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En studie for å lære mer om studiemedisinen kalt Inotuzumab Ozogamicin (InO) hos barn (1 til

23. september 2026 oppdatert av: Pfizer

EN PROSPEKTIV, RANDOMISERT, ÅPEN LABEL FASE 2-STUDIE FOR Å EVALUERE OVERLEGENHETEN TIL INOTUZUMAB OZOGAMICIN MONOTERAPI VERSUS ALLR3 FOR INDUKSJONSBEHANDLING AV HØYRISIKO FOR BARNE, FØRSTE TILBAKESKAP B-ORCELLE PÅREKKYKKE

This prospective, randomized, multicenter, open-label Phase 2 study is designed to evaluate the superiority of InO monotherapy vs ALLR3 after 1 cycle of induction treatment in paediatric participants (between 1 and <18 years) with High Risk (HR) or very high risk (VHR) first bone marrow relapse CD22-positive BCP ALL, and to evaluate the safety and tolerability, PK and long-term efficacy. Treatment with study intervention will end after induction therapy; follow-up will continue for up to 5 years from randomization.

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

This prospective, randomized, multicenter, open-label, Phase 2 study is designed to evaluate the superiority of InO monotherapy vs ALLR3, after 1 cycle of induction treatment in paediatric participants (between 1 and <18 years) with HR or VHR first bone marrow relapse CD22-positive BCP ALL, and to evaluate the safety and tolerability, PK and long-term efficacy. Treatment with study intervention will end after induction therapy; follow-up for efficacy and safety will continue for up to 5 years from randomization.

End of Treatment is defined as occurring upon recovery from 1 cycle of study therapy (Day 28 ± 2 days), or one day before initiation of new anticancer therapy, whichever occurs first.

Approximately 100 participants will be randomized (2:1) to receive 1 cycle of either InO monotherapy or ALLR3 (block 1) therapy during induction. Two interim analyses (approximately 50% and 75% enrollment) fort the primary outcome measure followed by a final analysis are planned to assess efficacy and futility (non-binding).

After completion of induction therapy (ie, study therapy), it is anticipated that the majority of responding participants will proceed immediately to consolidation therapy. Non-responders are expected to proceed with salvage therapy at the investigator's discretion. Participants responding to induction therapy are expected to proceed to SOC consolidation therapy upon recovery of blood counts, but no sooner than 7 days after last dose of study intervention.

All participants (responders and non-responders) will proceed to long-term follow-up for this study. All subsequent anticancer therapy will be determined by the treating physician.

Studietype

Intervensjonell

Registrering (Antatt)

