Inotuzumab Ozogamicin for Acute Lymphoblastic Leukemia

Recruiting at 1 trial location

Overseen By Sima C. Jeha

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: St. Jude Children's Research Hospital

Must not be taking: Chemotherapy, Targeted agents

Disqualifiers: SOS/VOD, HIV, Hepatitis B/C, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: up to 24 weeks

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests inotuzumab ozogamicin, a type of targeted therapy, for children and young adults with B-cell acute lymphoblastic leukemia (B-ALL). It focuses on those with residual cancer cells despite treatment. The aim is to determine if this drug can reduce or eliminate these remaining cells. Participants should have B-ALL that hasn't fully responded to at least two treatments or is relapsing, and their leukemia cells must display a specific marker called CD22. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group.

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if you need to stop your current medications, but it does mention that you must have recovered from the effects of previous cancer treatments. You may need to wait a certain period after your last treatment before joining the trial, depending on the type of therapy you received.

Is there any evidence suggesting that inotuzumab ozogamicin is likely to be safe for humans?

Research has shown that inotuzumab ozogamicin, also known as Besponsa, has been studied for safety in patients with B-cell precursor acute lymphoblastic leukemia (ALL), particularly for those whose cancer has not responded to treatment or has returned. Some studies have found that patients can tolerate this treatment well, though it carries certain risks.

The most common serious side effects include infections and fever. There is also a warning about a liver condition called veno-occlusive disease (VOD), which can occur, especially after a stem cell transplant. Despite these risks, many patients have managed the treatment well.

Inotuzumab ozogamicin is already approved for use in both adults and children with certain types of leukemia. This approval indicates that it has been tested for safety and effectiveness in other studies, demonstrating its safe use under medical supervision. However, those considering joining a trial should discuss the potential risks and benefits with a healthcare professional. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Inotuzumab Ozogamicin is unique because it specifically targets B cell acute lymphoblastic leukemia (B-ALL), which is a subtype of leukemia that can be stubborn against traditional treatments like chemotherapy. Unlike standard chemotherapy that attacks rapidly dividing cells in general, Inotuzumab Ozogamicin is an antibody-drug conjugate. This means it combines an antibody that precisely homes in on cancer cells with a potent anti-cancer drug, delivering a powerful punch directly to the leukemia cells. Researchers are excited about this treatment because it offers a more targeted approach, potentially resulting in fewer side effects and more effective results for patients whose cancer has relapsed or is resistant to other treatments.

What evidence suggests that inotuzumab ozogamicin might be an effective treatment for acute lymphoblastic leukemia?

Research has shown that inotuzumab ozogamicin, the treatment under study in this trial, may effectively treat acute lymphoblastic leukemia (ALL), particularly in cases where the disease returns or resists other treatments. Studies with adults who have relapsed or hard-to-treat ALL found that this treatment can lead to complete remission for many patients. Inotuzumab ozogamicin targets a protein called CD22, present in over 90% of B-cell ALL cases, making it a strong option for this condition. The drug uses a special antibody to deliver a powerful anti-cancer medicine directly to leukemia cells. This targeted method demonstrated significant success in previous adult trials, and researchers hope it will also benefit children with similar conditions. ^{3 6 7 8 9}

Who Is on the Research Team?

Sima Jeha, MD

Principal Investigator

St. Jude Children's Research Hospital

Are You a Good Fit for This Trial?

This trial is for children under 22 with B-lymphoblastic leukemia showing CD22 expression and persistent or rising MRD between 0.1% and 4.99%. They must have tried at least two prior treatments, may have had one bone marrow transplant, need proper liver and kidney function, no severe concurrent conditions, not pregnant or breastfeeding, and agree to use contraception.

Inclusion Criteria

My kidneys work well enough for my age.

My leukemia cells show CD22 on their surface.

It has been more than 42 days since my CAR-T cell therapy.

Exclusion Criteria

I agree to use birth control during and for 30 days after the study.

