Gene Therapy for Wiskott-Aldrich Syndrome (NCT01515462) | Clinical Trial Compass
CompletedPhase 1/2
Gene Therapy for Wiskott-Aldrich Syndrome
Italy8 participantsStarted 2010-04-20
Plain-language summary
This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.
Who can participate
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
. Diagnosis of WAS defined by genetic mutation and at least one of the following criteria:
. No HLA-identical sibling donor
. Negative search for a matched unrelated donor (10/10) or an adequate unrelated cord blood donor (5-6/6) within 4-6 months
. Parental/guardian/patient signed informed consent.
Exclusion criteria
. Patients positive for HIV-infection.
. Patients affected by neoplasia.
. Patients with cytogenetic alterations typical of MDS/AML.
. Patients with end-organ functions or any other severe disease which, in the judgement of the investigator, would make the patient inappropriate for entry into this study.
. Patients who underwent an allogeneic haematopoietic stem cell transplantation in the previous 6 months.
. Patients who underwent an allogeneic haematopoietic stem cell transplantation with evidence of residual cells of donor origin.
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
Safety of Reduced Conditioning Regimen
Timeframe: Follow up phase - Median duration: 11.1 years (range: 8.01 -13.3 years)
2
Safety of Lentivirus Gene Transfer Into HSC
Timeframe: after 48 hours after Telethon003 infusion
3
Sustained Engraftment of Genetically Corrected Haematopoietic Stem Cells in Peripheral Blood and/or in Bone Marrow
Timeframe: at 1 year after Telethon003 infusion
4
Presence of Detectable Vector-derived WASP
Timeframe: Median duration: 11.1 years (range: 8.01 -13.3 years)
5
Improved T-cell Functions
Timeframe: Follow up phase - Median duration: 11.1 years (range: 8.01 -13.3 years)
6
Antigen-specific Responses to Vaccination
Timeframe: Follow up phase - Median duration: 11.1 years (range: 8.01 -13.3 years)