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An open-label study to investigate ECUR-506 in male children less than 9 months of age with neonatal onset OTC deficiency
EU CTIS ID: 2023-506180-34-01
What this study is testing
To assess the safety, tolerability and efficacy of up to three dose levels of ECUR-506 following IV administration of a single dose.
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male sex
- Gestational or adjusted (corrected) gestational age ≥ 37 weeks
- Age at screening is 24 hours to 7 months
- Genetically confirmed OTC deficiency (OTCD) defined by the following: • Genetic confirmation of an OTC variant (pathogenic or likely pathogenic) associated with severe neonatal OTCD as defined below or has the same OTC variant as a family member who had severe neonatal OTCD within first week of life • Note: a prenatal genetic diagnosis will be confirmed post-birth and prior to dosing.
- Severe neonatal OTCD defined by the following: • Documented hyperammonemic crisis with elevated ammonia level of >560 μmol/L and clinical symptoms that include but are not limited to lethargy, poor feeding, coma, seizure and/or other neurologic sequelae) within first week of life and currently receiving treatment with dietary protein restriction and nitrogen scavenger therapy.
- Current or historical (within 2 weeks prior to Screening) biochemical profile consistent with OTCD: below LLN of plasma citrulline/arginine and urine orotic aciduria at time of diagnosis. Note: This is not applicable for a participant expectantly managed.
You likely can't join if
- Neonatal diagnosis of severe to profound Hypoxic Ischemic Encephalopathy (based on standard HIE metrics) due to birth injury
- Requiring urgent liver transplant due to liver failure as assessed by the principal investigator
- Contiguous gene deletion syndrome involving the OTC gene and including at least the CYBB gene on the telomeric side or the TSPAN7 gene on the centromeric side.
- Known or suspected major organ injury/dysfunction/anomalies (brain, heart, liver, kidneys) other than what is consistent with OTCD, based on routine medical assessments performed as part of standard care
- Treatment with any other gene therapy or gene editing therapy
- Co-enrollment in any other study unless approved by the sponsor
See the full eligibility criteria
Who can join
- Male sex
- Gestational or adjusted (corrected) gestational age ≥ 37 weeks
- Age at screening is 24 hours to 7 months
- Genetically confirmed OTC deficiency (OTCD) defined by the following: • Genetic confirmation of an OTC variant (pathogenic or likely pathogenic) associated with severe neonatal OTCD as defined below or has the same OTC variant as a family member who had severe neonatal OTCD within first week of life • Note: a prenatal genetic diagnosis will be confirmed post-birth and prior to dosing.
- Severe neonatal OTCD defined by the following: • Documented hyperammonemic crisis with elevated ammonia level of >560 μmol/L and clinical symptoms that include but are not limited to lethargy, poor feeding, coma, seizure and/or other neurologic sequelae) within first week of life and currently receiving treatment with dietary protein restriction and nitrogen scavenger therapy.
- Current or historical (within 2 weeks prior to Screening) biochemical profile consistent with OTCD: below LLN of plasma citrulline/arginine and urine orotic aciduria at time of diagnosis. Note: This is not applicable for a participant expectantly managed.
- Participant’s parent/legal authorized representative must be able to comprehend and be willing to provide a signed IRB/IEC) approved ICF which will include consent for participation in this 24- week protocol with immediate roll-over into the 14.5 year ECUR-LTFU protocol
- Weight ≥ 3.5 kg and ≤ 13.5 kg at screening
- Has received age-appropriate vaccinations.
What rules you out
- Neonatal diagnosis of severe to profound Hypoxic Ischemic Encephalopathy (based on standard HIE metrics) due to birth injury
- Requiring urgent liver transplant due to liver failure as assessed by the principal investigator
- Contiguous gene deletion syndrome involving the OTC gene and including at least the CYBB gene on the telomeric side or the TSPAN7 gene on the centromeric side.
- Known or suspected major organ injury/dysfunction/anomalies (brain, heart, liver, kidneys) other than what is consistent with OTCD, based on routine medical assessments performed as part of standard care
- Treatment with any other gene therapy or gene editing therapy
- Co-enrollment in any other study unless approved by the sponsor
- Any condition, that in the opinion of the Investigator, would compromise the safety of the participant or study data
- Documented vertical transmission of HepA/HepB/HepC
- 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
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
- Turkey
- Australia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom; Turkey; Australia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.