The Clinical trials registered in Clinicaltrial.gov that involve ERN Members and Affiliated partners in at least two Member States are listed below.
| Monitoring period | Title | Members and Affiliated Partners | Member State(s) |
|---|---|---|---|
| 2023 | Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies (SATISFY) | Assistance Publique-Hôpitaux de Paris, Hôpital Saint-Louis, Copenhagen University Hospital - Rigshospitalet and University Medical Center Utrecht. | France, Denmark, Netherlands. |
| 2023 | A Multicenter, open-label study to evaluate the safety, efficacy, pharmacokinetcs and pharmacodynamics of Emicizumab in patients with mild or moderate Hemophilia A without FVIII Inhibitors | University Hospital Leuven; Hospital Universitario Virgen del Rocío; Assistance Publique-Hôpitaux de Paris, Hôpital Necker-Enfants Malades; Academic Medical Center Amsterdam. | Belgium, Spain, France, Netherlands, |
| 2023 | Phase 3, Prospective, Multi-center, Open Label Study to Investigate Safety, Immunogenicity, and Hemostatic Efficacy of PEGylated Factor VIII (BAX 855) in Previously Untreated Patients (PUPs) and Minimally Treated Patients (MTPs) < 6 Years With Severe Hemophilia A (FVIII < 1%) | University Hospital Leuven; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan. | Belgium & Italy. |
| 2023 | A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF With or Without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed With Severe Von Willebrand Disease | University Hospital Leuven; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; IRCCS Ospedale Pediatrico Bambino Gesù, Roma. | Belgium & Italy. |
| 2023 | A multinational, open-label, randomised, controlled trial to investigate efficacy and safety of NNC0365-3769 (Mim8) in adults and adolescents with haemophilia A with or without inhibitors. | University Hospital Leuven; Academic Medical Center Amsterdam; Assistance Publique-Hôpitaux de Paris, Hôpital Necker-Enfants Malades; University Medical Center Utrecht; Vilnius University Hospital Santaros Klinikos; Hospices Civils de Lyon. | Belgium, Lithuania, Netherlands, France. |
| 2023 | PHASE IIIb, MULTICENTER, OPEN-LABEL, SINGLE-ARM STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SUBCUTANEOUS EMICIZUMAB IN PATIENTS FROM BIRTH TO 12 MONTHS OF AGE WITH HEMOPHILIA A WITHOUT INHIBITORS | University Hospital Leuven; Academic Medical Center Amsterdam; Assistance Publique-Hôpitaux de Paris, Hôpital Necker-Enfants Malades; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; Hospital Universitario Virgen del Rocío; Hospital de Sant Joan de Déu- Hospital de la Santa Creu i Sant Pau; Azienda Ospedaliero-Universitaria di Parma. | Belgium, Spain, France, Netherlands, Italy. |
| 2023 | A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels = 1 IU/dL Receiving Prophylactic FVIII Infusions. | University Hospital Leuven; Hospital Universitario Virgen del Rocío; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan. |
Belgium, Spain, Italy. |
| 2023 | Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene AAV5-hFIXco-Padua,AMT-061 administered to adult subjects with severe or moderately severe hemophilia B. | University Hospital Leuven; Academic Medical Center Amsterdam; University Medical Center Utrecht | Belgium & Netherlands. |
| 2023 | 10-day decitabine versus conventional chemotherapy ("3+7") followed by allografting in AML patients >= 60 years: a randomized phase III study of the EORTC Leukemia Group, CELG, GIMEMA and German MDS Study Group | University Hospital Liège; AOU Ospedali Riuniti "Umberto I - G.M. Lancisi-G. Salesi"; Vilnius University Hospital Santaros Klinikos; Radboud University Medical Center Nijmegen. | Belgium, Netherlands Italy, Lithuania. |
| 2023 | Extended Access of Momelotinib for Subjects with Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post essential Thrombocythemia Myelofibrosis (Post-PV/ET MF) | University Hospital Liège 81. S. Gerardo Hospital - Monza. | Belgium & Italy. |
