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ACUTE LYMPHOBLASTIC LEUKEMIAPhase 2NCT05748171

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

A Phase 2 study testing Inotuzumab ozogamicin and ALLR3 in people with ACUTE LYMPHOBLASTIC LEUKEMIA, run by Pfizer at 65 sites in 15 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

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… Read more about this study

Who is it for?
Ages 1 year to 17 years
Conditions the study is for
ACUTE LYMPHOBLASTIC LEUKEMIA
What is being tested?
Inotuzumab ozogamicin and ALLR3
Could I receive a placebo?
No, everyone receives an active treatment
How many people
100 participants
Where
65 sitesin 15 countries
Study dates
17 May 2023to 25 May 2036
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Male or female participants between 1 and <18 years of age.
  • 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).
  • 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.
Show all 19Show fewer
  • 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;
  • Prior history of thrombosis during corticosteroid use and/or asparaginase are eligible provided the patient receives anti-coagulant prophylaxis per institutional guidelines.
  • 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
  • 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)].
  • Prior allo-HSCT or CAR T-cell therapy.
  • Isolated extramedullary leukemia.
  • Philadelphia-chromosome positive ALL, ie. BCR-ABL/t(9;22) present.
  • Prior therapy with a calicheamicin-conjugated antibody (eg, InO or gemtuzumab ozogamicin).
  • Participants with active, uncontrolled bacterial, fungal, or viral infection.
  • Hypersensitivity/allergy to both PEG-ASP and Erwinia-ASP

Who cannot join

None listed.

Trial locations and contacts

65 sites in 15 countries. Contact the study team or a site near you to ask about taking part.

Pfizer CT.gov Call Center contact

1-800-718-1021

ClinicalTrials.gov_Inquiries@pfizer.com

65 sites in 15 countries

Germany14 sites, 14 recruiting

  • Charité Campus Virchow-Klinikum

    Berlin, 13353

    Recruiting

  • Universitaetsklinikum Duesseldorf

    Düsseldorf, 40225

    Recruiting

  • Universitaetsklinikum Essen

    Essen, North Rhine-Westphalia, 45122

    Recruiting

  • Universitätsklinikum Frankfurt Goethe-Universität

    Frankfurt am Main, Hesse, 60590

    Recruiting

  • Universitaetsklinikum Freiburg

    Freiburg im Breisgau, Baden-Wurttemberg, 79106

    Recruiting

  • Universitätsklinikum Gießen

    Giessen, 35392

    Recruiting

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246

    Recruiting

  • Medizinische Hochschule Hannover

    Hanover, Lower Saxony, 30625

    Recruiting

  • Medizinische Hochschule Hannover

    Hanover, 30625

    Recruiting

  • Universitätsklinikum Jena

    Jena, 07747

    Recruiting

  • Universitaetsklinikum Schleswig-Holstein Campus Kiel

    Kiel, Schleswig-Holstein, 24105

    Recruiting

  • Universitätsklinikum Münster - Albert Schweitzer Campus

    Münster, North Rhine-Westphalia, 48149

    Recruiting

  • Universitaetsklinikum Ulm

    Ulm, Baden-Wurttemberg, 89081

    Recruiting

  • Universitaetsklinikum Wuerzburg

    Würzburg, Bavaria, 97080

    Recruiting

Other trials in Germany

France11 sites, 11 recruiting

  • Bordeaux University Hospital - Pellegrin

    Bordeaux, Aquitaine, 33076

    Recruiting

  • Hôpital Jeanne de Flandre - CHRU

    Lille, NORD, 59037

    Recruiting

  • Institut d'Hématologie et d'Oncologie Pédiatrique

    Lyon, 69008

    Recruiting

  • CHU de Nantes - Hôpital Mère - Enfants

    Nantes, 44000

    Recruiting

  • Centre Hospitalier Universitaire de Nice - Hôpital l'Archet

    Nice, Alpes-maritimes, 06202

    Recruiting

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

    Paris, Paris, 75935

    Recruiting

  • Hôpital Armand Trousseau

    Paris, 75571

    Recruiting

  • CHRU De Rennes - Hôpital Sud

    Rennes, 35203

    Recruiting

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

    Strasbourg, Alsace, 67000

    Recruiting

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

    Toulouse, Haute-garonne, 31059

    Recruiting

  • Centre Hospitalier Régional Universitaire de Nancy - Hôpitaux de Brabois

    Vandœuvre-lès-Nancy, Meurthe-et-moselle, 54511

    Recruiting

Other trials in France

Italy9 sites, 9 recruiting

  • Policlinico "G. Rodolico"

