BridgeMD
An Open Label, Single Arm, Phase I/II Clinical Study of Autologous CD4+ T-Cells Edited Ex-Vivo at the CD40LG Locus by CRISPR/Cas9 and IDLV-based vector in Patients with X-linked Hyper IgM Syndrome Type 1 (HIGM1)
EU CTIS ID: 2025-524635-39-00
What this study is testing
To evaluate safety of the treatment with FT018 in patients with HIGM1
- Phase I and Phase II (Integrated)- First administration to humans
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
- Signature of informed consent
- Male subject
- ≥ 1year of age
- Lansky/Karnofsky ≥ 80%
- Genetic diagnosis of HIGM1
- Absent/reduced expression or functionality of CD40L on CD4+ T-cells upon in vitro stimulation with PMA/ionomycin
You likely can't join if
- CD40LG mutation upstream of the vector insertion site in CD40LG intron 1 or any other mutation not amenable to correction with gene editing
- Contraindication to vaccination included in the protocol or other medications/procedures foreseen in the protocol
- Exposure to any prior cell or gene therapies
- Documented HIV RNA, HCV RNA or HBV DNA positivity, presence of total syphilis antibodies and compliance to Tissue Directive (manufacturing issue)
- End-organ dysfunction or other severe disease or clinical condition which, in the judgment of the Investigator, would make the subject inappropriate for entry into this study
- Has previously undergone allogeneic hematopoietic stem cell transplantation and has evidence of residual cells of donor origin.
See the full eligibility criteria
- Signature of informed consent
- Male subject
- ≥ 1year of age
- Lansky/Karnofsky ≥ 80%
- Genetic diagnosis of HIGM1
- Absent/reduced expression or functionality of CD40L on CD4+ T-cells upon in vitro stimulation with PMA/ionomycin
- Requirement of chronic IgRT
- Good adherence to IgRT
- CD40LG mutation upstream of the vector insertion site in CD40LG intron 1 or any other mutation not amenable to correction with gene editing
- Contraindication to vaccination included in the protocol or other medications/procedures foreseen in the protocol
- Exposure to any prior cell or gene therapies
- Documented HIV RNA, HCV RNA or HBV DNA positivity, presence of total syphilis antibodies and compliance to Tissue Directive (manufacturing issue)
- End-organ dysfunction or other severe disease or clinical condition which, in the judgment of the Investigator, would make the subject inappropriate for entry into this study
- Has previously undergone allogeneic hematopoietic stem cell transplantation and has evidence of residual cells of donor origin.
- Patient enrolled in other clinical trials
- Patient unable to donate a sufficient number of lymphocytes for drug product manufacturing
- Systemic corticosteroid therapy or other immunosuppressive drugs that may interfere With apheresis or the DP
The study team makes the final eligibility decision.
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 male, 65+ years, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
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.