| 2023 | A phase 3, multicenter, double-blind, randomized, placebo-controlled study of ivosidenib or enasidenib in combination with induction therapy and consolidation therapy followed by maintenance therapy in patients with newly diagnosed acute myeloid leukemia or myelodysplastic syndrome with excess blasts-2, with an IDH1 or IDH2 mutation, respectively, eligible for intensive chemotherapy. | University Hospital Liège; Vilnius University Hospital Santaros Klinikos; Radboud University Medical Center Nijmegen; University Medical Center Utrecht; Universitätsklinikum Hamburg-Eppendorf. | Belgium, Netherlands , Lithuania , Germany. |
| 2023 | A PHASE 3, MULTICENTER, OPEN-LABEL, RANDOMIZED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF FEDRATINIB COMPARED TO BEST AVAILABLE THERAPY IN SUBJECTS WITH DIPSS INTERMEDIATE OR HIGH-RISK PRIMARY MYELOFIBROSIS, POST-POLYCYTHEMIA VERA MYELOFIBROSIS, OR POST-ESSENTIAL THROMBOCYTHEMIA MYELOFIBROSIS AND PREVIOUSLY TREATED WITH RUXOLITINIB |
University Hospital Liège; AOUI Verona; Spedali Civili di Brescia; AOU Policlinico Umberto I - Rome. | Belgium & Italy. |
| 2023 | A Randomized, Double-Blind, Phase 3 Study to Evaluate the Activity of Momelotinib (MMB) versus Danazol (DAN) in Symptomatic, Anemic Subjects with Primary Myelofibrosis (PMF), Post-Polycythemia Vera (PV) Myelofibrosis, or Post Essential Thrombocythemia (ET) Myelofibrosis who were Previously Treated with JAK Inhibitor Therapy |
University Hospital Liège; AOUI Verona; AOU Policlinico Umberto I - Rome; S. Gerardo Hospital - Monza. | Belgium & Italy. |
| 2023 | A phase 3, multicenter, open-label, randomized, study of gilteritinib versus midostaurin in combination with induction and consolidation therapy followed by one year maintenance in patients with newly diagnosed Acute Myeloid Leukemia (AML) or Myelodysplastic syndromes with excess blasts-2 (MDS-EB2) with FLT3 mutations eligible for intensive chemotherapy. | University Hospital Liège; Vilnius University Hospital Santaros Klinikos; Radboud University Medical Center Nijmegen; University Medical Center Utrecht; Universitätsklinikum Hamburg-Eppendorf. | Belgium, Netherlands, Lithuania, Germany. |
| 2023 | A Randomized, Open Label Phase 3 Study of Navitoclax in Combination with Ruxolitinib versus Ruxolitinib alone in Subjects with Myelofibrosis who have not been previously treated with JAK-2 inhibitor therapy (Transform-1) | University Hospital Liège; University Medical Center Utrecht; ASST Sette Laghi - Ospedale di Circolo, Varese; General Hospital of Athens "LAIKO”. | Belgium, Italy, Netherlands, Greece. |
| 2023 | A Phase 2, Open-label, Multicenter Study of TL-895 in Subjects with Relapsed/Refractory Myelofibrosis, Janus Kinase Inhibitor Intolerant Myelofibrosis and Janus Kinase Inhibitor treatment Ineligible Myelofibrosis. | University Hospital Liège; AUSL Romagna- Presidio Ospedaliero di Ravenna. | Belgium & Italy. |
| 2023 | A Phase 3, Randomized, Double-blind, Active-Control Study of CPI-0610 and Ruxolitinib vs. Placebo and Ruxolitinib in JAKi Treatment Naive MF Patients | University Hospital Liège; Universitätsklinikum Hamburg-Eppendorf; S. Gerardo Hospital - Monza; Azienda Ospedaliero-Universitaria S. Anna di Ferrara; Spedali Civili di Brescia. | Belgium, Italy & German. |
| 2023 | A Randomized, Double-blind, Placebo-controlled Phase 3 Study of Tamibarotene Plus Azacitidine Versus Placebo Plus Azacitidine in Newly Diagnosed Adult Patients Selected for RARA-positive Higher-risk Myelodysplastic Syndrome. | University Hospital Liège; AUSL Romagna- Presidio Ospedaliero di Ravenna; Azienda Ospedaliero-Universitaria S. Anna di Ferrara; Spedali Civili di Brescia; University Clinical Centre. | Belgium, Italy & Poland. |
| 2023 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Magrolimab versus Placebo in Combination with Venetoclax and Azacitidine in Newly Diagnosed, Previously Untreated Patients with Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy. | University Hospital Liège; AOU Policlinico Umberto I - Rome; AOU Ospedali Riuniti "Umberto I - G.M. Lancisi-G. Salesi”. | Belgium & Italy. |
