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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06255782
Additional trial details provided through ANZCTR are available at the end of this record.
Registration number
NCT06255782
Ethics application status
Date submitted
19/12/2023
Date registered
13/02/2024
Date last updated
15/06/2025
Titles & IDs
Public title
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
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Scientific title
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
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Secondary ID [1]
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OTC HOPE
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Secondary ID [2]
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ECUR-506-OTC-101
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Ornithine Transcarbamylase Deficiency
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Ornithine Transcarbamylase Deficiency Disease
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Ornithine Carbamoyltransferase Deficiency (Disorder)
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Urea Cycle Disorders, Inborn
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Neurological
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Other neurological disorders
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Metabolic and Endocrine
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Metabolic disorders
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Diet and Nutrition
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Other diet and nutrition disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Other - ECUR-506
Experimental: Low Dose Level - Participants will receive the Low Dose of ECUR-506 delivered one time via IV Infusion.
Experimental: High Dose Level - Participants will receive a higher dose of ECUR-506 delivered one time via IV infusion.
Experimental: Intermediate Dose Level - Additional Cohorts based on recommendations of DMC review of safety and efficacy data.
Treatment: Other: ECUR-506
ECUR-506 is a gene editing treatment delivering a gene encoding the editing enzyme and an OTC gene.
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Intervention code [1]
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Treatment: Other
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Treatment-emergent adverse events (incidence, severity, seriousness, and relatedness)
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Assessment method [1]
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Toxicity is graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. Adverse events (AEs) are reported based on clinical laboratory tests, vital signs, physical examinations, Pediatric Neurologist exam parameters, electrocardiograms, and any other medically indicated assessments from the time informed consent is signed through the end of the safety follow-up period. AEs are considered to be treatment emergent (TEAE) if they occur or worsen in severity following the administration of the study treatment. TEAEs are considered treatment-related if relationship to study drug is possibly related, probably related, or definitely related.
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Timepoint [1]
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Over 24 weeks post infusion
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Primary outcome [2]
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Urinalysis evaluations
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Assessment method [2]
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Toxicity is graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. Adverse events (AEs) are reported based on clinical laboratory tests, vital signs, physical examinations, electrocardiograms, and any other medically indicated assessments from the time informed consent is signed through the end of the safety follow-up period. AEs are considered to be treatment emergent (TEAE) if they occur or worsen in severity following the administration of the study treatment. TEAEs are considered treatment-related if relationship to study drug is possibly related, probably related, or definitely related.
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Timepoint [2]
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Assessed as change from baseline at pre-specified timepoints through Week 24 post infusion.
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Primary outcome [3]
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Achieving normal fasting plasma ammonia levels by EOS
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Assessment method [3]
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Fasting plasma ammonia levels that fall within normal limits for at least 75% of post-dose assessments.
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Timepoint [3]
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Over 24 weeks post infusion
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Primary outcome [4]
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Complete clinical response
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Assessment method [4]
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Discontinuation of scavenger medication for a minimum duration of 28 days without reductions in prescribed daily protein intake during this time period.
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Timepoint [4]
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Over 24 weeks post infusion
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Secondary outcome [1]
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Percent liver transduction
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Assessment method [1]
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Pharmacokinetics
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Timepoint [1]
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Assessed at Week 24
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Secondary outcome [2]
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Number of hyperammonemic crises (HAC)
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Assessment method [2]
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Pharmacodynamics and Efficacy
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Timepoint [2]
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Over 24 weeks post infusion
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Secondary outcome [3]
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qPCR measurement to evaluate the clearance of both vectors in body fluids over time
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Assessment method [3]
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Pharmacokinetics
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Timepoint [3]
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Over 24 weeks post infusion
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Secondary outcome [4]
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Scavenger drug dose per body surface area (BSA)
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Assessment method [4]
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Efficacy
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Timepoint [4]
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Over 24 weeks post infusion
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Secondary outcome [5]
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Protein allowance g/kg/day
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Assessment method [5]
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Efficacy
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Timepoint [5]
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Over 24 weeks post infusion
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Secondary outcome [6]
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Blood urea nitrogen measurements
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Assessment method [6]
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Pharmacodynamics
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Timepoint [6]
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Over 24 weeks post infusion
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Secondary outcome [7]
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Fasting plasma ammonia
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Assessment method [7]
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Efficacy
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Timepoint [7]
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Over 24 weeks post infusion
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Eligibility
Key inclusion criteria
Key
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.
