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Evaluating Inotuzumab Ozogamicin for Children with Relapsed Acute Lymphoblastic Leukemia

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 FIRST RELAPSE B-CELL PRECURSOR ACUTE LYMPHOBLASTIC LEUKAEMIA

PHASE2 · Pfizer · NCT05748171

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

PhasePHASE2
Study typeInterventional
Enrollment100 (estimated)
Ages1 Year to 17 Years
SexAll
SponsorPfizer (industry)
Drugs / interventionsgemtuzumab, CAR T
Locations76 sites (Vienna and 75 other locations)
Trial IDNCT05748171 on ClinicalTrials.gov

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.

Who should consider this trial

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.

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.

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 performed: Other studies have shown promising results with similar therapies, indicating potential for success in this approach.

Eligibility criteria

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 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.

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 this trial is running

Vienna and 75 other locations

+26 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: ACUTE LYMPHOBLASTIC LEUKEMIA, ALL, BCP ALL, High risk BCP ALL, Relapse ALL, Leukemia

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.