| 2023 | A Phase 2,Multicenter,Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab,With or Without Hydroxyurea/Hydroxycarbamide, in Sequential, Descending Age Groups of Pediatric Sickle Cell Disease Patients With Vaso-Occlusive Crisis. | University Hospital Liège; Hospital de Sant Joan de Déu- Hospital de la Santa Creu i Sant Pau; Assistance Publique-Hôpitaux de Paris, Hôpital Necker-Enfants Malades; AOU Modena; Hospital Universitari Vall d'Hebron; Charité Universitätsmedizin Berlin. | Belgium, Italy, Spain, France |
| 2023 | A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease | University Hospital Liège; Assistance Publique-Hôpitaux de Marseille; Assistance Publique-Hôpitaux de Paris, Hôpital Henri-Mondor; AORN A Cardarelli; IRCCS Ospedale Pediatrico Bambino Gesù, Roma; Erasmus MC: University Medical Center Rotterdam; University Medical Center Utrecht; AOU Modena. | Belgium, Italy, France. |
| 2023 | A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat (GRN163L) Versus Best Available Therapy (BAT) in Patients with Intermediate-2 or High-risk Myelofibrosis (MF) Relapsed / Refractory (R/R) to Janus Kinase (JAK)-Inhibitor. | University Hospital Liège; AUSL Romagna- Presidio Ospedaliero di Ravenna; S. Gerardo Hospital - Monza; Azienda Ospedaliero-Universitaria S. Anna di Ferrara. | Belgium, Italy. |
| 2023 | A Phase 3, Double-Blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Subjects with Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions. | University Hospital Liège; Spedali Civili di Brescia; AOU Policlinico Umberto I - Rome 62. AOUI Verona; ASST Sette Laghi - Ospedale di Circolo, Varese. | Belgium, Italy. |
| 2023 | Efficacy and safety of concizumab prophylaxis in patients with Hemophilia A or B Without Inhibitors (Explorer 8). | Varna Expert Center of coagulopathies and rare anemias; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan. | Bulgary, Italy. |
| 2023 | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT). | Gemeinschaftsklinikum Mittelrhein gGmbH; IRCCS Ospedale Pediatrico Bambino Gesù, Roma. | Germany, Italy. |
| 2023 | A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia. | Gemeinschaftsklinikum Mittelrhein gGmbH; IRCCS Ospedale Pediatrico Bambino Gesù, Roma | Germany, Italy. |
| 2023 | APOLLO: A randomized Phase III study to compare arsenic trioxide (ATO) combined to ATRA and idarubicin versus standard ATRA and anthracycLine-based chemotherapy (AIDA regimen) for patients with newLy diagnosed, high-risk acute promyelocytic leukemia. | Universitätsklinikum Carl Gustav Carus; Azienda Ospedaliero-Universitaria S. Anna di Ferrara; Spedali Civili di Brescia. | Germany, Italy. |
| 2023 | Gilead_5F9009_ENHANCE_Magrolimab: A Randomized, Double-blind, Multicenter Study Comparing Magrolimab in Combination With Azacitidine Versus Azacitidine Plus Placebo in Treatment-naïve Patients With Higher Risk Myelodysplastic Syndrome. | Universitätsklinikum Carl Gustav Carus; Spedali Civili di Brescia; ASST Sette Laghi - Ospedale di Circolo, Varese; S. Gerardo Hospital - Monza. | Germany, Italy. |
| 2023 | COMMANDS ACE-536-MDS-002: A Phase 3, Open -label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) versus Epoetin alfa for the Treatment of Anemia due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in ESA Native Subjects who require Red Blood Cell Transfusions. | Universitätsklinikum Carl Gustav Carus; IRCCS Clinical Institute Humanitas - Rozzano; AOU Policlinico Umberto I - Rome; Vilnius University Hospital Santaros Klinikos; Radboud University Medical Center Nijmegen. | Germany , Italy, Netherlands, Lithuania. |
| 2023 | ACE-536-LTFU-001: A Phase 3b, Open-label, Single-arm, Rollover Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials. | Universitätsklinikum Carl Gustav Carus; Assistance Publique-Hôpitaux de Paris, Hôpital Henri-Mondor; Aghia Sophia Childrens Hospital; General Hospital of Athens "LAIKO"; AORN A Cardarelli; Azienda Ospedaliero-Universitaria S. Anna di Ferrara; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; AOU S.Luigi Gonzaga; ASST Sette Laghi - Ospedale di Circolo, Varese. | Germany, Italy, Greece, France. |
