A Study of HY004 Treatment in Adult Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia (r/r B-ALL)

NCT06009107 | Withdrawn Phase 1 DMC

• 1 other identifier: HY004101
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a multi-center, phase I/II trial to evaluate the safety and efficacy of HY004 treatment in Adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-cell ALL).

Conditions

B-cell Acute Lymphoblastic Leukemia

Keywords

HY004; Cluster of differentiation antigen 19 and/or 22(CD19 and/or 22); CD19/22-directed CAR-T cells

Outcome Measures

Primary Outcomes (1)

• Overall Remission Rate (ORR)

  ORR is defined as Complete Remission (CR) and Complete Remission with Incomplete Blood Count Recovery (CRi) per NCCN classification, as determined by Independent Review Committee (IRC).

  at the end of Month 3

Secondary Outcomes (9)

• Overall Remission Rate (ORR)

  within 3 months

• Best overall response (BOR)

  up to 2 years

• Overall Remission Rate (ORR) with minimal residual disease (MRD) negativity

  at the end of Month 3

• Duration of remission (DOR)

  to data cutoff date

• Allogeneic Stem Cell Transplant (Allo-SCT) rate

  First infusion date of HY004 to data cutoff date(up to 2 years)

Other Outcomes (4)

• In vivo cellular Pharmacokinetic (PK) profile of HY004 in units of transgene copy number per genomic DNA (gDNA) amount.

  Up to 3 months(BM sample); Up to 2 years(Blood sample)

• In vivo cellular Pharmacokinetic (PK) profile of HY004 in units of percent of CAR-positive cells.

  Up to 3 months(BM sample); Up to 2 years(Blood sample)

• In vivo cellular pharmacodynamics (PD) profile of HY004.

  28 days

Study Arms (1)

Participant Group

EXPERIMENTAL

Participants with relapsed or refractory B-precursor acute lymphoblastic leukemia (r/r B-ALL) will receive conditioning chemotherapy (fludarabine 25-30 mg/m\^2 intravenously \[IV\] over 30 minutes on Day -5, Day -4, and Day -3 and cyclophosphamide 500 mg/m\^2 IV over 60 minutes on Day -5, Day -4), following a single IV infusion of chimeric antigen receptor (CAR) transduced autologous T cells(HY004).

Biological: HY004; Drug: Cyclophosphamide; Drug: Fludarabine Phosphate

Interventions

HY004 BIOLOGICAL

A single infusion of Autologous 2nd generation CD19/CD22-directed CAR-T cells administered intravenously.

Participant Group

Cyclophosphamide DRUG

Administered intravenously.

Participant Group

Fludarabine Phosphate DRUG

Administered intravenously.

Participant Group

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed written informed consent prior to any study procedures (patient and/or parent or legal guardian);
• Gender is not limited, and the age at the time of screening is ≥ 18 years old and ≤ 65 years old;
• Relapsed or refractory acute lymphoblastic leukemia (ALL);
• Documentation of CD19 and/orCD22 tumor expression demonstrated in bone marrow or peripheral blood within 3 months before screening;
• Bone marrow with ≥ 5% lymphoblasts by morphologic assessment at screening;
• ECOG score 0-1 points;
• Organ function requirements: All patients must have adequate renal and liver functions.

You may not qualify if:

• Active Central Nervous System (CNS) involvement by malignancy;
• Isolated extra-medullary disease relapse;
• Patients with Burkitt's lymphoma/leukemia;
• History of concomitant genetic syndrome;
• Patients with acute graft-versus-host disease (GVHD) or moderate-tosevere chronic GVHD within 4 weeks before screening; Patients with a history of allogeneic hematopoietic stem cell transplantation within 12 weeks before single collection;
• Active systemic autoimmune disease;
• Known infection with human immunodeficiency virus (HIV) or chronic infection with hepatitis B virus (HbsAg positive) or hepatitis C virus (anti- HCV positive);
• Patients with active infections at screening;
• Patients who have used CAR-T cell therapy before screening;
• Patients with an expected lifespan of less than 3 months.

Sponsors & Collaborators

• Juventas Cell Therapy Ltd.: lead

MeSH Terms

Conditions

Burkitt Lymphoma

Interventions

Cyclophosphamide; fludarabine phosphate

Condition Hierarchy (Ancestors)

Epstein-Barr Virus Infections; Herpesviridae Infections; DNA Virus Infections; Virus Diseases; Infections; Tumor Virus Infections; Lymphoma, B-Cell; Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Officials

• Jianxiang Wang M.D.

  Study Principal Investigator Institute of Hematology & Blood Diseases Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 18, 2023
• First Posted: August 24, 2023
• Study Start: June 30, 2025
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): December 30, 2027
• Last Updated: August 8, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share