Ensayos clínicos

ANEMIA FALCIFORME

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) (>12a).

Centros: Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez, Hospital Universitario La Paz (Madrid) – Dra. Marta Morado.

A Phase II, multicenter, randomized, open label two arm study comparing the effect of crizanlizumab + standard of care to standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle cell nephropathy (STEADFAST).

Centros: Hospital Universitario Gregorio Marañón (Madrid) – Dra. Gloria Pérez Rus, Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez.

PRAISE: An adaptive, randomized, placebo controlled, double-blind, multicenter study of oral FT-4202, a Pyruvate Kinase activator in patients with sickle cell disease

Centros: Hospital Universitario La Paz (Madrid) – Dra. Marta Morado, Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez.

AG946-C-001 A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Subjects with Sickle Cell Disease.

Centro: Hospital Clínico Universitario Virgen de la Arrixaca (Murcia) – Dr. Eduardo José Salido Fiérrez.

BO42452/ A Phase Ib Randomized, Placebo-Controlled Study Evaluating The Safety, Pharmacokinetics, Pharmacodynamics, And Efficacy Of Crovalimab For The Management Of Acute Uncomplicated Vaso-Cclusive Episodes (VOE) In Patients With Sickle Cell Disease (SCD)

Centro: Hospital Universitari Vall d´Hebron (Barcelona) – Dr. David Beneitez.

BO42451/ A Randomized Double-Blind Phase 2a Study Evaluating The Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Crovalimab As Adjunct Treatment In Prevention Of Acute Vaso-Occlusive Episodes (VOE) In Sickle Cell Disease (SCD)

Centro: Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez.

ANEMIA HEMOLÍTICA POR DÉFICIT DE PIRUVATO-KINASA

AG348-C-011/ An Open-Label, Multicenter, Extension Study Of AG-348 In Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled In AG-348 Studies

Centros: Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez, Hospital Universitario La Paz (Madrid) – Dra. Marta Morado, Hospital Clínico Universitario Virgen de la Arrixaca (Murcia) – Dr. Eduardo José Salido Fiérrez.

Pyruvate Kinase Deficiency global longitudinal registry (AG348-C-008 OBS DPK).

Centros: Hospital Universitario Vall d’Hebron (Barcelona), Hospital Universitario La Paz (Madrid), H. Sant Joan de Déu (Barcelona), H. de la Santa Creu i Sant Pau (Barcelona), Hospital Infantil Universitario Niño Jesús (Madrid)

Terapia génica para la Deficiencia de Piruvato Quinasa (PKD): ensayo clínico de fase I para evaluar la seguridad de la infusión de células CD34 + autólogas transducidas con un vector lentiviral que contiene el gen de piruvato quinasa de glóbulos rojos (CoRPK) con optimización de codones en sujetos adultos y pediátricos con PKD.

Centro: Hospital Infantil Universitario Niño Jesús (Madrid) – Dr. Julián Sevilla.

TALASEMIA

AG348-C-018. A phase 3 double blind, randomized, placebo controlled, multicenter, study evaluating the efficay and safety of Mitapivat in subjects with trasnfusion dependent alpha or beta thalassemia (ENERGIZE-T).

Centros: Hospital Universitario La Paz (Madrid) – Dra. Marta Morado, Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez, Hospital Clínico Universitario Virgen de la Arrixaca (Murcia) – Dr. Eduardo José Salido Fiérrez, Hospital Universitario Miguel Servet (Zaragoza) – Dra. Valle Recasens.

AG348-C-017. A phase 3 double blind, randomized, placebo controlled, multicenter, study evaluating the efficay and safety of Mitapivat in subjects with non-trasnfusion dependent alpha or beta thalassemia (ENERGIZE).

Centros: Hospital Universitario La Paz (Madrid) – Dra. Marta Morado, Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez, Hospital Clínico Universitario Virgen de la Arrixaca (Murcia) – Dr. Eduardo José Salido Fiérrez.

ANEMIA HEMOLÍTICA AUTOINMUNE

A Phase 2, Multicenter, Non-Randomized, Open-Label Study of RVT-1401 (Batoclimab) for the Treatment of Patients with Warm Autoimmune Hemolytic Anemia.

Centro: Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez.

A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

Centro: Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez.

MOM-M281-006: Eficacy and safety of M281 (Nipocalimab) in adults with warm autoimmune hemolytic anemia: a multicenter, rendomized, double-blind, placebo-controlled, study with a long-term open-label extension.

Centros: Hospital Universitario La Paz (Madrid) – Dra. Marta Morado, Hospital Universitario Príncipe de Asturias (Alcalá de Henares) – Dra. Monserrat López Rubio, Hospital Universitario Dr. Negrín (Gran Canarias) – Dra. Silvia Iñigo de la Iglesia, Hospital Universitario Miguel Servet (Zaragoza) – Dra. Valle Recasens.

