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

NCT06285825 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 2023-0864NCI-2024-01736
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Cytopenia

Outcome Measures

Primary Outcomes (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

  Through study completion; an average of 1 year.

Study Arms (1)

Emapalumab

EXPERIMENTAL

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.

Drug: Emapalumab

Interventions

Emapalumab DRUG

Given by Infusion

Emapalumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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)
• 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.

You may not qualify if:

• 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.
• 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

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• Sobi, Inc.: collaborator

Study Sites (1)

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

• MD Anderson Cancer Center

MeSH Terms

Conditions

Cytopenia

Interventions

Emapalumab

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Paolo Strati, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Paolo Strati, MD

CONTACT

(713) 745-1776; pstrati@mdanderson.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2024
• First Posted: February 29, 2024
• Study Start: July 5, 2024
• Primary Completion (Estimated): August 22, 2026
• Study Completion (Estimated): August 22, 2028
• Last Updated: May 5, 2026

Record last verified: 2026-05

Locations

US (1)