Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusio… (NCT07527975) | Clinical Trial Compass
By InvitationNot Applicable
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
United States, Spain, United Kingdom14 participantsStarted 2022-02-01
Plain-language summary
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
Who can participate
Sex
ALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Enrolled in one of the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).
. Received an autologous infusion of CD34+ enriched cells transduced ex vivo with LV vector carrying the FANCA gene, PGK-FANCA-WPRE (RP-L102), in the parent studies.
. Able to adhere to the study visit schedule and other protocol requirements.
. Has provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements.
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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
Survival in patients treated in the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).
Timeframe: From infusion in parent study to 15-years post-infusion.
2
Long term safety
Timeframe: From infusion in parent study to 15-years post-infusion.
3
Long-term persistence of the therapeutic LV (provirus) in hematopoietic cells in the bone marrow (BM) and blood.
Timeframe: From infusion in parent study to 15-years post-infusion.
4
Long-term clonality patterns.
Timeframe: From infusion in parent study to 15-years post-infusion.
5
Replication-competent lentivirus (RCL) in serum and peripheral blood cells.
Timeframe: From infusion in parent study to 15-years post-infusion.
6
Long-term stability and normalization of blood counts.
Timeframe: From infusion in parent study to 15-years post-infusion.
7
Phenotypic correction of BM and peripheral blood cells