| 2023 | GS-US-546-5857 Enhance2: A Phase 3, Randomized, Open-Label Study Evaluating the Safety and Efficacy of Magrolimab in Combination with Azacitidine versus Physician's Choice of Venetoclax in Combination with Azacitidine or Intensive Chemotherapy in Previously Untreated Patients with TP53 Mutant Acute Myeloid Leukemia. | Universitätsklinikum Carl Gustav Carus; ASST Sette Laghi - Ospedale di Circolo, Varese; AOU Ospedali Riuniti "Umberto I - G.M. Lancisi-G. Salesi". | Germany, Italy. |
| 2023 | A Treatment Study Protocol for Participants 0-45 Years With Acute Lymphoblastic Leukaemia. | Universitätsklinikum Hamburg-Eppendorf; Vilnius University Hospital Santaros Klinikos; University Medical Center Utrecht. | Germany, Netherlands, Lithuania. |
| 2023 | A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) (ASC4START). | Universitätsklinikum Hamburg-Eppendorf; General Hospital of Athens "LAIKO”. | Germany, Greece. |
| 2023 | Efficacy of Alternating Immunochemotherapy Consisting of R-CHOP + R-HAD vs R-CHOP Alone, Followed by Maintenance Therapy Consisting of Additional Lenalidomide + Rituximab vs Rituximab Alone for Older Patients With Mantle Cell Lymphoma. | Universitätsklinikum Hamburg-Eppendorf; Jules Bordet Institute; Maria Sklodowska-Curie National Research Institute of Oncology. | Germany, Belgium, Poland. |
| 2023 | A Study of Belantamab Mafodotin in Patients With Relapsed or Refractory AL Amyloidosis (EMN27). | Universitätsklinikum Heidelberg; Foundation IRCCS Polyclinic San Matteo, Pavia. | Germany, Italy. |
| 2023 | A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis. | Universitätsklinikum Heidelberg; Foundation IRCCS Polyclinic San Matteo, Pavia; University Clinical Centre. | Germany, Italy, Poland. |
| 2023 | A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis. | Universitätsklinikum Heidelberg; Foundation IRCCS Polyclinic San Matteo, Pavia; University Clinical Centre; Jules Bordet Institute. | Germany, Italy, Poland, Belgium. |
| 2023 | A Study to Evaluate the Efficacy and Safety of Daratumumab in Combination With Cyclophosphamide, Bortezomib and Dexamethasone (CyBorD) Compared to CyBorD Alone in Newly Diagnosed Systemic Amyloid Light-chain (AL) Amyloidosis | Universitätsklinikum Heidelberg; Foundation IRCCS Polyclinic San Matteo, Pavia; Jules Bordet Institute; AOU Policlinico Umberto I - Rome. | Germany, Italy, Belgium. |
| 2023 | A Phase 2, Multicohort Study of Daratumumab-Based Therapies in Participants with Amyloid Light Chain (AL) Amyloidosis. | Universitätsklinikum Heidelberg; Foundation IRCCS Polyclinic San Matteo, Pavia; AOU Policlinico Umberto I - Rome; University Medical Center Utrecht. | Germany , Italy, Netherlands. |
| 2023 | LUSPLUS trial - A phase IIIb, open-label, single arm study to evaluate the efficacy and safety of Luspatercept in patients with lower-risk MDS and ring-sideroblastic phenotype (MDS-RS). | Universitätsklinikum Leipzig; Hospital General Universitario Gregorio Marañón. | Germany, Spain. |
| 2023 | Caplacizumab and immunosuppressive therapy without first-line therapeutic plasmapheresis in adults with thrombotic thrombocytopenic in adults with thrombotic thrombocytopenic purpura. EFC16521. | Hospital General Universitario Gregorio Marañón; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan. | Spain, Italy. |
| 2023 | A Phase 1, Open-Label, Dose-Escalation and -Expansion Study of the Bruton Tyrosine Kinase-Targeted Protein-Degrader BGB-16673 in Patients With B-Cell Malignancies (BGB-16673-101). | Hospital General Universitario Gregorio Marañón; AOUI Verona. | Spain, Italy. |
| 2023 | ACTIVATE-KIDS-T: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period. | University Medical Center Utrecht; Hospital Universitari Vall d'Hebron. | Spain, Netherlands. |
| 2023 | ACTIVATE-KIDS: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period. | University Medical Center Utrecht; Hospital Universitari Vall d'Hebron. | Spain, Netherlands. |
| 2023 | ACTIVATE-KIDS: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period. | University Medical Center Utrecht; Hospital Universitari Vall d'Hebron. | Spain, Netherlands. |
| 2023 | An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease. | Hospital Universitari Vall d'Hebron; Charité Universitätsmedizin Berlin; Assistance Publique-Hôpitaux de Paris, Hôpital Henri-Mondor; IRCCS Ospedale Pediatrico Bambino Gesù, Roma; AOU S.Luigi Gonzaga. | France, Italy, Germany, Spain. |
