In Vivo PICX CAR-T Therapy for R/R Multiple Myeloma

In Vivo Prepared PICX Chimeric Antigen Receptor T-Cell Therapy for the Treatment of Relapsed/Refractory Multiple Myeloma

This study is an investigator-initiated, single-center, single-arm clinical study with a target population of patients with relapsed or refractory multiple myeloma.

It is an early exploratory clinical study evaluating the safety, tolerability, and preliminary efficacy of PICX Injection, an in vivo prepared CAR-T cell therapy, in the treatment of relapsed or refractory multiple myeloma.

Study Overview

• Status: Recruiting
• Conditions: Relapsed or Refractory Multiple Myeloma (RRMM)
• Intervention / Treatment: Biological: Invivo CAR-T

Detailed Description

This is a prospective, open-label, single-arm clinical investigation to assess the safety and efficacy of in vivo CAR-T cell therapy in patients with relapsed or refractory multiple myeloma.

Eligible subjects will be treated with PICX Injection as a single intravenous infusion. Post-infusion, subjects will remain hospitalized for close observation and undergo serial evaluations for adverse events and treatment response. Long-term follow-up will continue for up to 2 years to assess disease status and durability of response.

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Sanbin Wang, PhD; Phone Number: +86 13187424131; Email: sanbin1011@163.com

Study Locations

• China
  • Yunnan
    • Kunming, Yunnan, China
      • Recruiting
      • 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
      • Contact: Sanbin Wang, PhD; Phone Number: +86 13187424131; Email: sanbin1011@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age ≥ 18 years, male or female;

2. Confirmed diagnosis of multiple myeloma meeting at least one of the following criteria:

   1. Disease progression after at least 2 prior standard treatment regimens; or poor response to primary therapeutic agents (e.g., immunomodulatory agents, proteasome inhibitors)
   2. Disease progression within 18 months after first-line therapy
   3. Presence of features associated with high risk of disease relapse or progression (e.g., high-risk cytogenetic abnormalities);

3. At least one measurable disease indicator:

   1. Serum M-protein ≥ 0.5 g/dL
   2. Urine M-protein ≥ 200 mg/24 hours
   3. Involved serum free light chain (sFLC) ≥ 10 mg/dL with an abnormal serum free light chain κ/λ ratio
4. No evidence of extramedullary plasmacytoma (soft tissue plasmacytoma);
5. ECOG performance status score 0-2;
6. Expected survival period ≥ 3 months;

7. Adequate bone marrow function within 1 month prior to screening:

   1. Hemoglobin ≥ 60 g/L;
   2. Absolute neutrophil count (ANC) ≥ 0.5 × 10⁹/L;
   3. Platelet count (PLT) ≥ 50 × 10⁹/L;
   4. Lymphocyte count ≥ 0.5 × 10⁹/L;
   5. CD3-positive T-cell absolute count ≥ 0.15 × 10⁹/L;

8. Adequate vital organ function within 1 month prior to screening:

   1. Renal function: creatinine clearance rate (CrCl) ≥ 30 mL/min (calculated using the Cockcroft-Gault formula), or serum creatinine (Scr) ≤ 2.0 × upper limit of normal (ULN);
   2. Hepatic function: alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3.0 × ULN; total bilirubin (TBIL) ≤ 2.0 × ULN (except for patients with congenital hyperbilirubinemia such as Gilbert's syndrome, in which case direct bilirubin may be ≤ 1.5 × ULN);
   3. Cardiac function: left ventricular ejection fraction (LVEF) ≥ 40%; no clinically significant pericardial effusion; and no clinically significant electrocardiogram (ECG) abnormalities (e.g., severe arrhythmia, myocardial ischemia, conduction block);
   4. Pulmonary function: blood oxygen saturation (SpO₂) ≥ 90% without supplemental oxygen;
9. Women of childbearing potential must have a negative pregnancy test during the screening period and before study drug administration, and must not be lactating during the study;
10. Men and women of childbearing potential must agree to use effective contraceptive measures (excluding unreliable methods such as rhythm method) from the time of signing the informed consent form until 1 year after the last dose of study drug, and must agree not to donate sperm or eggs;
11. The subject or their legally authorized representative has signed the informed consent form (ICF), indicating understanding of the study purpose and procedures and voluntary participation.

