Ornithine Transcarbamylase DeficiencyNCT06805695
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
An observational study of Ornithine Transcarbamylase Deficiency and Ornithine Transcarbamylase Deficiency Disease, run by iECURE, Inc. at 4 sites in 3 countries.
Trial summary
This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol. Read more about this study
- Who is it for?
- Ages 7 to 15 months
- Conditions the study is for
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
- How many people
- 13 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 23 Dec 2024to Jul 2041
- Phase
- Observational
- 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 7 to 15 months
- All sexes
- No healthy volunteers
- Child
Study population: All participants from an iECURE parent treatment protocol, whether they received IP or not, will be consented to enter this LTFU study. Enrollment into the LTFU will begin upon either premature discontinuation from, or completion of, the parent treatment protocol.
Who may be able to join
- Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol
- Participant parent(s)/LAR is willing and able to adhere to the protocol requirements.
- Consent was obtained by the participants parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected.
Who cannot join
- 1. Participants who enroll into an interventional drug or gene therapy clinical trial utilizing an IP other than the IP provided in the parent protocol will be excluded from this protocol.
Trial locations and contacts
4 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 3 countries
United States2 sites, 2 recruiting
- Recruiting
Children's Hospital of Colorado, Anshutz Medical Campus
Aurora, Colorado, 80045
Lanie Dougherty, Lanie.Dougherty@childrenscolorado.org
- Recruiting
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Silvia Gunderson, silvia.gunderson@mssm.edu
France1 site, 1 recruiting
- Recruiting
Chu de Lyon - Hopital Femme Mere Enfant
Bron
Nathalie Guffon-fouilhoux, MD, nathalie.guffon-fouilhoux@chu-lyon.fr
United Kingdom1 site, 1 recruiting
- Recruiting
Great Ormond Street Hospital
London
Christopher Jackson, christopher.jackson@gosh.nhs.uk
About this study
This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol.
Read the detailed description
This is a 14.5 year LTFU protocol to assess long term safety and clinical response to IP as well as to capture natural disease history of participants not dosed with IP in a parent protocol. No IP is provided as part of this protocol. Participants will remain on standard of care medication as indicated and prescribed by their physicians.
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
- Being tested
- No Intervention
- 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, Liver Disease, Liver Transplant, Metabolism, Metabolic Diseases, Metabolism, Inborn Errors, Neonatal, Nervous System Diseases, NH4
- 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.
No InterventionOther
No Intervention
| Study group | Role | What they receive |
|---|---|---|
| Enrolled and Dosed | Previously dosed in a previous iECURE study. Receives: Other: No Intervention | |
| Enrolled but Not Dosed | Enrolled in a previous iECURE study, but not dosed. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
AE and SAE (incidence, severity, seriousness, and relatedness)
Safety
Measured: Over 14.5 years post dosing with ECUR-506
Change from baseline over 14.5 years post infusion in length
Length measured in cenitmeters
Measured: Over 14.5 years post dosing with ECUR-506
Change from baseline over 14.5 years post infusion in weight
Weight measured in kilograms
Measured: Over 14.5 years post dosing with ECUR-506
Urinalysis (Dip Stick) Evaluations
Urinalysis (Dip Stick) will be evaluated to monitor pathological changes to the participants urine and to monitor levels of Specific Gravity, pH, Glucose, Protein, Blood, Ketones, Bilirubin, Urobilinogen, Nitrite, Leukocyte esterase, red blood cell count, and white blood cell count as they relate to established local lab normal ranges.
Measured: Over 14.5 years post dosing with ECUR-506
Secondary outcomes (18)
qPCR measurement to evaluate the clearance of both vectors in blood over time.
Pharmacokinetics
Measured: Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in saliva over time.
Pharmacokinetics
Measured: Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in urine over time.
Pharmacokinetics
Measured: Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in feces over time.
