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CytopeniaPhase 2NCT06285825

A Pilot Study of Emapalumab for the Treatment of CAR T-Cell Therapy-Associated Prolonged Cytopenia

A Phase 2 study testing Emapalumab in people with Cytopenia, run by M.D. Anderson Cancer Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

To look at the safety and effectiveness of emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Cytopenia
What is being tested?
Emapalumab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
32 participants
Where
1 siteUnited States
Study dates
5 Jul 2024to 22 Aug 2028
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Eligible participants will be considered for inclusion in this study if they meet the following criteria based on tests obtained less than 4 weeks from first dose:
  • Relapsed or refractory FL (grade 1, 2, 3a and 3b), MCL, DLBCL, PMBCL, tFL, or HGBCL
  • Received standard of care therapy with axi-cel, tisa-cel or liso-cel
  • Age ≥ 18 years
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
  • Either in remission after CART and/or with refractory/progressive disease not requiring urgent treatment in the opinion of the treating physician
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  • Grade 3-4 cytopenia defined as either absolute neutrophil count of < 1.0x109/L, or platelet count < 50x109/L or haemoglobin< 8 g/dL starting or persisting between day 30 and day 90 after CART
  • Creatinine clearance (as estimated by Cockcroft Gault) ≥ 30 mL/min
  • Serum alanine transaminase (ALT) / aspartate transaminase (AST) ≤ 2.5 upper limit of normal (ULN), independently from etiology
  • Total bilirubin ≤1.5 mg/dL, or ≤ 3 UNLin participants with Gilbert's syndrome.
  • Baseline oxygen saturation > 92% on room air 11 Females of childbearing potential must have a negative serum or urine pregnancy test (females who have undergone surgical sterilization or who have been postmenopausal for at least 2 years are not considered to be of childbearing potential)
  • 12. Participant will be willing to undergo a bone marrow biopsy before and at the end of each cycle, and at time of severe cytopenia recurrence; archived samples can be accepted during the screening period in absence of intercurrent treatment 13. The effects of emapalumab on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for up to 6 months after the administration of emapalumab (whichever is administered last). (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). This includes all female patients, between the onset of menses (as early as 8 years of age) and 55 years unless the participants presents with an applicable exclusionary factor which may be one of the following:
  • Postmenopausal (no menses in greater than or equal to 12 consecutive months).
  • History of hysterectomy or bilateral salpingo-oophorectomy.
  • Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy).
  • History of bilateral tubal ligation or another surgical sterilization procedure.
  • Approved methods of birth control are as follows: Hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
  • Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 6 months after completion of emapalumab administration.
  • 14. Ability to understand and the willingness to sign a written informed consent document.
  • 15. Participant will need to test for latent tuberculosis infections using the purified protein derivative (PPD) or IFNƒÁ release assay and will be evaluated for tuberculosis risk factors within 3 months from initiation of treatment. We will administer tuberculosis prophylaxis to participants at risk for tuberculosis, or known to have a positive PPD test result, or positive IFNƒÁ release assay.

