Long-term follow-up for participants in an iECURE protocol
Official title: Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Run by iECURE, Inc. · NCT06805695 · Data updated 2026-11-12
This study looks at the long-term safety and effectiveness of a treatment for people who have already been part of an earlier study, while also tracking the health of those who didn’t receive the treatment.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 7 Months to 15 Months |
| Sex | All |
| Enrollment | 13 (estimated) |
| Sponsor | iECURE, Inc. Industry-sponsored |
| Locations | 4 sites (Aurora, Colorado and 3 other locations) |
| Status | Recruiting |
| Started | December 23, 2024 |
| Main results expected | July 2041 |
| Study ends | July 2041 |
| Registry record updated | November 5, 2026 |
| Trial ID | NCT06805695 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are individuals who have previously participated in an iECURE parent protocol, regardless of whether they received the investigational product.
✕Probably not a fit
Patients currently enrolled in other interventional drug or gene therapy trials utilizing different investigational products may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study aims to assess the long-term safety and effectiveness of an investigational product (IP) in participants who have previously been part of an iECURE protocol. It will also collect longitudinal data on the natural history of the disease in participants who were enrolled but not dosed with the IP. The study will last for 14.5 years, during which participants will continue their standard of care medications as prescribed by their physicians. No investigational product will be administered as part of this follow-up protocol.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term safety and clinical outcomes for patients with Ornithine Transcarbamylase Deficiency.
How similar studies have gone: While this study focuses on long-term follow-up, similar observational studies have shown success in capturing valuable data on chronic conditions, although the specific approach may be novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol 2. Participant parent(s)/LAR is willing and able to adhere to the protocol requirements. 3. Consent was obtained by the participants parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected. Exclusion Criteria: 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.
Where it's running
-
Children's Hospital of Colorado, Anshutz Medical Campus
Aurora, Colorado, United States Recruiting -
Icahn School of Medicine at Mount Sinai
New York, New York, United States Recruiting -
Chu de Lyon - Hopital Femme Mere Enfant
Bron, France Recruiting -
Great Ormond Street Hospital
London, United Kingdom Recruiting
Study contacts
- Study coordinator: George Diaz, M.D., Ph.D.
- Email: medinfo@iecure.com
- Phone: 1-877-694-3558
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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KRRO-121 for hyperammonemia in urea cycle disorders: a first-in-human study
This study tests KRRO-121, a treatment designed to help clear ammonia, in adults with urea cycle disorders and in healthy volunteers to see if it is safe and how it acts in the body.
-
Evaluating ARCT-810 for treating OTC deficiency
This study is testing a new treatment called ARCT-810 to see if it can help adolescents and adults with OTC deficiency feel better while they continue their usual care.
-
Gene therapy for children with Ornithine Transcarbamylase deficiency
This study is testing a new gene therapy for children with Ornithine Transcarbamylase deficiency to see if it can help their liver produce a missing enzyme and reduce dangerous ammonia levels in their blood.
-
Evaluating liver tissue in patients with urea cycle disorders
This study looks at liver tissue from people with urea cycle disorders to see how these conditions affect the liver, especially in those who have had a liver transplant or biopsy.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.