under 9 months old who have neonatal-onset Ornithine Transcarbamylase (OTC) deficiency. OTC deficiency is a genetic condition where the body can't properly remove ammonia, which can cause serious health problems. ECUR-506 aims to fix the genetic problem by delivering a working copy of the OTC gene. Researchers will be looking at how safe ECUR-506 is, how well it works, and the right dose to use. The study is open-label, meaning everyone involved will know which treatment is being given. We are looking to enroll about 20 participants.
- Study design
- This is an open-label study, meaning all participants and researchers will know which treatment is being given. It is planned to enroll about 20 male babies.
- What's involved
- Participants will receive a single IV infusion of ECUR-506. Physical exams and vital signs will be assessed at specific times throughout the study.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for treatment-emergent adverse events for over 24 weeks after the infusion. Physical exam and vital sign parameters will be assessed throughout the study's duration.
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An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
At a glance
Conditions
Where it's being run
12 sites across 10 statesStudy leadership
- George Diaz, M.D., Ph.D · STUDY_DIRECTOR · iECURE, Inc.
Who to contact
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What this trial measures
- Treatment-emergent adverse events (incidence, severity, seriousness, and relatedness)Over 24 weeks post infusion
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.
- Physical exam parametersAssessed 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.
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))
- Vital sign parametersAssessed 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.
Safety- (Includes PI assessment of Systolic Blood Pressure, Diastolic Blood Pressure, Pulse Rate, Respiratory Rate, Temperature)
- Pediatric neurologist exam parametersAssessed 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.
Safety- (Includes Pediatric Neurologist assessment of Neurological status by review of Cranial Nerve Function, Motor System Function, Sensory System Function, Primitive Reflexes.)
- Blood safety tests including hematology, serum chemistry, liver function tests, coagulation testsas change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Safety- (Blood Safety tests to be reviewed in relation to established normal ranges for each assessment for the applicable age group)
- Urinalysis evaluationsAssessed as change from baseline at pre-specified timepoints through Week 24 post infusion.
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.
- 12 lead ECG parametersas change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Safety- (Includes PI review of Heart Rate, PR Interval, RR Interval, QRS Duration, QT Interval, QTcF Interval, Overall Interpretation)
- Complete clinical responseOver 24 weeks post infusion
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.