Pharmacokinetics
Measured: Over 14.5 years post dosing with ECUR-506
Percent Liver Transduction
Pharmacokinetics
Measured: Over 14.5 years post dosing with ECUR-506
Number of hyperammonemic crises (HAC)
Pharmacodynamics and Efficacy
Measured: Over 14.5 years post dosing with ECUR-506
Among participants who experience HAC with associated neurological status change and are hospitalized: Asses daily ammonia levels for duration of hospitalization for each event
Pharmacodynamics and Safety
Measured: Over 14.5 years post dosing with ECUR-506
Among participants who experience HAC with associated neurological status change and are hospitalized: Assess duration of hospitalization for each event.
Pharmacodynamics and Safety
Measured: Over 14.5 years post dosing with ECUR-506
Among participants who experience HAC with associated neurological status change and are hospitalized: Assess requirement for ICU care.
Pharmacodynamics and Safety
Measured: Over 14.5 years post dosing with ECUR-506
Number of HAC with the following severities: a. Mild: adjustment of dietary protein intake and oral scavenger medication b. Moderate: cessation of dietary protein intake and initiation of IV scavenger therapy c. Severe: requirement for hemodialysis
Pharmacodynamics and Safety
Measured: Over 14.5 years post dosing with ECUR-506
Scavenger drug dose per body surface area (BSA)
Efficacy
Measured: Over 14.5 years post dosing with ECUR-506
Protein allowance g/kg
Efficacy
Measured: Over 14.5 years post dosing with ECUR-506
Concentration of blood urea nitrogen measurements
Pharmacodynamics
Measured: Over 14.5 years post dosing with ECUR-506
Time to liver transplant from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol)
Efficacy
Measured: Over 14.5 years post dosing with ECUR-506
Transplant free survival
Efficacy
Measured: Over 14.5 years post dosing with ECUR-506
Time to liver transplant or any cause of death from dosing to EOS.
Efficacy
Measured: Over 14.5 years post dosing with ECUR-506
Overall Survival: Survival measured from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol).
Efficacy
Measured: Over 14.5 years post dosing with ECUR-506
Time to any-cause death from dosing to EOS. Survival measured from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol).
Efficacy
Measured: Over 14.5 years post dosing with ECUR-506
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2025
- Study start23 Dec 2024
- Primary completionJul 2041 (estimated)
- Study completionJul 2041 (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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 13 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
- NCT06805695
- Sponsor's protocol ID
- ECUR-LTFU
- Status last verified
- Nov 2026
- First submitted
- 20 Dec 2024
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
- ClinicalTrials.gov ID
- NCT06805695
- Acronym / sponsor ID
- ECUR-LTFU
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, 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
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 13 participants (estimated)
- Lead sponsor
- iECURE, Inc.Industry (pharma, biotech, devices)
- Study start
- 23 Dec 2024 (actual)
- Primary completion
- Jul 2041 (estimated)
- Study completion
- Jul 2041 (estimated)
- First posted
- 3 Feb 2025 (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.
This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol.
Detailed description
This is a 14.5 year LTFU protocol to assess long term safety and clinical response to IP as well as to capture natural disease history of participants not dosed with IP in a parent protocol. No IP is provided as part of this protocol. Participants will remain on standard of care medication as indicated and prescribed by their physicians.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Enrolled and Dosed | – | Other: No InterventionPreviously dosed in a previous iECURE study. |
| Enrolled but Not Dosed | – | Other: No InterventionEnrolled in a previous iECURE study, but not dosed. |
Who may qualify
- Ages
- 7 to 15 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- All participants from an iECURE parent treatment protocol, whether they received IP or not, will be consented to enter this LTFU study. Enrollment into the LTFU will begin upon either premature discontinuation from, or completion of, the parent treatment protocol.
Key inclusion criteria (3 listed)
- Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol
- Participant parent(s)/LAR is willing and able to adhere to the protocol requirements.
- Consent was obtained by the participants parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected.
Key exclusion criteria (1 listed)
- 1. Participants who enroll into an interventional drug or gene therapy clinical trial utilizing an IP other than the IP provided in the parent protocol will be excluded from this protocol.
