Actively Recruiting
A Pilot Study of Emapalumab for the Treatment of CAR T-Cell Therapy-Associated Prolonged Cytopenia
Led by M.D. Anderson Cancer Center · Updated on 2026-05-18
32
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
S
Sobi, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of emapalumab to treat prolonged severe cytopenia in participants with large B-cell lymphoma (LBCL) who have received CAR T-cell therapy (CART). This pilot study aims to assess how well emapalumab works and its safety, while also exploring biomarkers that might show how participants respond or resist the treatment. The study is a Phase 2 interventional trial sponsored by M.D. Anderson Cancer Center. Participants will receive emapalumab by infusion and be assigned to one of two dose levels. Around 16 to 32 participants will be enrolled across these doses. The trial follows a stepwise approach: if enough participants show improvement at a dose level, the dose is considered effective, and the study may test a higher dose following the same approach. Dosing and enrollment continue based on participants' blood test improvements after treatment. During the study, participants will undergo blood tests and safety monitoring for about one year. Researchers will check for adverse events and evaluate the treatment's impact on blood cell counts. Bone marrow biopsies will be performed before and after each treatment cycle and if severe cytopenia recurs. Participants will also be monitored for infections and organ function. The total involvement includes screening, treatment, and follow-up visits to assess treatment effects and safety.
CONDITIONS
Brief Title
A Pilot Study of Emapalumab for the Treatment of CAR T-Cell Therapy-Associated Prolonged Cytopenia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 years or older
- Diagnosed with relapsed or refractory follicular lymphoma, mantle cell lymphoma, diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma, transformed follicular lymphoma, or high-grade B-cell lymphoma
- Received standard CAR T-cell therapies such as axi-cel, tisa-cel, or liso-cel
- ECOG performance status of 0, 1, or 2
- In remission after CAR T-cell therapy or with refractory/progressive disease not requiring urgent treatment
- Grade 3-4 cytopenia between day 30 and day 90 after CAR T-cell therapy, defined by specific low counts of neutrophils, platelets, or hemoglobin
- Creatinine clearance of at least 30 mL/min
- Liver enzymes (ALT/AST) less than or equal to 2.5 times the upper limit of normal
- Total bilirubin less than or equal to 1.5 mg/dL (or up to 3 mg/dL in participants with Gilbert's syndrome)
- Oxygen saturation above 92% on room air
- Negative pregnancy test for females of childbearing potential
- Willingness to undergo bone marrow biopsies before and after treatment cycles
- Agreement to use adequate contraception during the study and for 6 months after treatment
- Ability to understand and sign informed consent
- Testing for latent tuberculosis and willingness to receive prophylaxis if needed
You will not qualify if you...
- History of other malignancies except certain skin or localized cancers unless disease-free for 3 years
- History of Richter's transformation of chronic lymphocytic leukemia
- Grade 3-4 cytopenia before lymphodepleting chemotherapy for CAR T-cell therapy
- Grade 3-4 cytopenia caused by experimental CART products
- Bone marrow evidence of lymphoma, hypersplenism, HLH, or a second malignancy contributing to cytopenia
- Relapsed or progressive disease needing immediate anti-lymphoma treatment
- Active uncontrolled infections requiring IV antimicrobials
- Known HIV or active hepatitis B or C infections without undetectable viral loads
- Significant cardiac events within 6 months
- Primary immunodeficiency
- Recent symptomatic deep vein thrombosis or pulmonary embolism
- Severe allergic reactions to study agents
- Recent live vaccinations
- Pregnant or breastfeeding women
- Unwillingness to use birth control as required
- Medical conditions interfering with safety or efficacy assessments
- Inability to complete study visits or comply with study requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Duration varies depending on dosing schedule and participant response
Participants receive up to 2 planned doses of emapalumab by infusion to treat prolonged severe cytopenia after CAR T-cell therapy.
1 to 2 visits for dosing and assessments
Duration - Approximately 1 year
Participants are monitored for safety and adverse events for an average of 1 year after treatment completion.
Regular follow-up visits over 1 year
Trial Site Locations
Total: 1 location
1
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
P
Paolo Strati, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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