A Multicenter, Phase 1b, Open Label, Nonrandomized, Single Dose Study Evaluating the Safety, Tolerability and Activity of BIVV020 in Adults With Cold Agglutinin Disease.

Centro: Hospital Universitario La Paz (Madrid) – Dra. Marta Morado.

HEMOGLOBINURIA PAROXISTICA NOCTURNA

A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab.

Centro: Hospital Universitario Dr. Negrín (Gran Canarias) – Dra. Silvia Iñigo de la Iglesia

COMMODORE: A phase III, randomized, open-label, active-controlled, multicenter stydy evaluating the efficacy and safety of Crovalimab versus Eculizumab in adult and adolescent patients with paroxysmal nocturnal hemoglobinuria not previously treated wuth complement inhibitors.

Centros: Hospital Universitario La Paz (Madrid) – Dra. Marta Morado, Hospital Universitario Dr. Negrín (Gran Canarias) – Dra. Silvia Iñigo de la Iglesia, Hospital Universitario Basurto (Bilbao) – Dra. Cristina de Barrenetxea, Hospital Universitario Miguel Servet (Zaragoza) – Dra. Valle Recasens.

Ensayo multicéntrico, aleatorizado, abierto y controlado con comparador activo para evaluar la eficacia y la seguridad de LNP023 (Iptacopan) por vía oral dos veces al día en pacientes adultos con hemoglobinuria paroxística nocturna y anemia residual, a pesar del tratamiento con un anticuerpo anti-C5 por vía intravenosa.

Centro: Hospital Universitario Donostia (San Sebastián) – Dr. Carlos Vallejo.

A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH).

Centros: Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez.

BO42161/ A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Adult And Adolescent Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors

Centros: Hospital Universitari Vall d’Hebron (Barcelona) – Dr. David Beneitez.

SÍNDROME MIELODISPLÁSICO BAJO RIESGO

Efficacy and safety study 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 naïve subjects who require red blood cell transfusions (COMMANDS).

Centros: Instituto Catalan de Oncologia-Hospital Duran i Reynals (Barcelona), Hospital Universitari Vall d’Hebron (Barcelona), Hospital Virgen de las Nieves (Granada), Hospital General Universitario Gregorio Marañon (Madrid), Hospital Universitario 12 de Octubre (Madrid), Hospital Universitario Virgen De La Victoria (Málaga), C. H. de Orense (Ourense), Hospital Universitario de Salamanca (Salamanca), Hospital Clinico Universitario de Valencia (Valencia), Hospital Universitari i Politecnic La Fe de Valencia (Valencia)

Efficacy and safety of FG-4592 (Roxadustat) for treatment of anemia in patients with lower risk MDS with low red blood cell transfusion burden.

Centros: Hospital Universitario de Salamanca (Salamanca), H. de la Santa Creu i Sant Pau (Barcelona), Hospital Universitario Virgen del Rocío (Sevilla).

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.

Centros: Hospital Universitario Puerta del Mar (Cádiz), Hospital Universitario Puerta de Hierro Majadahonda (Madrid), Hospital Universitario Cruces (Barakaldo), Hospital Universitario Vall d’Hebron (Barcelona), Hospital Universitario La Paz (Madrid), Hospital Universitario de Salamanca (Salamanca), Hospital Universitari i Politecnic La Fe de Valencia (Valencia)

ANEMIA APLÁSICA ADQUIRIDA

Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia (SOAR)

Centro: Hospital Universitario Donostia (San Sebastián) – Dr. Carlos Vallejo

ANEMIA DE FANCONI

Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A (FANCOLEN-1).

Centro: Hospital Infantil Universitario Niño Jesús (Madrid) – Dr. Julián Sevilla

PTT CONGÉNITA

A Phase 3, Prospective, Randomized, Controlled, Open-label, Multicenter, 2 Period Crossover Study With a Single Arm Continuation Evaluating the Safety And Efficacy of BAX 930 (rADAMTS13) in the Prophylactic And On-demand Treatment of Subjects With Severe Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP])

Centros: Hospital Universitario Vall de Hebrón (Barcelona), Hospital Universitario La Paz (Madrid), H. Sant Joan de Déu (Barcelona), H. de la Santa Creu i Sant Pau (Barcelona)
Contacto: Dr. David Beneitez (H. Vall de Hebrón), Dra. Marta Morado (H.Universitario La Paz)

PYRUVATE KINASE DEFICIENCY GLOBAL LONGITUDINAL REGISTRY (AG348-C-008 OBS DPK)

A Phase 3, Prospective, Randomized, Controlled, Open-label, Multicenter, 2 Period Crossover Study With a Single Arm Continuation Evaluating the Safety And Efficacy of BAX 930 (rADAMTS13) in the Prophylactic And On-demand Treatment of Subjects With Severe Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP])