Exclusion Criteria:

1. Prior treatment with CAR-T therapy or other gene-modified cell therapy before screening;
2. Presence of active central nervous system (CNS) involvement at screening (including brain parenchymal, meningeal, or spinal meningeal involvement, or positive cerebrospinal fluid for tumor cells), or other CNS diseases;

3. Received the following anti-tumor therapies prior to PICX Injection infusion:

   1. Chemotherapy, combination therapy with proteasome inhibitors and immunomodulatory agents, or other systemic anti-tumor drug therapy within 14 days or at least 5 half-lives before infusion (excluding intrathecal chemotherapy, which must be discontinued at least 1 week prior to infusion);
   2. Radiotherapy to non-hematopoietic sites within 7 days, or to hematopoietic sites within 14 days before infusion;
   3. BCMA-targeting antibody-based therapy within 3 months before infusion;
4. Active or uncontrolled infection requiring systemic treatment at screening (including bacterial, viral, fungal, or other infections);

5. Presence of any of the following cardiac conditions:

   1. New York Heart Association (NYHA) Class III or IV congestive heart failure;
   2. Myocardial infarction, or coronary artery bypass grafting (CABG), or coronary stent placement within 6 months prior to screening;
   3. Clinically significant ventricular arrhythmia, or history of syncope of unknown cause (excluding vasovagal or dehydration-related);
   4. History of severe non-ischemic cardiomyopathy;

6. Presence of other clinically significant diseases or conditions, including:

   1. Primary immunodeficiency disease;
   2. Cerebrovascular accident or seizure within 6 months prior to screening;
   3. Definite cognitive impairment or psychiatric/behavioral abnormalities (e.g., dementia, altered mental status), or severe psychiatric disorders;
   4. Parkinson's disease, Parkinsonism, or other movement disorders;
7. Grade 2-4 acute graft-versus-host disease (GVHD) or moderate to severe chronic GVHD within 4 weeks prior to screening;

8. History of other malignancies other than multiple myeloma prior to screening, except for:

   1. Malignancies treated with curative intent with no known active disease for ≥ 2 years prior to enrollment;
   2. Adequately treated cervical carcinoma in situ, basal cell or squamous cell skin carcinoma, localized prostate cancer after radical surgery, or ductal carcinoma in situ after radical surgery;
9. Vaccination with live-attenuated vaccine within 4 weeks prior to screening;
10. Known severe hypersensitivity to PICX Injection or any of its formulation components;
11. Inability to establish venous access;
12. Other conditions deemed by the investigator to be unsuitable for participation in the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Invivo CAR-T	Biological: Invivo CAR-T; Patients were enrolled and given a single dose of CAR-T injection intravenously, hospitalized for observation over the following month, and followed up for observation over the following 2 years.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximal Tolerated Dose (MTD)	MTD will be determined based on Dose-Limiting Toxicity (DLTs) observed during the first 28 days of study treatment.	Up to 28 days after infusion
Incidence of Adverse Events (AE) after infusion	The frequency, severity, and laboratory findings of all adverse events/serious adverse events are included.	Up to 28 days after infusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response Rate (ORR)	ORR is defined as the proportion of subjects achieving stringent complete remission (sCR), complete remission (CR), very good partial response (VGPR), and partial response (PR) per IMWG response criteria (2016).	Day 28#Month 2#Month 3#Month 6#Month 12#Month 18#Month 24
Minimal Residual Disease (MRD) Negative Rate	MRD negativity rate is defined as the proportion of subjects achieving MRD-negative status as assessed by next-generation flow cytometry or sequencing.	Month 3#Month 6#Month 12#Month 18#Month 24
Time to Response (TTR)	TTR is defined as the time from CAR-T infusion to first documented response (PR or better).	Up to 24 months
Duration of Response (DOR)	DOR is defined as the time from first documented response (PR or better) to first documented disease progression or death from any cause.	up to 24 months
Progression-Free Survival (PFS)	PFS is defined as the time from CAR-T infusion to first documented disease progression or death from any cause.	up to 24 months
Overall Survival (OS)	OS is defined as the time from CAR-T infusion to death from any cause.	up to 24 months

Collaborators and Investigators

• Sponsor: Chongqing Precision Biotech Co., Ltd

Study record dates

Study Major Dates

• Study Start (Estimated): July 31, 2026
• Primary Completion (Estimated): April 30, 2028
• Study Completion (Estimated): July 31, 2028

Study Registration Dates

• First Submitted: July 14, 2026
• First Submitted That Met QC Criteria: July 17, 2026
• First Posted (Actual): July 20, 2026

Study Record Updates

• Last Update Posted (Actual): July 20, 2026
• Last Update Submitted That Met QC Criteria: July 17, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: CAR-T; malignant hematological tumors; in Vivo
• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Pathologic Processes; Neoplasms; Disease Attributes; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Neoplasms, Plasma Cell; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Hemorrhagic Disorders; Pathological Conditions, Signs and Symptoms; Hemic and Lymphatic Diseases; Recurrence; Multiple Myeloma
• Other Study ID Numbers: PBC027-2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No