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Minimum age
24
Hours
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Maximum age
7
Months
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Sex
Males
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Other
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
8/04/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/09/2026
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Actual
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Sample size
Target
8
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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The Children's Hospital at Westmead - Sydney
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Recruitment hospital [2]
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The Royal Children's Hospital - Melbourne
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Recruitment postcode(s) [1]
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- Sydney
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Recruitment postcode(s) [2]
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3052 - Melbourne
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Colorado
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Country [3]
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United States of America
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State/province [3]
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New York
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Country [4]
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Spain
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State/province [4]
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Barcelona
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Country [5]
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Spain
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State/province [5]
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Madrid
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Country [6]
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United Kingdom
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State/province [6]
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London
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Country [7]
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United Kingdom
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State/province [7]
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Newcastle Upon Tyne
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
iECURE, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief 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 1/2/3, open-label, multicenter, safety, efficacy, 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, tolerability, and efficacy of up to three dose levels of ECUR-506 following intravenous (IV) administration of a single dose.
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Trial website
https://clinicaltrials.gov/study/NCT06255782
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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George Diaz, M.D., Ph.D
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Address
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iECURE, Inc.
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Country
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0
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Phone
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Fax
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Email
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Contact person for public queries
Name
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George Diaz, M.D., Ph.D.
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Address
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Country
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Phone
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1-877-694-3558
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Fax
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0
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06255782
Additional trial details provided through ANZCTR
Accrual to date
Recruiting in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC
Recruitment hospital [1]
188
The Children's Hospital at Westmead
Recruitment hospital [2]
189
The Royal Childrens Hospital
Recruitment postcode(s) [1]
187
2145
Recruitment postcode(s) [2]
188
3052
Funding & Sponsors
Primary sponsor
Commercial sector/Industry
Primary sponsor name
iECURE Inc
Primary sponsor address
1777 Sentry Parkway West
Building 14, Suite 200
Blue Bell PA, 19422
Primary sponsor country
United States of America
Other collaborator category [1]
104
Commercial sector/Industry
Name [1]
104
Fortrea Australia Pty ltd
Address [1]
104
Suite 3.02, Level 3, Building A, 97 Waterloo Road, Macquarie Corporate Centre, Macquarie Park, NSW 2113
Country [1]
104
Australia
Ethics approval
Ethics application status
Approved
Ethics committee name [1]
77
Sydney Children's Hospitals Network Human Research Ethics Committee
Address [1]
77
Email:
[email protected]
(preferred) Mailing Addresses: The Children’s Hospital at Westmead, Kids Research Building, Corner Hawkesbury Road and Hainsworth Street, Westmead NSW 2145. Locked Bag 4001, Westmead NSW 2145
Country [1]
77
Australia
Date submitted for ethics approval [1]
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20/10/2023
Approval date [1]
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24/11/2023
Ethics approval number [1]
77
2023/ETH02015
Public notes
Contacts
Principal investigator
Title
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Dr
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Name
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Shanti Balsubramaniam
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Address
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The Children's Hospital at Westmead, Corner Hawkesbury Road and Hainsworth Street, Westmead NSW 2145, Australia
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Country
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Australia
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Phone
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+61 02 7825 0201
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Fax
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Email
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[email protected]
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Contact person for public queries
Title
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Mr
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Name
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George Diaz M.D. Ph.D.
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Address
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1777 Sentry Parkway West Building 14, Suite 200 Blue Bell PA, 19422
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Country
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United States of America
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Phone
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+1 877 694 3558
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Title
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Mr
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Name
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George Diaz M.D. Ph.D.
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Address
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1777 Sentry Parkway West Building 14, Suite 200 Blue Bell PA, 19422
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Country
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United States of America
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Phone
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+1 877 694 3558
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Fax
447
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Email
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[email protected]
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Download to PDF