Ignite Creation Date:
2024-10-26 @ 3:36 PM
Last Modification Date:
2024-10-26 @ 3:36 PM
Study NCT ID:
NCT06533579
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-07-24
Brief Title:
Gene Therapy for B-Cell Acute Lymphoblastic Leukemia
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Phase 12 Safety Dose-finding and Pharmacokinetics Study of VNX-101 Gene Therapy in Patients With Relapsed or Refractory CD19 B-Cell Acute Lymphoblastic Leukemia
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a Phase 12 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
Detailed Description:
VNX-101 is an investigational adeno-associated virus AAV gene therapy developed to express a secreted anti-CD19anti-CD3 scFv diabody termed GP101 GP101 binds both cluster of differentiation CD19 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 native T-cell receptor
In this 2-part study dose-finding data from Part 1 of the study n12 patients with marrow blasts 5 will be used determine the dose for Part 2 in patients at higher disease burden marrow blasts 50 Part 1 is a dose-finding PK study in adults 18 years old designed to determine the minimal dose that achieves target PK serum levels of GP101 at steady state 8-week timepoint without dose-limited toxicities defined as the recommended Part 2 dose RP2D Prior to VNX-101 dosing subjects may undergo standard of care chemotherapy to meet dosing criteria Part 2 n14 will be opened following data safety monitoring board review of Part 1 data and is designed to determine the safety and pharmacokinetics PK of VNX-101 at the RP2D in a broader array of subjects with higher leukemic burden ie bone marrow blasts 50 The age range for Part 2 will be expanded to include subjects 13 years old Patients will be followed for safety and efficacy up to 5 years post VNX-101 dosing Long-term follow-up assessments for safety will be conducted for 6 to 15 years post VNX-101 dosing
In this 2-part study dose-finding data from Part 1 of the study n12 patients with marrow blasts 5 will be used determine the dose for Part 2 in patients at higher disease burden marrow blasts 50 Part 1 is a dose-finding PK study in adults 18 years old designed to determine the minimal dose that achieves target PK serum levels of GP101 at steady state 8-week timepoint without dose-limited toxicities defined as the recommended Part 2 dose RP2D Prior to VNX-101 dosing subjects may undergo standard of care chemotherapy to meet dosing criteria Part 2 n14 will be opened following data safety monitoring board review of Part 1 data and is designed to determine the safety and pharmacokinetics PK of VNX-101 at the RP2D in a broader array of subjects with higher leukemic burden ie bone marrow blasts 50 The age range for Part 2 will be expanded to include subjects 13 years old Patients will be followed for safety and efficacy up to 5 years post VNX-101 dosing Long-term follow-up assessments for safety will be conducted for 6 to 15 years post VNX-101 dosing
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None