Autologous HuCART19 T Cells Manufactured Using the CliniMACS Prodigy Platform for Pediatric B-ALL (huCART19 Prodigy)
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age0-29
SponsorStephan Grupp MD PhD
About this trial
This study will determine the safety and efficacy of moving to a second-generation manufacturing process using the CliniMACS Prodigy platform to manufacture huCART19 cells for patients with B cell Acute Lymphoblastic Leukemia (B-ALL).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Signed Informed Informed Consent
Disqualifiers
Active hepatitis B or active hepatitis C
HIV infection
Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy.
Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.
Trial design
Design model
Sequential
Treatments tested in this trial
Autologous Humanized CD19-Directed Chimeric Antigen Receptor T-Cells (huCART19)
Biological/VaccineThe investigational agent in this protocol is humanized CART19 cells (huCART19). Autologous T cells will be engineered to express an extracellular single chain antibody (scFv) with specificity for CD19. This will be expected to redirect specificity of the transduced T cells for cells that express CD19, a molecule that is restricted in expression on the surface of the malignant cells and on normal B cells.
Treatment groups
115 Participants
are divided into 2 treatment groupsGroup A: Dose Escalation ArmExperimental treatment 1 intervention
Group B: Dose Expansion ArmsExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Safety of huCART19 Administration
The safety of the administering Humanized Cd19-Directed Chimeric Antigen Receptor T-Cells (huCART9) will be measured by the monitoring the frequency and severity of adverse events in patients with advanced or refractory CD19+ hematologic malignancies, including those previously treated with cell therapy.
Time frame
5 years2
Efficacy of huCART19 Administration
The efficacy of huCART9 will be measured by the evaluating the overall response rate in patients with advanced or refractory CD19+ hematologic malignancies, including those previously treated with cell therapy.
Time frame
5 yearsSecondary outcomes
1
Manufacturing Feasibility
Manufacturing feasibility will be measured by the percentage of manufactured products that do not meet release criteria for vector transduction efficiency, T cell product purity, viability, sterility, or due to tumor contamination.
Time frame
5 years2
Safety of huCART19 as measured by ≥ Grade 3 toxicity rate
Safety of huCART19 as measured by ≥ Grade 3 toxicity rate (toxicity that is possibly attributed to huCART19) that is unmanageable, unexpected, and unrelated to chemotherapy.
Time frame
5 years3
Anti-tumor response due to huCART19 cell infusions
For subjects with detectable disease, measure anti-tumor response due to huCART19 cell infusions, as defined by the presence of medullary (morphologic or Minimal Residual Disease (MRD-level disease) and/or extramedullary disease at 1-month post-infusion.
Time frame
5 years4
Remission Rate
Overall remission rate will be measured by the proportion of treated subjects who achieve complete morphologic remission at Day 28 post huCART19 infusion.
Time frame
5 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Stephan Grupp MD PhD
Lead sponsor
Children's Hospital of Philadelphia
Sponsor institution
Children's Hospital of Philadelphia
Collaborator
Source ClinicalTrials.gov