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A Long-term Follow-up Study for Subjects Previously Treated with Autologous ex vivo Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
EU CTIS ID: 2024-518369-92-00
What this study is testing
- To characterize the long-term safety of Telethon003 treatment. - To characterize the long-term clinical efficacy of Telethon003 treatment.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- The patient has been treated with Telethon003
- The patient or, if applicable, the patient’s parent(s)/legal guardian(s), are able and willing to provide informed consent.
- Willingness to attend the study visit schedule and be compliant with the protocol.
You likely can't join if
- Participation in an ongoing clinical study with an investigational drug (other to gene therapy study or early access, object for this study) prior to enrolment in this study or willingness to participate in another interventional clinical trial. If in deviation to this exclusion criteria, a subject took or takes another investigational drug or other available therapy (e.g. hematopoietic stem cell transplant) he will be followed anyway to collect safety events.
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.