At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- No Intervention (other)
- How long the study runs
- Study runs about 199 months (dates as stated)
- About the drug or intervention
- No Intervention — other: No Intervention
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Observing health over time
- Ages
- 7 Months to 15 Months
- Who
- All
- Number of participants
- 13
- Started
- 2024-12-23
- Last checked
- 2026-08
Plain English Summary
What is this study?
- • Testing a new treatment for ornithine transcarbamylase deficiency
- • Clinical study - 13 participants
- • 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
Who can take part?
- • Ages 7 Months to 15 Months
- • Diagnosed with ornithine transcarbamylase deficiency
Where?
- • London - Great Ormond Street Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
More detail
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 7 Months - 15 Months
- Who can join: All genders
What the study is looking for
- ✓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...
Who cannot take part
- ✗1\. Participants who enroll into an interventional drug or gene therapy clinical trial utilizing an IP other than...
See the full criteria
Where Is This Study? (1 UK site)
Great Ormond Street Hospital
London, United Kingdom
How to Get in Touch
George Diaz, M.D., Ph.D.
Sponsor contactCONTACT