Emapalumab for Low Blood Cell Count

Overseen By Paolo Strati, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: M.D. Anderson Cancer Center

Must not be taking: IV antimicrobials

Disqualifiers: History of malignancy, HIV, 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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 16 weeks

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the safety and effectiveness of the drug emapalumab (Gamifant) for individuals with a specific type of persistent low blood cell count following CAR T-cell therapy. It targets patients with certain types of lymphoma who experience severe, prolonged low blood cell counts after treatment. Participants should have undergone specific cancer therapies and not require immediate treatment for their disease. The trial tests two different doses of emapalumab to determine which is most effective and safe. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to important findings.

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

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor to get a clear answer based on your specific situation.

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

Research has shown that emapalumab has been tested in patients with primary hemophagocytic lymphohistiocytosis (HLH). In these studies, 53% of patients experienced serious side effects, with infections and digestive system bleeding being the most common. Although emapalumab has been used before, it can cause serious side effects. Prospective participants should consider these findings when deciding to join a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for low blood cell count aim to stimulate the production of blood cells or suppress the immune system's attack on these cells. Emapalumab works differently by specifically targeting and neutralizing interferon-gamma, a protein involved in the immune system's attack on blood cells. This unique mechanism could potentially reduce inflammation and protect blood cells more effectively than current treatments. Researchers are excited because this targeted approach might offer a more precise treatment option with fewer side effects, especially for patients who don't respond well to standard therapies.

What evidence suggests that emapalumab might be an effective treatment for low blood cell count?

Research has shown that emapalumab may help treat certain blood-related conditions. Specifically, studies have found that emapalumab can improve levels of white blood cells, neutrophils, and platelets in patients with refractory macrophage activation syndrome. In another study, 63% of patients responded well to emapalumab, and 70% of those patients successfully received a stem cell transplant. Although this is not directly related to low blood cell counts from CAR T-cell therapy, these findings suggest that emapalumab might help stabilize blood cell levels. This trial will explore emapalumab's potential to assist with severe low blood cell counts, with participants receiving one of two dose levels based on their enrollment timing. ^{6 7 8 9 10}

Who Is on the Research Team?

Paolo Strati

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

This trial is for individuals with LBCL who have severe, prolonged low blood cell counts after CAR T-cell therapy. Specific eligibility details are not provided, but typically participants must meet certain health standards and not have conditions that could interfere with the study or their safety.

Inclusion Criteria

Baseline oxygen saturation > 92% on room air

I have not had a menstrual period for at least 12 months.

Serum alanine transaminase (ALT) / aspartate transaminase (AST) ≤ 2.5 upper limit of normal (ULN), independently from etiology

Exclusion Criteria

Participants will be ineligible for this study if they meet the following criteria:

I am pregnant or breastfeeding.

Any medical condition likely to interfere with assessment of safety or efficacy of study treatment in the investigator's opinion

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive emapalumab at one of two dose levels for the treatment of prolonged severe cytopenia

16 weeks

Regular visits for dose administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term follow-up

Participants are monitored for adverse events and long-term outcomes

Up to 1 year

What Are the Treatments Tested in This Trial?

Interventions

• Emapalumab

Trial Overview The trial is testing emapalumab to see if it's safe and can help treat people with long-lasting severe cytopenia (low white blood cells, anemia, low platelet count) following CAR T-cell therapy for LBCL.

How Is the Trial Designed?

Group I: Emapalumab Experimental Treatment 1 Intervention

If you are found to be eligible to take part in this research study, you will be assigned to 1 of 2 dose levels of emapalumab, based on when you enroll on the study. Up to 16 participants will receive each dose level of the study drug. * The first group of 5 participants will be enrolled at the lower dose level. * Depending on the safety data seen in this first group, the next 11 participants will be enrolled at the lower dose. * After reviewing the early safety data from that first group of 16 participants, the study team will enroll the next 5 participants at the higher dose level and be checked for serious side effects. * Depending on the safety information from the first 5 participants in the higher dose level group, the other 11 will then be enrolled.

Emapalumab is already approved in United States for the following indications:

Approved in United States as Gamifant for:

• Primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Sobi, Inc.

Industry Sponsor

Trials

14

Recruited

1,000+

Published Research Related to This Trial

In a phase 2 study involving subjects with rheumatoid arthritis, mavrilimumab significantly improved disease activity, with 55.7% of treated participants achieving a meaningful reduction in Disease Activity Score compared to 34.7% in the placebo group.

The treatment was generally safe, with most adverse events being mild to moderate, and no serious infections or significant hypersensitivity reactions reported, indicating a favorable safety profile for mavrilimumab.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of mavrilimumab in subjects with rheumatoid arthritis. Burmester, GR., Weinblatt, ME., McInnes, IB., et al.[2022]

Mavrilimumab, a GM-CSF receptor-α antibody, showed significant clinical benefits in a Phase II trial for moderate rheumatoid arthritis, particularly in the 100 mg dosage group compared to placebo.

