Ornithine Transcarbamylase DeficiencyPhase 3NCT06255782
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
A Phase 3 study testing ECUR-506 in people with Ornithine Transcarbamylase Deficiency and Ornithine Transcarbamylase Deficiency Disease, run by iECURE, Inc. at 12 sites in 4 countries.
Trial 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 detoxifying of ammonia. Read more about this study
- Who is it for?
- Ages 24 hours to 7 months, men only
- Conditions the study is for
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder)
- What is being tested?
- ECUR-506
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 12 sitesin 4 countries
- Study dates
- 8 Apr 2024to Dec 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 24 hours to 7 months
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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 Inclusion Criteria #7 or has the same OTC variant as a family member who had severe neonatal OTCD within first week of life.
Show all 9Show fewer
- 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 and nitrogen scavenger therapy.
- 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.
Who cannot join
- 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
Show all 10Show fewer
- 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
Trial locations and contacts
12 sites in 4 countries.
12 sites in 4 countries
United States6 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
Children's Hospital of Colorado, Anshutz Medical Campus
Aurora, Colorado, 80045
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
UCLA Mattel Children's Hospital
Los Angeles, California, 90095
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Oregon Health and Science University
Portland, Oregon, 97239
Australia2 sites
The Royal Children's Hospital
Melbourne, Victoria, 3052
The Children's Hospital at Westmead
Sydney, New South Wales
Spain2 sites
Hopsital Sant Joan de Deu
Barcelona, 08950
Hospital Universitario 12 de Octubre
Madrid, 28041
United Kingdom2 sites
Great Ormond Street Hospital
London
The Newcastle upon Tyne Hospitals NHS Foundation Trust- Great North Children's Hospital
Newcastle upon Tyne
About this study
Official 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
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. The most severe form presents shortly after birth and occurs more commonly in boys than girls.
This is a Phase 1/2/3, open-label, multicenter study evaluating the safety, efficacy, and dose of ECUR-506 in male babies with neonatal-onset OTC deficiency. The primary objective 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.
Read the detailed description
The study drug, ECUR-506, is an investigational gene editing therapy. Gene editing is an approach used to repair, replace, or introduce functional copies of genes that are not working properly. ECUR-506 contains a functional copy of the OTC gene, along with a gene to encode an editing enzyme that enables insertion of the OTC gene into the genome. The study drug is administered as a single IV infusion. Because genes cannot enter cells on their own, ECUR-506 uses a delivery system based on adeno-associated virus (AAV), a commonly used viral vector, to transport the genetic material into cells.
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
- Being tested
- ECUR-506
- Keywords
- Amino Acid Metabolism, Inborn Errors, Ammonia, Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Central Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, High Ammonia, Hyperammonemia, Inborn, Inborn Errors, Inherited Metabolic Disorders, Liver Disease, Liver Transplant, Metabolism, Metabolic Diseases, Metabolism, Inborn Errors, Neonatal, Nervous System Diseases
- Medical subject headings
- Ornithine Carbamoyltransferase Deficiency Disease, Urea Cycle Disorders, Inborn, Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Central Nervous System Diseases, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ECUR-506Genetic
ECUR-506 is a gene editing treatment delivering a gene encoding the editing enzyme and an OTC gene.
| Study group | Role | What they receive |
|---|---|---|
| Low Dose Level | Experimental | Participants will receive the Low Dose of ECUR-506 delivered one time via IV Infusion. Receives: Genetic: ECUR-506 |
| Intermediate Dose Level | Experimental | Participants will receive an intermediate dose of ECUR-506 delivered on time via IV infusion Receives: Genetic: ECUR-506 |
| High Dose Level | Experimental | Participants will receive a higher dose of ECUR-506 delivered one time via IV infusion. Receives: Genetic: ECUR-506 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent adverse events (incidence, severity, seriousness, and relatedness)
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.
Measured: Over 24 weeks post infusion
Physical exam parameters
Safety- (Includes PI assessment of General Appearance, Dermatological, HEENT, Lymphatic, Respiratory, Cardiovascular, Gastrointestinal, Musculoskeletal, Neurological (Cranial Nerve Function, Motor System Function, Sensory System Function, Primitive Reflexes))
Measured: Assessed as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Vital sign parameters
Safety- (Includes PI assessment of Systolic Blood Pressure, Diastolic Blood Pressure, Pulse Rate, Respiratory Rate, Temperature)
Measured: Assessed as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Pediatric neurologist exam parameters
Safety- (Includes Pediatric Neurologist assessment of Neurological status by review of Cranial Nerve Function, Motor System Function, Sensory System Function, Primitive Reflexes.)
Measured: Assessed as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Blood safety tests including hematology, serum chemistry, liver function tests, coagulation tests
Safety- (Blood Safety tests to be reviewed in relation to established normal ranges for each assessment for the applicable age group)
Measured: as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Urinalysis evaluations
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.
