Actively Recruiting
Phase I Clinical Trial of Fresh, Allogeneic, Second-generation CD19-CAR T Cells for Children and Young Adults With Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia
Led by Bambino Gesù Hospital and Research Institute · Updated on 2025-12-02
24
Participants Needed
1
Research Sites
778 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating fresh, donor-derived CD19-directed second-generation Chimeric Antigen Receptor T (CAR T) cells in pediatric and young adult patients with relapsed or refractory B-cell precursor Acute Lymphoblastic Leukemia (BCP-ALL). This phase 1, open-label, single-center study aims to assess the safety, determine the recommended dose, and gather preliminary evidence of efficacy in patients who have relapsed after or before allogeneic hematopoietic stem cell transplantation (alloHSCT) with a fully matched donor available. Participants receive a single intravenous infusion of CD19-CAR_Lenti_ALLO cells on Day 0 following a lymphodepletion regimen with fludarabine and cyclophosphamide given on days -5, -4, and -3. Patients are assigned to two cohorts based on donor HLA matching: cohort A with fully matched family or unrelated donors and cohort B with haploidentical donors. Dose levels vary by cohort, and an additional lower dose level may be explored if dose-limiting toxicities occur. During the study, participants will be closely monitored for safety, including dose-limiting toxicities over 28 days, and for graft-versus-host disease occurrence and severity over one year. Researchers will also evaluate treatment responses such as complete response and minimal residual disease negativity. Follow-up includes survival assessment over one year. The study involves detailed clinical evaluations and laboratory tests to ensure thorough monitoring of each participant.
CONDITIONS
Brief Title
Allogeneic Second-generation CD19-CAR T Cells for Pediatric Relapsed/Refractory B-ALL
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients diagnosed with CD19-expressing B-cell Acute Lymphoblastic Leukemia relapse
- Relapse after allogeneic hematopoietic stem cell transplantation (alloHSCT) or relapsed/refractory disease with failure of frontline therapy and at least two rescue treatments including CD19/CD22 monoclonal antibody
- Availability of a fully matched related donor
- CD19+ cell count of at least 50 cells/mcL or minimal residual disease (MRD) of at least 10^-4
- Voluntary informed consent; for those under 18, legal guardian consent and verbal assent for those 12 or older when appropriate
- Clinical performance status: Karnofsky score ≥ 60% if older than 16 years; Lansky score ≥ 60% if 16 years or younger
- Willingness to use birth control from enrollment through 4 months after lymphodepletion
- Negative pregnancy test for females of child-bearing potential
You will not qualify if you...
- Pregnant or lactating women
- Severe uncontrolled infections
- HIV or active hepatitis C or B infection
- Life expectancy less than 6 weeks or rapidly progressing disease compromising study completion
- Liver function with total bilirubin > 4 times upper limit of normal or transaminases > 6 times upper limit
- Kidney function with serum creatinine > 3 times upper limit for age
- Blood oxygen saturation below 90%
- Left ventricular ejection fraction below 45% by echocardiogram
- Congestive heart failure, cardiac arrhythmia, psychiatric illness, or social situations limiting study compliance or posing risk
- Active grade 2-4 acute or chronic graft-versus-host disease requiring steroids or immunosuppressive therapy
- Relapse occurring less than 60 days after alloHSCT
- Recent systemic steroids (≥ 2 mg/kg prednisone) or chemotherapy within 2 weeks before infusion
- Recent anti-thymocyte globulin or Alemtuzumab within 8 weeks before infusion
- Recent immunosuppressive agents within 2 weeks before infusion
- Radiation therapy completed less than 2 weeks before infusion
- Other investigational anti-cancer agents within 30 days before infusion
- Exceptions include intrathecal chemotherapy with recovery and stable physiologic steroid replacement doses
- Donors must meet conventional eligibility for allogeneic donation as required by law
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 3 days
Participants receive lymphodepletion chemotherapy with fludarabine and cyclophosphamide before CAR T cell infusion.
Daily visits for 3 days
Duration - Single day
Participants receive a single intravenous infusion of donor-derived CD19-CAR T cells on Day 0.
1 infusion visit (in-person)
Duration - Up to 1 year
Participants are monitored for safety, efficacy, and occurrence of graft-versus-host disease and other outcomes after treatment.
Regular follow-up visits for up to 1 year
Trial Site Locations
Total: 1 location
1
Ospedale Pediatrico Bambino Gesù
Rome, Italy, Italy, 00165
Actively Recruiting
Research Team
F
Franco Locatelli, MD, PhD
F
Francesca del Bufalo, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here