100

Fase

  • Fase 2

Kontakter og plasseringer

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

Studiekontakt

Studiesteder

  • Belgia
    • Bruxelles-capitale, Région de
      • Brussels, Bruxelles-capitale, Région de, Belgia, 1200
        • Rekruttering
        • Cliniques universitaires Saint-Luc
    • Oost-vlaanderen
      • Ghent, Oost-vlaanderen, Belgia, 9000
        • Rekruttering
        • UZ Gent
    • Vlaams-brabant
      • Leuven, Vlaams-brabant, Belgia, 3000
        • Rekruttering
        • UZ Leuven
  • Danmark
    • Capital Region
      • Copenhagen, Capital Region, Danmark, DK-2100
        • Rekruttering
        • Rigshospitalet
  • Finland
      • Helsinki, Finland, 00290
        • Rekruttering
        • Helsinki university hospital
  • Frankrike
      • Lyon, Frankrike, 69008
        • Rekruttering
        • Institut d'Hématologie et d'Oncologie Pédiatrique
      • Nantes, Frankrike, 44000
        • Rekruttering
        • CHU de Nantes - Hôpital Mère - Enfants
      • Paris, Frankrike, 75571
        • Rekruttering
        • Hôpital Armand Trousseau
      • Rennes, Frankrike, 35203
        • Rekruttering
        • CHRU De Rennes - Hôpital Sud
    • Alpes-maritimes
      • Nice, Alpes-maritimes, Frankrike, 06202
        • Rekruttering
        • Centre Hospitalier Universitaire de Nice - Hôpital l'Archet
    • Alsace
      • Strasbourg, Alsace, Frankrike, 67000
        • Rekruttering
        • CHU Strasbourg-Hautepierre, Service d'hematologie oncologie pediatrique, pediatrie 3
    • Aquitaine
      • Bordeaux, Aquitaine, Frankrike, 33076
        • Rekruttering
        • Bordeaux University Hospital - Pellegrin
    • Haute-garonne
      • Toulouse, Haute-garonne, Frankrike, 31059
        • Rekruttering
        • CHU de Toulouse - Hôpital des Enfants - Hemato-Immuno-Oncologie
    • Meurthe-et-moselle
      • Vandœuvre-lès-Nancy, Meurthe-et-moselle, Frankrike, 54511
        • Rekruttering
        • Centre Hospitalier Régional Universitaire de Nancy - Hôpitaux de Brabois
    • NORD
      • Lille, NORD, Frankrike, 59037
        • Rekruttering
        • Hôpital Jeanne de Flandre - CHRU
    • Paris
      • Paris, Paris, Frankrike, 75935
        • Rekruttering
        • Assistance Publique - Hopitaux de Paris (AP-HP) - Hopital Robert Debre - Centre Hospitalo Universita
  • Israel
    • Central District
      • Petah Tikva, Central District, Israel, 49202
        • Rekruttering
        • Schneider Children's Medical Center
      • Ramat Gan, Central District, Israel, 5265601
        • Rekruttering
        • The Edmond and Lily Safra Children's Hospital; The Chaim Sheba Medical Center
    • Northern District
      • Haifa, Northern District, Israel, 3109601
        • Rekruttering
        • Rambam Health Care Campus
    • TELL ABĪB
      • Tel Aviv, TELL ABĪB, Israel, 6423906
        • Rekruttering
        • Tel-Aviv Sourasky Medical Center Dana-Dwek Children's Hospital
  • Italia
      • Pavia, Italia, 27100
        • Rekruttering
        • Fondazione IRCCS Policlinico San Matteo
      • Torino, Italia, 10126
        • Rekruttering
        • Ospedale Regina Margherita
      • Trieste, Italia, 34137
        • Rekruttering
        • IRCCS Materno Infantile Burlo Garofolo
    • Campania
      • Naples, Campania, Italia, 80123
        • Rekruttering
        • Azienda Ospedaliera di Rilievo Nazional Santobono Pausilipon
    • Liguria
      • Genoa, Liguria, Italia, 16147
        • Rekruttering
        • IRCCS Istituto Giannina Gaslini
    • Lombardy
      • Monza, Lombardy, Italia, 20900
        • Rekruttering
        • Fondazione IRCCS San Gerardo dei Tintori
    • ROMA
      • Rome, ROMA, Italia, 00165
        • Rekruttering
        • Ospedale Pediatrico Bambino Gesù IRCCS
    • Sicily
      • Catania, Sicily, Italia, 95123
        • Rekruttering
        • Policlinico "G. Rodolico"
    • Veneto
      • Padova, Veneto, Italia, 35128
        • Rekruttering
        • Azienda Ospedale - Università Padova
  • Nederland
      • Utrecht, Nederland, 3584 CS
        • Rekruttering
        • Prinses Maxima Centrum voor Kinderoncologie
  • Norge
      • Oslo, Norge, 0372
        • Rekruttering
        • Oslo Universitetssykehus Rikshospitalet
