Hematology
Anemia
From patients with PKD (Pyruvate Kinase)
>10 pediatric with Pyruvate Kinase Deficiency (PKD)
Cohort with Sickle cell disease with STA
Samples from Sickle cell disease with STA
From patients with G6PD (Blood -Urine – Saliva)
80 pediatric and 20 adult with G6P Deficiency
From patients with Red blood cell membrane anomalies (Blood – Urine – Saliva)
35 pediatric with Red blood cell membranes anomalies
Models from patients with Human erythroid progenitor cell line (K562)
Protocols for genome editing of hematopoietic stem cells for ex vivo gene therapy of SCD
Protocols for lentiviral transduction of hematopoietic stem cells for ex vivo gene therapy of SCD
Samples from patients with Sickle cell disease (Blood – Urine – Saliva – Red blood cells)
Samples from patients with Sickle cell disease and Vasocclusive Crisis (VOCs)
Recombinant lentiviral vector for stem cell-based gene therapy of Sickle cell disorder
Bifunctional lentiviral vectors allowing the bs-globin silencing and expression of an anti-sickling HBB and uses thereof for gene therapy of sickle cell disease
Masitinib for the treatment of sickle cell disease
>1000 pediatric and adult (ESCORT-HU) with Sickle cell disease
>1000 pediatric and adult with Sickle cell disease with Vasocclusive Crisis (VOCs)
NCT04707235 – ESCORT-HU Extension: European Sickle Cell Disease Cohort – Hydroxyurea – Extension Study
NCT04628585 – Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy
NCT06019208 – GenoMed4ALL: Improving SCD Classification and Prognosis by AI
NCT05470270 – Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
NCT05084521 – Interest of Famotidine in Children With Sickle Cell Disease
NCT05053932 – Long-term Comparative Cerebrovascular Outcome After Transplantation vs Standard Care in Sickle Cell Anemia
From patients with Congenital dyserythropoiesis (Blood – Urine – Saliva)
2 Isolated patients (Congenital dyserythropoiesis)
From patients with Beta-thalassemia
From patients with CD34+ hematopoietic stem/progenitor cells (HSPCs – mobilized or cord blood-derived)
From patients with Human erythroid progenitor cell line (K562)
Human erythroid progenitor cell line (Beta-thalassemia)
Samples From patients with CD34+ hematopoietic stem/progenitor cells (HSPCs – mobilized or cord blood-derived)
Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies
Bifunctional vectors allowing BCL11A silencing and expression of an anti-sickling HBB and uses thereof for gene therapy of β-hemoglobinopathies
30 pediatric and adults (Beta-thalassemia)
Coagulation disorders
From patients with Hemophilia A
250 pediatric and adult with Hemophilia A
NCT04370054 – Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
NCT04431726 – A Study to Evaluate the Efficacy Safety Pharmacokinetics and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
NCT05053139 – A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors
NCT03938792 – Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
NCT04384341 – Haemophilia and Bone Loss – PHILEOS Study
NCT05145127 – Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
NCT05611801 – A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
NCT05135559 – Open-label Study Investigating Efficacy Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors (Explorer10)
NCT05605678 – Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)
NCT05789524 – The Efficacy and Safety of SerpinPC in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
From patients with Hemophilia B
40 pediatric Hemophilia B
NCT03938792 – Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
NCT04384341 – Haemophilia and Bone Loss – PHILEOS Study
NCT05145127 – Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
NCT05611801 – A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
NCT05135559 – Open-label Study Investigating Efficacy Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors (Explorer10)
