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Clinical Trials in Spain / NCT06255782
Active, not recruiting Phase 3

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

NCT06255782 · tracked via the Priya Life Science Spain tracker
Sponsor
iECURE, Inc
Phase
Phase 3
Started
2024-04-08
Last updated
2026-09-30

Condition(s) studied

Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency DiseaseOrnithine Carbamoyltransferase Deficiency (Disorder)Urea Cycle Disorders, Inborn

Investigational drug(s) / intervention(s)

ECUR-506

ECUR-506: ECUR-506 is a gene editing treatment delivering a gene encoding the editing enzyme and an OTC gene.

Study summary

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.

Eligibility

Sex
MALE
Min age
24 Hours
Max age
7 Months
Healthy volunteers
No
Key Inclusion Criteria: 1. Male sex 2. Gestational or adjusted (corrected) gestational age ≥ 37 weeks 3. Age at screening is 24 hours to 7 months 4. Weight ≥ 3.5 kg and ≤ 13.5 kg at screening 5. Has received age-appropriate vaccinations 6. Genetically confirmed OTCD defined by genetic confirmation of an OTC variant (pathogenic or likely pathogenic) associated with severe neonatal OTCD defined below in Inclusion Criteria #7 or has the same OTC variant as a family member who had severe neonatal OTCD within first week of life. 7. Severe neonatal OTCD defined by hyperammonemic crisis with elevated ammonia level of \>560 μmol/L and clinical symptoms within first week of life, and currently receiving treatment with both dietary protein restriction and nitrogen scavenger therapy. 8. Current or historical biochemical profile consistent with OTCD 9. Participant's parent(s)/LAR must be able to comprehend and be willing to provide a signed IRB/IEC-approved ICF. Key Exclusion Criteria: 1. Neonatal diagnosis of severe to profound Hypoxic Ischemic Encephalopathy due to birth injury 2. Requiring urgent liver transplant due to liver failure as assessed by the PI. 3. Contiguous gene deletion involving the OTC gene and including at least the CYBB gene on the telomeric side or the TSPAN7 gene on the centromeric side. 4. Known or suspected major organ injury/dysfunction/anomalies. 5. Vital sign and laboratory abnormalities outside of reference ranges. 6. Treatment with any other gene therapy or gene editing therapy 7. Co-enrollment in any other study unless approved by the sponsor. 8. Any condition, that in the opinion of the Investigator, would compromise the safety of the participant or study data 9. Documented vertical transmission of HepA/HepB/HepC 10. Documented in-utero teratogen, substance, and/or alcohol exposure, which in the opinion of the Investigator may increase the participant's risk of developmental delays, congenital anomalies, and/or significant medical complications

Primary outcome measure(s)

  • 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 parameters — 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.
    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 parameters — 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.
    Safety- (Includes PI assessment of Systolic Blood Pressure, Diastolic Blood Pressure, Pulse Rate, Respiratory Rate, Temperature)
  • Pediatric neurologist exam parameters — 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.
    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 tests — 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- (Blood Safety tests to be reviewed in relation to established normal ranges for each assessment for the applicable age group)
  • Urinalysis evaluations — Assessed 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 parameters — 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 review of Heart Rate, PR Interval, RR Interval, QRS Duration, QT Interval, QTcF Interval, Overall Interpretation)
  • Complete clinical response — Over 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.

Trial sites (12)

FacilityCityRegionStatus
UCLA Mattel Children's Hospital Los Angeles California
Children's Hospital of Colorado, Anshutz Medical Campus Aurora Colorado
Emory University School of Medicine Atlanta Georgia
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
Icahn School of Medicine at Mount Sinai New York New York
Oregon Health and Science University Portland Oregon
The Children's Hospital at Westmead Sydney New South Wales
The Royal Children's Hospital Melbourne Victoria
Hopsital Sant Joan de Deu Barcelona Spain
Hospital Universitario 12 de Octubre Madrid 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
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 NCT06255782 on ClinicalTrials.gov ↗ ← All trials in Spain