      • Oslo, Norge, 0379
        • Rekruttering
        • Radium Hospital
  • Slovakia
    • Bratislava Region
      • Bratislava, Bratislava Region, Slovakia, 833 40
        • Rekruttering
        • Narodny ustav detskych chorob
  • Spania
      • Madrid, Spania, 28046
        • Rekruttering
        • Hospital Universitario La Paz
      • Santiago de Compostela, Spania, 15706
        • Rekruttering
        • CHUS - Hospital Clinico Universitario
      • Seville, Spania, 41013
        • Rekruttering
        • Hospital Universitario Virgen Del Rocio
      • Valencia, Spania, 46026
        • Rekruttering
        • Hospital Universitari i Politecnic La Fe
    • A Coruña [LA Coruña]
      • Santiago de Compostela, A Coruña [LA Coruña], Spania, 15706
        • Rekruttering
        • CHUS - Hospital Clinico Universitario
    • Barcelona [barcelona]
      • Barcelona, Barcelona [barcelona], Spania, 08035
        • Rekruttering
        • Hospital Universitari Vall d'Hebron
      • Esplugues de Llobregat, Barcelona [barcelona], Spania, 08950
        • Rekruttering
        • Hospital Sant Joan de Déu
    • Madrid, Comunidad de
      • Madrid, Madrid, Comunidad de, Spania, 28009
        • Rekruttering
        • Hospital Infantil Universitario Niño Jesús
    • Murcia
      • El Palmar, Murcia, Spania, 30120
        • Rekruttering
        • Hospital Clinico Universitario Virgen de la Arrixaca
  • Sveits
      • Zurich, Sveits, 8008
        • Rekruttering
        • Kinderspital Zürich
    • Canton of Bern
      • Bern, Canton of Bern, Sveits, 3010
        • Rekruttering
        • Inselspital Bern
    • Canton of Vaud
      • Lausanne, Canton of Vaud, Sveits, 1011
        • Rekruttering
        • CHUV (centre hospitalier universitaire vaudois)
  • Sverige
      • Stockholm, Sverige, 17067
        • Rekruttering
        • Astrid Lindgrens Barnsjukhus
    • Skåne LÄN [se-12]
      • Lund, Skåne LÄN [se-12], Sverige, 22185
        • Rekruttering
        • Skånes Universitetssjukhus Lund
    • Västra Götalands LÄN [se-14]
      • Gothenburg, Västra Götalands LÄN [se-14], Sverige, 416 85
        • Rekruttering
        • Sahlgrenska Universitetssjukhuset Östra
  • Tsjekkia
      • Prague, Tsjekkia, 150 00
        • Rekruttering
        • Fakultni Nemocnice Motol a Homolka
    • Brno-město
      • Brno, Brno-město, Tsjekkia, 613 00
        • Rekruttering
        • Detska nemocnice FN Brno
  • Tyskland
      • Berlin, Tyskland, 13353
        • Rekruttering
        • Charité Campus Virchow-Klinikum
      • Düsseldorf, Tyskland, 40225
        • Rekruttering
        • Universitaetsklinikum Duesseldorf
      • Giessen, Tyskland, 35392
        • Rekruttering
        • Universitätsklinikum Gießen
      • Hamburg, Tyskland, 20246
        • Rekruttering
        • Universitaetsklinikum Hamburg-Eppendorf
      • Hanover, Tyskland, 30625
        • Rekruttering
        • Medizinische Hochschule Hannover
      • Jena, Tyskland, 07747
        • Rekruttering
        • Universitätsklinikum Jena
    • Baden-Wurttemberg
      • Freiburg im Breisgau, Baden-Wurttemberg, Tyskland, 79106
        • Rekruttering
        • Universitaetsklinikum Freiburg
      • Ulm, Baden-Wurttemberg, Tyskland, 89081
        • Rekruttering
        • Universitaetsklinikum Ulm
    • Bavaria
      • Würzburg, Bavaria, Tyskland, 97080
        • Rekruttering
        • Universitaetsklinikum Wuerzburg
    • Hesse
      • Frankfurt am Main, Hesse, Tyskland, 60590
        • Rekruttering
        • Universitätsklinikum Frankfurt Goethe-Universität
    • Lower Saxony
      • Hanover, Lower Saxony, Tyskland, 30625
        • Rekruttering
        • Medizinische Hochschule Hannover
    • North Rhine-Westphalia
      • Essen, North Rhine-Westphalia, Tyskland, 45122
        • Rekruttering
        • Universitaetsklinikum Essen
      • Münster, North Rhine-Westphalia, Tyskland, 48149
        • Rekruttering
        • Universitätsklinikum Münster - Albert Schweitzer Campus
    • Schleswig-Holstein
      • Kiel, Schleswig-Holstein, Tyskland, 24105
        • Rekruttering
        • Universitaetsklinikum Schleswig-Holstein Campus Kiel
  • Østerrike
      • Vienna, Østerrike, 1090
        • Rekruttering
        • St. Anna Kinderspital

