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Acute Lymphoblastic LeukemiaPhase 2NCT03913559

Inotuzumab Ozogamicin for Children With MRD Positive CD22+ Lymphoblastic Leukemia

A Phase 2 study testing Inotuzumab ozogamicin, Methotrexate and Hydrocortisone in people with Acute Lymphoblastic Leukemia, run by St. Jude Children's Research Hospital at 2 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This trial is a limited multi-center, Phase II study to evaluate inotuzumab ozogamicin (Besponsa) in pediatric patients with MRD positive CD22-positive B-lymphoblastic leukemia (B-ALL). Read more about this study

Who is it for?
Ages Up to 21 years
Conditions the study is for
Acute Lymphoblastic Leukemia
What is being tested?
Inotuzumab ozogamicin, Methotrexate and Hydrocortisone
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
5 participants
Where
2 sitesUnited States
Study dates
14 May 2019to 15 Sep 2025
Phase
Phase 2
Are there results?
None posted in the registry

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 Up to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age
  • Participants must be < 22 years of age.
  • Diagnosis
  • Participants must have B-ALL with persistent or rising MRD between 0.1 and 4.99% without extramedullary disease following at least two prior induction attempts, relapse or after hematopoietic stem cell transplant
  • Leukemia blasts demonstrating surface expression of CD22
  • Performance Level
Show all 23Show fewer
  • Karnofsky or Lansky performance score ≥ 50% (corresponding to ECOG Score of ≥ 2). The Lansky performance score should be used for participants < 16 years and the Karnofsky performance score for participants ≥ 16 years.
  • Prior Therapy
  • Patients must have fully recovered from the acute toxic effects of all prior anticancer therapy, defined as resolution of all such toxicities to ≤ Grade 2 or lower per the inclusion/exclusion criteria prior to entering this study.
  • At least 14 days must have elapsed since the completion of cytotoxic therapy, with the exception of standard maintenance therapy and steroids.
  • At least 7 days must have elapsed since completion of therapy with a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period prior to enrollment must be extended beyond the time during which adverse events are known to occur.
  • At least 3 half-lives must have elapsed since prior therapy that included a monoclonal antibody with the exception of blinatumomab. Patients must have been off blinatumomab infusion for at least 7 days and all drug related toxicity must have resolved to Grade 2 or lower as outlined in the inclusion/exclusion criteria.
  • At least 42 days must have elapsed since CAR-T cell therapy.
  • Participant has received ≤ 1 prior bone marrow transplant.
  • At least 90 days have elapsed since bone marrow transplant and participant is off immune suppression for ≥ 2 weeks, if applicable with no evidence of active GVHD.
  • At least 2 weeks must have elapsed since local XRT (small port); ≥ 3 months must have elapsed if prior cranial or craniospinal XRT was received, if ≥ 50% of the pelvis was irradiated, or if TBI was received; ≥ 6 weeks must have elapsed if other substantial bone marrow irradiation was given.
  • Organ Function Requirements
  • Adequate renal function defined as glomerular filtration rate ≥ 60 cc/min/1.73m2 or serum creatinine based on age as follows:
  • Age: <6 months; maximum serum creatinine (mg/dL): 0.4 (male, female); Age: 6 months to <1 year; maximum serum creatinine (mg/dL): 0.5 (male, female); Age: 1 to < 2 years; maximum serum creatinine (mg/dL): 0.6 (male, female); Age: 2 to < 6 years; maximum serum creatinine (mg/dL): 0.8 (male, female); Age: 6 to <10 years; maximum serum creatinine (mg/ dL): 1 (male, female); Age: 10 to <13 years; maximum serum creatinine (mg/dL): 1.2 (male, female); Age: 13 to <16 years; maximum serum creatinine (mg/dL): 1.5 (male), 1.4 (female); Age: ≥ 16 years; maximum serum creatinine (mg/dL): 1.7 (male), 1.4 (female)
  • Adequate hepatic function defined as:
  • Direct bilirubin ≤ 1.4 mg/dL (if total bilirubin > 1.4 mg/dL) and
  • AST or ALT ≤ 3 x ULN for age.
  • Adequate cardiac function defined as shortening fraction of ≥ 27% or ejection fraction ≥ 45%.