| 2023 | An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies. | Hospital Universitari Vall d'Hebron; Assistance Publique-Hôpitaux de Paris, Hôpital Henri-Mondor; AORN A Cardarelli; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; University Medical Center Utrecht. | Spain, Netherlands, France, Italy. |
| 2023 | A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE) Study AG348-C-017. |
Hospital Universitari Vall d'Hebron; Assistance Publique-Hôpitaux de Paris, Hôpital Henri-Mondor; General Hospital of Athens "LAIKO"; Aghia Sophia Childrens Hospital; AORN A Cardarelli; Azienda Ospedaliero-Universitaria S. Anna di Ferrara; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; Academic Medical Center Amsterdam; Erasmus MC: University Medical Center Rotterdam; University Medical Center Utrecht; AOU Modena; AOU S.Luigi Gonzaga. | Spain,Netherlands, France, Italy, |
| 2023 | A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (ENERGIZE-T). | Hospital Universitari Vall d'Hebron; Assistance Publique-Hôpitaux de Paris, Hôpital Henri-Mondor; General Hospital of Athens "LAIKO"; Aghia Sophia Childrens Hospital; Azienda Ospedaliero-Universitaria S. Anna di Ferrara; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; Academic Medical Center Amsterdam; Erasmus MC: University Medical Center Rotterdam; University Medical Center Utrecht; AOU Modena; Assistance Publique-Hôpitaux de Marseille. | Spain, Netherlands, France, Italy. |
| 2023 | A Phase II, multicenter, randomized, open label two arm study evaluating the effect of crizanlizumab + standard of care and standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle cell nephropathy (STEADFAST). |
Hospital Universitari Vall d'Hebron; Assistance Publique-Hôpitaux de Paris, Hôpital Henri-Mondor. | Spain, France. |
| 2023 | A phase III, Multicenter, Randomized, Double-blind Study to Assess Efficacy and Safety of Two Doses of Crizanlizumab versus placebo, with or without Hydroxyurea/ Hydroxycarbamide Therapy, in Adolescent and Adult Sickle Cell Disease Patients with Vaso-Occlusive Crises (STAND). | Hospital Universitari Vall d'Hebron; Assistance Publique-Hôpitaux de Paris, Hôpital Henri-Mondor; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; Assistance Publique-Hôpitaux de Marseille; Academic Medical Center Amsterdam. | Spain,Netherlands, France, Italy, |
| 2023 | Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (XTEND-ed). | Hospital de Sant Joan de Déu- Hospital de la Santa Creu i Sant Pau; Assistance Publique-Hôpitaux de Marseille; Hospices Civils de Lyon; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; Academic Medical Center Amsterdam; University Medical Center Utrecht. | Spain, Netherlands, France, Italy. |
| 2023 | A Phase 3, Open-label Interventional Study of an Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A (XTEND-1). | Hospital de Sant Joan de Déu- Hospital de la Santa Creu i Sant Pau; Assistance Publique-Hôpitaux de Marseille; CHU de Lille; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; San Bortolo Hospital - Vicenza | Spain, France, Italy. |
| 2023 | CROSSWALK-a (BO42452) : "Phase IB randomized, placebo-controlled study evaluating the safety, pharmacokinetics, pharmacodynamics, and efficacy of crovalimab for the management of ACUTE uncomplicated vaso-occlusive episodes (VOE) in patients with sickle cell disease (SCD)" - (PI and coordinator: Pr Bartolucci, sponsor Roche). |
Assistance Publique-Hôpitaux de Paris, Hôpital Henri-Mondor; AOUI Verona. | France, Italy. |
| 2023 | CROSSWALK-c (BO42451) : "A Randomized Double-Blind Phase IIA Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease (SCD)" - (PI and coordinator : Pr Bartolucci, sponsor Roche). | Assistance Publique-Hôpitaux de Paris, Hôpital Henri-Mondor; AOUI Verona. | France, Italy. |
| 2023 | A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD). | Assistance Publique-Hôpitaux de Paris, Hôpital Necker-Enfants Malades; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; IRCCS Ospedale Pediatrico Bambino Gesù, Roma; Erasmus MC: University Medical Center Rotterdam. | France, Italy, Netherlands. |
