Gene Therapy For B-Cell Acute Lymphoblastic Leukemia

Overview

About this study

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).

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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
* AAV specified capsid 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

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 3/20/2025. Questions regarding updates should be directed to the study team contact.
 

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Kebede Begna, M.D.

Contact us for the latest status

Contact information:

Cancer Center Clinical Trials Referral Office

(855) 776-0015

Rochester, Minn.

Mayo Clinic principal investigator

Antoine Saliba, M.D.

Contact us for the latest status

Contact information:

Cancer Center Clinical Trials Referral Office

(855) 776-0015

More information

Publications

Publications are currently not available