SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
China10 participantsStarted 2024-06-30
Plain-language summary
This is a Phase I/II gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous lentiviral gene transfer protocol.
Who can participate
Age range
1 Month – 1 Year
Sex
MALE
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
. Diagnosis of SCID-X1 based on:
. No available HLA identical related donor.
. With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus; disseminated BCG infection.
. No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia.
. No prior allogeneic stem cell transplantation.
. Life expectancy ≥ 3 months.
. Documented to be negative for HIV infection.
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.
. Written, informed consent obtained prior to any study-specific procedures.
Exclusion criteria
. No available molecular diagnosis confirming SCID-X1.
. Existence of an available HLA-identical related donor.
. Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease.
. Current treatment with any chemotherapeutic agent (becomes eligible if not on treatment for at least 1 month).
. Patients with evidence of infection with HIV-1 or 2.
. Presence of a medical condition indicating that survival will be less than 4 weeks such as the requirement for mechanical ventilation, severe failure of a major organ system, or evidence of a serious, progressive infection that is refractory to medical treatment.
. Current treatment with any immunosuppressive agent, excluding corticosteroids.
. Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.