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Un estudio para evaluar la tolerabilidad de ianalumab (VAY736) con la elección del investigador de agonista del receptor de trombopoyetina (IC TPO-RA) en pacientes con trombocitopenia inmune primaria (ITP) (VAY2EXPLORE)

5 de noviembre de 2026 actualizado por: Novartis Pharmaceuticals

Estudio de fase 2 abierto para evaluar la tolerabilidad de ianalumab (VAY736) con la elección de agonista del receptor de trombopoyetina del investigador (IC TPO-RA) en pacientes con púrpura trombocitopénica idiopática (PTI) previamente tratados con al menos un tratamiento (VAY2EXPLORE)

The purpose of this study is to investigate the tolerability of ianalumab (9 mg/kg) with investigator's choice thrombopoietin receptor agonist (IC TPO-RA) in participants diagnosed with primary immune thrombocytopenia (ITP) who have been treated with at least one but no more than four prior treatments, and with no change in IC TPO-RA dose in at least the last 14 days prior to the start of ianalumab.

Descripción general del estudio

Estado

Reclutamiento

Condiciones

Intervención / Tratamiento

Descripción detallada

The study will include an exploratory cohort of participants with primary Evans syndrome (ES) for whom IC TPO-RA therapy is appropriate per investigator's assessment.

The study will consist of a 28-day screening period; a 16-week treatment period; an IC TPO-RA tapering period during which all participants will be monitored for 16 weeks. All participants will then continue to be followed for another 60-weeks (15 months) of long-term safety follow-up period.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

164

Fase

  • Fase 2

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Copia de seguridad de contactos de estudio

Ubicaciones de estudio

  • Estados Unidos
    • Arizona
      • Chandler, Arizona, Estados Unidos, 85224
        • Reclutamiento
        • Ironwood Cancer and Research Centers
        • Investigador principal:
          • Anu Batra
        • Contacto:
    • Arkansas
      • Fayetteville, Arkansas, Estados Unidos, 72703
        • Reclutamiento
        • Highlands Oncology Group
        • Investigador principal:
          • Joseph Thaddeus Beck
        • Contacto:
          • Connor Ray
          • Número de teléfono: +1 479 587 1700
          • Correo electrónico: JRay@hogonc.com
    • California
      • Torrance, California, Estados Unidos, 90502
        • Reclutamiento
        • Harbor-UCLA Medical Center
        • Investigador principal:
          • Sarah Tomassetti
        • Contacto:
    • Connecticut
      • New Haven, Connecticut, Estados Unidos, 06520
        • Reclutamiento
        • Yale Cancer Center
        • Investigador principal:
          • Yevgeniya Foster
        • Contacto:
      • Stamford, Connecticut, Estados Unidos, 06902
        • Reclutamiento
        • Stamford Hospital
        • Investigador principal:
          • Joshua Dower
        • Contacto:
    • Florida
      • Jacksonville, Florida, Estados Unidos, 32207
        • Reclutamiento
        • Baptist MD Anderson Cancer Center
        • Contacto:
        • Investigador principal:
          • Jorge Antunez de Mayolo
      • Margate, Florida, Estados Unidos, 33063
        • Reclutamiento
        • DH Cancer Research Center LLC
        • Investigador principal:
          • Mazyar Rouhani
        • Contacto:
      • Miami, Florida, Estados Unidos, 33136
        • Reclutamiento
        • University Of Miami
        • Contacto:
        • Investigador principal:
          • Gerald Soff
      • Orlando, Florida, Estados Unidos, 32803
        • Reclutamiento
        • Advent Health Orlando
        • Contacto:
        • Investigador principal:
          • Carlos A Alemany
    • Illinois
      • Hinsdale, Illinois, Estados Unidos, 60521
        • Reclutamiento
        • Hope And Healing Care
        • Investigador principal:
          • Srilata Gundala
        • Contacto:
    • Louisiana
      • Covington, Louisiana, Estados Unidos, 70433
        • Reclutamiento
        • Pontchartrain Cancer Center
        • Investigador principal:
          • David Oubre
        • Contacto:
    • Minnesota
      • Rochester, Minnesota, Estados Unidos, 55905
        • Reclutamiento
        • Mayo Clinic Rochester
        • Contacto:
        • Investigador principal:
          • Alexandra Paula Wolanskyj-Spinner
    • Missouri
      • Kansas City, Missouri, Estados Unidos, 64108
        • Reclutamiento
        • University Health Kansas City
        • Investigador principal:
          • Anuj Shrestha
        • Contacto:
    • Montana
      • Billings, Montana, Estados Unidos, 59102
        • Reclutamiento
        • Intermountain Healthcare
        • Investigador principal:
          • Patrick Cobb
        • Contacto:
    • New Jersey
      • East Brunswick, New Jersey, Estados Unidos, 08816
      • Summit, New Jersey, Estados Unidos, 07901
        • Reclutamiento
        • Summit Medical Group
        • Contacto:
        • Investigador principal:
          • Dipen Patel
    • New York
      • The Bronx, New York, Estados Unidos, 10467
        • Reclutamiento
        • Montefiore Medical Center
        • Investigador principal:
          • Irina Murakhovskaya
        • Contacto:
    • North Carolina
      • Durham, North Carolina, Estados Unidos, 27710
        • Reclutamiento
        • Duke University Medical Center
        • Investigador principal:
          • Ara Metjian
        • Contacto:
    • Pennsylvania
      • Philadelphia, Pennsylvania, Estados Unidos, 19104
        • Reclutamiento
        • Hospital of the University of Pennsylvania Health System
        • Investigador principal:
          • Adam Cuker
        • Contacto:
      • Pittsburgh, Pennsylvania, Estados Unidos, 15224
        • Reclutamiento
        • Western Pennsylvania Hospital
        • Contacto:
        • Investigador principal:
          • Deep Shah
    • South Dakota
      • Sioux Falls, South Dakota, Estados Unidos, 57106
        • Reclutamiento
        • Avera Cancer Institute
        • Investigador principal:
          • Waqas Jehangir
        • Contacto:
    • Texas
      • Fort Worth, Texas, Estados Unidos, 76104
        • Reclutamiento
        • JPS health Network
        • Investigador principal:
          • Kalyani Narra
        • Contacto:
    • Utah
      • Salt Lake City, Utah, Estados Unidos, 84106
        • Reclutamiento
        • Utah Cancer Specialists
        • Contacto:
        • Investigador principal:
          • DiSean Kendall