    Catania, Sicily, 95123

    Recruiting

  • IRCCS Istituto Giannina Gaslini

    Genoa, Liguria, 16147

    Recruiting

  • Fondazione IRCCS San Gerardo dei Tintori

    Monza, Lombardy, 20900

    Recruiting

  • Azienda Ospedaliera di Rilievo Nazional Santobono Pausilipon

    Naples, Campania, 80123

    Recruiting

  • Azienda Ospedale - Università Padova

    Padova, Veneto, 35128

    Recruiting

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100

    Recruiting

  • Ospedale Pediatrico Bambino Gesù IRCCS

    Rome, ROMA, 00165

    Recruiting

  • Ospedale Regina Margherita

    Torino, 10126

    Recruiting

  • IRCCS Materno Infantile Burlo Garofolo

    Trieste, 34137

    Recruiting

Other trials in Italy

Spain9 sites, 9 recruiting

  • Hospital Universitari Vall d'Hebron

    Barcelona, Barcelona [barcelona], 08035

    Recruiting

  • Hospital Clinico Universitario Virgen de la Arrixaca

    El Palmar, Murcia, 30120

    Recruiting

  • Hospital Sant Joan de Déu

    Esplugues de Llobregat, Barcelona [barcelona], 08950

    Recruiting

  • Hospital Infantil Universitario Niño Jesús

    Madrid, Madrid, Comunidad de, 28009

    Recruiting

  • Hospital Universitario La Paz

    Madrid, 28046

    Recruiting

  • CHUS - Hospital Clinico Universitario

    Santiago de Compostela, A Coruña [LA Coruña], 15706

    Recruiting

  • CHUS - Hospital Clinico Universitario

    Santiago de Compostela, 15706

    Recruiting

  • Hospital Universitario Virgen Del Rocio

    Seville, 41013

    Recruiting

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026

    Recruiting

Other trials in Spain

Israel4 sites, 4 recruiting

  • Rambam Health Care Campus

    Haifa, Northern District, 3109601

    Recruiting

  • Schneider Children's Medical Center

    Petah Tikva, Central District, 49202

    Recruiting

  • The Edmond and Lily Safra Children's Hospital; The Chaim Sheba Medical Center

    Ramat Gan, Central District, 5265601

    Recruiting

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

    Tel Aviv, TELL ABĪB, 6423906

    Recruiting

Other trials in Israel

Belgium3 sites, 3 recruiting

  • Cliniques universitaires Saint-Luc

    Brussels, Bruxelles-capitale, Région de, 1200

    Recruiting

  • UZ Gent

    Ghent, Oost-vlaanderen, 9000

    Recruiting

  • UZ Leuven

    Leuven, Vlaams-brabant, 3000

    Recruiting

Other trials in Belgium

Sweden3 sites, 3 recruiting

  • Sahlgrenska Universitetssjukhuset Östra

    Gothenburg, Västra Götalands LÄN [se-14], 416 85

    Recruiting

  • Skånes Universitetssjukhus Lund

    Lund, Skåne LÄN [se-12], 22185

    Recruiting

  • Astrid Lindgrens Barnsjukhus

    Stockholm, 17067

    Recruiting

Other trials in Sweden

Switzerland3 sites, 3 recruiting

  • Inselspital Bern

    Bern, Canton of Bern, 3010

    Recruiting

  • CHUV (centre hospitalier universitaire vaudois)

