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Clinical Trial 20592

Cancer Type: Malignant Hematology
Study Type: Treatment
NCT#: NCT04404660

Phase: Phase I/II
Principal Investigator: Shah, Bijal

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Study Title

An Open-Label, Multi-Centre, Phase Ib/II Study Evaluating the Safety and Efficacy of AUTO1, a CAR T Cell Treatment Targeting CD19, in Adult Patients with Relapsed or Refractory B Cell Acute Lymphoblastic Leukaemia


This is a Phase Ib/II study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting CD19 in adult patients with relapsed or refractory B cell acute lymphoblastic leukemia (ALL).


Phase Ib Primary Objective: -to evaluate the safety of AUTO1 Phase II: Primary Objective -to evaluate the clinical efficacy of AUTO1 Phase Ib Secondary Objectives: -to evaluate the feasibility of manufacturing and administering AUTO1 -to evaluate the clinical efficacy of AUTO1 -to evaluate the expansion and persistence of AUTO1 Phase II Secondary Objectives: -to evaluate the clinical efficacy of AUTO1 -to assess the safety and tolerability of AUTO1 -to evaluate the feasibility of manufacturing and administering AUTO1 -to evaluate the expansion and persistence of AUTO1 -to evaluate the duration of B cell aplasia -to evaluate Patient Reported Outcome and Quality of Life -to evaluate health care resource utilization for the management of AUTO1 related toxicity Exploratory Objectives: -to determine the proportion of patients who achieve MRD-negative CR using next generation sequencing -to determine ORR and DoR in patients who have received prior blinatumomab -to determine ORR in patients with ≤20% and >20% blasts prior to pre-conditioning as assess by IRRC -to determine the time course and magnitude of cytokine release evaluated using an appropriate assay -to assess antibody and/or T cell mediated immune responses against AUTO1 -to characterize the relationship between the CAR-T cell phenotype/genomics and persistence in vivo -seek any relationship between parameters of activity and tumor burden (e.g. % blast cells in BM, or EM site), level of CD19 expression (flow cytometry or immunohistochemistry [IHC]), CAR T cell phenotype -seek any relationship between incidence and severity of CRS, neurotoxicity, or other toxicity and tumour burden (e.g. % blast cells in BM, or EM site), level of CD19 expression (IHC or flow cytometry), CAR T Cell phenotype -to evaluate CSF for cytokine and CAR T cell levels in patients experiencing neurologic toxicity whenever feasible



Chemotherapy (NOS); Immunotherapy


AUTO1 (); cyclophosphamide (); cytoxan (cyclophosphamide); fludarabine (Fludarabine phosphate)

Inclusion Criteria

  • Age 18 years or older Age 18 years or older
  • ECOG performance status of 0 or 1
  • Relapsed or refractory B cell ALL
  • Patients with Ph+ ALL are eligible if intolerant to TKI, failed two lines of any TKI, or failed one line of second-generation TKI, or if TKI is contraindicated
  • Documented CD19 positivity within 1 month of screening
  • Phase Ib: Primary Cohort IA: Presence of >5% blasts in BM at screening
  • Phase Ib: Exploratory Cohort IB: MRD-positive defined as >1E-4 and > Phase II: Primary Cohort IIA: Presence of >5% blasts in BM at screening
  • Phase II: Exploratory Cohort IIB: MRD-positive defined as >1E-4 and > Adequate renal, hepatic, pulmonary, and cardiac function

  • Exclusion Criteria

  • Phase Ib (Cohort IA and Cohort IB) and Phase II Cohort IIA only ALL with isolated EM disease
  • Diagnosis of Burkitt's leukaemia/lymphoma or CML lymphoid in blast crisis
  • History or presence of clinically relevant CNS pathology
  • > Presence of CNS-3 disease or CNS-2 disease with neurological changes
  • Presence of active or uncontrolled fungal, bacterial, viral, or other infection requiring systemic antimicrobials for management
  • Active or latent Hepatitis B virus or active Hepatitis C virus
  • Human Immunodeficiency Virus (HIV), HTLV-1, HTLV-2, syphilis positive test
  • Prior CD19 targeted therapy other than blinatumomab. Patients who have experienced Grade 3 or higher neurotoxicity following blinatumomab.

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