Denne side blev automatisk oversat, og nøjagtigheden af ​​oversættelsen er ikke garanteret. Der henvises til engelsk version for en kildetekst.

En undersøgelse til vurdering af tolerabiliteten af Ianalumab (VAY736) i kombination med forskerens valg af trombopoietinreceptoragonist (IC TPO-RA) hos patienter med primær immun trombocytopeni (ITP) (VAY2EXPLORE)

5. november 2026 opdateret af: Novartis Pharmaceuticals

En fase 2, åben-label undersøgelse til evaluering af tolerabiliteten af ianalumab (VAY736) med forskerens valg af trombopoietinreceptoragonist (IC TPO-RA) hos patienter med primær immun trombocytopeni (ITP), der tidligere er behandlet med mindst én behandling (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.

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

164

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Undersøgelse Kontakt Backup

Studiesteder

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

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

18 år og ældre (Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

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

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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).
Biologisk: ianalumab

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

Andre navne:
  • VAY736
Medicin: thrombopoietin receptor agonist (TPO-RA)

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

Andre navne:
  • romiplostim
  • eltrombopag
  • avatrombopag
Eksperimentel: 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).
Biologisk: ianalumab

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

Andre navne:
  • VAY736
Medicin: thrombopoietin receptor agonist (TPO-RA)

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

Andre navne:
  • romiplostim
  • eltrombopag
  • avatrombopag

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
(Main cohort: Primary immune thrombocytopenia (ITP)): Percentages of participants who are tolerable to ianalumab (9 mg/kg)
Tidsramme: 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

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
(Main cohort: Primary immune thrombocytopenia (ITP)): Incidence rate of Adverse Events
Tidsramme: 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
Tidsramme: 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)
Tidsramme: 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
Tidsramme: 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).

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Novartis Pharmaceuticals

Efterforskere

  • Studieleder: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

4. august 2026

Primær færdiggørelse (Anslået)

18. januar 2029

Studieafslutning (Anslået)

31. august 2030

Datoer for studieregistrering

Først indsendt

12. februar 2026

Først indsendt, der opfyldte QC-kriterier

12. februar 2026

Først opslået (Faktiske)

19. februar 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

6. november 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

5. november 2026

Sidst verificeret

1. november 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • CVAY736Q1US01

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

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.

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Abonner