Centros: Hospital Universitario Cruces (Barakaldo) – Dr. Rafael Del Orbe, Hospital General Universitario de Alicante – Dr. Pascual Marco, Hospital Universitari i Politecnic La Fe de Valencia (Valencia) – Dr. Santiago Bonanad

A PHASE III, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO
EVALUATE THE EFFICACY AND SAFETY OF AG-348 IN NOT REGULARLY TRANSFUSED
ADULT SUBJECTS WITH PYRUVATE KINASE DEFICIENCY (AG348-C-006 F III DPK)

Centros: Hospital Universitario Vall de Hebrón (Barcelona) y Hospital Universitario La Paz (Madrid),H. de la Santa Creu i Sant Pau (Barcelona)
Contacto: Dr. David Beneitez (H. Vall de Hebrón), Dra. Marta Morado (H.Universitario La Paz), Dr. Ángel Remacha (H. de la Santa Creu i Sant Pau)

AN OPEN-LABEL, MULTICENTER, EXTENSION STUDY OF AG-348 IN ADULT
SUBJECTS WITH PYRUVATE KINASE DEFICIENCY PREVIOUSLY ENROLLED IN AG-348
STUDIES (AG348-C-011 FIII EXTENSIÓN DPK)

Centro: Hospital Universitario Vall de Hebrón (Barcelona) y Hospital Universitario La Paz (Madrid)
Contacto: Dr. David Beneitez (H. Vall de Hebrón), Dra. Marta Morado (H.Universitario La Paz)

lentiviral-mediated gene therapy of fanconi anemia patients subtype a (fancolen-1)

Centro: Hospital infantil Universitario Niño Jesus, Madrid, Spain
Contacto: Julian Sevilla

paricalcitol improves anemia of inflamation (pieraid)

Centro: Hospital Sin Espases, Mallorca
Contacto: Miguel Giovanni Uriol Rivera

USEFULNESS OF FECAL IMMUNOCHEMICAL TEST IN IRON DEFICIENCY ANEMIA (IDAFIT) (IDAFIT)

Centro: Hospital Universitario de Canarias

EFFECTS OF IRON THERAPY IN HEART FAILURE WITH PRESERVED EJECTION FRACTION AND IRON DEFICIENCY (PREFER-HF) (PREFER-HF)

Centro: Institut de Recerca Biomèdica de Lleida
Contacto: José Luis Morales Rull, MD, PhD.

ELTROMBOPAG COMBINED WITH CYCLOSPORINE AS FIRST LINE THERAPY IN PATIENTS WITH SEVERE ACQUIRED APLASTIC ANEMIA (SOAR)

Centro: San Sebastián, Madrid.

EFFICACY AND SAFETY OF FG-4592 FOR TREATMENT OF ANEMIA IN PATIENTS WITH LOWER RISK MDS WITH LOW RED BLOOD CELL TRANSFUSION BURDEN

Centros: Investigational Site Recruiting, Sabadell, Barcelona, Spain, 08208 / Investigational Site Recruiting, Barcelona, Catalunya, Spain, 08003 / Investigational Site Recruiting, Pamplona, Navarra, Spain, 31008 / Investigational Site Recruiting, Barcelona, Spain, 08041 / Investigational Site Recruiting, Madrid, Spain, 28034 / Investigational Site Recruiting Sevilla, Spain, 41009 / Investigational Site Recruiting Sevilla, Spain, 41013. H. de la Santa Creu i Sant Pau (Barcelona).

EFFICACY AND SAFETY STUDY 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 NAÏVE SUBJECTS WHO REQUIRE RED BLOOD CELL TRANSFUSIONS (COMMANDS)

Centro: Instituto Catalan de Oncologia-Hospital Duran i Reynals Not yet recruiting, Barcelona, Spain, 08908 / Hospital Universitari Vall d’Hebron Not yet recruiting, Barcelona, Spain, 8035 / Hospital Virgenes de las Nieves Not yet recruiting, Granada, Spain, 18014 / Hospital General Universitario Gregorio Maranon Not yet recruiting, Madrid, Spain, 28007 / Hospital Universitario 12 de Octubre Not yet recruiting, Madrid, Spain, 28041 / Hospital Universitario Virgen De La Victoria Not yet recruiting, Malaga, Spain, 29010 / C. H. de Orense Not yet recruiting, Ourense, Spain, 32005 / Hospital Universitario de Salamanca Not yet recruiting, Salamanca, Spain, 37007 / Hospital Clinico Universitario de Valencia Not yet recruiting, Valencia, Spain, 46010 / Hospital Universitari i Politecnic La Fe de Valencia Not yet recruiting, Valencia, Spain, 46026

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