Deze pagina is automatisch vertaald en de nauwkeurigheid van de vertaling kan niet worden gegarandeerd. Raadpleeg de Engelse versie voor een brontekst.

Een studie om de verdraagbaarheid van ianalumab (VAY736) te beoordelen in combinatie met de door de onderzoeker gekozen trombopoïetinerceptoragonist (IC TPO-RA) bij patiënten met primaire immuuntrombocytopenie (ITP) (VAY2EXPLORE)

5 november 2026 bijgewerkt door: Novartis Pharmaceuticals

Een fase 2 open-label studie om de verdraagbaarheid van Ianalumab (VAY736) te evalueren in combinatie met de door de onderzoeker gekozen trombopoïetine-receptoragonist (IC TPO-RA) bij patiënten met primaire immuuntrombocytopenie (ITP) die eerder met ten minste één behandeling zijn behandeld (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.

Studie Overzicht

Toestand

Werving

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

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.

Studietype

Ingrijpend

Inschrijving (Geschat)

164

Fase

  • Fase 2

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Contact Back-up

Studie Locaties

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

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

18 jaar en ouder (Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

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

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: 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).
Biologisch: ianalumab

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

Andere namen:
  • VAY736
Geneesmiddel: thrombopoietin receptor agonist (TPO-RA)

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

Andere namen:
  • romiplostim
  • eltrombopag
  • avatrombopag
Experimenteel: 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).
Biologisch: ianalumab

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

Andere namen:
  • VAY736
Geneesmiddel: thrombopoietin receptor agonist (TPO-RA)

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

Andere namen:
  • romiplostim
  • eltrombopag
  • avatrombopag

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
(Main cohort: Primary immune thrombocytopenia (ITP)): Percentages of participants who are tolerable to ianalumab (9 mg/kg)
Tijdsspanne: 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

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
(Main cohort: Primary immune thrombocytopenia (ITP)): Incidence rate of Adverse Events
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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).

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Novartis Pharmaceuticals

Onderzoekers

  • Studie directeur: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

4 augustus 2026

Primaire voltooiing (Geschat)

18 januari 2029

Studie voltooiing (Geschat)

31 augustus 2030

Studieregistratiedata

Eerst ingediend

12 februari 2026

Eerst ingediend dat voldeed aan de QC-criteria

12 februari 2026

Eerst geplaatst (Werkelijk)

19 februari 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

6 november 2026

Laatste update ingediend die voldeed aan QC-criteria

5 november 2026

Laatst geverifieerd

1 november 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • CVAY736Q1US01

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

JA

Beschrijving IPD-plan

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.

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

Abonneren