Measured: Assessed as change from baseline at pre-specified timepoints through Week 24 post infusion.
12 lead ECG parameters
Safety- (Includes PI review of Heart Rate, PR Interval, RR Interval, QRS Duration, QT Interval, QTcF Interval, Overall Interpretation)
Measured: as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Complete clinical response
Discontinuation of scavenger medication for a minimum duration of 28 days without reductions in prescribed daily protein intake during this time period by end of study.
Measured: Over 24 weeks post infusion
Secondary outcomes (14)
Number of HAEs/person-year
Key Secondary Endpoint: Pharmacodynamics and Efficacy
Measured: Day 1 post dose through Week 24
qPCR measurement to evaluate the clearance of both vectors in body fluids over time
Supportive Secondary Endpoint: Pharmacokinetics
Measured: Over 24 weeks post infusion
Incidence of hyperammonemic episode (HAE)
Supportive Secondary Endpoint: Pharmacodynamics and Efficacy
Measured: Over 24 weeks post infusion
Incidence and number of hyperammonemic episodes (HAE/HAC) resulting in hospitalization
Supportive Secondary Endpoint: Pharmacodynamics and Efficacy
Measured: Over 24 weeks post infusion
Overall and by hospitalization severity (Mild: adjustment of dietary protein intake and oral scavenger medication / Moderate: cessation of dietary protein intake and initiation of IV scavenger therapy / Severe: requirement for hemodialysis)
Supportive Secondary Endpoint: Pharmacodynamics and Efficacy.
Measured: Will be assessed Day 1 post dose through Wk 24 on all enrolled and dosed participants
Duration of hospitalization for each HAE/HAC
Supportive Secondary Endpoint: Pharmacodynamics and Efficacy
Measured: Over 24 weeks post infusion
Requirement for Intensive Care Unit (ICU) care during hospitalization for each HAE/HAC
Supportive Secondary Endpoint: Pharmacodynamics and Efficacy
Measured: Over 24 weeks post infusion
Time to liver transplant from dosing to end of study (EOS)
Supportive Secondary Endpoint: Efficacy
Measured: Assessed as change from baseline at pre-specified timepoints through Week 24 post infusion.
Transplant free survival
Supportive Secondary Endpoint: Efficacy
Measured: Time to lever transplant or any-cause death from dosing to EOS
Overall survival
Supportive Secondary Endpoint: Efficacy
Measured: Time to any-cause death from dosing to EOS
Achieving and maintaining complete clinical response through end of study
Supportive Secondary Endpoint: Efficacy
Measured: Over 24 weeks post infusion
Scavenger drug dose
Supportive Secondary Endpoint: Efficacy
Measured: Over 24 weeks post infusion
Dietary protein intake g/kg/day
Supportive Secondary Endpoint: Efficacy
Measured: Supportive Secondary: Over 24 weeks post infusion
Blood urea nitrogen measurements
Supportive Secondary Endpoint: Pharmacodynamics
Measured: Over 24 weeks post infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2024
- Study start8 Apr 2024
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ornithine Transcarbamylase DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Dose escalation with dose staggering. Dose selection will be based on an assessment of the totality of the safety and efficacy.
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- iECURE, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- George Diaz, M.D., Ph.D, Study director, iECURE, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06255782
- Sponsor's protocol ID
- ECUR-506-OTC-101
- Other IDs
- OTC-HOPE
- Status last verified
- Sep 2026
- First submitted
- 19 Dec 2023
- Last updated
- 30 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT06255782
- Acronym / sponsor ID
- ECUR-506-OTC-101
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
- Keywords
- Amino Acid Metabolism, Inborn Errors, Ammonia, Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Central Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- iECURE, Inc.Industry (pharma, biotech, devices)
- Study start
- 8 Apr 2024 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 13 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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. The most severe form presents shortly after birth and occurs more commonly in boys than girls.
This is a Phase 1/2/3, open-label, multicenter study evaluating the safety, efficacy, and dose of ECUR-506 in male babies with neonatal-onset OTC deficiency. The primary objective 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.
Detailed description
The study drug, ECUR-506, is an investigational gene editing therapy. Gene editing is an approach used to repair, replace, or introduce functional copies of genes that are not working properly. ECUR-506 contains a functional copy of the OTC gene, along with a gene to encode an editing enzyme that enables insertion of the OTC gene into the genome. The study drug is administered as a single IV infusion. Because genes cannot enter cells on their own, ECUR-506 uses a delivery system based on adeno-associated virus (AAV), a commonly used viral vector, to transport the genetic material into cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Dose escalation with dose staggering. Dose selection will be based on an assessment of the totality of the safety and efficacy.
| Arm | Type | Receives |
|---|---|---|
| Low Dose Level | Experimental | Genetic: ECUR-506Participants will receive the Low Dose of ECUR-506 delivered one time via IV Infusion. |
| Intermediate Dose Level | Experimental | Genetic: ECUR-506Participants will receive an intermediate dose of ECUR-506 delivered on time via IV infusion |
| High Dose Level | Experimental | Genetic: ECUR-506Participants will receive a higher dose of ECUR-506 delivered one time via IV infusion. |
Who may qualify
- Ages
- 24 hours to 7 months
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ECUR-506ECUR-506 is a gene editing treatment delivering a gene encoding the editing enzyme and an OTC gene. | Genetic | High Dose Level, Intermediate Dose Level, Low Dose Level |
Primary outcome
Treatment-emergent adverse events (incidence, severity, seriousness, and relatedness)
Time frame: Over 24 weeks post infusion
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.
