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New treatment option for Ornithine Transcarbamylase Deficiency
Official title An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
ClinicalTrials.gov ID: NCT06255782
What this study is testing
What is ECUR-506?
ECUR-506 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for ornithine transcarbamylase deficiency.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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 detoxifying of ammonia. Individuals with OTC deficiency can develop elevated levels of ammonia in the blood, potentially resulting in severe consequences, including cumulative and irreversible neurological damage, coma, and death.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 24 to 7, men only
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
- Weight ≥ 3.5 kg and ≤ 13.5 kg at screening
- Has received age-appropriate vaccinations
You likely can't join if
- Neonatal diagnosis of severe to profound Hypoxic Ischemic Encephalopathy due to birth injury
- Requiring urgent liver transplant due to liver failure as assessed by the PI.
- 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.
- Known or suspected major organ injury/dysfunction/anomalies.
- Vital sign and laboratory abnormalities outside of reference ranges.
- Treatment with any other gene therapy or gene editing therapy
See the full eligibility criteria
- Male sex
- Gestational or adjusted (corrected) gestational age ≥ 37 weeks
- Age at screening is 24 hours to 7 months
- Weight ≥ 3.5 kg and ≤ 13.5 kg at screening
- Has received age-appropriate vaccinations
- Genetically confirmed OTCD defined by genetic confirmation of an OTC variant (pathogenic or likely pathogenic) associated with severe neonatal OTCD defined below in #7 or has the same OTC variant as a family member who...
- Severe neonatal OTCD defined by hyperammonemic crisis with elevated ammonia level of \>560 μmol/L and clinical symptoms within first week of life, and currently receiving treatment with both dietary protein restriction...
- Current or historical biochemical profile consistent with OTCD
- Participant's parent(s)/LAR must be able to comprehend and be willing to provide a signed IRB/IEC-approved ICF. Key
- Neonatal diagnosis of severe to profound Hypoxic Ischemic Encephalopathy due to birth injury
- Requiring urgent liver transplant due to liver failure as assessed by the PI.
- 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.
- Known or suspected major organ injury/dysfunction/anomalies.
- Vital sign and laboratory abnormalities outside of reference ranges.
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Aurora, Colorado, United States
- Atlanta, Georgia, United States
- Chicago, Illinois, United States
- New York, New York, United States
- Portland, Oregon, United States
- Sydney, New South Wales, Australia
- Melbourne, Victoria, Australia
- Barcelona, Spain
- Madrid, Spain
- London, United Kingdom
- Newcastle upon Tyne, United Kingdom
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, 24 hours to 7 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Aurora, Colorado, United States; Atlanta, Georgia, United States; Chicago, Illinois, United States; New York, New York, United States; Portland, Oregon, United States and 6 more location(s). 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 ClinicalTrials.gov; the study team decides eligibility.