| 2023 | Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX. | Assistance Publique-Hôpitaux de Paris, Hôpital Necker-Enfants Malades; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan. | France, Italy. |
| 2023 | Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors. | Assistance Publique-Hôpitaux de Paris, Hôpital Necker-Enfants Malades; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan. | France, Italy. |
| 2023 | BAXALTA 071102: studio di fase 3, prospettico, multicentrico, non controllato, in aperto, per determinare l'efficacia, la sicurezza e la tollerabilità di rVWF in procedure chirurgiche di elezione e di emergenza,e la farmacocinetica di rVWF in bambini con diagnosi di malattia di von Willebrand in forma grave. | CHU de Lille; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; AOU Careggi, Florence. | France, Italy. |
| 2023 | BIOMARIN 270+A28:J38-302 :A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector¿ Mediated Gene Transfer of Human Factor VIII at a dose of 4E13 vg/kg in Hemophilia A Patients with Residual FVIII Levels = 1 IU/dL Receiving Prophylactic FVIII Infusions. | CHU de Lille; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; AOU Careggi, Florence. | France, Italy. |
| 2023 | NN7769-4514 (FRONTIER2): A multinational, open-label, randomised, controlled trial to investigate efficacy and safety of NNC0365-3769 (Mim8) in adults and adolescents with haemophilia A with or without inhibitors. | CHU de Lille; AOU Careggi, Florence; Centro Hospitalar do Porto, EPE. | France, Italy, Portugal. |
| 2023 | Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants Who Require Regular RBC Transfusions Due to Beta (β)-Thalassemia. | Aghia Sophia Childrens Hospital; AOU S.Luigi Gonzaga; AORN A Cardarelli. | Greece, Italy. |
| 2023 | A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants With Alpha (α)-Thalassemia. | Aghia Sophia Childrens Hospital; AOU S.Luigi Gonzaga. | Greece, Italy. |
| 2023 | A Phase 1b, Open Label, Global, Multicenter, Dose Determination, Randomized Dose Expansion Study to Determine the Maximum Tolerated Dose, Assess the Safety and Tolerability, Pharmacokinetics and Preliminary Efficacy of Iberdomide (CC-220) in Combination With R-CHOP-21 and CC-99282 in Combination With R-CHOP-21 for Subjects With Previously Untreated Aggressive B-cell Lymphoma. | General Hospital of Athens "LAIKO"; Maria Sklodowska-Curie National Research Institute of Oncology. | Greece, Poland. |
| 2023 | A Phase 2b Open-label Study of Selinexor (KPT-330) in Patients With Relapsed/Refractory Diffuse Large B-Cell Lymphoma (DLBCL). | General Hospital of Athens "LAIKO"; Maria Sklodowska-Curie National Research Institute of Oncology; Jules Bordet Institute. | Greece, Poland, Belgium. |
| 2023 | A phase 3b, multi-center, open-label, treatment optimization study of oral asciminib in patients with Chronic Myelogenous Leukemia in chronic phase (CML-CP) previously treated with 2 or more tyrosine kinase inhibitors. | General Hospital of Athens "LAIKO"; AOUI Verona. | Greece, Italy. |
| 2023 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Tafasitamab Plus Lenalidomide in Addition to Rituximab Versus Lenalidomide in Addition to Rituximab in Patients With Relapsed/Refractory (R/R) Follicular Lymphoma Grade 1 to 3a or R/R Marginal Zone Lymphoma. | General Hospital of Athens "LAIKO"; Maria Sklodowska-Curie National Research Institute of Oncology. | Greece, Poland. |
| 2023 | Abbvie M16-109: "A Phase 2 Open-Label Study Evaluating Tolerability and Efficacy of Navitoclax Alone or in Combination with Ruxolitinib in Subjects with Myelofibrosis". | General Hospital of Athens "LAIKO"; ASST Sette Laghi - Ospedale di Circolo, Varese. | Greece, Italy. |
| 2023 | A Randomized, Open-Label, Phase 3 Study Evaluating Efficacy and Safety of Navitoclax in Combination With Ruxolitinib Versus Best Available Therapy in Subjects With Relapsed/Refractory Myelofibrosis (TRANSFORM-2). | General Hospital of Athens "LAIKO"; S. Gerardo Hospital - Monza; AOU Policlinico Umberto I - Rome 92. University Clinical Centre; ASST Sette Laghi - Ospedale di Circolo, Varese. | Greece, Italy , Poland. |
