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

ID#: NCT06255782

Age: 24 hours - 7 months

Gender: Male

Healthy Subjects: No

Study Phase: Phase 1/Phase 2

Recruitment Status: Recruiting

Start Date: April 08, 2024

End Date: September 01, 2026

Contact Information:
George Diaz, M.D., Ph.D.
1-877-694-3558
Trial Recruitment
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 detoxification of ammonia. Individuals with OTC deficiency can build-up excess levels of ammonia in their blood, potentially resulting in devastating consequences, including cumulative and irreversible neurological damage, coma and death. The severe form of the condition emerges shortly after birth and is more common in boys than girls. This is a Phase 1/2, open-label, multicenter, safety and dose finding study of ECUR-506 in male babies with neonatal onset OTC deficiency. The primary objective of this study is to evaluate the safety and tolerability of multiple dose levels of ECUR-506 following intravenous (IV) administration of a single dose.
Eligibility: Key

Inclusion Criteria:

1. Male sex

2. Gestational age ≥ 37 weeks

3. Age at screening is 24 hours to 7 months*

4. Weight ≥ 3.5 kg and ≤ 10.0 at screening

5. Has received all age-appropriate vaccinations

6. Genetically confirmed OTCD

7. Severe neonatal OTCD defined by hyperammonemic crisis 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

4. Known or suspected major organ injury/dysfunction/anomalies.

5. Treatment with any other gene therapy or gene editing therapy

6. Co-enrollment in any other clinical study with an investigational product prior to or during the duration of this trial would require the participant to be withdrawn from this study

7. Any condition, that in the opinion of the Investigator, would compromise the safety of the participant or study data

8. Documented vertical transmission of HSV, HIV, or HepA/HepB/HepC

9. 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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