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En studie for å vurdere toleransen av Ianalumab (VAY736) sammen med forskerens valg av trombopoietinreseptoragonist (IC TPO-RA) hos pasienter med primær immun trombocytopeni (ITP) (VAY2EXPLORE)

5. november 2026 oppdatert av: Novartis Pharmaceuticals

En fase 2 åpen studie for å evaluere tolerabiliteten til Ianalumab (VAY736) sammen med behandlerens valg av trombopoietinreseptoragonist (IC TPO-RA) hos pasienter med primær immun trombocytopeni (ITP) som tidligere har blitt behandlet med minst 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.

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Detaljert 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.

Studietype

Intervensjonell

Registrering (Antatt)

164

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studer Kontakt Backup

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

18 år og eldre (Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: 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 navn:
  • VAY736
Legemiddel: thrombopoietin receptor agonist (TPO-RA)

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

Andre navn:
  • romiplostim
  • eltrombopag
  • avatrombopag
Eksperimentell: 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 navn:
  • VAY736
Legemiddel: thrombopoietin receptor agonist (TPO-RA)

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

Andre navn:
  • romiplostim
  • eltrombopag
  • avatrombopag

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
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
Tiltaksbeskrivelse
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).

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Novartis Pharmaceuticals

Etterforskere

  • Studieleder: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

4. august 2026

Primær fullføring (Antatt)

18. januar 2029

Studiet fullført (Antatt)

31. august 2030

Datoer for studieregistrering

Først innsendt

12. februar 2026

Først innsendt som oppfylte QC-kriteriene

12. februar 2026

Først lagt ut (Faktiske)

19. februar 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

6. november 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

5. november 2026

Sist bekreftet

1. november 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • CVAY736Q1US01

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (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.

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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