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

1 år til 17 år (Barn)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

Inclusion Criteria:

  1. Male or female participants between 1 and <18 years of age.

  2. Morphologically confirmed diagnosis of first relapse HR or VHR BCP ALL; HR first relapse is defined as relapse occurring within 18 to 30 months of original diagnosis of ALL or within 6 months of completion of primary therapy, and lacking any identified very high-risk genetic abnormalities (Groeneveld-Krentz et al, 2019) (ie, KMT2A::AFF1 fusion [t(4;11)(q21;q23)], TCF3-HLF fusion [t(17;19)(q22;p13)], TCF3-PBX1 fusion [t(1;19)(q23;p13.3)], hypodiploidy [<40 chromosomes] or masked low hypodiploidy (Molina et al, 2021), TP53 alteration). VHR first relapse is defined as relapse within 18 months of original diagnosis of ALL and/or with any of the following genetic abnormalities at original diagnosis or at relapse (Groeneveld-Krentz et al, 2019) (ie, KMT2A::AFF1 fusion [t(4;11)(q21;q23)], TCF3-HLF fusion [t(17;19)(q22;p13)], TCF3-PBX1 fusion [t(1;19)(q23;p13.3)], hypodiploidy [<40 chromosomes] or masked low hypodiploidy (Molina et al, 2021), TP53 alteration).

    • CD22-positive ALL as defined by local institution;
    • Bone marrow involvement of ≥ 5% leukemic blasts (≥ M2 status).

  3. Adequate serum chemistry parameters:

    • An eGFR in participants 1 to <2 years of age, or eCrCl in those 2 to <18 years of age, ≥30 mL/min using the recommended formula in Section 10.10.2.
    • AST and ALT ≤5 × institutional ULN at the time of randomization or pre-cytoreduction/general anesthesia; (Refer to Appendix 9 for France-specific requirement on the ALT/AST threshold);
    • Total bilirubin ≤1.5 × institutional ULN unless the participant has documented Gilbert's syndrome;

  4. Prior history of thrombosis during corticosteroid use and/or asparaginase are eligible provided the patient receives anti-coagulant prophylaxis per institutional guidelines.

  5. Cardiac shortening fraction ≥ 30% by echocardiogram or ejection fraction >50% by MUGA.

6 Participants with combined bone marrow and testicular relapse are eligible assuming orchiectomy is performed prior to randomization or is planned at the end of induction therapy.

5.2. Exclusion Criteria

  1. Any history of prior or ongoing hepatic SOS or prior liver failure [defined as severe acute liver injury with encephalopathy and impaired synthetic function (INR of ≥1.5)].
  2. Prior allo-HSCT or CAR T-cell therapy.
  3. Isolated extramedullary leukemia.
  4. Philadelphia-chromosome positive ALL, ie. BCR-ABL/t(9;22) present.
  5. Prior therapy with a calicheamicin-conjugated antibody (eg, InO or gemtuzumab ozogamicin).
  6. Participants with active, uncontrolled bacterial, fungal, or viral infection.
  7. Hypersensitivity/allergy to both PEG-ASP and Erwinia-ASP

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: Inotuzumab ozogamicin

Each participant in the InO arm will receive 1 course (3 doses) of InO, as follows:

  • Day 1: 0.8 mg/m2
  • Days 8 (±1 day) and Day 15 (±1 day): 0.5 mg/m2/dose
Legemiddel: Inotuzumab ozogamicin

Inotuzumab ozogamicin (BESPONSA™) is a CD22 targeted antibody drug conjugate (ADC) approved in several countries for the treatment of adults with relapsed or refractory B cell precursor acute lymphoblastic leukemia (ALL). The approved starting dose is 1.8mg/m2/cycle.

Andre navn:
  • Besponsa
Aktiv komparator: ALLR3

Mitoxantrone 10 mg/m2 on Days 1 and 2 Vincristine 1.5 mg/m2 (max single dose 2 mg) administered on Days 3, 10, 17 and 24 Dexamethasone 20 mg/m2/day administered orally (or IV) divided into two daily doses (maximum 40 mg/day) as two 5-day blocks on Days 1-5 and Days 15-19.

PEG-asparaginase 1000 units/m2 IV administered on Days 3 and 17. In case of hypersensitivity/allergic reaction to PEG-asparaginase, each dose of PEG-asparaginase will be replaced by Erwinia-asparaginase at a dose of 20,000 units/m² IV or IM every other day for a total of 6 doses
Legemiddel: ALLR3

The ALLR3 chemotherapy regimen (vincristine, mitoxantrone, dexamethasone, and PEG-asparaginase [or erwinia-asparaginase in the event of an allergic reaction to PEG-asparaginase]) has been adopted by pediatric oncology groups as treatment for pediatric relapsed/refractory (R/R) acute lymphoblastic leukemia (ALL)

