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
ESTADO CLÍNICO
Reclutando
FASE/S DEL ENSAYO
Fase I-II
PATOLOGÍA
Anemias Carenciales, Enfermedad de células falciformes, Linfoma Folicular, Trasplante de progenitores hematopoyéticos, Terapia celular y T-CARs, --> Otros, Linfoma no Hodgkin, Leucemia mieloide aguda, Insuficiencias medulares y aplasia medular, Anemias Congénitas y Hemolíticas, Linfoma
FECHA DE APERTURA
Marzo, 2020
FECHA DE CIERRE
,
CRITERIOS DE INCLUSIÓN
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.
CRITERIOS DE EXCLUSIÓN
There are no criteria for exclusion in this study.
DESCRIPCIÓN
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.
PALABRAS CLAVE
anaemia, anemia, anemia aplásica, anemia de fanconi, anemia hemolítica, aplastic anemia, autologous, autologous transplant, blastic plasmacytoid dendritic cell neoplasm (bpdcn), complement inhibitor, diffuse large b-cell lymphoma (dlbcl) activated b-cell type, diffuse large b-cell lymphoma (dlbcl) germinal center b-cell type, diffuse large b-cell lymphoma (dlbcl), non germinal center b-cell type, fanconi anemia, gene therapy, hematopoietic stem cell transplant, hemolytic anemia, langerhans cell histiocytosis, mantle cell lymphoma, non germinal center b-cell type, non-langerhans cell histiocytosis, ph + acute lymphoblastic leukemia, sickle cell disease, stem cell
