GD2-CART01 (iC9-GD2-CAR T-cells): Following a lymphodepleting treatment with conventional chemotherapy, patients will be treated with 1.0 to 6.0 x 10⁶/kg GD2 Chimeric Antigen Receptor (CAR) positive T cells, infused i.v. as a single dose
Study summary
The purpose of this study is to test the safety and efficacy of iC9-GD2-CAR T-cells, a third generation (4.1BB-CD28) CAR T cell treatment targeting GD2 in paediatric or young adult patients affected by relapsed/refractory malignant central nervous system (CNS) tumors. In order to improve the safety of the approach, the suicide gene inducible Caspase 9 (iC9) has been included.
Eligibility
Sex
ALL
Min age
6 Months
Max age
30 Years
Healthy volunteers
No
Inclusion Criteria:
1. Imaging assessments performed within 14 days of start of treatment
2. Age: 6months-30years
3. Measurable or evaluable disease on at least 2 dimensions on MRI at the time of treatment enrollment
4. Karnofsky/Lansky≥60
5. Recoverfromthetoxiceffectsofpreviousradiationandchemotherapies:grade4and or 3 non-hematologic toxicities must have resolved to grade ≤ 2; in presence of chronic complications (i.e. treatment-associated thrombocytopenia), patient must be clinically stable, according to the opinion of the treating physicians, and meet all other eligibility criteria
6. Positioning of an implantable intraventricular access device (CodmanHolterRickham reservoir, Integra LifeSciences, NJ, U.S.A) and a microdialysis probe (71 high cutoff microdialysis bolt catheter, M Dialysis AB, Stockholm Sweden)
7. Written and signed informed consent from patients, parents or legal guardians. For subjects \< 18 year-old their legal guardian must give informed consent. In addition, pediatric subjects will be included in age-appropriate discussion and written informed assent will be obtained for those greater than or equal to 7 years of age, when appropriate
8. Patients of childbearing or child-fathering potential must be willing to practice birth control from the time of enrollment on this study and for four months after receiving the preparative regimen
9. Females of childbearing potential must have a negative pregnancy test because of the potentially dangerous effects on the fetus
Exclusion Criteria:
1. Pregnant or lactating women
2. Severe,uncontrolledactiveinfections
3. HIV or active HCV and/or HBV infection
4. Rapidly progressive disease with life expectancy \< 6 weeks
5. Historyofgrade3or4hypersensitivitytomurineprotein-containingproducts
6. Hepatic function: inadequate liver function defined as total bilirubin \> 4x upper limit of normal (ULN) or transaminase (ALT and AST) \> 6 x ULN based on age and laboratory specific normal ranges
7. Renal function: serum creatinine \> 3x ULN for age
8. Blood oxygen saturation \< 90%
9. Cardiac function: left ventricular ejection fraction lower than 45% by ECHO
10. Marrow function: absolute neutrophils count (ANC) lower than 500/mm3 and/or platelets lower than 20.000 (not reached by transfusion)
11. Congestive heart failure, cardiac arrhythmia, psychiatric illness, or social situations that would limit compliance with study requirements or in the opinion of the principal investigator (PI) would pose an unacceptable risk to the subject. 12.Concurrent or recent prior therapies, before infusion:
1. If receiving glucocorticoids, patient must be on a stable or weaning dose for at least 7 days prior to infusion. Recent or current use of inhaled/topical/non- absorbable steroids is not exclusionary. Subjects receiving steroid therapy at physiologic replacement doses only are allowed provided there has been no increase in dose for at least 2 weeks prior to starting apheresis
2. Systemic chemotherapy in the 3 weeks preceding infusion
3. Immunosuppressive agents less than or equal to 30 days
4. Radiation therapy must have been completed at least 6 weeks prior to enrollment
5. Otheranti-neoplasticinvestigationalagentscurrentlyorwithin30dayspriorto start of protocol therapy
13.Patient-derived GD2-CART01 production failure: vitality \<80%, CD3+ cells \<80%, CD3+ CAR+ cells \<20%, CD3+ CAR+ antitumor activity \<60% in functional co-culture assay at an Effector: Target ratio 1:1, viable CAR+ cells upon AP1903 exposition \>20%, RCR positivity, Vector Copy Number \>10, non-sterility, endotoxin contamination (\> 1 EU/ml)
Primary outcome measure(s)
Safety and definition of the MTD/RD — 4 weeks after CAR T cell infusion To evaluate the safety of the infusion of iC9-GD2-CAR-T cells at different escalating/de-escalating doses and establish the dose limiting toxicity (DLT) and the maximum tolerated dose/recommended dose (MTD/RD) of the cellular product
Trial sites (1)
Facility
City
Region
Status
Ospedale Pediatrico Bambino Gesù
Roma
Italy
Recruiting
More Bambino Gesù Hospital and Research Institute trials in Italy
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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