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 |
|---|---|---|
| No InterventionNo Intervention | Other | Enrolled and Dosed, Enrolled but Not Dosed |
Primary outcome
AE and SAE (incidence, severity, seriousness, and relatedness)
Time frame: Over 14.5 years post dosing with ECUR-506
Safety
Change from baseline over 14.5 years post infusion in length
Time frame: Over 14.5 years post dosing with ECUR-506
Length measured in cenitmeters
Change from baseline over 14.5 years post infusion in weight
Time frame: Over 14.5 years post dosing with ECUR-506
Weight measured in kilograms
Urinalysis (Dip Stick) Evaluations
Time frame: Over 14.5 years post dosing with ECUR-506
Urinalysis (Dip Stick) will be evaluated to monitor pathological changes to the participants urine and to monitor levels of Specific Gravity, pH, Glucose, Protein, Blood, Ketones, Bilirubin, Urobilinogen, Nitrite, Leukocyte esterase, red blood cell count, and white blood cell count as they relate to established local lab normal ranges.
18 secondary outcomes
qPCR measurement to evaluate the clearance of both vectors in blood over time.
Time frame: Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in saliva over time.
Time frame: Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in urine over time.
Time frame: Over 14.5 years post dosing with ECUR-506
qPCR measurement to evaluate the clearance of both vectors in feces over time.
Time frame: Over 14.5 years post dosing with ECUR-506
Percent Liver Transduction
Time frame: Over 14.5 years post dosing with ECUR-506
Number of hyperammonemic crises (HAC)
Time frame: Over 14.5 years post dosing with ECUR-506
Among participants who experience HAC with associated neurological status change and are hospitalized: Asses daily ammonia levels for duration of hospitalization for each event
Time frame: Over 14.5 years post dosing with ECUR-506
Among participants who experience HAC with associated neurological status change and are hospitalized: Assess duration of hospitalization for each event.
Time frame: Over 14.5 years post dosing with ECUR-506
Among participants who experience HAC with associated neurological status change and are hospitalized: Assess requirement for ICU care.
Time frame: Over 14.5 years post dosing with ECUR-506
Number of HAC with the following severities: a. Mild: adjustment of dietary protein intake and oral scavenger medication b. Moderate: cessation of dietary protein intake and initiation of IV scavenger therapy c. Severe: requirement for hemodialysis
Time frame: Over 14.5 years post dosing with ECUR-506
Scavenger drug dose per body surface area (BSA)
Time frame: Over 14.5 years post dosing with ECUR-506
Protein allowance g/kg
Time frame: Over 14.5 years post dosing with ECUR-506
Concentration of blood urea nitrogen measurements
Time frame: Over 14.5 years post dosing with ECUR-506
Time to liver transplant from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol)
Time frame: Over 14.5 years post dosing with ECUR-506
Transplant free survival
Time frame: Over 14.5 years post dosing with ECUR-506
Time to liver transplant or any cause of death from dosing to EOS.
Time frame: Over 14.5 years post dosing with ECUR-506
Overall Survival: Survival measured from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol).
Time frame: Over 14.5 years post dosing with ECUR-506
Time to any-cause death from dosing to EOS. Survival measured from Day 1 (Parent Protocol) for participants who are dosed and Day -1 (Parent Protocol) for participants who are not dosed to end of study EOS (LTFU Protocol).
Time frame: Over 14.5 years post dosing with ECUR-506
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 5 Nov 2026.
- Individual participant dataSharing planned: Undecided
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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.
George Diaz, M.D., Ph.D study director
iECURE, Inc.
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Ornithine Transcarbamylase Deficiency and Ornithine Transcarbamylase Deficiency Disease, run by iECURE, Inc. at 4 sites in 3 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 3 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, France, 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.
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?
Contact George Diaz, M.D., Ph.D. (1-877-694-3558, medinfo@iecure.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
AE and SAE (incidence, severity, seriousness, and relatedness) (time frame: Over 14.5 years post dosing with ECUR-506). The study lists 4 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 5 Nov 2026.