What side effects are associated with this type of intervention?

Common treatments for Hairy Cell Leukemia, such as Rituximab/Ruxience and Moxetumomab pasudotox-tdfk, have several known side effects. Rituximab/Ruxience can cause infusion-related reactions (fever, chills, low blood pressure), infections, low blood cell counts, and cardiac issues. Moxetumomab pasudotox-tdfk can lead to capillary leak syndrome, hemolytic uremic syndrome, and infusion-related reactions like fever and hypotension. Both treatments require careful monitoring for severe and potentially life-threatening toxicities.

What prior FDA approvals exist?

For the treatment of Hairy Cell Leukemia (HCL), the FDA has approved several drugs. Rituximab, an antibody that binds to CD20 on cancerous B cells, is commonly used in HCL treatment. Moxetumomab pasudotox-tdfk, a recombinant immunotoxin targeting CD22, has also been approved for HCL, providing targeted cell killing. These treatments are part of a broader strategy to manage HCL by targeting specific proteins on the surface of cancerous white blood cells.

What other types of research exist?

Promising novel alternatives for Hairy Cell Leukemia treatment in 2024 include: 1) Venetoclax, a BCL-2 inhibitor that has shown efficacy in other B-cell malignancies; 2) Ibrutinib, a BTK inhibitor with potential benefits in B-cell cancers; 3) CAR-T cell therapies targeting CD19 or other specific antigens on HCL cells. These therapies represent advancements in targeted and immunotherapy approaches, offering new options for patients.

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Immunotoxin

Moxetumomab + Rituximab/Ruxience for Hairy Cell Leukemia

Q: What is the mechanism of action of this treatment?

Hairy Cell Leukemia (HCL) treatments often include Rituximab/Ruxience and Moxetumomab pasudotox-tdfk. Rituximab/Ruxience are monoclonal antibodies that bind to the CD20 protein on the surface of cancerous B-cells, marking them for destruction by the immune system. Moxetumomab pasudotox-tdfk is an immunotoxin that targets CD22 on B-cells, delivering a toxin that kills the cancer cells. These mechanisms are crucial for HCL patients as they specifically target and eliminate malignant cells, potentially leading to better treatment outcomes and fewer side effects compared to non-targeted therapies.

Phase 1

Waitlist Available

Led By Robert J Kreitman, M.D.

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age greater than or equal to 18 years as the disease under study, HCL/HCLv, has not been reported in children < age 18.

ECOG performance status less than or equal to2 (Karnofsky greater than or equal to 60%)

Must not have

Patients with a history of both thromboembolism and known congenital hypercoagulable conditions.

History of an allogeneic bone marrow transplant.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group.

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the safety of combining two drugs, Rituximab/Ruxience and Moxetumomab pasudotox, to treat hairy cell leukemia or a variant of it. Eligible participants must be 18 or older and the cancer must not have responded to standard therapy.

Who is the study for?

Adults over 18 with Hairy Cell Leukemia (HCL) or its variant, who haven't responded to standard therapy. They must have certain blood count levels, be Pseudomonas-immunotoxin naive, and have adequate organ function. Participants should not be pregnant or breastfeeding and must use effective contraception. Those with a history of certain medical conditions like heart disease are excluded.

What is being tested?

The trial is testing the safety of combining Moxetumomab pasudotox-tdfk (Lumoxiti) with Rituximab/Ruxience in patients with relapsed HCL. The treatment involves up to eight cycles of these drugs administered intravenously, along with medications to prevent allergic reactions and instructions for hydration and aspirin intake.

What are the potential side effects?

Potential side effects may include allergic reactions related to infusion, increased risk of infections due to immune system suppression by the drugs, possible liver enzyme elevations indicating liver stress or damage, fatigue from anemia caused by bone marrow suppression, and other non-specified drug-related effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 years old or older.

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I can take care of myself but might not be able to do heavy physical work.

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I have been diagnosed with hairy cell leukemia.

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I need treatment for low blood counts or symptoms from an enlarged spleen or HCL mass.

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My kidney function is within the required range.

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I have HCL or HCLv and have been treated with a purine analog. If I have HCL, I've also had another therapy if my first treatment worked for more than 2 years and 1 month.

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My kidney function is normal based on creatinine levels or clearance.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a history of blood clots and a genetic condition that makes my blood clot more easily.

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I have had a bone marrow transplant from another person.

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I have a history of blood clotting disorders.

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I am not pregnant or breastfeeding during the study.

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I do not have brain metastases.

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I do not have any serious ongoing illnesses that would stop me from following the study's requirements.

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I have a detached retina or choroid.

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I do not have an active cancer needing treatment, except for minor skin cancers.

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I have not had radioimmunotherapy in the last 2 years.

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I am taking more than 0.625 mg/day of estrogen.

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I do not have severe blood clotting issues.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group.

This trial's timeline: 3 weeks for screening, Varies for treatment, and date treatment consent signed to date off study, approximately 11 months and 13 days for the first group, and 4 months and 3 days for the second group. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Recommended Safe Dose of Moxetumomab Pasudotox-tdfk

Recommended Safe Dose of Rituximab/Ruxience

Secondary study objectives

Duration of Response (DOR)

Number of Participants Who Are Minimal Residual Disease (MRD)-Free

Number of Participants Whose Cancer Shrinks or Disappears After Treatment

Other study objectives

Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)

Number of Participants With a Dose-limiting Toxicity (DLT)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Moxetumomab - Dose Expansion 40 mcg/kgExperimental Treatment7 Interventions

Arm 1 and Arm 2 Moxetumomab Pasudotox-tdfk + Ruxience

Group II: Moxetumomab - Dose Escalation 30 mcg/kgExperimental Treatment7 Interventions

Arm 1 Moxetumomab Pasudotox-tdfk + Rituximab

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Famotidine

2005

Completed Phase 4

~1220

Diphenhydramine

2002

Completed Phase 4

~1210

Rituximab

1999

Completed Phase 4

~2990

Dexamethasone

2007

Completed Phase 4

~2650

Acetaminophen

2017

Completed Phase 4

~2030

Aspirin

2014

Completed Phase 4

~55580

0 / 18Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Hairy Cell Leukemia (HCL) treatments often include Rituximab/Ruxience and Moxetumomab pasudotox-tdfk. Rituximab/Ruxience are monoclonal antibodies that bind to the CD20 protein on the surface of cancerous B-cells, marking them for destruction by the immune system. Moxetumomab pasudotox-tdfk is an immunotoxin that targets CD22 on B-cells, delivering a toxin that kills the cancer cells. These mechanisms are crucial for HCL patients as they specifically target and eliminate malignant cells, potentially leading to better treatment outcomes and fewer side effects compared to non-targeted therapies.

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