Physical exam parameters
Time frame: Assessed as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Safety- (Includes PI assessment of General Appearance, Dermatological, HEENT, Lymphatic, Respiratory, Cardiovascular, Gastrointestinal, Musculoskeletal, Neurological (Cranial Nerve Function, Motor System Function, Sensory System Function, Primitive Reflexes))
Vital sign parameters
Time frame: Assessed as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Safety- (Includes PI assessment of Systolic Blood Pressure, Diastolic Blood Pressure, Pulse Rate, Respiratory Rate, Temperature)
Pediatric neurologist exam parameters
Time frame: Assessed as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Safety- (Includes Pediatric Neurologist assessment of Neurological status by review of Cranial Nerve Function, Motor System Function, Sensory System Function, Primitive Reflexes.)
Blood safety tests including hematology, serum chemistry, liver function tests, coagulation tests
Time frame: as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Safety- (Blood Safety tests to be reviewed in relation to established normal ranges for each assessment for the applicable age group)
Urinalysis evaluations
Time frame: Assessed as change from baseline at pre-specified timepoints through Week 24 post infusion.
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.
12 lead ECG parameters
Time frame: as change from baseline at pre-specified timepoints as described in the SOE throughout the duration of the study on all enrolled and dosed participants.
Safety- (Includes PI review of Heart Rate, PR Interval, RR Interval, QRS Duration, QT Interval, QTcF Interval, Overall Interpretation)
Complete clinical response
Time frame: Over 24 weeks post infusion
Discontinuation of scavenger medication for a minimum duration of 28 days without reductions in prescribed daily protein intake during this time period by end of study.
14 secondary outcomes
Number of HAEs/person-year
Time frame: Day 1 post dose through Week 24
qPCR measurement to evaluate the clearance of both vectors in body fluids over time
Time frame: Over 24 weeks post infusion
Incidence of hyperammonemic episode (HAE)
Time frame: Over 24 weeks post infusion
Incidence and number of hyperammonemic episodes (HAE/HAC) resulting in hospitalization
Time frame: Over 24 weeks post infusion
Overall and by hospitalization severity (Mild: adjustment of dietary protein intake and oral scavenger medication / Moderate: cessation of dietary protein intake and initiation of IV scavenger therapy / Severe: requirement for hemodialysis)
Time frame: Will be assessed Day 1 post dose through Wk 24 on all enrolled and dosed participants
Duration of hospitalization for each HAE/HAC
Time frame: Over 24 weeks post infusion
Requirement for Intensive Care Unit (ICU) care during hospitalization for each HAE/HAC
Time frame: Over 24 weeks post infusion
Time to liver transplant from dosing to end of study (EOS)
Time frame: Assessed as change from baseline at pre-specified timepoints through Week 24 post infusion.
Transplant free survival
Time frame: Time to lever transplant or any-cause death from dosing to EOS
Overall survival
Time frame: Time to any-cause death from dosing to EOS
Achieving and maintaining complete clinical response through end of study
Time frame: Over 24 weeks post infusion
Scavenger drug dose
Time frame: Over 24 weeks post infusion
Dietary protein intake g/kg/day
Time frame: Supportive Secondary: Over 24 weeks post infusion
Blood urea nitrogen measurements
Time frame: Over 24 weeks post infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2026.
- Individual participant dataSharing planned: No
Related trials for Ornithine Transcarbamylase Deficiency
RecruitingPhase 1/2
Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD
Urea Cycle Disorders, Inborn, Urea Cycle Disorders
RecruitingObservational study
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
Active, not recruitingObservational study
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
Ornithine Transcarbamylase (OTC) Deficiency
Active, not recruitingObservational study
Liver Disease in Urea Cycle Disorders
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
George Diaz, M.D., Ph.D study director
iECURE, Inc.
12 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ECUR-506 in people with Ornithine Transcarbamylase Deficiency and Ornithine Transcarbamylase Deficiency Disease, run by iECURE, Inc. at 12 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 30 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Australia, Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment-emergent adverse events (incidence, severity, seriousness, and relatedness) (time frame: Over 24 weeks post infusion). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Sep 2026; AzTrials last refreshed its copy on 26 Oct 2026.