Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem a… (NCT06155500) | Clinical Trial Compass
Active — Not RecruitingPhase 1
Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
United States4 participantsStarted 2024-04-16
Plain-language summary
CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy.
Who can participate
Age range
18 Years – 100 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Patients must have received gene therapy treatment from the parent treatment protocol (CADPT03A12101).
. Patients must provide informed consent prior to their entry into this study.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a long-term follow-up study for people who already received OTQ923 gene-edited stem cell therapy, does my treatment history make me eligible to be followed in this study, or is it only for patients who were treated in the original trial?
2This study is specifically tracking delayed adverse events that may be related to the OTQ923 genome-editing therapy — what kinds of late-appearing side effects are they watching for, and how would those affect my ongoing care?
3Since the trial is in Phase 1 and is no longer actively enrolling new participants, what does that mean for how much is still unknown about the long-term safety of this type of genome-edited stem cell treatment?
4How long would I be expected to participate in this follow-up study, and what does ongoing monitoring actually involve in terms of visits, tests, or check-ins?
5If delayed adverse events are found in participants of this study, how might that information change the treatment options you'd recommend for someone with sickle cell disease like me?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy