Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Recruiting
Observational Study
Ornithine Transcarbamylase Deficiency
Ornithine Transcarbamylase Deficiency Disease
Urea Cycle Disorders, Inborn
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 7 Months – 15 Months
- Sex
- Any
- Study type
- Observational
- Participants needed
- 13 (estimated)
- Sponsor
- iECURE, Inc. · Industry
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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.
Trial Locations
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Eligibility 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 appl…
Contacts