An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
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
iECURE, Inc.
20 participants
Apr 8, 2024
INTERVENTIONAL
Conditions
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
Inclusion Criteria8
- Male sex
- Gestational or adjusted (corrected) gestational age ≥ 37 weeks
- Age at screening is 24 hours to 7 months
- Weight ≥ 3.5 kg and ≤ 13.5 kg at screening
- Has received age-appropriate vaccinations
- 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.
- Current or historical biochemical profile consistent with OTCD
- Participant's parent(s)/LAR must be able to comprehend and be willing to provide a signed IRB/IEC-approved ICF.
Exclusion Criteria10
- Neonatal diagnosis of severe to profound Hypoxic Ischemic Encephalopathy due to birth injury
- Requiring urgent liver transplant due to liver failure as assessed by the PI.
- 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.
- Known or suspected major organ injury/dysfunction/anomalies.
- Vital sign and laboratory abnormalities outside of reference ranges.
- Treatment with any other gene therapy or gene editing therapy
- Co-enrollment in any other study unless approved by the sponsor.
- Any condition, that in the opinion of the Investigator, would compromise the safety of the participant or study data
- Documented vertical transmission of HepA/HepB/HepC
- 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
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Interventions
ECUR-506 is a gene editing treatment delivering a gene encoding the editing enzyme and an OTC gene.
Locations(12)
View Full Details on ClinicalTrials.gov
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NCT06255782