Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

Launched by IECURE, INC. · Jan 28, 2025

Keywords

ClinConnect Summary

This clinical trial, called the Long-term Follow-up (LTFU) Study, is focused on understanding the long-term safety and effectiveness of a treatment for a rare genetic condition known as Ornithine Transcarbamylase Deficiency (OTC Deficiency). The study is looking at both participants who have received the treatment in a previous study and those who were enrolled but did not receive the treatment. By collecting this information, researchers hope to learn more about how this condition affects individuals over time and how well the treatment works in the long run.

To be eligible for this study, participants must have previously taken part in a related research study and either completed it or decided not to continue. Additionally, participants’ parents or legal representatives need to agree to follow the study's guidelines and provide consent. Participants in this study can expect regular check-ins where their health and well-being will be monitored. This study is currently recruiting individuals aged between 21 and 45 years and welcomes all genders. If you or someone you know is interested in this study, it could be an important opportunity to contribute to our understanding of this condition and its treatment.

Gender

ALL

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 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.