Evaluating Inotuzumab Ozogamicin for Children with Relapsed Acute Lymphoblastic Leukemia
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
Run by Pfizer · NCT05748171 · Data updated 2026-11-12
This study is testing if a new drug called Inotuzumab Ozogamicin can help children with relapsed acute lymphoblastic leukemia do better than the usual treatment.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Ages | 1 Year to 17 Years |
| Sex | All |
| Enrollment | 100 (estimated) |
| Sponsor | Pfizer Industry-sponsored |
| Drugs / interventions | gemtuzumab, CAR T |
| Locations | 65 sites (Vienna and 64 other locations) |
| Status | Recruiting |
| Started | May 17, 2023 |
| Main results expected | May 21, 2033 |
| Study ends | May 25, 2036 |
| Registry record updated | September 24, 2026 |
| Trial ID | NCT05748171 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are children aged 1 to under 18 years with a confirmed diagnosis of first relapse high-risk CD22-positive BCP ALL.
✕Probably not a fit
Patients with very high-risk genetic abnormalities or those who do not meet the specific inclusion criteria for the trial may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This Phase 2 clinical trial aims to compare the effectiveness of Inotuzumab Ozogamicin (InO) monotherapy against standard ALLR3 treatment in children aged 1 to under 18 years who have experienced a first relapse of high-risk CD22-positive acute lymphoblastic leukemia (ALL). The study will involve approximately 100 participants who will be randomized to receive either treatment after one cycle of induction therapy. The trial will assess the safety, tolerability, pharmacokinetics, and long-term efficacy of InO, with follow-up continuing for up to five years post-randomization. The primary focus is on evaluating the superiority of InO in achieving better treatment outcomes compared to standard therapy.
Why it matters
Potential benefit: If successful, this treatment could provide a more effective option for children with relapsed acute lymphoblastic leukemia, potentially improving survival rates.
How similar studies have gone: Other studies have shown promising results with similar therapies, indicating potential for success in this approach.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
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St. Anna Kinderspital
Vienna, Austria Recruiting -
Cliniques universitaires Saint-Luc
Brussels, Bruxelles-capitale, Région de, Belgium Recruiting -
UZ Gent
Ghent, Oost-vlaanderen, Belgium Recruiting -
UZ Leuven
Leuven, Vlaams-brabant, Belgium Recruiting -
Detska nemocnice FN Brno
Brno, Brno-město, Czechia Recruiting -
Fakultni Nemocnice Motol a Homolka
Prague, Czechia Recruiting
Show 44 more locations
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Rigshospitalet
Copenhagen, Capital Region, Denmark Recruiting -
Helsinki university hospital
Helsinki, Finland Recruiting -
Centre Hospitalier Universitaire de Nice - Hôpital l'Archet
Nice, Alpes-maritimes, France Recruiting -
CHU Strasbourg-Hautepierre, Service d'hematologie oncologie pediatrique, pediatrie 3
Strasbourg, Alsace, France Recruiting -
Bordeaux University Hospital - Pellegrin
Bordeaux, Aquitaine, France Recruiting -
CHU de Toulouse - Hôpital des Enfants - Hemato-Immuno-Oncologie
Toulouse, Haute-garonne, France Recruiting -
Centre Hospitalier Régional Universitaire de Nancy - Hôpitaux de Brabois
Vandœuvre-lès-Nancy, Meurthe-et-moselle, France Recruiting -
Hôpital Jeanne de Flandre - CHRU
Lille, Nord, France Recruiting -
Assistance Publique - Hopitaux de Paris (AP-HP) - Hopital Robert Debre - Centre Hospitalo Universita
Paris, Paris, France Recruiting -
Institut d'Hématologie et d'Oncologie Pédiatrique
Lyon, France Recruiting -
CHU de Nantes - Hôpital Mère - Enfants
Nantes, France Recruiting -
Hôpital Armand Trousseau
Paris, France Recruiting -
CHRU De Rennes - Hôpital Sud
Rennes, France Recruiting -
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, Germany Recruiting -
Universitaetsklinikum Ulm
Ulm, Baden-Wurttemberg, Germany Recruiting -
Universitaetsklinikum Wuerzburg
Würzburg, Bavaria, Germany Recruiting -
Universitätsklinikum Frankfurt Goethe-Universität
Frankfurt am Main, Hesse, Germany Recruiting -
Medizinische Hochschule Hannover
Hanover, Lower Saxony, Germany Recruiting -
Universitaetsklinikum Essen
Essen, North Rhine-Westphalia, Germany Recruiting -
Universitätsklinikum Münster - Albert Schweitzer Campus
Münster, North Rhine-Westphalia, Germany Recruiting -
Universitaetsklinikum Schleswig-Holstein Campus Kiel
Kiel, Schleswig-Holstein, Germany Recruiting -
Charité Campus Virchow-Klinikum
Berlin, Germany Recruiting -
Universitaetsklinikum Duesseldorf
Düsseldorf, Germany Recruiting -
Universitätsklinikum Gießen
Giessen, Germany Recruiting -
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, Germany Recruiting -
Medizinische Hochschule Hannover
Hanover, Germany Recruiting -
Universitätsklinikum Jena
Jena, Germany Recruiting -
Schneider Children's Medical Center
Petah Tikva, Central District, Israel Recruiting -
The Edmond and Lily Safra Children's Hospital; The Chaim Sheba Medical Center
Ramat Gan, Central District, Israel Recruiting -
Rambam Health Care Campus
Haifa, Northern District, Israel Recruiting -
Tel-Aviv Sourasky Medical Center Dana-Dwek Children's Hospital
Tel Aviv, Tell Abīb, Israel Recruiting -
Azienda Ospedaliera di Rilievo Nazional Santobono Pausilipon
Naples, Campania, Italy Recruiting -
IRCCS Istituto Giannina Gaslini
Genoa, Liguria, Italy Recruiting -
Fondazione IRCCS San Gerardo dei Tintori
Monza, Lombardy, Italy Recruiting -
Ospedale Pediatrico Bambino Gesù IRCCS
Rome, Roma, Italy Recruiting -
Policlinico "G. Rodolico"
Catania, Sicily, Italy Recruiting -
Azienda Ospedale - Università Padova
Padova, Veneto, Italy Recruiting -
Fondazione IRCCS Policlinico San Matteo
Pavia, Italy Recruiting -
Ospedale Regina Margherita
Torino, Italy Recruiting -
IRCCS Materno Infantile Burlo Garofolo
Trieste, Italy Recruiting -
Prinses Maxima Centrum voor Kinderoncologie
Utrecht, Netherlands Recruiting -
Oslo Universitetssykehus Rikshospitalet
Oslo, Norway Recruiting -
Radium Hospital
Oslo, Norway Recruiting -
Narodny ustav detskych chorob
Bratislava, Bratislava Region, Slovakia Recruiting
+15 more sites. See ClinicalTrials.gov for the full list.
Study contacts
- Study coordinator: Pfizer CT.gov Call Center
- Email: ClinicalTrials.gov_Inquiries@pfizer.com
- Phone: 1-800-718-1021
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.