A Study to Learn More About the Study Medicine Called Inotuzumab Ozogamicin (InO) in Children (1 to <18 Years) With First Relapse ALL

NCT ID: NCT05748171

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-05-17

Study Completion Date

2036-11-04

Brief Summary

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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) 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.

Detailed Description

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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 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.

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.

Conditions

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ACUTE LYMPHOBLASTIC LEUKEMIA

Keywords

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ALL BCP ALL High risk BCP ALL Relapse ALL Leukemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Participants will be randomized at 2:1 ratio, so that approximately 67 participants will receive InO and 33 participants will receive the comparator treatment regimen, ALLR3
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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

Group Type EXPERIMENTAL

Inotuzumab ozogamicin

Intervention Type DRUG

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.

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

Group Type ACTIVE_COMPARATOR

ALLR3

Intervention Type DRUG

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)

Interventions

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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.

Intervention Type DRUG

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)

Intervention Type DRUG

Other Intervention Names

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Besponsa R3

Eligibility Criteria

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Inclusion Criteria

1. Male or female participants between 1 and \<18 years of age.
2. Morphologically confirmed diagnosis of first relapse HR 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).

* 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;
* 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.

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
Minimum Eligible Age

1 Year

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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St. Anna Kinderspital

Vienna, , Austria

Site Status RECRUITING

Cliniques universitaires Saint-Luc

Brussels, Bruxelles-capitale, Région de, Belgium

Site Status RECRUITING

UZ Gent

Ghent, Oost-vlaanderen, Belgium

Site Status RECRUITING

UZ Leuven

Leuven, Vlaams-brabant, Belgium

Site Status RECRUITING

Detska nemocnice FN Brno

Brno, Brno-město, Czechia

Site Status RECRUITING

Fakultni nemocnice v Motole

Prague, , Czechia

Site Status RECRUITING

Rigshospitalet

Copenhagen, Capital Region, Denmark

Site Status RECRUITING

Helsinki university hospital

Helsinki, , Finland

Site Status RECRUITING

Centre Hospitalier Universitaire de Nice - Hôpital l'Archet

Nice, Alpes-maritimes, France

Site Status RECRUITING

CHU Strasbourg-Hautepierre, Service d'hematologie oncologie pediatrique, pediatrie 3

Strasbourg, Alsace, France

Site Status RECRUITING

Bordeaux University Hospital - Pellegrin

Bordeaux, Aquitaine, France

Site Status RECRUITING

CHU de Toulouse - Hôpital des Enfants - Hemato-Immuno-Oncologie

Toulouse, Haute-garonne, France

Site Status RECRUITING

Hôpital Arnaud de Villeneuve - CHU Montpellier

Montpellier, Hérault, France

Site Status RECRUITING

Centre Hospitalier Universitaire de Nantes - Hôpital Femme-Enfant-Adolescent Chu De Nantes

Nantes, Loire-atlantique, France

Site Status RECRUITING

Centre Hospitalier Régional Universitaire de Nancy - Hôpitaux de Brabois

Vandœuvre-lès-Nancy, Meurthe-et-moselle, France

Site Status RECRUITING

Hôpital Jeanne de Flandre

Lille, NORD, France

Site Status RECRUITING

Assistance Publique - Hopitaux de Paris (AP-HP) - Hopital Robert Debre - Centre Hospitalo Universita

Paris, Paris, France

Site Status RECRUITING

Institut d'Hématologie et d'Oncologie Pédiatrique

Lyon, , France

Site Status RECRUITING

Hôpital Armand Trousseau

Paris, , France

Site Status RECRUITING

CHRU De Rennes - Hôpital Sud

Rennes, , France

Site Status RECRUITING

Universitaetsklinikum Freiburg

Freiburg im Breisgau, Baden-Wurttemberg, Germany

Site Status RECRUITING

Universitaetsklinikum Tuebingen

Tübingen, Baden-Wurttemberg, Germany

Site Status RECRUITING

Universitaetsklinikum Ulm

Ulm, Baden-Wurttemberg, Germany

Site Status RECRUITING

Universitaetsklinikum Wuerzburg

Würzburg, Bavaria, Germany

Site Status RECRUITING

Universitätsklinikum Frankfurt Goethe-Universität

Frankfurt am Main, Hesse, Germany

Site Status RECRUITING

Medizinische Hochschule Hannover

Hanover, Lower Saxony, Germany

Site Status RECRUITING

Universitaetsklinikum Essen

Essen, North Rhine-Westphalia, Germany

Site Status RECRUITING

Universitätsklinikum Münster - Albert Schweitzer Campus

Münster, North Rhine-Westphalia, Germany

Site Status RECRUITING

Universitaetsklinikum Schleswig-Holstein Campus Kiel

Kiel, Schleswig-Holstein, Germany

Site Status RECRUITING

Charité Campus Virchow-Klinikum

Berlin, , Germany

Site Status RECRUITING

Universitaetsklinikum Duesseldorf

Düsseldorf, , Germany

Site Status RECRUITING

Universitätsklinikum Gießen

Giessen, , Germany

Site Status RECRUITING

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, , Germany

Site Status RECRUITING

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status RECRUITING

Universitätsklinikum Jena

Jena, , Germany

Site Status RECRUITING

Pécsi Tudományegyetem Klinikai Központ

Pécs, Baranya, Hungary

Site Status RECRUITING

Borsod-Abaúj-Zemplén Megyei Központi Kórház és Egyetemi Oktatókórház

Miskolc, Borsod-Abauj Zemplen county, Hungary

Site Status RECRUITING

Semmelweis Egyetem

Budapest, , Hungary

Site Status RECRUITING

Schneider Children's Medical Center

Petah Tikva, Central District, Israel

Site Status RECRUITING

The Edmond and Lily Safra Children's Hospital The Chaim Sheba Medical Center Department of Pediatric

