ECUR-506 for Ornithine Transcarbamylase Deficiency

Recruiting at 8 trial locations

Overseen By George Diaz, M.D., Ph.D.

Age: < 18

Sex: Male

Trial Phase: Phase 1 & 2

Sponsor: iECURE, Inc.

Must not be taking: Gene therapy

Disqualifiers: Hypoxic Ischemic Encephalopathy, Liver failure, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

13 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether participants must stop taking their current medications. Please consult with the study team for guidance.

What is the purpose of this trial?

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.

Research Team

George Diaz, M.D., Ph.D

Principal Investigator

iECURE, Inc.

Eligibility Criteria

This trial is for male babies under 9 months old with a severe form of OTC deficiency, which causes dangerous ammonia build-up in the blood. Babies must weigh between 3.5 and 10 kg, have had all vaccinations appropriate for their age, be born after at least 37 weeks of pregnancy, and have genetically confirmed OTCD.

Inclusion Criteria

My parent or legal guardian can understand and is willing to sign the consent form.

I had a severe ammonia increase in my first week of life.

My blood tests show I may have ornithine transcarbamylase deficiency.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous (IV) administration of ECUR-506 at varying dose levels

1 day

1 visit (in-person)

Safety Follow-up

Participants are monitored for treatment-emergent adverse events and other safety parameters

24 weeks

Multiple visits (in-person and virtual)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• ECUR-506

Trial Overview The study tests ECUR-506's safety and tolerability on baby boys with neonatal onset OTC deficiency. It's an early-stage trial (Phase 1/2) where participants receive one dose of the drug intravenously to see how they react to different dose levels.

Participant Groups

Group I: Cohort 2 Experimental Treatment 1 Intervention

Participants will receive a higher dose of ECUR-506 delivered one time via IV infusion.

Group II: Cohort 1 Experimental Treatment 1 Intervention

Participants will receive the Low Dose of ECUR-506 delivered one time via IV Infusion.

Group III: Additional Cohorts Experimental Treatment 1 Intervention

Additional Cohorts based on recommendations of DMC review of safety and efficacy data.

ECUR-506 is already approved in United States, United Kingdom for the following indications:

Approved in United States as ECUR-506 for:

• Neonatal onset ornithine transcarbamylase (OTC) deficiency

Approved in United Kingdom as ECUR-506 for:

• Neonatal onset ornithine transcarbamylase (OTC) deficiency

Who Is Running the Clinical Trial?

iECURE, Inc.

Lead Sponsor

Trials

2

Recruited

30+