The drug's unique mechanism of targeting GM-CSF signaling, along with its demonstrated efficacy and safety, suggests it could effectively induce remission and improve overall health outcomes for patients with rheumatoid arthritis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mavrilimumab, a human monoclonal GM-CSF receptor-α antibody for the management of rheumatoid arthritis: a novel approach to therapy. Nair, JR., Edwards, SW., Moots, RJ.[2019]

In a study of 17 patients undergoing allogeneic stem cell transplantation (allo-SCT), major mismatches in neutrophil antigens (NA1 and NA2) did not significantly affect the engraftment of neutrophils or the incidence of post-engraftment neutropenia.

The findings suggest that using leukocyte depletion filters and administering G-CSF after transplantation may mitigate the impact of NA2 antigen mismatches on neutrophil engraftment, although the study's small sample size limits the statistical power of these conclusions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Little impact of donor/recipient major mismatch for neutrophil-specific antigen NA2 on neutrophil recovery after allogeneic SCT. Inukai, T., Goi, K., Tezuka, T., et al.[2009]

Autoantibodies against the FcgammaRIIIb receptor can prolong the survival of neutrophils (PMNs) by triggering anti-apoptotic signals, which may help in non-organ-specific diseases.

The mechanism involves signaling through FcgammaRIIIb and CD11b, leading to the production of granulocyte colony-stimulating factors that delay apoptosis, suggesting a potential therapeutic target for modulating immune responses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pathogenic effects of anti-Fc gamma receptor IIIb (CD16) on polymorphonuclear neutrophils in non-organ-specific autoimmune diseases. Youinou, P., Durand, V., Renaudineau, Y., et al.[2019]

A new genotyping assay using polymerase chain reaction has been developed to accurately identify the granulocyte antigens NA1 and NA2, which are important in various clinical conditions like neonatal neutropenia and transfusion-related complications.

The assay showed a 100% concordance rate with traditional serologic typing, demonstrating its reliability, and revealed specific gene frequencies for NA1 and NA2 across different ethnic groups, which can aid in understanding population-specific risks in related medical conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Determination of neutrophil antigen gene frequencies in five ethnic groups by polymerase chain reaction with sequence-specific primers. Hessner, MJ., Curtis, BR., Endean, DJ., et al.[2022]

Citations

1. sobi.com sobi.com/en/press-releases/fda-approves-gamifantr-emapalumab-lzsg-first-ever-treatment-adults-and-children-macrophage-activation-syndrome-stills-disease-2342991

FDA approves Gamifant® (emapalumab-lzsg) as first-ever ... Approval based on the pooled analysis of our pivotal EMERALD and NI-0501-06 studies, showing 54% of patients treated with Gamifant achieved ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12558795/

Low-dose emapalumab treatment in refractory macrophage ... During the follow-up period, WBC, neutrophil, and platelet counts initially increased after treatment of emapalumab, and were subsequently ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02069899

NCT02069899 | A Study for Long-term Follow-up of ... The aim of this study is to monitor the long-term safety profile of emapalumab in participants who have previously received at least one dose of emapalumab.

4. gamifant.com gamifant.com/phlh/results

Primary HLH efficacy data Gamifant was proven to be effective in a clinical trial · 63% (17/27) overall response rate (ORR) with Gamifant · 70% of patients (19/27) proceeded to HSCT ...

5. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2018/761107Orig1s000MultidisciplineR.pdf

761107Orig1s000 - accessdata.fda.gov No NI-0501-related alterations to immune cell levels in peripheral blood. ... supports that efficacy results can be attributed to emapalumab ...

6. gamifant.com gamifant.com/phlh/safety

Safety Data | Gamifant® (emapalumab-lzsg) For HCPs Serious adverse reactions were reported in 53% of patients. The most common serious adverse reactions (≥3%) included infections, gastrointestinal hemorrhage ...

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2018/761107lbl.pdf

GAMIFANT (emapalumab-lzsg) injection, - accessdata.fda.gov The safety data described in this section reflect exposure to GAMIFANT in which 34 patients with untreated primary HLH and previously treated patients with ...

8. gamifant.com gamifant.com/sites/default/files/2025-07/Full-Prescribing-Information.pdf

Full prescribing Information The safety data described in this section reflect exposure to GAMIFANT in which 34 patients with untreated primary HLH and previously treated patients with ...

9. uhcprovider.com uhcprovider.com/content/dam/provider/docs/public/policies/comm-medical-drug/gamifant-emapalumab-lzsg.pdf

Gamifant® (Emapalumab-Lzsg) Study NI-0501-05, was a multicenter follow-up study to collect safety and outcome data for patients who received emapalumab through NI-0501-04 and compassionate ...

10. rxlist.com rxlist.com/gamifant-drug.htm

Gamifant (Emapalumab-lzsg Injection): Side Effects, Uses ... Safety and effectiveness of GAMIFANT have been established in pediatric patients, newborn and older, with primary HLH that is reactivated or refractory to ...