Clinical trial NCT06417398: Preliminary Clinical Study of UTAA09 Injection in the Treatment of Relapsed/Refractory Autoimmune Diseases
Official title: Preliminary Clinical Study of UTAA09 Injection in the Treatment of Relapsed/Refractory Autoimmune Diseases
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: EARLY_PHASE1
Conditions: Systemic Lupus Erythematosus
Idiopathic Inflammatory Myopathies
Systemic Sclerosis
Sjogren's Syndrome
Rheumatoid Arthritis
Immune Thrombocytopenia
Primary Biliary Cholangitis
Lead sponsor: PersonGen BioTherapeutics (Suzhou) Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2024-05-16
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To evaluate the safety of UTAA09 injection in the treatment of relapsed/refractory (R/R) autoimmune disease (AID).

To evaluate the pharmacokinetic (PK) profile of UTAA09 injection in patients with R/R AID.

To evaluate the pharmacodynamic (PD) characteristics of UTAA09 injection in patients with R/R AID.

To evaluate the initial efficacy of UTAA09 injection in the treatment of R/R AID subjects.

To evaluate the immunogenicity of UTAA09 injection in R/R AID subjects.

Detailed description: This clinical trial was designed as a single-arm, open-label, single-center, investigator-initiated early-stage clinical study to evaluate the safety of UTAA09 injection in patients with relapsed/refractory AID. After signing the informed consent letter, qualified subjects were screened for infusion of UTAA09 injection, and their blood was collected before and after infusion for pharmacokinetics, pharmacodynamics, immunogenicity, safety and other evaluation. In addition to the baseline period, therapeutic efficacy was evaluated at a frequency of 28d, 2m, 3m, 4m, 5m, 6m, 8m, 10m, 12m after cell transfusion, and tumors were evaluated until disease progression (PD), new anti-disease therapy, death, intolerable toxicity, investigator decision, or subject's voluntary withdrawal, whichever occurred first.

All adverse events were recorded from the beginning of the subject's elutriation pre-treatment (if it occurred) until 3 months after the subject received cell transfusion or disease progression/recurrence or initiation of a new anti-disease therapy or the end of the study, whichever occurred first, and 3 months after cell transfusion or disease progression/recurrence or initiation of a new anti-disease therapy (whichever occurred first) until the end of the study. Only adverse events associated with the study product were collected

Eligibility criteria: Inclusion Criteria:

(2) Expected survival time ≥3 months; (3) Subjects with recurrent/refractory autoimmune disease who have failed standard treatment or lack effective treatment, including but not limited to systemic lupus erythematosus, idiopathic inflammatory myopathy, systemic sclerosis, Sjogren's syndrome, rheumatoid arthritis, connective tissue disease-associated interstitial lung disease, immune thrombocytopenia, primary biliary cholangitis, etc.

(4) Liver and kidney function, cardiopulmonary function meet the following requirements:

* Creatinine ≤1.5×ULN; (2) Electrocardiogram showed no clinically significant abnormal bands;

  * Blood oxygen saturation \>91% in non-oxygen state;

    * Total bilirubin ≤2×ULN; ALT and AST≤2.5 x ULN; ALT and AST abnormalities due to disease, such as liver infiltration or bile duct obstruction, were determined to be less than 5×ULN. If Gilbert syndrome is diagnosed, the total bilirubin index can be relaxed to ≤3.0×ULN and the direct bilirubin ≤1.5×ULN.

      (5) no serious mental disorders; (6) Can understand this test and have signed the informed consent.

Exclusion Criteria:

1. Malignant tumors other than R/R AID disease in the 5 years prior to screening, except for adequately treated cervical carcinoma in situ, basal cell or squamous cell skin cancer, local prostate cancer after radical surgery, and breast ductal carcinoma in situ after radical surgery;
2. Hepatitis B surface antigen (HBsAg) positive; Hepatitis B core antibody (HBcAb) positive and peripheral blood hepatitis B virus (HBV) DNA titer detection is not within the normal reference value range; Hepatitis C virus (HCV) Antibody positive and peripheral blood hepatitis C virus (HCV) RNA positive; Human immunodeficiency virus (HIV) Antibody positive; Syphilis positive;
3. Serious heart disease, including but not limited to unstable angina, myocardial infarction or bypass or stent surgery (within 6 months prior to screening), congestive heart failure (NYHA classification ≥III), and severe arrhythmia;
4. Systemic diseases that are deemed unstable by researchers: including but not limited to severe liver, kidney, or metabolic diseases that require drug treatment;
5. Active or uncontrollable infections (except mild genitourinary and upper respiratory tract infections) that require systemic treatment within 7 days prior to administration;
6. Pregnant or lactating women, and female subjects who plan pregnancy within 2 years after cell transfusion or male subjects whose partners plan pregnancy within 2 years after cell transfusion;
7. Patients who received CAR-T therapy or other gene-modified cell therapy before screening;
8. Participated in other clinical studies 1 month before screening;
9. Evidence of central nervous system invasion during subject screening;
10. Mental patients with depression or suicidal thoughts;
11. Those who received live vaccine within 28 days prior to screening;
12. Situations considered unsuitable for inclusion by other researchers.

Primary outcomes:
measure: AE
timeFrame: 3 months after cell reinfusion or disease progression/recurrence or start of new anti-disease therapy (whichever occurs first)
Secondary outcomes:
measure: Peak Plasma Concentration (Cmax）
timeFrame: 3 months after cell reinfusion disease progression/recurrence or start of new anti-disease therapy (whichever occurs first)
measure: Area under the plasma concentration versus time curve (AUC)
timeFrame: 3 months after cell reinfusion/28d after cell reinfusion
measure: Content of CD19 positive cells in peripheral blood
timeFrame: every visits after infusion up to 2 years
Other outcomes:
Locations:
Study officials:
affiliation: The First Affiliated Hospital of Soochow University
name: jian wu, Doctor
role: PRINCIPAL_INVESTIGATOR
References: