Junta
de Andalucía

Ensayos Clínicos

LEUCEMIA MIELOBLÁSTICA AGUDA

Primera línea

• QUIWI: A 2:1 randomized phase II trial to compare the efficacy and safety of standard chemotherapy plus quizartinib versus standard chemotherapy plus placebo in adult patients with newly diagnosed FLT3 wild-type AML.
• COVALENT-101. Novel covalent menin inhibitor: BMF-219 – for AML/ALL/DLBCL/MM/CLL
• LAVA CD123: A PHASE III RANDOMIZED MULTICENTER OPEN-LABEL TRIAL OF TAGRAXOFUSP IN COMBINATION WITH VENETOCLAX/AZACITIDINE (VEN/AZA) VERSUS VEN/AZA ALONE IN ADULTS WITH PREVIOUSLY UNTREATED ACUTE MYELOID LEUKEMIA (AML) WHO ARE INELIGIBLE FOR INTENSIVE CHEMOTHERAPY
• AC220-168: A Phase 3, Double-Blind, Placebo-Controlled Study of Quizartinib Administered in Combination with Induction and Consolidation Chemotherapy, and Administered as Maintenance Therapy in Adult Patients with Newly Diagnosed FLT3-ITD (-) Acute Myeloid Leukemia.

En recaída/refractaria

• BGB-11417-104: A Phase 1b/2, Open-Label, Dose Finding, and Expansion Study of the Bcl-2 Inhibitor BGB-11417 in Patients With Myeloid Malignancies
• ARO-013: Phase III Randomized, ouble-blind, Placebo-controlled Study Investigating the Efficacy of the Addition of Crenolanib to Salvage Chemotherapy Versus Salvage Chemotherapy Lone in Subjects ≤ 75 Years of Age with Relapsed/Refractory FLT3 Mutated Acute Myeloid Leukemia
• KO-MEN-001: Fase I/IIa del inhibidor KO-539 de menina-MLL (KMT2A) en pacientes afectados de leucemia mieloide aguda recidivante o refractaria.
• CC-91633-AML-001: A phase I, open-label, dose-finding study of CC-91633 (BMS-986397) in subjects with relapsed or refractory acute myeloid leukemia or relapsed or refractory higher-risk myeloysplastic syndromes
• AB18001: título: Estudio título: “Estudio de fase I/II para evaluar la seguridad, la farmacocinética y la eficacia de la administración intravenosa diaria de AB8939 en pacientes con leucemia mieloide aguda recidivante/refractaria.
• Sellas SLSG18301.A Randomized, Open-Label Study of the Efficacy and Safety of Galinpepimut-S (GPS) Maintenance Monotherapy Compared to Investigator’s Choice of Bes t Available Therapy in Subjects with Acute Myeloid Leukemia Who Have Achieved Complete Remission After Second-Line Salvage T
• CA-4948-102: Phase 1/2a open-label, single arm dose escalation and expansion study of orally-administered emavusertib (CA-4948) as monotherapy and in combination with azacitidine or venetoclax in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS)
• SNDX-5613-0700_2636/0009 : Phase 2 Study in patients with relapsed/refractory AML/ALL who harbor NPM1 or KMT2Ar mutations: revumenib
• FLAG-QUIDA: Estudio multicéntrico, prospectivo, no aleatorizado,  fase  I-II para evaluar la eficacia y la seguridad de la combinación de quizartinib oral con el esquema de quimioterapia FLAG-IDA en pacientes con leucemia mieloide aguda en primera recidiva o refractarios (LMA R/R)
• RIVER 52: Multicenter, Open-Label Clinical Trial of RVU120 in Patients with Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia with or without NPM1 Mutation (RIVER-52)
• RIVER 81: A Multicenter, Open-Label, Dose-Finding Clinical Trial to Assess the Safety, Pharmacokinetics, Pharmacodynamics, and Clinical Efficacy of RVU120 in Combination with Venetoclax in Participants with Acute Myeloid Leukemia Who Failed Prior Therapy with Venetoclax and a Hypomethylating Agent (RIVER-81)
• NM32-2668: A First-in-Human Phase 1/2 Study of NM32-2668 (Anti-ROR1/Anti-CD3/Anti-HAS Tri-Specific Antibody) in Adult Patients with Advanced Hematological Tumors.
• LAVA CD123 – Phase 1 study in Acute Myelocytic Leukemia (AML)/MDS
• KO-MEN-008: Phase 1 Study to Determine the Safety and Tolerability of Ziftomenib Combinations for the Treatment of KMT2A-rearranged or NPM1-mutant Relapsed/Refractory Acute Myeloid Leukemia
• KO-MEN-007: Phase 1 study of venetoclax/azacitidine or venetoclax in combination with ziftomenib (KO-539) or standard induction cytarabine/daunorubicin (7+3) chemotherapy in combination with ziftomenib for the treatment of patients with acute myeloid leukemia
• FLORENTINE: A PHASE 1, OPEN-LABEL, DOSE ESCALATION AND DOSE EXPANSION STUDY OF CLN-049 FOR THE TREATMENT OF ACUTE MYELOID LEUKEMIA (AML) PATIENTS WITH MEASURABLE RESIDUAL DISEASE (MRD)

SÍNDROMES MIELODISPLÁSICOS

• SY-1425-301: 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
• KER-050: A Phase 2, Open-Label, Ascending Dose Study of KER-050 for the Treatment of Anemia in Patients with Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)
• AG946-C-002: A Phase 2a/2b, Multicenter, Proof of Concept, Efficacy, and Safety Study of AG 946 in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes
• ASTX727-10: A Phase 2 Study of Oral Decitabine/Cedazuridine in Combination With Magrolimab for Previously Untreated Subjects With Intermediate- to Very High-Risk Myelodysplastic Syndromes (MDS)
• CNST0610C208: A Phase 2, Open-Label, Randomized Dose Optimization Study on Pwlabresib as Monotherapy and In Combination with Luspatercept for The Treatment of Anemia Due to Very Low, Low or Intermediate Risk Myelodysplastic Syndrome in Patients Who Requiere Red Blood Cell Transfusions.

LEUCEMIA LINFOBLÁSTICA AGUDA

• EspHALL: International phase 3 trial in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbones
• IntReALL: International Study for Treatment of High Risk Childhood Relapsed ALL 2010
• 20180251: A Phase 1b Open-label Study to Investigate the Safety and Pharmacokinetics of Administration of Subcutaneous Blinatumomab for the Treatment of adults with Relapsed or Refractory B cell Precursor Acute
• Lymphoblastic Leukemia (R/RBALL)
• 2215-CL-0603: Phase 1/2, Multicenter, Open-Label, Single Arm, Dose Escalation and Expansion Study of Gilteritinib (ASP2215) Combined with Chemotherapy in Children, Adolescents and Young Adults with FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)
• AMGEN 20190360: Phase 3 Randomized, Controlled Study of Blinatumomab Alternating With Low-intensity Chemotherapy Versus Standard of Care for Older Adults With Newly Diagnosed Philadelphia-negative B-cell Precursor Acute Lymphoblastic Leukemia With Safety Run-in.
• B1931036: Estudio prospectivo, aleatorizado, abierto, en fase II para evaluar la superioridad de inotuzumab ozogamicina en monoterapia frente a ALLR3 para el tratamiento de inducción de la LLA infantil de precursores de linfocitos B en primera recidiva de alto riesgo.
• KO-MEN-001 sub3: Evaluation of Ziftomenib in Patients with Relapsed or Refractory Acute Lymphoblastic Leukemia (ALL)
• INTERFANT-21: International collaborative treatment protocol for infants under one year with KMT2A-rearranged acute lymphoblastic leukemia or mixed phenotype acute leukemia
• INTERFANT-06: Treatment Protocol for Infants Under 1 Year With KMT2A-rearranged ALL or Mixed Phenotype Acute Leukemia

LEUCEMIA PROMIELOCÍTICA

• TUD-APOLLO-064

MIELOMA MÚLTIPLE

Nuevo diagnóstico:

• “Tratamiento de inducción con bortezomib, melfalán y prednisona (VMP) seguido de lenalidomida y dexametasona (Rd) frente a carfilzomib, lenalidomida y dexametasona (KRd) más/menos daratumumab, 18 ciclos, seguido de tratamiento de consolidación y mantenimiento con lenalidomida y daratumumab: un ensayo clínico de fase III, multicéntrico, aleatorizado para pacientes adultos mayores, de entre 65 y 80 años, con buen estado general y mieloma múltiple de nuevo diagnóstico.
• GEM-CESAR. Estudio fase II multicéntrico de carfilzomib, lenalidomida y dexametasona (KRd) como inducción, seguido de melfalán a altas dosis y trasplante autólogo de células progenitoras de sangre periférica, consolidación con KRd y mantenimiento con lenalidomida y dexametasona en pacientes de edad inferior o igual a 70 años con Mieloma Múltiple asintomático de alto riesgo de progresión a Mieloma sintomático.
• GEM-IBERDARAX. Multicenter, phase II, national and open-label study to evaluate Iberdomide-dexamethasone alone or in combination with standard MM treatment regimens in transplant ineligible newly diagnosed patients
• Ensayo de fase III en pacientes con MMND candidatos a TASPE para comparar VRD extendido más intervención temprana de rescate vs. Isatuximab-VRD vs. Isatuximab-VID
• GEM TECTAL. An open label, multicenter, Phase 2, pilot study, evaluating early treatment with bispecific T-cell redirectors (teclistamab and talquetamab) in the frontline therapy of newly diagnosed high-risk multiple myeloma

Recaída

• Selinexor, Bortezomib and Daratumumab in Multiple Myeloma. An Open-label, Multicenter, Phase 2 trial of selinexor (KPT-330), bortezomib and low-dose dexamethasone plus daratumumab (SELIBORDARA) for the treatment of patients with refractory or relapsed and refractory multiple myeloma
• M13-494. con título “A Phase 3, Multicenter, Randomized, Open Label Study of Venetoclax and Dexamethasone Compared with Pomalidomide and Dexamethasone in SUbjects with t(11;14)-Positive Relapsed or Refractory Multiple Myeloma”, 94_
• 64407564MMY1001-Part 3 TALMMY. A Phase 1/2, First-in-Human, Open-Label, Dose Escalation Study of Talquetamab, a Humanized GPRC5D x CD3 Bispecific Antibody, in Subjects with Relapsed or Refractory Multiple Myeloma
• DREAMM 8. Estudio aleatorizado de fase III, multicéntrico, abierto, para evaluar la eficacia y seguridad de Belantamab Mafodotin en combinación con Pomalidomida y Dexametasona (B-Pd) versus Pomalidomida más Bortezomib y Dexametasona (PVd) en participantes con mieloma múltiple recidivante / refractario (DREAMM8).
• IKEMA -EFC15246. Randomized, open label, multicenter study assessing the clinical benefit of isatuximab combined with carfilzomib (Kyprolis®) and dexamethasone versus carfilzomib with dexamethasone in patients with relapsed and/or refractory multiple myeloma previously treated with 1 to 3 prior lines
• C1071005 magnetismm5. AN OPEN-LABEL, 3-ARM, MULTICENTER, RANDOMIZED PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ELRANATAMAB (PF-06863135) MONOTHERAPY AND ELRANATAMAB + DARATUMUMAB VERSUS DARATUMUMAB + POMALIDOMIDE + DEXAMETHASONE IN PARTICIPANTS WITH RELAPSED/REFRACTORY MULTIPLE MYELOMA WHO HAVE RECEIVED AT LEAST 2 PRIOR LINES OF THERAPY INCLUDING LENALIDOMIDE AND A PROTEASOME INHIBITOR
• A Phase 3 Randomized Study Comparing Teclistamab in Combination with Daratumumab SC (Tec-Dara) versus Daratumumab SC, Pomalidomide, and Dexamethasone (DPd) or Daratumumab SC, Bortezomib, and Dexamethasone (DVd) in Participants with Relapsed or Refractory Multiple Myeloma
• BGB-11417-105- 34027. A Phase 1b/2 Dose-Escalation and Cohort-Expansion Study to Determine the Safety and Efficacy of BGB-11417 in Combination With Dexamethasone and Carfilzomib/ Dexamethasone in Patients With Relapsed/Refractory Multiple Myeloma and t(11;14)
• A phase 1/2 multicenter, open-label, dose-escalation study of IDP-121 in patients with relapsed/refractory hematologic malignancies

AMILOIDOSIS

• CAEL101 301. A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment-Naïve Patients with Mayo Stage IIIb AL Amyloidosis
• CAEL101 302. A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment-Naïve Patients with Mayo Stage IIIa AL Amyloidosis

MIELOFIBROSIS

• M20-178: Estudio de fase 3 aleatorizado y abierto para evaluar la eficacia y la seguridad de navitoclax combinado con ruxolitinib en comparación con el mejor tratamiento disponible en pacientes con mielofibrosis en recaída/refractario (TRANSFORM-2).
• ACE-536-MF-002: Estudio de fase 3, doble ciego y aleatorizado para comparar la eficacia y la seguridad de luspatercept (ACE-536) frente a placebo en pacientes con
• mielofibrosis asociada a neoplasia mieloproliferativa en tratamiento concomitante con un inhibidor de JAK2 y que necesitan transfusion de eritrocitos
• PAC203/PAC303 : A Randomized, Controlled Phase 3 Study of Pacritinib Versus Physician’s Choice in Patients with Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post Essential Thrombocythemia Myelofibrosis with Severe Thrombocytopenia (platelet counts <50,000/µL)

ANEMIA HEMOLÍTICA

• Efficacy and Safety of M281 in Adults with Warm Autoimmune, Hemolytic Anemia: A Multicenter, Randomized, Double-blind, Placebo-controlled Study with a Long-term Open-label Extension
• A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY, WITH A SAFETY AND DOSE CONFIRMATION RUN-IN PERIOD, TO EVALUATE THE EFFICACY AND SAFETY OF OBEXELIMAB IN PATIENTS WITH WARM AUTOIMMUNE HEMOLYTIC ANEMIA (SAPHIARE)

TROMBOCITOPENIA FETAL

• Double-blind, Randomized, Placebo-controlled Study Evaluating the Safety and Efficacy of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) in At-risk Pregnancies Randomized Controlled Study of Nipocalimab in At-risk Pregnancies for FNAIT FREESIA-1

PÚRPURA TROMBOCITOPÉNICA IDIOPÁTICA

• A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia (ITP). Solo abierta la cohorte de menores de edad
• A phase III, randomized, double-blind study of ianalumab (VAY736) versus placebo in addition to first-line corticosteroids in primary immune thrombocytopenia (VAYHIT1)
• A multicentre, randomized, open-label study of romiplostim plus dexamethasone vs dexamethasone in patients with newly diagnosed primary immune thrombocytopenia

PÚRPURA TROMBOCITOPÉNICA TROMBÓTICA

• An open-label, single-arm, multicenter study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy without first-line therapeutic
• plasma exchange in adults with immune-mediated thrombotic thrombocytopenic purpura
• -A Phase 2b, multicenter, randomized, double-blind study of safety and efficacy of TAK-755 (rADAMTS13) with minimal to no plasma exchange (PEX) in the treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP)

DREPANOCITOSIS

• A 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) (CROSSWALK-a)
• 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)

HEMOFILIA

• GENA 22 Nuwiq for Perioperative management Of patients With haemophilia A on Emicizumab Regular prophylaxis study (NuPOWER)
• GENA 23 Nuwiq Dosing and outcomes In the ManagEment of women/girls with haemophilia A Needing FVIII treatment for Surgery – an International, Open-label, Non-controlled study (NuDIMENSION)

TRASPLANTE

• ALXN1210-TMA-313 _A Phase 3, Open-label, Randomized, Multicenter Study of Ravulizumab in Adult and Adolescent Participants who have Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplant (HSCT)
• ARES trial_EvAluation of the efficacy of MaaT013 as salvage theRapy in acute GVHD patiEntS with gastrointestinal involvement, refractory to ruxolitinib; a multi-center open-label phase III trial
• EQUATOR_A Phase 3, Randomized, Double-Blind, Placebo-Controlled Multicenter Study of Itolizumab in Combination with Corticosteroids for the Initial Treatment of Acute Graft Versus Host Disease.
• EFC17757_A randomized, double-blind, multicenter, Phase 3 study to evaluate efficacy and safety of belumosudil in combination with corticosteroids versus placebo in combination with corticosteroids in participants at least 12 years of age with newly diagnosed chronic graft versus host disease (cGVHD)

TERAPIA CELULAR/CART

• ATA-129-EBV-302. Multicenter, Open Label, Phase 3 Study of Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Subjects with Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease after Failure of Rituximab or Rituximab and Chemotherapy.
• ATA129-EBV-205_An Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects with Epstein-Barr Virus-associated Diseases
• GC-LTFU-001_Long-term follow up protocol for subjects treated with gene-modified T cells
• T reg4cGVHD_Phase II Trial of Donor Regulatory T-cells for Steroid-Refractory Chronic Graft-versus-Host-Disease in patients who do not obtain complete remission with ruxolitinib
• CYTB323A12101_Phase I, open label, multicenter, dose escalation study of YTB323 in adult patients with CLL/SLL and DLBCL
• INMUNOTERAPIA CON LINFOCITOS T DIFERENCIADOS, ADULTOS, AUTÓLOGOS, DE SANGRE PERIFÉRICA, SELECCIONADOS MEDIANTE EXPRESIÓN DE CD62L, EXPANDIDOS Y TRANSDUCIDOS (MODIFICADOS GENÉTICAMENTE) MEDIANTE UN VECTOR LENTIVIRAL PARA QUE EXPRESEN UN RECEPTOR QUIMÉRICO CON ESPECIFICIDAD ANTI-CD19 ASOCIADO A SECUENCIAS COESTIMULADORAS 4-1-BB Y CD3Ζ EN PACIENTES CON LINFOMA NO-HODGKIN B
• CART-ARI FSJD
• Estudio fase 2 de la infusión de linfocitos T diferenciados autólogos de sangre periférica expandidos y transducidos con un lentivirus para expresar un receptor antigénico quimérico con especificidad anti-CD19 (A3B1) conjugado con las regiones coestimuladoras 4-1BB y CD3z (células ARI-0001) en niños y adolescentes de 0 a 18 años con leucemia linfoblástica aguda CD19+ resistente o refractaria a tratamiento
• ZUMA22_a randomized Phase 3 study of Axi-cel vs Standard of care in relapsed/refractory FL
• M2020-371_A pivotal Phase II randomised, multi-centre, open-label study to evaluate the efficacy and safety of MB-CART2019.1 compared to standard of care therapy in participants with relapsed/refractory diffuse large B-cell lymphoma (R-R DLBCL), who are not eligible for high-dose chemotherapy and autologous stem cell transplantation
• Nathali_Open label dose-escalation and dose-expansion study to evaluate the safety, expansion, persistence and clinical activity of UCART20x22 (allogeneic engineered T-cells expressing antiCD20 and anti-CD22 Chimeric Antigen Receptors) in subjects with relapsed or refractory B-Cell Non-Hodgkin’s Lymphoma (B-NHL)
• CARTITUDE 6_A Phase 3 Randomized Study Comparing Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) followed by Ciltacabtagene Autoleucel versus Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) followed by Autologous Stem Cell Transplant (ASCT) in Participants with Newly Diagnosed Multiple Myeloma who are Transplant Eligible