Phase I Clinical Trial of CART Cell Therapy for Refractory/Relapsed Acute Lymphoblastic Leukemia in Children, Adolescents and Young Adults

Launched by INSTITUTO DE INVESTIGACIÓN HOSPITAL UNIVERSITARIO LA PAZ · Nov 25, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called CART cell therapy for children, adolescents, and young adults with a specific type of leukemia that has not responded to standard treatments. The researchers want to find out if two different types of CART cells can be safely used to help these patients. One type targets a protein called CD19, while the other targets a different protein called CD22. The trial is open to young people who have been diagnosed with either B-cell or T-cell acute lymphoblastic leukemia that has returned or has not improved with previous treatments.

To participate in this trial, patients must meet certain requirements. They should have a specific type of leukemia that has not responded to regular chemotherapy and should be willing to consider a stem cell transplant if the treatment works. Participants will undergo evaluations to ensure their overall health is stable enough for the trial. If eligible, they can expect close monitoring throughout the study to assess how well the treatment works and its safety. It's important to know that this trial is not yet recruiting participants, so there will be a waiting period before enrollment begins.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• * ARM A: CD19+/- CD22+ B-ALL with relapsed or refractory disease not responding to conventional chemotherapy and with no other curative therapy available. Treatment with previous CART CD19 therapy is permitted, but is not mandatory, OR:
• • ARM B: T-ALL with relapsed or refractory disease not responding to conventional chemotherapy and with no other curative therapy available.
• • Patients diagnosed with ALL must be suitable for allogeneic HSCT and willing to proceed to transplant if the CART treatment induces complete remission and the investigator believes it is the best option.
• • For ARM B there must be a suitable haploidentical donor (following local standard operating procedures).
• • Lansky (age \<16 years) or Karnofsky (age ≥16 years) score of 50 or greater.
• • Life expectancy greater than 12 weeks.
• • Absolute neutrophil count (ANC) ≥ 500/μL unless, in the opinion of the investigator, cytopenia is due to underlying leukemia and is potentially reversible with leukemia therapy.
• • Platelet count ≥ 50,000/μL unless, in the opinion of the investigator, cytopenia is due to underlying leukemia and is potentially reversible with leukemia therapy.
• • Absolute lymphocyte count ≥ 100/μL.
• • Adequate renal, hepatic, pulmonary, and cardiac function.
• • Adequate venous access and absence of contraindications for lymphoapheresis
• • Patients with a seizure disorder may be enrolled if well controlled with anticonvulsants.
• • Patients or patients' legal representative, parent(s), or guardian able to provide written informed consent.
• Exclusion Criteria:
• • Enrolled in another clinical trial in the previous 4 weeks.
• • Active infection requiring systemic medical therapy including clinically significant viral infection or uncontrolled viral reactivation of EBV, CMV, adenovirus, BK-virus, HHV-6 or Aspergillus.
• • Any of the following cardiac criteria: cardiac echocardiography with LVSF\<30% or LVEF\<40%; or clinically significant pericardial effusion.
• • Presence of CNS-3 disease or uncontrolled seizure disorder.
• • Active immunosuppressive therapy with the exception of prednisone 10 mg/day (or equivalent), within 7 days prior to enrolment.
• • GFR \<30 ml/min or bilirubin \>3 times the upper limit of normality (unless due to Gilbert's syndrome).
• • Any other condition that, in the opinion of the PI, may interfere with the efficacy and/or safety evaluation of the trial.
• • Pregnant or lactating women.
• • Sexually active patients must be willing to utilize one of the more effective birth control methods for at least 12 months after the infusion and until CAR-T cells are no longer present on two consecutive tests. Male partner should use a condom. Women of child-bearing potential are defined as all women physiologically capable of becoming pregnant.
• • Sexually active males should use a condom during intercourse for at least 12 months after the infusion and until CAR-T cells are no longer present on two consecutive tests.