Tandem CD19/CD22 CAR T-cells as potential therapy for children and young adults with high-risk r/r B-ALL.
Berta González-Martínez, Víctor Galán-Gómez, Alfonso Navarro-Zapata...
https://pubmed.ncbi.nlm.nih.gov/40753722Actively Recruiting
Phase 1
Age: 0 - 30Years
All Genders
ID06709469
Phase I Trial of CART Cell Therapy for Refractory or Relapsed Acute Lymphoblastic Leukemia in Children, Adolescents, and Young Adults Evaluating Feasibility and Safety
Led by Instituto de Investigación Hospital Universitario La Paz · Updated on 2025-09-22
10
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the feasibility and safety of two different anti-CD19 chimeric antigen receptor T cell (CART) therapies for children and young adults with relapsed or refractory acute lymphoblastic leukemia (ALL). This study focuses on patients with CD19+ B-cell ALL or T-cell ALL who have limited treatment options and aims to address the high relapse rates seen after current CART therapies. The trial is a phase I study designed to explore new treatment approaches for these hard-to-treat leukemias. The trial involves two treatment groups based on disease biomarkers. One group receives autologous dual CD19/CD22 CART cells, administered as two intravenous doses with specific timing and dosing guidelines. The other group receives allogeneic CART-NKG2D cells given in up to three intravenous doses spaced 48 hours apart, depending on patient response. Treatments are tailored based on the patient's leukemia type and biomarker expression, with careful monitoring for cytokine-release syndrome. Participants will undergo regular assessments including laboratory tests to monitor immune cell levels, antigen expression, and cytokine profiles over an average of five years. Safety is closely tracked through adverse event reporting, with a focus on the severity and frequency of side effects related to the CART therapies. Additional tests include DNA methylation profiling and antibody presence to understand the treatments' effects. The study requires participants to have adequate organ function and provides long-term follow-up to evaluate treatment persistence and overall response.
CONDITIONS
Brief Title
Phase I Clinical Trial of CART Cell Therapy for Refractory/Relapsed Acute Lymphoblastic Leukemia in Children, Adolescents and Young Adults
Who Can Participate
Age: 0 - 30Years
All Genders
Eligibility Criteria
You may qualify if you...
- Children and young adults with relapsed or refractory CD19+/- CD22+ B-ALL not responding to chemotherapy and no other curative options
- Children and young adults with relapsed or refractory T-ALL not responding to chemotherapy and no other curative options
- Suitable for allogeneic stem cell transplant and willing to proceed if remission is achieved
- For T-ALL patients, a suitable haploidentical donor must be available
- Lansky (under 16) or Karnofsky (16 or older) score of 50 or greater
- Life expectancy longer than 12 weeks
- Absolute neutrophil count of 500/µL or higher unless reversible with therapy
- Platelet count of 50,000/µL or higher unless reversible with therapy
- Absolute lymphocyte count of 100/µL or higher
- Adequate kidney, liver, lung, and heart function
- Adequate venous access and no contraindications for lymphoapheresis
- Patients with well-controlled seizure disorders
- Ability to provide written informed consent or have a legal representative do so
You will not qualify if you...
- Participation in another clinical trial within the past 4 weeks
- Active infections requiring systemic treatment, including significant viral infections or uncontrolled viral reactivation
- Cardiac issues such as left ventricular shortening fraction below 30% or ejection fraction below 40%, or significant pericardial effusion
- CNS-3 disease or uncontrolled seizure disorder
- Active immunosuppressive therapy except low-dose prednisone within 7 days prior to enrollment
- Glomerular filtration rate below 30 ml/min or bilirubin more than three times the upper limit unless due to Gilbert's syndrome
- Any condition that may interfere with trial safety or effectiveness as judged by the investigator
- Pregnant or breastfeeding women
- Sexually active patients must use effective birth control for at least 12 months after infusion and until CAR-T cells are undetectable on two tests
- Male partners must use condoms for at least 12 months after infusion until CAR-T cells are undetectable
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to several days depending on dosing schedule and response
Participants receive infusions of CAR T-cell therapies targeting their specific leukemia type, with dosing depending on the treatment arm and participant response to cytokine-release syndrome.
1 to 3 infusion visits over approximately 1 week
Follow-up
Duration - Up to 5 years
Participants are monitored for safety, adverse events, and treatment response over an extended period after CAR T-cell therapy.
Regular visits during follow-up as scheduled by the study team
Trial Site Locations
Total: 1 location
1
Hospital Universitario La Paz
Madrid, Madrid, Spain, 28046
Actively Recruiting
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Research Team
A
Antonio Pérez Martínez, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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