Ramat Gan, Central District, Israel

Site Status RECRUITING

Rambam Health Care Campus

Haifa, Northern District, Israel

Site Status RECRUITING

Tel-Aviv Sourasky Medical Center Dana-Dwek Children's Hospital

Tel Aviv, TELL ABĪB, Israel

Site Status RECRUITING

Azienda Ospedaliera di Rilievo Nazional Santobono Pausilipon

Napoli, Campania, Italy

Site Status RECRUITING

IRCCS Istituto Giannina Gaslini

Genoa, Liguria, Italy

Site Status RECRUITING

Fondazione IRCCS San Gerardo dei Tintori

Monza, Lombardy, Italy

Site Status RECRUITING

Ospedale Pediatrico Bambino Gesù IRCCS

Rome, ROMA, Italy

Site Status RECRUITING

Policlinico "G. Rodolico"

Catania, Sicily, Italy

Site Status RECRUITING

Azienda Ospedale - Università Padova

Padua, Veneto, Italy

Site Status RECRUITING

IRCCS - AOU di Bologna

Bologna, , Italy

Site Status RECRUITING

Azienda di Rilievo Nazionale e Alta Specializzazione Civico Di Cristina Benfratelli

Palermo, , Italy

Site Status RECRUITING

Fondazione IRCCS Policlinico San Matteo

Pavia, , Italy

Site Status RECRUITING

Ospedale Regina Margherita

Torino, , Italy

Site Status RECRUITING

Ospedale Infantile Burlo Garofolo

Trieste, , Italy

Site Status RECRUITING

Prinses Maxima Centrum voor Kinderoncologie

Utrecht, , Netherlands

Site Status RECRUITING

Oslo Universitetssykehus Rikshospitalet

Oslo, , Norway

Site Status RECRUITING

Radium Hospital

Oslo, , Norway

Site Status RECRUITING

Szpital Uniwersytecki nr 1 im. dr. A. Jurasza w Bydgoszczy

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland

Site Status RECRUITING

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu

Wroclaw, Lower Silesian Voivodeship, Poland

Site Status RECRUITING

Narodny ustav detskych chorob

Bratislava, Bratislava Region, Slovakia

Site Status RECRUITING

CHUS - Hospital Clinico Universitario

Santiago de Compostela, A Coruña [LA Coruña], Spain

Site Status RECRUITING

Hospital Universitari Vall d'Hebron

Barcelona, Barcelona [barcelona], Spain

Site Status RECRUITING

Hospital Sant Joan de Déu

Esplugues de Llobregat, Barcelona [barcelona], Spain

Site Status RECRUITING

Hospital Infantil Universitario Niño Jesús

Madrid, Madrid, Comunidad de, Spain

Site Status RECRUITING

Hospital Clinico Universitario Virgen de la Arrixaca

El Palmar, , Spain

Site Status RECRUITING

Hospital Universitario La Paz

Madrid, , Spain

Site Status RECRUITING

CHUS - Hospital Clinico Universitario

Santiago de Compostela, , Spain

Site Status RECRUITING

Hospital Universitario Virgen Del Rocio

Seville, , Spain

Site Status RECRUITING

Hospital Universitari i Politecnic La Fe

Valencia, , Spain

Site Status RECRUITING

Skånes Universitetssjukhus Lund

Lund, Skåne LÄN [se-12], Sweden

Site Status RECRUITING

Sahlgrenska Universitetssjukhuset Östra

Gothenburg, Västra Götalands LÄN [se-14], Sweden

Site Status RECRUITING

Astrid Lindgrens Barnsjukhus

Stockholm, , Sweden

Site Status RECRUITING

Inselspital Bern

Bern, Canton of Bern, Switzerland

Site Status RECRUITING

CHUV (centre hospitalier universitaire vaudois)

Lausanne, Canton of Vaud, Switzerland

Site Status RECRUITING

Kinderspital Zürich

Zurich, , Switzerland

Site Status RECRUITING

Countries

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Greece Austria Belgium Czechia Denmark Finland France Germany Hungary Israel Italy Netherlands Norway Poland Slovakia Spain Sweden Switzerland

Central Contacts

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Pfizer CT.gov Call Center

Role: CONTACT

Phone: 1-800-718-1021

Email: [email protected]

Related Links

Access external resources that provide additional context or updates about the study.

https://pmiform.com/clinical-trial-info-request?StudyID=B1931036

To obtain contact information for a study center near you, click here.

Other Identifiers

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2023-509810-13-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

B1931036

Identifier Type: -

Identifier Source: org_study_id