Who cannot join

  • Participants will be ineligible for this study if they meet the following criteria:
  • History of malignancy other than nonmelanoma skin cancer or localized carcinoma (e.g., cervix, bladder, breast, prostate) unless disease free for at least 3 years.
  • History of Richter's transformation of chronic lymphocytic leukemia (CLL)
  • Presence of grade 3-4 cytopenia before initiation of lymphodepleting chemotherapy for CART
  • Grade 3-4 cytopenia due to use of experimental non-commercially available CART product
  • Bone marrow evidence of LBCL, hypersplenism, HLH, or a second malignancy (e.g., MDS) that could be contributing to cytopenia, with no alternative, identifiable potential cause for the cytopenia.
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  • Relapsed or progressive disease requiring immediate initiation of anti-lymphoma treatment
  • Known presence of fungal, bacterial, viral, or other infection that is uncontrolled or requiring IV antimicrobials for management, including leishmania infection. Simple UTI and uncomplicated bacterial pharyngitis are permitted if responding to active treatment and after consultation with the Principal investigator.
  • Known history of infection with HIV or hepatitis B (HBsAg positive) or hepatitis C virus (anti-HCV positive). A history of HIV, hepatitis B or hepatitis C is permitted if the viral load is undetectable per quantitative PCR and/or nucleic acid testing. For participants with a history of Hepatitis B or C, standard of care monitoring for viral reactivation will be conducted. Subjects with a history of Hepatitis B will be required to undergo hepatitis B reactivation prophylaxis unless contraindicated.
  • History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, or other clinically significant cardiac disease within 6 months of enrolment
  • History of primary immunodeficiency
  • History of symptomatic deep vein thrombosis or symptomatic pulmonary embolism within 1 month of enrolment considered significant in the opinion of the principal investigator
  • Any medical condition likely to interfere with assessment of safety or efficacy of study treatment in the investigator's opinion
  • History of severe immediate hypersensitivity reaction to any of the agents used in this study, including E coli-derived proteins.
  • Receipt of BCG vaccine within 12 weeks prior screening or receipt of a live or attenuated (other than BCG) vaccine within 4 weeks prior to screening
  • Women of child-bearing potential who are pregnant or breastfeeding.
  • Women of child-bearing potential and men with partners who are of child-bearing potential who are not willing to practice birth control as noted in section 4.1 from the time of consent through 6 months after the completion of emapalumab.
  • In the investigator's judgment, the subject is unlikely to complete all protocol-required study visits or procedures, including follow-up visits, or comply with the study requirements for participationTrial Treatments

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Paolo Strati, MD contact

(713) 745-1776

pstrati@mdanderson.org

United States1 site, 1 recruiting

  • MD Anderson Cancer Center

    Houston, Texas, 77030

    Paolo Strati, MD, 713-745-1776, pstrati@mdanderson.org

    Paolo Strati, MD

    Recruiting

Other trials in United States

About this study

To look at the safety and effectiveness of emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART.

Read the detailed description

Primary Objectives:

  • To evaluate the efficacy of emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART.

Secondary Objectives:

  • To evaluate safety and tolerability of emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART.

Exploratory Objective:

  • To investigate biomarkers of response and resistance to emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART.
Conditions
Cytopenia
Being tested
Emapalumab
Medical subject headings
Cytopenia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EmapalumabDrug

    Given by Infusion

Study groups in NCT06285825
Study groupRoleWhat they receive
EmapalumabExperimentalTo 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 2 planned doses will be studied at this time, with about 16-32 total participants enrolled at all dose levels. More dose levels may be studied, if needed. * If at least 3 of the first 5 participants show improvement in their blood test results, 11 more participants will be enrolled at that dose level. * If at least 9 of the first 16 participants enrolled show improvement, that dose will be considered effective. * If fewer than 3 of the first 5 participants show improvement in their blood test results, the study team will move to a higher dose of the drug and repeat the same approach. * Even if a dose level is claimed to be effective, the study team may still choose to test a next higher dose level. If the next higher dose level is tested, enrollment at that dose level will follow the same pattern as the first dose level.
Receives: Drug: Emapalumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety and adverse events (AEs)

    Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

    Measured: Through study completion; an average of 1 year.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Feb 2024
  2. Study start5 Jul 2024
  3. Primary completion22 Aug 2028 (estimated)
  4. Study completion22 Aug 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
32 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06285825
Sponsor's protocol ID
2023-0864
Other IDs
NCI-2024-01736
Status last verified
Aug 2026
First submitted
22 Feb 2024
Last updated
7 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Emapalumab in people with Cytopenia, run by M.D. Anderson Cancer Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 20 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 7 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Paolo Strati, MD ((713) 745-1776, pstrati@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety and adverse events (AEs) (time frame: Through study completion; an average of 1 year.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06285825.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2026.

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