BridgeMD
A single arm, open label, multicenter, single-dose, phase 2b clinical study evaluating efficacy and safety of gene therapy using autologous CD34+ hematopoietic stem cells transduced with the GLOBE lentiviral vector using an improved transduction protocol in subjects with transfusion-dependent beta-thalassemia.
EU CTIS ID: 2025-522160-32-00
What this study is testing
To evaluate the clinical efficacy of FT007 infusion in terms of transfusion independence in pediatric and adult patients with transfusion dependent beta-thalassemia.
- Therapeutic exploratory (Phase II)
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
- Male and female adults/adolescents/children diagnosed with transfusion-dependent β-thalassemia (homozygous or compound heterozygous). At least 2 out of the 9 patients must have B0/B0 or B0/B0- like genotype. In case the genetic diagnosis available at screening wasn’t performed in a certified laboratory (check under PI’s or delegated investigator’s responsibility), the genetic diagnosis will be repeated at clinical sites during the screening phase
- Documented history of at least 100 mL/kg/year or 10 U/year of packed red blood cell transfusions in each of the 2 years prior to signing informed consent.
- Age ≥ 18 years and ≤ 35 years for Group 1, Age ≥ 3 years and ≤ 35 years for Group 2.
- Karnofsky Index or Lansky ≥ 80%
- Adequate cardiac, renal, hepatic and pulmonary functions resulting in eligibility to undergo autologous HSCT
- Low risk thrombophilic screen and negative history of significant previous thrombotic events
You likely can't join if
- Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long active agents)
- Severe, active viral, bacterial or fungal infection at eligibility evaluation
- Current or prior malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or exceptional family history of familial cancer syndromes
- Current or prior immunodeficiency disorder.
- For patients until the age of 14 years only: availability of an HLA-matched family donor
- Previous allogeneic hematopoietic stem cell transplantation.
See the full eligibility criteria
- Male and female adults/adolescents/children diagnosed with transfusion-dependent β-thalassemia (homozygous or compound heterozygous). At least 2 out of the 9 patients must have B0/B0 or B0/B0- like genotype. In case the genetic diagnosis available at screening wasn’t performed in a certified laboratory (check under PI’s or delegated investigator’s responsibility), the genetic diagnosis will be repeated at clinical sites during the screening phase
- Documented history of at least 100 mL/kg/year or 10 U/year of packed red blood cell transfusions in each of the 2 years prior to signing informed consent.
- Age ≥ 18 years and ≤ 35 years for Group 1, Age ≥ 3 years and ≤ 35 years for Group 2.
- Karnofsky Index or Lansky ≥ 80%
- Adequate cardiac, renal, hepatic and pulmonary functions resulting in eligibility to undergo autologous HSCT
- Low risk thrombophilic screen and negative history of significant previous thrombotic events
- Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long active agents)
- Severe, active viral, bacterial or fungal infection at eligibility evaluation
- Current or prior malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or exceptional family history of familial cancer syndromes
- Current or prior immunodeficiency disorder.
- For patients until the age of 14 years only: availability of an HLA-matched family donor
- Previous allogeneic hematopoietic stem cell transplantation.
- Previous gene therapy treatment (gene addition or gene editing).
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 all sexes, 0-17 years, 18-64 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.