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

18 años y mayores (Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

Key Inclusion Criteria:

  • Signed informed consent must be obtained prior to participation in the study.
  • Patients aged 18 years and older on the day of signing the informed consent.
  • ITP cohort only: Confirmed diagnosis of primary ITP that has previously responded to corticosteroid treatment or IVIG treatment but the response was not sustained (response is defined as a platelet count ≥ 50 G/L).
  • ITP cohort only: Received at least one prior treatment for ITP.
  • ITP cohort only: Patients with a platelet count < 100 G/L who are receiving a TPO-RA. Patients may already be receiving a TPO-RA or may start a TPO-RA at the time of screening. All patients should be on a stable dose of TPO-RA for at least 14 days prior to first dose of ianalumab. Note: during the screening period, a documented assessment of platelets < 100 G/L is mandatory for enrollment. For patients who received rescue medication before screening, platelet count results obtained prior to the start of the rescue therapy should be used to assess eligibility if collected within 14 days prior to screening.
  • ES cohort only: Patients with clinical diagnosis of primary ES with active thrombocytopenia (< 100 G/L) with warm autoimmune hemolytic anemia (wAIHA) for whom a TPO-RA is appropriate per Investigator.
  • ES cohort only: Inadequate response to or relapse after treatment with corticosteroid therapy.
  • ES cohort only: Diagnosis confirmed by current or past positive direct antiglobulin test (DAT) (IgG+, with or without C3+) and evidence of hemolysis.
  • ES cohort only: any supportive care treatment administered for wAIHA must be stable for at least 4 weeks prior to enrollment.

Key Exclusion Criteria:

  • Patients being treated with TPO-RA for > 6 months.

  • Current life-threatening bleeding (related to thrombocytopenia).

  • Prior splenectomy within 6 months of first administration of ianalumab.

  • Patients with the following laboratory abnormalities:

    • Neutrophils: < 1000/mm3
    • Serum creatinine > 1.5 × upper limit of normal (ULN)
    • Aspartate aminotransferase (AST) > 3.0 × ULN
    • Alanine aminotransferase (ALT) > 3.0 × ULN
    • Immunoglobulin G (IgG) < 5 g/L
    • ITP cohort only: hemoglobin < 10 g/L, total bilirubin > 1.5 × ULN

  • Patients with significantly compromised liver disease (Child-Pugh 7 to 9) and decompensated liver disease (Child-Pugh 10 to 15).

  • Treatment with a B-cell depleting therapy (e.g. rituximab or anti-B cell Activating Factor (e.g. belimumab) within 12 weeks prior to the first administration of ianalumab. Patients who are refractory to rituximab will be excluded from this trial, where refractory is defined as:

    ~ Patients who have not achieved a response (defined as platelet count ≥ 30 G/L and at least doubling from baseline within 12 weeks in the absence of rescue therapy) following completion of a standard course of rituximab

  • History of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes.

  • Known history of primary or secondary immunodeficiency, or a positive human immunodeficiency virus (HIV) enzyme-linked immunosorbent assay (ELISA) and Western blot) test result.

  • Patients exposed to more than 4 prior treatments for ITP.

  • ITP cohort only: Diagnosis of secondary thrombocytopenia.

