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

Launched by IECURE, INC. · Feb 2, 2024

Keywords

ClinConnect Summary

This clinical trial, called OTC-HOPE, is investigating a new treatment called ECUR-506 for male babies under 9 months old who have a serious condition known as Ornithine Transcarbamylase (OTC) deficiency. OTC deficiency is an inherited disorder that affects the liver's ability to remove ammonia from the blood, which can build up to dangerous levels and lead to severe health issues, especially in newborns. The main goal of this study is to see if ECUR-506 is safe and how well it is tolerated when given through an intravenous (IV) dose.

To participate in this trial, boys must be between 24 hours and 7 months old, weigh between 3.5 kg and 10 kg, and have a confirmed diagnosis of severe OTC deficiency. They should also have received all necessary vaccinations. The trial is currently recruiting participants, and parents or guardians will need to sign consent forms to allow their children to take part. If enrolled, families can expect close monitoring for safety as the researchers evaluate the treatment's effects. This study is important because it aims to find better ways to manage a condition that can have very serious consequences for affected infants.

Gender

MALE

Eligibility criteria

• 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
• • 7. Severe neonatal OTCD defined by hyperammonemic crisis with elevated ammonia level of \>560 μmol/L and clinical symptoms within first week of life
• • 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 abnormalities
• • 6. Laboratory abnormalities outside of laboratory normal ranges for urinalysis, complete blood count, and comprehensive metabolic panel that are attributable to comorbidities unrelated to OTCD
• • 7. Treatment with any other gene therapy or gene editing therapy
• • 8. Co-enrollment in any other clinical study unless approved by the sponsor.
• • 9. Any condition, that in the opinion of the Investigator, would compromise the safety of the participant or study data
• • 10. Documented vertical transmission of HepA/HepB/HepC
• • 11. 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