Inability or unwillingness of research participant or legal guardian/representative to give written informed consent

I have had a condition where small veins in my liver were blocked.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive inotuzumab ozogamicin intravenously on days 1, 8, and 15 of each 28-day cycle, with up to six cycles possible

up to 24 weeks

3 visits per cycle (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

1 year

What Are the Treatments Tested in This Trial?

Interventions

• Inotuzumab Ozogamicin

Trial Overview The study tests inotuzumab ozogamicin (Besponsa) effectiveness in pediatric patients with MRD positive CD22-positive B-ALL who still have a small number of leukemia cells after treatment. It aims to see if this drug can help achieve undetectable levels of these cells.

How Is the Trial Designed?

Group I: Inotuzumab ozogamicin Experimental Treatment 7 Interventions

Experimental: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

Inotuzumab Ozogamicin is already approved in European Union, United States for the following indications:

Approved in European Union as Besponsa for:

• Relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)

Approved in United States as Besponsa for:

• Relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL) in adult and pediatric patients 1 year and older

Who Is Running the Clinical Trial?

St. Jude Children's Research Hospital

Lead Sponsor

Trials

451

Recruited

5,326,000+

Pfizer

Industry Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Citations

1. besponsa.pfizerpro.com besponsa.pfizerpro.com/efficacy/adult-clinical-data%E2%80%94ino-vate

BESPONSA® (inotuzumab ozogamicin) CR/CRi | Safety Info Efficacy and safety analysis by age cohort of inotuzumab ozogamicin in patients with relapsed or refractory acute lymphoblastic leukemia enrolled in INO-VATE.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12533514/

Inotuzumab ozogamicin in adult acute lymphoblastic leukemia Outcomes have been historically poor in adults with relapsed/refractory (R/R) B-cell ALL, with a 5-year overall survival (OS) rate of <10% with ...

3. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/1422/531484/Safety-and-Efficacy-Results-of-Dose-Adjusted

Safety and Efficacy Results of Dose-Adjusted Inotuzumab ... The results of this study indicate that dose-adjusted InO is a well-tolerated and effective treatment option for B-ALL adults with or without chemotherapy or ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT01564784

NCT01564784 | A Study Of Inotuzumab Ozogamicin ... This study will compare the efficacy, in terms of complete responses and overall survival, of inotuzumab ozogamicin versus investigator's choice of chemotherapy ...

5. besponsa.pfizerpro.com besponsa.pfizerpro.com/efficacy/adult-clinical-trial

BESPONSA® (inotuzumab ozogamicin) Clinical Trial Learn about INO-VATE ALL, a Phase 3 clinical trial of BESPONSA® in adult patients with Ph- or Ph+ relapsed or refractory ALL. See risks & benefits.

6. besponsa.pfizerpro.com besponsa.pfizerpro.com/safety/adult-safety-data

BESPONSA® (inotuzumab ozogamicin) Safety Information Adult Safety Data · BESPONSA has a BOXED WARNING for VOD and post-HSCT NRM · The most common (≥2%) serious adverse reactions (ARs) were infection, febrile ...

7. ema.europa.eu ema.europa.eu/en/documents/product-information/besponsa-epar-product-information_en.pdf

Besponsa, INN-inotuzumab ozogamicin Adverse reactions included treatment-emergent, all-causality events that commenced on, or after Cycle 1 Day 1 within 42 days after the final dose of BESPONSA, ...

8. aetna.com aetna.com/cpb/medical/data/900_999/0919.html?ref=counterforcehealth.org

Inotuzumab Ozogamicin (Besponsa) - Medical Clinical ... Besponsa is indicated for the treatment relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL) in adult and pediatric patients ...

9. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40120068/

INO-CD22: A multicenter, real-world study of inotuzumab ... Conclusions: Despite the limitations of retrospective studies, the INO-CD22 study highlights the favorable safety profile and clinical activity ...