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Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) in patients with Fanconi Anaemia Subtype A: FANCOLEN-I
EU CTIS ID: 2024-511523-33-00
What this study is testing
To assess survival in subjects treated in the parent study (FANCOLEN-1) To evaluate long term (LT) safety following infusion of hematopoietic cells transduced with the therapeutic lentiviral LV vector. To determine long term (LT) persistence of the therapeutic LV (provirus) in hematopoietic cells in the bone marrow (BM) & blood, & evaluate potential correlations between provirus/transgene persistence & hematologic stability To determine long term (LT) clonality patterns beyond the 3-year follow-up stipulated in parent study. To evaluate, when relevant , replication competent lentivirus (RCL) in serum and peripheral blood (PB) cells To determine long-term (LT) stability & normalization of blood counts in subjects after RP-L102 infusion To determine the phenotypic correction of BM and PB cells in in long-term follow-up after gene therapy. To enable preliminary assessment of the incidence of hematologic malignancies and solid organ tumors.
- Phase I and Phase II (Integrated)- 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
- Subjects must meet all the following criteria to be included in the study: 1. Was enrolled in the clinical phase 1/2 study FANCOLEN-I. 2. Received infusion of autologous CD34+ enriched gene corrected hematopoietic cells in clinical phase 1/2 study FANCOLEN-I. 3. Is willing and able to adhere to the study visit schedule and other protocol requirements. 4. Provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements. Patients who have undergone allogeneic HSCT (either because of bone marrow failure or leukemia/MDS) will also be followed in this protocol. Evaluations for VCN in HSCT recipients will not be performed if 3 prior assessments did not indicate presence of provirus (transgene) in any evaluated cell population.
You likely can't join if
- There are no criteria for exclusion in this study.
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. The study team makes the final eligibility decision.
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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.