Who cannot join

  • History of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) of any severity.
  • Concurrent chemotherapy or targeted anti-cancer agents, other than intrathecal therapy.
  • Patient with concurrent severe and/or uncontrolled medical conditions that, in the opinion of the investigator, may impair participation in the study or the evaluation of safety and/or efficacy.
  • Known HIV infection or active hepatitis B (defined as hepatitis B surface antigen-positive) or C (defined as hepatitis C antibody-positive).
  • Pregnant or lactating (female participant of childbearing potential must have negative serum or urine pregnancy test required within 7 days prior to start of treatment).
  • Male or female participant of reproductive potential must agree to use appropriate methods of contraception for the duration of study treatment and for at least 30 days after last dose of protocol treatment.
  • Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105

  • Rady Children's Hospital San Diego

    San Diego, California, 92123

Other trials in United States

About this study

This trial is a limited multi-center, Phase II study to evaluate inotuzumab ozogamicin (Besponsa) in pediatric patients with MRD positive CD22-positive B-lymphoblastic leukemia (B-ALL).

Some patients with newly diagnosed ALL maintain low levels of MRD, despite achieving complete remission with less than 5% blasts in the bone marrow. Others experience re-emergence of low level MRD or increasing levels of MRD on therapy or post-transplant. New approaches are needed to achieve undetectable MRD in these high-risk patients.

Read more

Inotuzumab ozogamicin is an antibody-drug conjugate composed of a humanized IgG subtype 4 monoclonal CD22-targeted antibody linked to calicheamicin, a potent anti-tumor antibiotic. CD22 is expressed in more than 90% of patients with B-cell ALL, making it an attractive target in this patient population. Inotuzumab ozogamicin has demonstrated exceptional activity in adults with relapsed or refractory B-ALL.

Primary Objective

  • Assess the efficacy of inotuzumab ozogamicin in patients with MRD positive CD22+ B-ALL with 0.1 - 4.99% blasts in bone marrow.

Secondary Objectives

  • Study the safety of inotuzumab ozogamicin when used in patients with MRD - positive CD22+ B-ALL with < 5 % blasts in bone marrow.
  • Estimate the incidence, severity, and outcome of hepatotoxicity and sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) in patients during inotuzumab ozogamicin and following subsequent treatment, including hematopoietic stem cell transplant (HSCT).
Read the detailed description

The drug will be administered intravenously on days 1, 8, and 15 of each 28-day cycle. Patients who do not meet the definition of treatment failure after the first cycle may receive up to five additional cycles of therapy. .

After completion of study treatment, patients are followed for 1 year.

Being tested
Inotuzumab ozogamicin, Methotrexate, Hydrocortisone, Cytarabine, Diphenhydramine
Keywords
B-cell Acute Lymphoblastic Leukemia
Medical subject headings
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Burkitt Lymphoma

What participants receive

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

  • Inotuzumab ozogamicinDrug

    Also called Besponsa, CMC-544

    dose: 0.5 mg/m2 IV over 60 minutes, days: 1, 8 and 15 every 4 weeks (28 days per cycle) for up to 6 cycles.

