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

Phase 1

Active, not recruiting

• Conditions: Ornithine Transcarbamylase Deficiency; Ornithine Transcarbamylase Deficiency Disease; Ornithine Carbamoyltransferase Deficiency (Disorder); Urea Cycle Disorders, Inborn

• Registration Number: NCT06255782
• Lead Sponsor: iECURE, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-12-19
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Active, not recruiting
• Sex: Male
• Target Recruitment: 13

Inclusion Criteria

Key Inclusion Criteria:<br><br> 1. Male sex<br><br> 2. Gestational age = 37 weeks<br><br> 3. Age at screening is 24 hours to 7 months*<br><br> 4. Weight = 3.5 kg and = 10.0 at screening<br><br> 5. Has received all age-appropriate vaccinations<br><br> 6. Genetically confirmed OTCD<br><br> 7. Severe neonatal OTCD defined by hyperammonemic crisis within first week of life<br><br> 8. Current or historical biochemical profile consistent with OTCD<br><br> 9. Participant's parent(s)/LAR must be able to comprehend and be willing to provide a<br> signed IRB/IEC-approved ICF.<br><br>Key Exclusion Criteria:<br><br> 1. Neonatal diagnosis of severe to profound Hypoxic Ischemic Encephalopathy due to<br> birth injury<br><br> 2. Requiring urgent liver transplant due to liver failure as assessed by the PI.<br><br> 3. Contiguous gene deletion involving the OTC gene<br><br> 4. Known or suspected major organ injury/dysfunction/anomalies.<br><br> 5. Treatment with any other gene therapy or gene editing therapy<br><br> 6. Co-enrollment in any other clinical study with an investigational product prior to<br> or during the duration of this trial would require the participant to be withdrawn<br> from this study<br><br> 7. Any condition, that in the opinion of the Investigator, would compromise the safety<br> of the participant or study data<br><br> 8. Documented vertical transmission of HSV, HIV, or HepA/HepB/HepC<br><br> 9. Documented in-utero teratogen, substance, and/or alcohol exposure, which in the<br> opinion of the Investigator may increase the participant's risk of developmental<br> delays, congenital anomalies, and/or significant medical complications

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Treatment-emergent adverse events (incidence, severity, seriousness, and relatedness);Urinalysis evaluations

Secondary Outcome Measures

Name	Time	Method
Percent liver transduction;Number of hyperammonemic crises (HAC);qPCR measurement to evaluate the clearance of both vectors in body fluids over time;Scavenger drug dose per body surface area (BSA);Protein allowance g/kg;Blood urea nitrogen measurements