- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT07715630
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.
Studieoversikt
Status
Intervensjon / Behandling
Detaljert beskrivelse
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.
Studietype
Registrering (Antatt)
Fase
- Fase 1
Kontakter og plasseringer
Studiekontakt
- Navn: Sanbin Wang, PhD
- Telefonnummer: +86 13187424131
- E-post: sanbin1011@163.com
Studiesteder
-
-
Yunnan
-
Kunming, Yunnan, Kina
- Rekruttering
- 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
-
Ta kontakt med:
- Sanbin Wang, PhD
- Telefonnummer: +86 13187424131
- E-post: sanbin1011@163.com
-
-
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Voksen
- Eldre voksen
Tar imot friske frivillige
Beskrivelse
Inclusion Criteria:
- Age ≥ 18 years, male or female;
Confirmed diagnosis of multiple myeloma meeting at least one of the following criteria:
- Disease progression after at least 2 prior standard treatment regimens; or poor response to primary therapeutic agents (e.g., immunomodulatory agents, proteasome inhibitors)
- Disease progression within 18 months after first-line therapy
- Presence of features associated with high risk of disease relapse or progression (e.g., high-risk cytogenetic abnormalities);
At least one measurable disease indicator:
- Serum M-protein ≥ 0.5 g/dL
- Urine M-protein ≥ 200 mg/24 hours
- Involved serum free light chain (sFLC) ≥ 10 mg/dL with an abnormal serum free light chain κ/λ ratio
- No evidence of extramedullary plasmacytoma (soft tissue plasmacytoma);
- ECOG performance status score 0-2;
- Expected survival period ≥ 3 months;
Adequate bone marrow function within 1 month prior to screening:
- Hemoglobin ≥ 60 g/L;
- Absolute neutrophil count (ANC) ≥ 0.5 × 10⁹/L;
- Platelet count (PLT) ≥ 50 × 10⁹/L;
- Lymphocyte count ≥ 0.5 × 10⁹/L;
- CD3-positive T-cell absolute count ≥ 0.15 × 10⁹/L;
Adequate vital organ function within 1 month prior to screening:
- 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);
- 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);
- 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);
- Pulmonary function: blood oxygen saturation (SpO₂) ≥ 90% without supplemental oxygen;
- 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;
- 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;
- 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:
- Prior treatment with CAR-T therapy or other gene-modified cell therapy before screening;
- 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;
Received the following anti-tumor therapies prior to PICX Injection infusion:
- 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);
- Radiotherapy to non-hematopoietic sites within 7 days, or to hematopoietic sites within 14 days before infusion;
- BCMA-targeting antibody-based therapy within 3 months before infusion;
- Active or uncontrolled infection requiring systemic treatment at screening (including bacterial, viral, fungal, or other infections);
Presence of any of the following cardiac conditions:
- New York Heart Association (NYHA) Class III or IV congestive heart failure;
- Myocardial infarction, or coronary artery bypass grafting (CABG), or coronary stent placement within 6 months prior to screening;
- Clinically significant ventricular arrhythmia, or history of syncope of unknown cause (excluding vasovagal or dehydration-related);
- History of severe non-ischemic cardiomyopathy;
Presence of other clinically significant diseases or conditions, including:
- Primary immunodeficiency disease;
- Cerebrovascular accident or seizure within 6 months prior to screening;
- Definite cognitive impairment or psychiatric/behavioral abnormalities (e.g., dementia, altered mental status), or severe psychiatric disorders;
- Parkinson's disease, Parkinsonism, or other movement disorders;
- Grade 2-4 acute graft-versus-host disease (GVHD) or moderate to severe chronic GVHD within 4 weeks prior to screening;
History of other malignancies other than multiple myeloma prior to screening, except for:
- Malignancies treated with curative intent with no known active disease for ≥ 2 years prior to enrollment;
- 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;
- Vaccination with live-attenuated vaccine within 4 weeks prior to screening;
- Known severe hypersensitivity to PICX Injection or any of its formulation components;
- Inability to establish venous access;
- Other conditions deemed by the investigator to be unsuitable for participation in the study.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: In vivo CAR-T
|
Pasienter ble inkludert og gitt en enkelt dose CAR-T-injeksjon intravenøst, innlagt for observasjon i løpet av den påfølgende måneden, og fulgt opp for observasjon i løpet av de påfølgende 2 årene.
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Maximal Tolerated Dose (MTD)
Tidsramme: Up to 28 days after infusion
|
MTD will be determined based on Dose-Limiting Toxicity (DLTs) observed during the first 28 days of study treatment.
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Up to 28 days after infusion
|
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Incidence of Adverse Events (AE) after infusion
Tidsramme: Up to 28 days after infusion
|
The frequency, severity, and laboratory findings of all adverse events/serious adverse events are included.
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Up to 28 days after infusion
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Objective Response Rate (ORR)
Tidsramme: Day 28#Month 2#Month 3#Month 6#Month 12#Month 18#Month 24
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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
|
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Minimal Residual Disease (MRD) Negative Rate
Tidsramme: Month 3#Month 6#Month 12#Month 18#Month 24
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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
|
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Time to Response (TTR)
Tidsramme: Up to 24 months
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TTR is defined as the time from CAR-T infusion to first documented response (PR or better).
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Up to 24 months
|
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Duration of Response (DOR)
Tidsramme: up to 24 months
|
DOR is defined as the time from first documented response (PR or better) to first documented disease progression or death from any cause.
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up to 24 months
|
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Progression-Free Survival (PFS)
Tidsramme: up to 24 months
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PFS is defined as the time from CAR-T infusion to first documented disease progression or death from any cause.
|
up to 24 months
|
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Overall Survival (OS)
Tidsramme: up to 24 months
|
OS is defined as the time from CAR-T infusion to death from any cause.
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up to 24 months
|
Samarbeidspartnere og etterforskere
Studierekorddatoer
Studer hoveddatoer
Studiestart (Antatt)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
- Vaskulære sykdommer
- Kardiovaskulære sykdommer
- Patologiske prosesser
- Neoplasmer
- Sykdomsattributter
- Sykdommer i immunsystemet
- Neoplasmer etter histologisk type
- Hematologiske sykdommer
- Lymfoproliferative lidelser
- Immunproliferative lidelser
- Neoplasmer, plasmacelle
- Hemostatiske lidelser
- Paraproteinemier
- Blodproteinforstyrrelser
- Hemoragiske lidelser
- Patologiske tilstander, tegn og symptomer
- Hemic og lymfatiske sykdommer
- Tilbakefall
- Multippelt myelom
Andre studie-ID-numre
- PBC027-2
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
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