Leukapheresis for CAR or Adoptive Cell Therapy Manufacturing

Launched by NATIONAL CANCER INSTITUTE (NCI) · Jul 21, 2017

Keywords

ClinConnect Summary

This study is not testing a new drug right away. It’s an observational program intended to identify people who might benefit from future CAR-T cell therapy and to collect and store a sample of their white blood cells (through a process called leukapheresis) for use in later CAR-T or other adoptive cell therapy studies at NIH. The main goal is to see what proportion of participants go on to enroll in a CAR-T study within about 12 months after cell collection. Researchers will also look at safety during the collection procedure and, over time, how many participants enroll in CAR-based therapies by disease type.

To be eligible, people would be ages 3 to 65 who have leukemia or lymphoma that has relapsed after standard treatment or did not respond to it, and who are likely to benefit from future CAR therapy. Participants undergo a screening visit and then leukapheresis, which lasts about 4–6 hours and may involve a central line. The collected cells are frozen for future use. This study does not provide CAR-T treatment itself during participation, but it helps prepare for potential future trials. It is conducted at the NIH Clinical Center in Bethesda, Maryland, and is currently enrolling by invitation.

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Eligibility criteria

• * INCLUSION CRITERIA:
• • Age: \>= 3 and \<= 65 years
• • Weight \>= 15 kg
• • Confirmation of cancer diagnosis provided by disease-specific assessment (e.g., flow cytometry, PCR) or H\&E verification.
• * Disease Status:
• • Relapsed/refractory cancer that has failed at least one standard regimen and are not in remission at the time of leukapheresis, OR
• • Previously treated patients without detectable disease at the time of leukapheresis but at high-relapse risk.
• * Potentially eligible for future NIH-CAR or other adoptive cell therapy based on the following:
• • Adequate performance status: Patients \> 10 years of age: Karnofsky \>= 50%; Patients \<= 10 years of age: Lansky scale \>= 50%
• * Adequate organ function:
• • absolute neutrophil count \>750/mcL\*
• • platelets \>=30,000/mcL\*
• • total bilirubin \<=2 X ULN (except in the case of subjects with documented Gilbert s disease \> 3x ULN)
• • AST(SGOT)/ALT(SGPT)\<=20 X institutional upper limit of normal for age and laboratory normal ranges
• • creatinine within age adjusted normal institutional limits (see below) OR
• • creatinine clearance \>= 60 mL/min/1.73 m\^2 for creatinine levels above institutional normal.
• • Age (Years): \<=5; Maximum Serum Creatine (mg/dL): 0.8
• • Age (Years): 5 \< age \<= 10; Maximum Serum Creatine (mg/dL): 1.0
• • Age (Years): \>10; Maximum Serum Creatine (mg/dL): 1.2
• • Cytopenias deemed to be disease-related and not therapy-related are exempt from this exclusion.
• • Patients, parents/guardians, legally authorized representative (LAR), or durable power of attorney must be able to give consent and sign the Informed Consent Document.
• EXCLUSION CRITERIA:
• • Transfusion refractory thrombocytopenia such that platelet count cannot be adequately supported with transfusions to be at \>=30,000/mcL
• • Active DIC, bleeding or coagulopathy which cannot be corrected with minimal intervention
• • Rapidly progressive disease or hyperleukocytosis \>= 50,000 blasts/mcL
• • Symptomatic, uncontrolled or severe intercurrent illness that would compromise the ability to tolerate CAR or adoptive cell therapy-based toxicity
• • Subjects must have recovered from the acute side effects of their prior therapy, such that eligibility criteria are met. Cytopenias deemed to be disease-related and not therapyrelated are exempt from this exclusion.
• • Pregnant or nursing (lactating) individuals, where pregnancy is defined as the state of after conception and until the termination of gestation, confirmed by a positive hCG laboratory test at screening
• • Active or latent hepatitis B or active hepatitis C, or any uncontrolled infection at screening
• • Human Immunodeficiency Virus (HIV) infection at screening (The experimental treatments being evaluated depend upon an intact immune system. Patients who are HIV seropositive can have decreased immune competence and thus be less responsive to the experimental treatment and more susceptible to its toxicities)
• • Any patient that in the opinion of the investigator is not medically stable to undergo the leukapheresis procedure or will not comply with the visit schedules or procedures