Go back to trials list
A Phase 1/2 Safety, Dose-finding, and Pharmacokinetics Study of VNX-101 Gene Therapy in Patients With Relapsed or Refractory CD19+ B-Cell Acute Lymphoblastic Leukemia
Description
This is a Phase 1/2, first-in-human, open-label, dose-escalating trial designed to assess the safety and efficacy of VNX-101 in patients with relapsed or refractory CD19+ B-cell acute lymphoblastic leukemia (ALL).VNX-101 is an investigational adeno-associated virus (AAV) gene therapy developed to express a secreted anti-CD19/anti-CD3 scFv diabody (termed GP101). GP101 binds both cluster of differentiation (CD)19 and CD3, inducing T-cells to kill both benign and malignant B-cells. Following a single intravenous (IV) infusion, the vector induces the liver and key tissues to continuously secrete GP101 into the bloodstream, resulting in long-term, consistent serum levels of GP101. Potential advantages of VNX-101 over autologous CAR-T therapy include it is off-the-shelf, provides a gentle onset of action, does not require lymphodepletion chemotherapy, engages all T-cells continuously (including those freshly produced from the bone marrow), and utilizes highly efficient signaling through the
Trial Eligibility
Inclusion Criteria: * Age: Part 1: 18-90 years of age, Part 2: 13-90 years of age * Relapsed B-cell ALL with bone marrow blasts \>= 5% * Refractory B-cell ALL as defined in the protocol * Bone marrow blasts requirement (flow cytometry): Part 1: \>0.01% to \<5% prior to VNX-101 dosing, Part 2: \>0.01% to \<50% prior to VNX-101 dosing. * Ineligible or declined CAR-T therapy or failed to respond or relapsed after such therapy * If prior blinatumomab treatment, cells remain CD19+ and not refractory to blinatumomab * AAVrh74 total antibody \<1:400 * Protocol-specified ranges for renal, liver, cardiac and pulmonary function * Protocol-specified ranges for hematology parameters Exclusion Criteria: * Hepatoxicity (AST or ALT \> 2x upper limit of normal) * History of thrombotic microangiopathy or cardiomyopathy, or evidence of sensory neuropathy * Pregnant or nursing (lactating) women * Acute Graft versus Host Disease (GvHD): Grade 2-4 or chronic GvHD of any grade * History of hypersensitivity to corticosteroids or history of corticosteroid-related toxicity * Chemotherapy given within the protocol-specified discontinuation timelines Other Inclusion/Exclusion criteria to be applied per protocol.
Study Info
Organization
Vironexis Biotherapeutics Inc.
Primary Outcome
Treatment emergent adverse events (TEAEs) and treatment-emergent serious events (TESAEs)
Interventions
Locations Recruiting
Colorado Blood Cancer Institute
United States, Colorado, Denver
Oncology Hematology Care
United States, Ohio, Cincinnati
TriStar BMT
United States, Tennessee, Nashville
Interested in joining this trial?
Our dedicated patient navigators are here to guide you through the validation and enrollment process with ease.
Get the latest thought leadership on your Chronic Eosinophilic Leukemia delivered straight to your inbox
Subscribe to the weekly newsletter for news, stories, clinical trial updates, and helpful resources and events with cancer experts.