  • MethotrexateDrug

    Also called Trexall®

    Intrathecal (IT) therapy

  • HydrocortisoneDrug

    Also called Cortisol

    Intrathecal (IT) therapy

  • CytarabineDrug

    Also called Ara-C

    Intrathecal (IT) therapy

  • DiphenhydramineDrug

    Also called Benadryl

    1 mg/kg (max 50 mg) IV

  • AcetaminophenDrug

    Also called Tylenol

    10 mg/kg (max 650 mg) PO x 1

  • MethylprednisoloneDrug

    Also called Solu-Medrol

    1 mg/kg IV x 1

Study groups in NCT03913559
Study groupRoleWhat they receive
Inotuzumab ozogamicinExperimentalExperimental:Inotuzumab Ozogamicin (InO) Patients with B cell acute lymphoblastic leukemia (B-ALL) that is showing early signs of relapsing (coming back) or is not responding to treatment (refractory). Interventions:methotrexate, hydrocortisone and cytarabine into the central nervous system (called triple intrathecal chemotherapy or IT chemotherapy) during this study. Premedication: diphenhydramine, acetaminophen and methylprednisolone
Receives: Drug: Inotuzumab ozogamicin; Drug: Methotrexate; Drug: Hydrocortisone; Drug: Cytarabine; Drug: Diphenhydramine; Drug: Acetaminophen; Drug: Methylprednisolone

What is being measured

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

  1. Treatment response Cycle1 - count

    Number of patients that reach MRD negative at the end of cycle 1

    Measured: At the end of cycle 1 (each cycle is 28 days)

  2. Treatment Response Cycle 1 - percentage

    Percentage of patients that reach MRD negative at the end of cycle 1

    Measured: At the end of cycle 1 (each cycle is 28 days)

  3. Treatment Response Cycle 2 - count

    Number of patients that reach MRD negative at the end of cycle 2

    Measured: At the end of cycle 2 (each cycle is 28 days)

  4. Treatment Response Cycle 2 - percentage

    Percentage of patients that reach MRD negative at the end of cycle 2

    Measured: At the end of cycle 2 (each cycle is 28 days)

Secondary outcomes (4)

  1. Occurrence of death - count

    Number of patient deaths that occur at any time during observation on study

    Measured: up to 30 days from last dose of inotuzumab ozogamicin or up to 30 days post-transplant among the patients who proceed to transplant

  2. Occurrence of death - percentage

    Percentage of patient deaths that occur at any time during observation on study

    Measured: up to 30 days from last dose of inotuzumab ozogamicin or up to 30 days post-transplant among the patients who proceed to transplant

  3. Occurrence of Veno-occlusive disease (VOD) - count

    Number of patients that develop VOD at any time during observation on study

    Measured: up to 30 days from last dose of inotuzumab ozogamicin or up to 30 days post-transplant among the patients who proceed to transplant

  4. Occurrence of Veno-occlusive disease (VOD) - percentage

    Percentage of patients that develop VOD at any time during observation on study

    Measured: up to 30 days from last dose of inotuzumab ozogamicin or up to 30 days post-transplant among the patients who proceed to transplant

Timeline

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

  1. First posted12 Apr 2019
  2. Study start14 May 2019
  3. Primary completion15 Sep 2025
  4. Study completion15 Sep 2025

Why it stopped: Closed due to slow accrual

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
5 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03913559
Sponsor's protocol ID
INOMRD
Other IDs
NCI-2019-01062
Status last verified
Feb 2026
First submitted
9 Apr 2019
Last updated
12 Feb 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.

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

  • Shi Z, Zhu Y, Zhang J, Chen B. Monoclonal antibodies: new chance in the management of B-cell acute lymphoblastic leukemia. Hematology. 2022 Dec;27(1):642-652. doi: 10.1080/16078454.2022.2074704.

    PubMed 35622074

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Inotuzumab ozogamicin, Methotrexate and Hydrocortisone in people with Acute Lymphoblastic Leukemia, run by St. Jude Children's Research Hospital at 2 sites in United States.

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 23 inclusion and 7 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: stopped early, as of the sponsor's last update on 12 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. 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?

The study was stopped early (reason given: Closed due to slow accrual). Results are not available in the source record.

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

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Treatment response Cycle1 - count (time frame: At the end of cycle 1 (each cycle is 28 days)). The study lists 4 primary outcomes in all. 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 NCT03913559, which also lists 2 linked publications.

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 12 Feb 2026.

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