Inaticabtagene Autoleucel Injection in the Treatment of Autoimmune Hemolytic Anemia After Three or More Lines of Therapy

NCT07091370 | Not Yet Recruiting Phase 1

• 1 other identifier: HY001106
• Study Type: interventional
• Target: 12
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a multicenter, open-label, single-arm Phase I clinical trial to evaluate the safety and tolerability of Inaticabtagene Autoleucel Injection treatment in autoimmune hemolytic anemia that has failed at least three lines of treatment.

Conditions

AIHA - Cold Autoimmune Hemolytic Anemia

Keywords

AIHA; CarT

Outcome Measures

Primary Outcomes (2)

• Incidence of Treatment-related Adverse Events

  Therapy-related adverse events (AE), including severe adverse events (SAE) and laboratory outliers with clinical significance, will be recorded and assessed according to the Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0)

  Up to 28 days post-infusion

• The safe dosage for a single infusion of Inaticabtagene Autoleucel Injection

  The safe dosage for AIHA patients will be evaluated by comprehensively assessing the Overall Response Rate (ORR) and the incidence of adverse events (AEs).

  Up to 28 days post-infusion

Secondary Outcomes (3)

• Overall Remission Rate (ORR)

  Sixs months after cell reinfusion

• Changes in laboratory test indicators

  Up to 24 months post-infusion

• Drug-free Remission (DFR)

  Up to 24 months post-infusion

Study Arms (1)

Inaticabtagene Autoleucel Injection

EXPERIMENTAL

A conditioning chemotherapy regimen of fludarabine and cyclophosphamide will be administered followed by investigational treatment, Inaticabtagene Autoleucel Injection.

Drug: Inaticabtagene Autoleucel Injection

Interventions

Inaticabtagene Autoleucel Injection DRUG

The dose was incremented according to the "3+3" principle, and the three dose levels A, B and C were given in sequence at one time, which were respectively: A-1 dose group: 0.25×10\^6 CAR-T live cells /kg body weight Group A (initial dose) : 0.5×10\^6 CAR-T live cells /kg body weight Group B: 1.0×10\^6 CAR-T live cells /kg body weight Group C: 1.5×10\^6 CAR-T live cells /kg body weight

Inaticabtagene Autoleucel Injection

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age range: 18-75 years (including 18 and 75 years), regardless of gender.
• Clinically diagnosed with AIHA or Evans, with hemoglobin lower than 100g/L and symptoms related to anemia according to the diagnostic criteria of the "Diagnosis and Treatment Guidelines for Autoimmune Hemolytic Anemia in Chinese Adults (2023 Edition)" and the "Chinese Expert Consensus on the Diagnosis and Treatment of Evans Syndrome (2024 Edition)". And meet the diagnostic criteria of failure in at least three lines of treatment as follows:
• Patients diagnosed with warm resistance type AIHA (wAIHA)/mixed AIHA (mAIHA) /Evans syndrome, the following conditions must be met: first-line glucocorticoid treatment failed, second-line rituximab treatment failed and failure of third-line treatment measures (including any one or more of splenectomy, cyclosporine, cyclophosphamide, azathioprine, mycophenolate mofetil, fludarabine, bortezomib, etc.) Patients diagnosed with cold-resistant type AIHA (cAIHA), that is, cold agglutinin disease (CAD), the following conditions must be met: first-line rituximab treatment failed, second-line rituximab ± bendamustine/fludarabine failed and failure of third-line treatment measures (including any one or more of bortezomib, cyclosporine, cyclophosphamide, mycophenolate mofetil, azathioprine, etc.)
• Women of childbearing potential must have a negative blood pregnancy test during the screening period. Any male and female patients of childbearing potential must agree to use an effective method of contraception throughout the study and for at least 2 year following infusion of CAR-T cells. Childbearing potential is biologically capable of bearing a living baby and sexually active. Female patients who were not of childbearing potential (ie, met at least 1 of the following criteria):
• Hysterectomy or oophorectomy, or medically confirmed ovarian failure, or medically confirmed postmenopausal (cessation of menses for at least 12 consecutive months in the absence of pathological or physiological causes).
• Adequate organ function according to the following criteria:
• Aspartate aminotransferase (AST) ≤ 3 times of upper limit of normal (ULN); Alanine aminotransferase (ALT) ≤ 3 times ULN; Serum creatinine ≤ 1.5 times ULN, or creatinine clearance ≥ 60 mL/min (Cockcroft and Gault formula); International normalized ratio (INR) ≤ 1.5 times ULN, and activated partial thromboplastin time (APTT) ≤ 1.5 times the ULN; Must have minimal pulmonary reserve and oxygen saturation \> 93% in a nonoxygenated state;
• The Eastern Cancer Consortium (ECOG) performance status score is 0-2.

You may not qualify if:

• Previously diagnosed proliferative tumors of the lymphatic system;
• Secondary AIHA caused by drugs or infections;
• Pregnancy or breastfeeding;
• Previously undergone organ or hematopoietic stem cell transplantation;
• A history of new thrombosis or organ infarction within 6 months prior to screening;
• The active stage diagnosed as a connective tissue disease;
• Suffering from hereditary hemolytic diseases or other acquired hemolytic diseases;
• Combined with active infections (such as sepsis, bacteremia, mycosis, uncontrolled pulmonary infection and active tuberculosis, etc.);
• Positive for hepatitis B surface antigen (HBsAg) and/or hepatitis Be antigen (HBeAg); Hepatitis Be antibody (HBe-Ab) and/or hepatitis B core antibody (HBc-Ab) are positive, and the HBV-DNA copy number is greater than the measurable lower limit; Positive for hepatitis C (HCV) antibody; Positive for human immunodeficiency virus (HIV) antibody; Those with positive syphilis (TP) test;
• Major surgery that was assessed as unsuitable by the investigators within 4 weeks before screening;
• Patients with concurrent active malignancy within the past five years, those with a history of malignancy but cuired are eligible.
• The patient's heart meets any of the following conditions:
• Left ventricular ejection fraction (LVEF) ≤45%;
• New York Heart Association (NYHA) grade III or IV congestive heart failure or active heart disease;
• Severe arrhythmias requiring treatment (excluding atrial fibrillation and paroxysmal supraventricular tachycardia);

Sponsors & Collaborators

• Juventas Cell Therapy Ltd.: lead

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Jun Shi, Dr.

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Junyin Yu

CONTACT

13920424844; Yujunyin@juventas.cn

Xiaofeng Pan, Dr.

CONTACT

panxiaofeng@juventas.cn

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: July 21, 2025
• First Posted: July 29, 2025
• Study Start: December 27, 2025
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): March 30, 2028
• Last Updated: September 22, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share