    Lausanne, Canton of Vaud, 1011

    Recruiting

  • Kinderspital Zürich

    Zurich, 8008

    Recruiting

Other trials in Switzerland

Czechia2 sites, 2 recruiting

  • Detska nemocnice FN Brno

    Brno, Brno-město, 613 00

    Recruiting

  • Fakultni Nemocnice Motol a Homolka

    Prague, 150 00

    Recruiting

Other trials in Czechia

Norway2 sites, 2 recruiting

  • Oslo Universitetssykehus Rikshospitalet

    Oslo, 0372

    Recruiting

  • Radium Hospital

    Oslo, 0379

    Recruiting

Other trials in Norway

Austria1 site, 1 recruiting

  • St. Anna Kinderspital

    Vienna, 1090

    Recruiting

Other trials in Austria

Denmark1 site, 1 recruiting

  • Rigshospitalet

    Copenhagen, Capital Region, DK-2100

    Recruiting

Other trials in Denmark

Finland1 site, 1 recruiting

  • Helsinki university hospital

    Helsinki, 00290

    Recruiting

Other trials in Finland

Netherlands1 site, 1 recruiting

  • Prinses Maxima Centrum voor Kinderoncologie

    Utrecht, 3584 CS

    Recruiting

Other trials in Netherlands

Slovakia1 site, 1 recruiting

  • Narodny ustav detskych chorob

    Bratislava, Bratislava Region, 833 40

    Recruiting

Other trials in Slovakia

About this study

Official title: A PROSPECTIVE, RANDOMIZED, OPEN-LABEL PHASE 2 STUDY TO EVALUATE THE SUPERIORITY OF INOTUZUMAB OZOGAMICIN MONOTHERAPY VERSUS ALLR3 FOR INDUCTION TREATMENT OF CHILDHOOD HIGH-RISK OR VERY HIGH-RISK FIRST RELAPSE B-CELL PRECURSOR ACUTE LYMPHOBLASTIC LEUKAEMIA

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.

Read the detailed description

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.

Being tested
Inotuzumab ozogamicin, ALLR3
Keywords
ALL, BCP ALL, High risk BCP ALL, Relapse ALL, Leukemia, Very high risk BCP ALL
Medical subject headings
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Inotuzumab ozogamicinDrug

    Also called Besponsa

    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.

  • ALLR3Drug

    Also called R3

    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)

Study groups in NCT05748171
Study groupRoleWhat they receive
Inotuzumab ozogamicinExperimentalEach 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
Receives: Drug: Inotuzumab ozogamicin
ALLR3Active comparatorMitoxantrone 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
Receives: Drug: ALLR3

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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)

    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

    Measured: After 1 treatment cycle: Day 28 +/- 2 days

Secondary outcomes (9)

  1. Event Free Survival (EFS)

    EFS will be summarized using Kaplan-Meier methods and displayed graphically by treatment arm.

    Measured: 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

  2. Duration of Response (DoR) for Participants Who Achieved CR/CRp/CRi

    DoR will be summarized using Kaplan-Meier methods.

    Measured: 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

  3. Rate of hematopoietic stem cell transplantation (HSCT)

    HSCT rate will be summarized by descriptive analyses (ie, percentage of participants who underwent HSCT after treatment).

    Measured: Up to 5 years from randomization

  4. Overall Survival (OS)

    OS will be summarized by treatment arm using Kaplan-Meier methods.

    Measured: From start of treatment to date of death due to any cause: up to 5 years from randomization

  5. Number of participants reporting an Adverse Event (AE)

    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.

    Measured: From time of informed consent up to a minimum of 60 calendar days after the last dose of study drug.

  6. Pharmacokinetics (PK) parameter: InO Cmax

    Descriptive summary statistics will be provided for InO serum concentrations at scheduled visits.

    Measured: 1 treatment cycle: 28 days

  7. Number of Adverse Events (AE) reported by severity

    AEs will be graded by the investigator according to the CTCAE (Common Terminology Criteria for Adverse Events) version 4.03.

    Measured: From time of informed consent up to a minimum of 60 calendar days after the last dose of study drug.

  8. Pharmacokinetics (PK) parameter: InO trough levels

    Descriptive summary statistics will be provided for InO serum concentrations at scheduled visits.

    Measured: 1 treatment cycle: 28 days

  9. Rate of Chimeric antigen receptor (CAR) T-cell therapy

    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).

    Measured: Up to 5 years from randomisation

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Feb 2023
  2. Study start17 May 2023
  3. Primary completion21 May 2033 (estimated)
  4. Study completion25 May 2036 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
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
Masking
None (open label)
Enrolment
100 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05748171
Sponsor's protocol ID
B1931036
Other IDs
2023-509810-13-00, 2023
Status last verified
Sep 2026
First submitted
20 Jan 2023
Last updated
24 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: 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.

Publications

  • Davis KL, Yao CC, Zimmerman JAO, Rau RE. Immunotherapy in B-Cell Acute Lymphoblastic Leukemia. J Natl Compr Canc Netw. 2025 Dec;23(12):e257067. doi: 10.6004/jnccn.2025.7067.

    PubMed 41671463

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Inotuzumab ozogamicin and ALLR3 in people with ACUTE LYMPHOBLASTIC LEUKEMIA, run by Pfizer at 65 sites in 15 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 24 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 65 sites in Germany, France, Italy, Spain, Israel and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Pfizer CT.gov Call Center (1-800-718-1021, ClinicalTrials.gov_Inquiries@pfizer.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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) (time frame: After 1 treatment cycle: Day 28 +/- 2 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05748171, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2026.

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