NCT05605678 – Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)
NCT05789524 – The Efficacy and Safety of SerpinPC in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
NCT04272554 – AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
NCT05086575 – Observatory of Patients With Haemophilia B Treated by IdElvion®
NCT05789537 – A Study of SerpinPC in Participants With Hemophilia B (HemB) With Inhibitors
Tumor of hemapoietic and lymphoid tissues
NCT04920617 – DPX-Survivac and Pembrolizumab With and Without Intermittent Low-Dose Cyclophosphamide in Subjects With Relapsed/Refractory Diffuse Large B-Cell Lymphoma
Blasts derived from patients with CLL
Mice – CLL grafted mice (blasts)
Tumor grafting in mice (CLL)
Patient cohort
Blasts derived from patients with CML
Mice – CML grafted mice (blasts)
Tumor grafting in mice (CML)
Patient cohort
Blasts derived from ALL patients
Cell lines from patients with ALL
Mice – ALL grafted mice (blasts)
Tumor grafting in mice
Patient cohort
NCT03750994 – Economic Evaluation of Innovative Molecular Analyses in Onco-haematology (PRME-K 2016)
Blood from patients with mastocytosis
Methods and pharmaceutical compositions for the treatment of mast cell diseases
Methods and pharmaceutical compositions for the treatment of systemic mastocytosis
>500 Pediatric and adult (Mastocytosis)
NCT04910685 – (HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
NCT06186856 – Epidemiological Data on Mast Cell Pathologies in France
NCT04703192 – Valemetostat Tosylate (DS-3201b) an Enhancer of Zeste Homolog (EZH) 1/2 Dual Inhibitor for Relapsed/Refractory Peripheral T-Cell Lymphoma (VALENTINE-PTCL01)
NCT04984837 – Study of Lacutamab in Peripheral T-cell Lymphoma
NCT05444712 – Transplantation After Complete Response In Patients With T-cell Lymphoma
Mice – MDS grafted mice (bone marrow – blasts)
Tumor grafting in mice (MDS)
Xpo1 antagonist kpt-251 for the treatment of myelodysplastic syndrome
Patient cohort
NCT03750994 – Economic Evaluation of Innovative Molecular Analyses in Onco-haematology (PRME-K 2016)
NCT05181735 – Study Evaluating Combination of Luspatercept in LR-MDS Without RS Having Failed or Being Ineligible to ESA
Blasts derived from patients with mantle cell lymphoma
Lymphoma cell lines
Mantle cell lymphoma grafted mice (blasts)
Tumor grafting in mice (Mantle cell lymphoma)
Patient cohort
NCT05471843 – Study of BGB-11417 Monotherapy in Participants With Relapsed or Refractory Mantle Cell Lymphoma
NCT04802590 – Study of Ibrutinib + CD20 Antibody and Venetoclax in Patients With Untreated Mantle Cell Lymphoma
Bone marrow from patients with mast cell leukemia
Mice – Mast cell leukemia grafted mice (bone marrow)
Protocol with Mast cell leukemia grafted mice (bone marrow)
Tumor grafting in mice (Mast cell leukemia)
Patient cohort
Patient-derived EATL cells grafted mice
RCD2 patient-derived EATL cells
RCD2 patient-derived EATL cells
Mice – Human PDX in NOD SCID mice
Blasts derived from patients with AML
Cell lines from patients with AML
Human AML cell lines (AML)
Mice – AML grafted mice (blasts)
Tumor grafting in mice (AML)
Patient cohort associated with GOELAMS
NCT03750994 – Economic Evaluation of Innovative Molecular Analyses in Onco-haematology (PRME-K 2016)
NCT05020665 – Entospletinib Plus Intensive Induction/Consolidation Chemotherapy in Newly Diagnosed NPM1-mutated AML
NCT05193448 – A Non-interventional Ambispective Real-world Cohort of rEfractory and reLapsed (R/R) FLT3 Mutated Acute MyEloid Leukemia (AML) Patients Treated With Gilteritinib in FrANCE
NCT05070208 – Multicenter Retrospective Observatory of Patients With Acute Myeloid Leukemia to Core Binding Factor
NCT04777916 – Prospective Non-interventional Study of Adult Patients With Acute Myeloid Leukemia (AML)
NCT05260528 – CPX-351 vs Intensive Chemotherapy in Patients With de Novo Intermediate or Adverse Risk AML Stratified by Genomics