| 2023 | A Phase 2 Open-label Clinical Study to Evaluate the Efficacy and Safety of Zilovertamab Vedotin (MK-2140) in Participants With Relapsed or Refractory Diffuse Large B-Cell Lymphoma (waveLINE-004). | General Hospital of Athens "LAIKO"; Maria Sklodowska-Curie National Research Institute of Oncology. | Greece, Poland. |
| 2023 | SLSG18-301 A Randomized, Open-Label Study of the Efficacy and Safety of Galinpepimut-S (GPS) Maintenance Monotherapy Compared to Investigator's Choice of Best Available Therapy in Subjects with Acute Myeloid Leukemia Who Have Achieved Complete Remission After Second-Line Salvage Therapy. | General Hospital of Athens "LAIKO"; University Clinical Centre; AUSL Romagna- Presidio Ospedaliero di Ravenna. | Greece, Italy, Poland. |
| 2023 | A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients with Advanced Systemic Mastocytosis. | General Hospital of Athens "LAIKO"; AOUI Verona. | Greece, Italy. |
| 2023 | Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study). | General Hospital of Athens "LAIKO"; University Clinical Centre. | Greece, Poland. |
| 2023 | COVALENT-101: A phase 1 first-in-human dose-escalation and dose-expansion study of BMF-219, an oral, covalent, menin inhibitor, in adult patients with acute leukemia (AL), diffuse large B-cell lymphoma (DLBCL), multiple myeloma (MM), and chronic lymphocytic leukemia (CLL)/ small lymphocytic lymphoma (SLL). | AOU Ospedali Riuniti "Umberto I - G.M. Lancisi-G. Salesi"; Radboud University Medical Center Nijmegen. | Italy, Netherlands. |
| 2023 | Study Comparing Zanubrutinib + Rituximab Versus Bendamustine + Rituximab in Participants With Untreated Mantle Cell Lymphoma. | Jules Bordet Institute; AOUI Verona; AOU Policlinico Umberto I - Rome. | Italy, Belgium. |
| 2023 | A Phase 1b/2 Study of BMS-986158 Monotherapy and in Combination with Either Ruxolitinib or Fedratinib in Participants with DIPSS Intermediate or High Risk Myelofibrosis. | AOUI Verona; Spedali Civili di Brescia; University Clinical Centre. | Italy, Poland. |
| 2023 | DREAMM 7: A Multicenter, Open-Label, Randomized Phase III Study to Evaluate the Efficacy and Safety of the Combination of Belantamab Mafodotin, Bortezomib, and Dexamethasone (B-Vd) Compared With the Combination of Daratumumab, Bortezomib and Dexamethasone (D-Vd) in Participants With Relapsed/Refractory Multiple Myeloma. | AUSL Romagna- Presidio Ospedaliero di Ravenna; Spedali Civili di Brescia; Maria Sklodowska-Curie National Research Institute of Oncology. | Italy, Poland. |
| 2023 | A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa (FREEDOM). | Hospital de Sant Joan de Déu- Hospital de la Santa Creu i Sant Pau; Azienda Ospedaliero-Universitaria di Parma; University Medical Center Utrecht. | Italy, Netherlands, Spain. |
| 2023 | A Phase 2b Randomized Study to Assess the Efficacy and Safety of the Combination of Ublituximab + Umbralisib With or Without Bendamustine and Umbralisib Alone in Patients With Previously Treated Non-Hodgkin's Lymphoma. | Azienda Ospedaliero-Universitaria S. Anna di Ferrara; Maria Sklodowska-Curie National Research Institute of Oncology; AOU Policlinico Umberto I - Rome. | Italy, Poland. |
| 2023 | A Phase 3 Open label,Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients < 12 Years of Age With Severe Hemophilia A. | Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; Academic Medical Center Amsterdam. | Italy, Netherlands. |
| 2023 | A Phase III Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. (COMMODORE 2). | Hospital de Sant Joan de Déu- Hospital de la Santa Creu i Sant Pau; Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; Vilnius University Hospital Santaros Klinikos. | Spain, Italy, Lithuania. |
| 2023 | A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia (BEYOND). | Foundation IRCCS Ca'Granda Ospedale Maggiore Policlinico, Milan; Aghia Sophia Childrens Hospital. | Italy, Greece. |
