The Safety and Efficacy of VGO-Cs01p in Patients With CD7-positive Relapsed/Refractory Acute T-lymphoblastic Leukemia

NCT06849401 | Not Yet Recruiting Early Phase 1

• 1 other identifier: VGO-Cs01p-001
• Study Type: interventional
• Target: 9
• Locations: 0 countries
• Sites: N/A

Brief Summary

To learn if the VGO-Cs01p can help to control CD7-positive relapsed/refractory acute T-lymphoblastic leukemia (R/R T-ALL) in children.

Conditions

T-ALL

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events

  An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.

  1 year post the first VGO-Cs01p infusion

Secondary Outcomes (11)

• ORR（Objective response rate）

  28 days and 1 year post the first VGO-Cs01p infusion

• MRD (Minimal/Measurable Residual Diseas)

  1 year post the first VGO-Cs01p infusion

• DOR (Duration of response)

  1 year post the first VGO-Cs01p infusion

• LFS (Leukemia-Free Survival)

  1 year post the first VGO-Cs01p infusion

• OS (Overall survival)

  1 year post the first VGO-Cs01p infusion

Study Arms (1)

VGO-Cs01p

EXPERIMENTAL

Up to three sequential VGO-Cs01p dose levels （4e7、1.2e8、3.6e8 CAR-NK cells/kg）are planned. Each subject will accept six doses of VGO-Cs01p

Biological: VGO-Cs01p

Interventions

VGO-Cs01p BIOLOGICAL

Off-the-shelf NK cell products derived from human embryonic stem cells (hESCs)

VGO-Cs01p

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age ≥2 and ≤18 years old, male or female;
• Subjects who have relapse or refractory T-cell lymphoblastic leukemia (T-ALL) according to the standards of the NCCN Clinical Practice Guidelines for Acute Lymphoblastic Leukemia (2024.V6);
• Meets the criteria for recurrent or refractory T-ALL, including: a) Recurrent: Reappearance of blasts in peripheral blood or bone marrow (\>25%) after complete remission or occurrence of extramedullary disease, and ineffectiveness of other treatments； b) Primary Refractory: Appearance of blasts in bone marrow ≥5% after 2 months standard induction chemotherapy, and no other treatment can be used as judged by the investigator;
• After one cycle of other treatments (such as Olverembatinib combined with APG-125), the blasts remain≥5%;
• Cell immunophenotyping confirmation of CD7 positive blasts \>80%;
• Estimated survival period \>12 weeks;
• Eastern Cooperative Oncology Group (ECOG) performance status score ≤1 or KPS \> 60;
• Left ventricular ejection fraction ≥50%;
• Pulmonary function ≤ Grade 1 dyspnea (CTCAE v5.0), normal oxygen saturation without oxygen supplementation;
• TBil ≤ 3×ULN, AST and ALT ≤ 5×ULN, creatinine ≤ 1.6 mg/dl within 1 week prior to enrollment;
• Negative serum pregnancy test for fertile women; fertile non-abstinent female patients must agree to use an effective contraceptive method from screening to 1 year after cell infusion. Fertile male patients' partners must agree to use effective contraception from screening to 1 year after cell infusion, and should not donate semen or sperm throughout the study;
• The subject or their legal guardian voluntarily participates in the study, understands the information, purpose, and risks described in the informed consent form, and can provide a signed and dated informed consent form;
• The subject and/or their parents or their legal guardian should voluntary and able to comply with all requirements of the trial.

You may not qualify if:

• Extramedullary involvement of the central nervous system or testicular at screening.
• Patients with a history of severe CNS diseases, such as uncontrolled seizures, stroke, severe brain damage resulting in speech impairment, psychiatric disorders, etc;
• NYHA functional class III or IV heart failure;
• Presence of disseminated intravascular coagulation;
• Presence of severe autoimmune diseases or immune deficiency diseases;
• Active GVHD requiring systemic treatment;
• Presence of other severe diseases, presence of gastrointestinal ulcers or active gastrointestinal bleeding, currently undergoing anticoagulant or antiplatelet therapy, or judged by the investigator to pose unacceptable surgical or anesthesia risks;
• Currently receiving systemic steroids or other immunosuppressive therapy prior to screening, and still need long-term use after enrollment as judged by the investigator (excluding inhaled or local use);
• History or concurrent active malignant tumors within 3 years prior to enrollment;
• Active HBV or HCV infection (HBV-DNA positive or HCV-RNA positive), HIV positive, or positive syphilis test;
• Other severe or persistent active infections;
• Adverse events related to previous systemic immunotherapy (including other investigational drugs or medical device interventions) prior to enrollment have not reduced to grade 1 severity or returned to baseline;
• Platelet count remains low after intervention treatment (meeting clinical transfusion criteria) prior to enrollment；
• Discontinuation of immunosuppressive agents for less than 2 weeks;
• Participation in CAR-T cell therapy or gene therapy at any time prior to screening;

Sponsors & Collaborators

• Shanghai Jiao Tong University School of Medicine: lead
• Shanghai Vitalgen BioPharma Co., Ltd.: collaborator

MeSH Terms

Conditions

Precursor T-Cell Lymphoblastic Leukemia-Lymphoma

Condition Hierarchy (Ancestors)

Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Central Study Contacts

Hui Zhang

CONTACT

15821333007; zhang-hui@scmc.com.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief Physician

Study Record Dates

• First Submitted: February 21, 2025
• First Posted: February 27, 2025
• Study Start: July 1, 2025
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: May 15, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will share