Long-term follow-up for participants in an iECURE protocol
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
iECURE, Inc. · NCT06805695
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 |
|---|---|
| Enrollment | 13 (estimated) |
| Ages | 7 Months to 15 Months |
| Sex | All |
| Sponsor | iECURE, Inc. (industry) |
| Locations | 1 site (London) |
| Trial ID | NCT06805695 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are individuals who have previously participated in an iECURE parent protocol, regardless of whether they received the investigational product.
Not a fit: Patients currently enrolled in other interventional drug or gene therapy trials utilizing different investigational products may not benefit from this study.
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 performed: 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.
Eligibility criteria
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 this trial is running
London
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn, Amino Acid Metabolism, Inborn Errors, Ammonia, Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn