BridgeMD
Phase I/II clinical trial of autologous hematopoietic stem cell gene therapy in rag1-deficient severe combined immunodeficiency
EU CTIS ID: 2023-510204-50-00
What this study is testing
To evaluate the effect of RAG1 gene therapy on overall survival To evaluate the efficacy of RAG1 gene therapy in achieving reconstitution of the T and B cell immune system in patients with RAG1-SCID at 6 months To evaluate the safety and tolerability of the RAG1 gene therapy product, including identification of short- and long-term adverse events
- Human Pharmacology (Phase I)- Other
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
- RAG1-deficient SCID as confirmed by genetic analysis
- Peripheral blood T cells < 300/μL and/or naïve T cells < 1/μL
- Age < 2 years
- Age at least 8 weeks by the time of busulfan and fludarabine administration
- Lack of an available HLA-matched donor (HLA-identical sibling or 10/10 (A, B, C, DR, DQ) allele-matched (un)related donor)
- Signed informed consent (parental or guardian)
You likely can't join if
- Omenn syndrome
- Previous allogeneic HSCT
- Significant organ dysfunction/co-morbidity (including but not limited to): Mechanical ventilation, Shortening fraction on echocardiogram <25%, Renal failure defined as dialysis dependence, uncontrolled seizure disorder.)
- Any other condition that the investigator considers is a contraindication to collection and/or infusion of transduced cells for that individual or indicate patient's inability to follow the protocol, for example contraindication f to busulfan, major congenital abnormalities, ineligible to receive anaesthesia, or documented refusal or inability of the family to return for scheduled visits.
- Human immunodeficiency virus (HIV) infection or Human T-cell Leukemia Virus (HTLV) infection.
See the full eligibility criteria
- RAG1-deficient SCID as confirmed by genetic analysis
- Peripheral blood T cells < 300/μL and/or naïve T cells < 1/μL
- Age < 2 years
- Age at least 8 weeks by the time of busulfan and fludarabine administration
- Lack of an available HLA-matched donor (HLA-identical sibling or 10/10 (A, B, C, DR, DQ) allele-matched (un)related donor)
- Signed informed consent (parental or guardian)
- Able to return to the local HSCT centre for follow-up (per protocol) during the 2-year study and the 15-year long-term off study review
- Absence of peripheral blood naïve CD4+ T cells
- Omenn syndrome
- Previous allogeneic HSCT
- Significant organ dysfunction/co-morbidity (including but not limited to): Mechanical ventilation, Shortening fraction on echocardiogram <25%, Renal failure defined as dialysis dependence, uncontrolled seizure disorder.)
- Any other condition that the investigator considers is a contraindication to collection and/or infusion of transduced cells for that individual or indicate patient's inability to follow the protocol, for example contraindication f to busulfan, major congenital abnormalities, ineligible to receive anaesthesia, or documented refusal or inability of the family to return for scheduled visits.
- Human immunodeficiency virus (HIV) infection or Human T-cell Leukemia Virus (HTLV) infection.
The study team makes the final eligibility decision.
Where it's taking place
- Australia
- Turkey
- United Kingdom
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. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Australia; Turkey; United Kingdom. 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.