Andre navn:
  • R3

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Minimum Residual Disease (MRD) Negativity in participants achieving complete response (CR), complete response with incomplete platelet count recovery (CRp), or complete response with incomplete count recovery (CRi)
Tidsramme: After 1 treatment cycle: Day 28 +/- 2 days

MRD negativity status is determined based on the minimum MRD percentage between the date of CR/CRp/CRi and end of treatment test as assessed by RQ-PCR, with reflex to FC result if MRD is non-evaluable by RQ-PCR
After 1 treatment cycle: Day 28 +/- 2 days

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Event Free Survival (EFS)
Tidsramme: From study start to first event (progression, relapse, failure to achieve CR/CRp/CRi by the end of induction, MRD persistence prior to HSCT [hematopoietic stem cell transplant], second malignancy, or death): up to 5 years from randomization

EFS will be summarized using Kaplan-Meier methods and displayed graphically by treatment arm.
From study start to first event (progression, relapse, failure to achieve CR/CRp/CRi by the end of induction, MRD persistence prior to HSCT [hematopoietic stem cell transplant], second malignancy, or death): up to 5 years from randomization
Duration of Response (DoR) for Participants Who Achieved CR/CRp/CRi
Tidsramme: From date of first response to date of first event (objective progression, relapse as determined by investigator assessment, MRD persistence prior to HSCT, or death due to any cause, whichever occurs first): up to 5 years from End of Treatment

DoR will be summarized using Kaplan-Meier methods.
From date of first response to date of first event (objective progression, relapse as determined by investigator assessment, MRD persistence prior to HSCT, or death due to any cause, whichever occurs first): up to 5 years from End of Treatment
Rate of hematopoietic stem cell transplantation (HSCT)
Tidsramme: Up to 5 years from randomization

HSCT rate will be summarized by descriptive analyses (ie, percentage of participants who underwent HSCT after treatment).
Up to 5 years from randomization
Overall Survival (OS)
Tidsramme: From start of treatment to date of death due to any cause: up to 5 years from randomization

OS will be summarized by treatment arm using Kaplan-Meier methods.
From start of treatment to date of death due to any cause: up to 5 years from randomization
Number of participants reporting an Adverse Event (AE)
Tidsramme: From time of informed consent up to a minimum of 60 calendar days after the last dose of study drug.

The number and percentage of participants who experienced any AE, SAE (Serious Adverse Event), treatment related AE, and treatment related SAE will be summarized according to worst toxicity grades.
From time of informed consent up to a minimum of 60 calendar days after the last dose of study drug.
Pharmacokinetics (PK) parameter: InO Cmax
Tidsramme: 1 treatment cycle: 28 days

Descriptive summary statistics will be provided for InO serum concentrations at scheduled visits.
1 treatment cycle: 28 days
Number of Adverse Events (AE) reported by severity
Tidsramme: From time of informed consent up to a minimum of 60 calendar days after the last dose of study drug.

AEs will be graded by the investigator according to the CTCAE (Common Terminology Criteria for Adverse Events) version 4.03.
From time of informed consent up to a minimum of 60 calendar days after the last dose of study drug.
Pharmacokinetics (PK) parameter: InO trough levels
Tidsramme: 1 treatment cycle: 28 days

Descriptive summary statistics will be provided for InO serum concentrations at scheduled visits.
1 treatment cycle: 28 days
Rate of Chimeric antigen receptor (CAR) T-cell therapy
Tidsramme: Up to 5 years from randomisation

CAR T-cell therapy rate will be summarized by descriptive analyses (ie, the number, percent of participants who underwent CAR T-cell therapy after treatment).
Up to 5 years from randomisation

Samarbeidspartnere og etterforskere

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

Sponsor

Pfizer

Etterforskere

  • Studieleder: Pfizer CT.gov Call Center, Pfizer

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

Hjelpsomme linker

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)

17. mai 2023

Primær fullføring (Antatt)

21. mai 2033

Studiet fullført (Antatt)

25. mai 2036

Datoer for studieregistrering

Først innsendt

20. januar 2023

Først innsendt som oppfylte QC-kriteriene

17. februar 2023

Først lagt ut (Faktiske)

28. februar 2023

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

24. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

23. september 2026

Sist bekreftet

1. september 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • B1931036
  • 2023-509810-13-00 (Registeridentifikator: CTIS (EU))
  • 2023 (U.S. NIH-stipend/kontrakt)

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

JA

IPD-planbeskrivelse

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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