  • ITP cohort: Use of immunosuppressant drugs other than corticosteroids or rituximab.

  • ES cohort only: Diagnosis of secondary ES.

  • ES cohort only: Life-threatening hemolysis.

  • ES cohort only: patients with autoimmune hemolytic anemia other than wAIHA

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: Aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Main cohort: Primary immune thrombocytopenia (ITP)

All participants will be assigned to ianalumab 9 mg/kg plus investigator's choice thrombopoietin receptor agonist (IC TPO-RA).
Biológico: ianalumab

9 mg per kilogram infusion every 4 weeks (Q4W) for 16 weeks

Otros nombres:
  • VAY736
Droga: thrombopoietin receptor agonist (TPO-RA)

IC TPO-RAs will be administered according to the respective United States Prescribing Information (USPIs)

Otros nombres:
  • romiplostim
  • eltrombopag
  • avatrombopag
Experimental: Exploratory cohort: Primary Evans syndrome (ES)

All participants will be assigned to ianalumab 9 mg/kg plus investigator's choice thrombopoietin receptor agonist (IC TPO-RA).
Biológico: ianalumab

9 mg per kilogram infusion every 4 weeks (Q4W) for 16 weeks

Otros nombres:
  • VAY736
Droga: thrombopoietin receptor agonist (TPO-RA)

IC TPO-RAs will be administered according to the respective United States Prescribing Information (USPIs)

Otros nombres:
  • romiplostim
  • eltrombopag
  • avatrombopag

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
(Main cohort: Primary immune thrombocytopenia (ITP)): Percentages of participants who are tolerable to ianalumab (9 mg/kg)
Periodo de tiempo: Up to Week 16

The proportion of participants who tolerate ianalumab (9 mg/kg) is defined as those who do not experience any of the following events during the combination treatment period (up to Week 16):

  • Discontinuation due to an adverse event (AE) unrelated to efficacy
  • Adverse events leading to dose reduction or dose rate reduction
  • Adverse events resulting in study drug interruption.
Up to Week 16

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
(Main cohort: Primary immune thrombocytopenia (ITP)): Incidence rate of Adverse Events
Periodo de tiempo: From Week 1 Day 1 (first dose of ianalumab) to the end of study (EOS), an average of 4 years

The distribution of adverse events will be conducted through the analysis of frequencies for treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs), based on the monitoring of relevant clinical and laboratory safety parameters.
From Week 1 Day 1 (first dose of ianalumab) to the end of study (EOS), an average of 4 years
(Main cohort: Primary immune thrombocytopenia (ITP)): Percentage of participants with platelet count ≥ 30 G/L, ≥ 50 G/L, ≥ 100 G/L
Periodo de tiempo: Baseline, and at each scheduled study visit until the end of study (EOS), an average of 2 years

Platelet count will be measured as part of routine hematology safety assessments
Baseline, and at each scheduled study visit until the end of study (EOS), an average of 2 years
(Main cohort: Primary immune thrombocytopenia (ITP)): Change from baseline in platelet count for prespecified subgroups (<30, 30 to 50, 50 to <100 G/L)
Periodo de tiempo: Baseline, and at each scheduled study visit until the end of study (EOS), an average of 2 years

Platelet count will be measured as part of routine hematology safety assessments
Baseline, and at each scheduled study visit until the end of study (EOS), an average of 2 years
(Main cohort: Primary immune thrombocytopenia (ITP)): Percentage of participants with successful IC TPO-RA tapering
Periodo de tiempo: From Week 17 Day 1 (W17D1) through Week 33 Day 1 (W33D1).

Tapering of investigator's choice thrombopoietin receptor agonist (IC TPO-RA) will be evaluated based on the proportion of participants who successfully discontinue IC TPO-RA without requiring rescue therapy or new immune thrombocytopenia (ITP) treatments by the end of the tapering period.

A participant is considered successfully tapered if all of the following criteria are met:

  • Discontinuation of IC TPO-RA before Week 33 Day 1 (W33D1) with at least two consecutive platelet counts ≥ 30 G/L from separate visits.
  • No use of rescue treatment or initiation of new ITP therapy in the 4 weeks prior to W33D1.
From Week 17 Day 1 (W17D1) through Week 33 Day 1 (W33D1).

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Novartis Pharmaceuticals

Investigadores

  • Director de estudio: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

4 de agosto de 2026

Finalización primaria (Estimado)

18 de enero de 2029

Finalización del estudio (Estimado)

31 de agosto de 2030

Fechas de registro del estudio

Enviado por primera vez

12 de febrero de 2026

Primero enviado que cumplió con los criterios de control de calidad

12 de febrero de 2026

Publicado por primera vez (Actual)

19 de febrero de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

6 de noviembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

5 de noviembre de 2026

Última verificación

1 de noviembre de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • CVAY736Q1US01

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

SÍ

Descripción del plan IPD

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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