Emapalumab for Low Blood Cell Count
Recruiting in Palo Alto (17 mi)
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: M.D. Anderson Cancer Center
Must not be taking: IV antimicrobials
Disqualifiers: History of malignancy, HIV, others
No Placebo Group
Prior Safety Data
Approved in 1 Jurisdiction
Trial Summary
What is the purpose of this trial?To look at the safety and effectiveness of emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART.
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.
What makes the drug Emapalumab unique for treating low blood cell count?
Emapalumab is unique because it specifically targets and neutralizes interferon gamma, a protein involved in inflammation, which is different from other treatments that may not target this pathway. This mechanism can be particularly beneficial in conditions where excessive inflammation is a problem.
12345Eligibility Criteria
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.
I am 18 years old or older.
+20 more
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
+15 more
Trial Timeline
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
Participant Groups
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.
1Treatment groups
Experimental Treatment
Group I: EmapalumabExperimental Treatment1 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
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
MD Anderson Cancer CenterHouston, TX
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Who Is Running the Clinical Trial?
M.D. Anderson Cancer CenterLead Sponsor
Sobi, Inc.Industry Sponsor
References
Mavrilimumab, a human monoclonal GM-CSF receptor-α antibody for the management of rheumatoid arthritis: a novel approach to therapy. [2019]Mavrilimumab , formerly known as CAM-3001, a GM-CSF receptor-α antibody, is the first human monoclonal antibody to be used in Phase II studies (2011) to modulate the innate immunity pathway targeting GM-CSF signaling in moderate rheumatoid arthritis (RA).
Pathogenic effects of anti-Fc gamma receptor IIIb (CD16) on polymorphonuclear neutrophils in non-organ-specific autoimmune diseases. [2019]The receptors FcgammaRIIIb and FcgammaRIIa for the Fc portion of IgG are naturally expressed in polymorphonuclear neutrophils (PMN). Autoantibodies (Ab) against FcgammaRIIIb exist in patients with non-organ-specific disease. These may be categorized, based on the results of an indirect immunofluorescence (IIF) test for the detection of anti-membrane-bound FcgammaRIIIb autoAbs, and an enzyme-linked immunosorbent assay for that of soluble FcgammaRIIIb-recognizing autoAbs. The IIF+ autoAbs are not cytotoxic, and prolong the survival of the cells. The autoAb-triggered anti-apoptotic signal may be transduced through FcgammaRIIa and/or CD11b, the beta-chain of the neighboring complement receptor type 3. However, FcgammaRIIIb appears to be as competent as FcgammaRIIa, because the results obtained using the respective monoclonal Abs are additive. Soluble FcgammaRIIIb binds to CD11b and produces similar effects, suggesting that autoAb-stimulated FcgammaRIIIb can work in concert with CD11b. Anti-FcgammaRIIIb-conditioned supernatant of PMNs induces the transcription of messenger RNA for granulocyte colony-stimulating factor (CSF) and granulocyte-macrophage CSF, followed by the release of these anti-apoptotic factors. The delay in apoptosis is accompanied by a down-regulated expression of Bax. Thus, apoptosis of aged PMNs can be modulated by signaling through FcgammaRIIIb, which may occur in patients with PMN-binding anti-FcgammaRIIIb autoAbs.
Determination of neutrophil antigen gene frequencies in five ethnic groups by polymerase chain reaction with sequence-specific primers. [2022]The granulocyte antigens NA1 and NA2 are the two recognized allelic forms of Fc gamma receptor IIIB. These antigens are clinically relevant, because they are the most frequent targets of neutrophil antibodies in alloimmune neonatal neutropenia, transfusion-related acute lung injury, and chronic benign autoimmune neutropenia of infancy.
Little impact of donor/recipient major mismatch for neutrophil-specific antigen NA2 on neutrophil recovery after allogeneic SCT. [2009]The Fcgamma receptor IIIb (FcgammaRIIIb), a receptor for the Fcgamma region of IgG, is specifically expressed on neutrophils. It has two allelic polymorphisms, NA1 and NA2, which are highly immunogenic and act as targets in alloimmune or autoimmune neutropenia. Thus, neutrophil antigens (NA) compatibility of donor/recipient pairs might be expected to affect the engraftment of neutrophils after allogeneic SCT (allo-SCT). Here, the impact of NA compatibility of 17 patients and their donors undergoing allo-SCT with a myeloablative regimen was determined. Leukocyte depletion filters were used for all transfusions before and post-SCT; most patients received G-CSF after transplant. Major mismatches for NA1 and NA2 were present in 1 and 7 patient/donor pairs, respectively. These eight patients receiving NA major-mismatched allo-SCT were compared with nine patients who received NA compatible allo-SCT. Engraftment of neutrophils and the incidence of post-engraftment neutropenia were found to be identical in the two groups. Despite the limitations in statistical power because of the small number of patients analyzed, these observations suggest that the major mismatching for NA2 antigen has little impact on the engraftment of neutrophils after myeloablative allo-SCT, at least in patients transfused using leukocyte depletion filters and receiving G-CSF after transplantation.
Efficacy and safety of mavrilimumab in subjects with rheumatoid arthritis. [2022]Mavrilimumab, a human monoclonal antibody targeting the alpha subunit of the granulocyte-macrophage colony-stimulating factor receptor, was evaluated in a phase 2 randomised, double-blind, placebo-controlled study to investigate efficacy and safety in subjects with rheumatoid arthritis (RA).