| 2023 | Clinical Benefit of SAR650984, Bortezomib, Lenalidomide and Dexamethasone Combination in NDMM Patients Not Eligible for Transplant (IMROZ). | Vilnius University Hospital Santaros Klinikos; Maria Sklodowska-Curie National Research Institute of Oncology; Spedali Civili di Brescia. | Italy, Lithuania, Poland. |
| 2023 | Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria. | Vilnius University Hospital Santaros Klinikos; Radboud University Medical Center Nijmegen; AOU Policlinico Umberto I - Rome. | Italy, Lithuania, Netherlands. |
| 2023 | GEN3013-01, A Phase 1/2, Open-Label, Dose-Escalation Trial of GEN3013 in Patients with Relapsed, Progressive or Refractory B-Cell Lymphoma. | University Medical Center Utrecht; Maria Sklodowska-Curie National Research Institute of Oncology. | Netherlands, Poland. |
| 2023 | Monumental-1, Anti GPRC5D duobody studie, A Phase 1, First-in-Human, Open-Label, Dose Escalation Study of JNJ-64407564, a Humanized DuoBody® Antibody, in Subjects with Relapsed or Refractory Multiple Myeloma. |
90. University Medical Center Utrecht 91. Maria Sklodowska-Curie National Research Institute of Oncology. | Netherlands, Poland. |
| 2023 | MajesTEC-9, A Phase 3 Randomized Study Comparing Teclistamab Monotherapy Versus Pomalidomide, Bortezomib, Dexamethasone (PVd) or Carfilzomib, Dexamethasone (Kd) in Participants With Relapsed or Refractory Multiple Myeloma Who Have Received 1 to 3 Prior Lines of Therapy, Including an Anti-CD38 Monoclonal Antibody and Lenalidomide. |
University Medical Center Utrecht; ASST Sette Laghi - Ospedale di Circolo, Varese. | Netherlands, Italy. |
| 2023 | A Phase 2, Open-Label, 2-Cohort, Multicenter Study of INCB050465, a PI3Kδ Inhibitor, in Relapsed or Refractory Mantle Cell Lymphoma Previously Treated With or Without a BTK Inhibitor (CITADEL-205). | Maria Sklodowska-Curie National Research Institute of Oncology; Jules Bordet Institute. | Poland, Belgium. |
| 2023 | Improvement of Outcome in Elderly Patients or Patients Not Eligible for High-dose Chemotherapy With Aggressive NHL in First Relapse/Progression by Adding Nivolumab to Gemcitabine, Oxaliplatin Plus Rituximab in Case of B-cell Lymphoma. | Maria Sklodowska-Curie National Research Institute of Oncology; Jules Bordet Institute. | Poland, Belgium. |
| 2023 | REGENERON_ R1979- ONC-1625: "AN OPEN-LABEL STUDY TO ASSESS THE ANTI-TUMOR ACTIVITY AND SAFETY OF REGN1979, AN ANTI-CD20 X ANTI-CD3 BISPECIFIC ANTIBODY, IN PATIENTS WITH RELAPSED OR REFRACTORY FOLLICULAR LYMPHOMA”. | Maria Sklodowska-Curie National Research Institute of Oncology; ASST Sette Laghi - Ospedale di Circolo, Varese; University Clinical Centre. | Poland, Italy. |
| 2023 | A Phase 3 Open-Label, Randomized Study of LOXO-305 Versus Investigator's Choice of Idelalisib Plus Rituximab or Bendamustine Plus Rituximab in BTK Inhibitor Pretreated Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (BRUIN CLL-321). | Maria Sklodowska-Curie National Research; Institute of Oncology; University Clinical Centre; Spedali Civili di Brescia. | Poland, Italy. |
| 2023 | Study With Elranatamab Versus Lenalidomide in Patients With Newly Diagnosed Multiple Myeloma After Transplant (MagnetisMM-7). | Maria Sklodowska-Curie National Research Institute of Oncology; University Clinical Centre; AOU Policlinico Umberto I - Rome. | Poland, Italy. |
| 2023 | A Phase 1/2 Study of CPI-0610, a Small Molecule Inhibitor of BET Proteins: Phase 1 (Dose Escalation of CPI-0610 in Patients With Hematological Malignancies) and Phase 2 (Dose Expansion of CPI-0610 With and Without Ruxolitinib in Patients With Myelofibrosis and Essential Thrombocytopenia). | ASST Sette Laghi - Ospedale di Circolo, Varese; University Clinical Centre. | Poland, Italy. |
| 2023 | Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants Who Require Regular RBC Transfusions Due to Beta (β)-Thalassemia. | Aghia Sophia Childrens Hospital; AOU S.Luigi Gonzaga. | Italy, Greece. |
| 2023 | MDS3001: A Study to Evaluate Imetelstat (GRN163L) in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) that is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment. | ASST Sette Laghi - Ospedale di Circolo, Varese; Universitätsklinikum Carl Gustav Carus. | Germany, Italy. |