En undersøgelse af YTS109-celleinjektion hos forsøgspersoner med recidiverende/refraktær autoimmun hæmolytisk anæmi
Sikkerheden og effektiviteten af YTS109-celleinjektion til patienter med recidiverende/refraktær autoimmun hæmolytisk anæmi efter at have modtaget tre eller flere behandlingslinjer.
This is a Phase I, single-arm, open-label, dose-escalation and dose-expansion study. The primary objective is to evaluate the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of YTS109 START T-cell therapy in patients with autoimmune hemolytic anemia who have failed ≥3 lines of therapy.
Studieoversigt
Status
Status
Betingelser
Betingelser
Intervention / Behandling
Intervention / Behandling
Undersøgelsestype
Undersøgelsestype
Tilmelding (Anslået)
Tilmelding
Fase
Fase
- Fase 1
Kontakter og lokationer
Studiesteder
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Tianjin Municipality
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Tianjin, Tianjin Municipality, Kina, 301617
- Regenerative Medicine Center
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Deltagelseskriterier
Berettigelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria:
- Age ≥12 years, regardless of gender.
- Diagnosis of AIHA or Evans syndrome [including warm antibody, mixed AIHA and cold antibody AIHA (Cold agglutinin disease)].
- Failure or intolerance to at least 3 lines of therapy: glucocorticoids and/or rituximab, and any one of the following treatments (splenectomy, cyclosporine, cyclophosphamide, azathioprine, mycophenolate mofetil, bendamustine, fludarabine, bortezomib, etc.Biologics, including anti-CD38 monoclonal antibody, BTK inhibitor, Syk inhibitor and complement inhibitor) (HGB < 100g/L).
- Adequate organ function: a. Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3×ULN. b. Creatinine clearance (CrCl) (Cockcroft-Gault formula) ≥60ml/min. c.Blood oxygen saturation (SpO2) ≥92%.
- ECOG performance status≤2
- Subjects of childbearing potential will be required to follow contraception requirements from the time of enrollment until the end of the 12-month safety follow-up period.
- The subjects voluntarily participate in the study, sign the informed consent, demonstrate good compliance, and cooperate with follow-up.
Exclusion Criteria:
- Diagnosis of lymphoproliferative tumor
- Other hereditary or acquired hemolytic diseases (Secondary AIHA caused by drugs or infection)
- The platelet count in peripheral blood<30×10^9/L
- Pregnant or breast-feeding subjects
- Receive any of the following treatments within the specified time before cell infusion: a.anti-CD20 monoclonal antibodies <12 weeks, b.sutimlimab or other marketed biologics <5 half-lives,c.plasma exchange <4 weeks, d.post-splenectomy <12 weeks, e. BTK inhibitors, anti-CD38 monoclonal antibody, Syk inhibitors, BAFF inhibitors < 5 half-lives.
- Previously received organ or stem cell transplantation
- History of new thrombosis or organ infarction in the past 6 months
- Diagnosis of the active stage of the connective tissue disease.
- Have active infections, such as sepsis, bacteremia, fungemia, uncontrolled pulmonary infection and active tuberculosis, etc.
- Positive hepatitis B surface antigen (HBsAg) or hepatitis B e antigen (HBeAg); positive hepatitis B e antibody (HBe-Ab) or hepatitis B core antibody (HBc-Ab), and the HBV-DNA copy number is above the lower limit of the measurable capacity; positive hepatitis C (HCV) antibody; positive human immunodeficiency virus (HIV) antibody; positive syphilis test.
- Underwent major surgery within 4 weeks before screening, as determined by the investigator to be unsuitable for enrollment.
- Have malignant tumors within 5 years before enrollment, except tumors with negligible risk of metastasis or death and curable tumors, such as adequately treated cervical carcinoma in situ, cutaneous basal cell carcinoma, etc.
- Have any of the following cardiovascular diseases: a.Left ventricular ejection fraction (LVEF) ≤45%, b. presence of active heart disease or congestive heart failure (New York Heart Association [NYHA] Class III or IV)), c.severe arrhythmias requiring treatment, d.have myocardial infarction, bypass surgery, or stent placement within the 6 months before the study, e.other heart diseases judged by the researcher to be unsuitable for enrollment.
- Have a history of live attenuated vaccines within 6 weeks before enrollment.
- Have a history of epilepsy or other active central nervous system diseases.
- Have an allergy to the ingredients of the medicine used in this study.
- Previously received CAR-T cell therapy.
- Patients considered to be ineligible for the study by the investigator for reasons other than the above.
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Antal våben
Våben og indgreb
Deltagergruppe / ArmDeltagergruppe / Arm |
Intervention / BehandlingIntervention / Behandling |
|---|---|
|
Eksperimentel: YTS109
Participants will receive YTS109 cell infusion after preconditioning, and they need to be closely monitored for 24 hours following START-T cell infusion. |
In this study, subjects will receive YTS109 Cell Injection(0.5-1E6 STAR+T cell/kg) once. |
Hvad måler undersøgelsen?
Primære resultatmål
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Dosisbegrænsende toksicitet
Tidsramme: Inden for 28 dage efter infusion
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Inden for 28 dage efter infusion
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Objektiv responsrate (ORR) for hver dosisgruppe
Tidsramme: Inden for 4 uger efter infusion
|
Inden for 4 uger efter infusion
|
|
|
The incidence and frequency of treatment-emergent adverse events
Tidsramme: Within 12 months after infusion
|
Safety assessments are conducted using the NCI-CTCAE version 5.0 standards. |
Within 12 months after infusion
|
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Best overall response rate (BOR) of each dose group
Tidsramme: Within 12 weeks after infusion
|
BOR is determined as the most favorable response observed after cell infusion, until either disease relapse or the completion of a specified observation period. |
Within 12 weeks after infusion
|
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Time to response (TTR)
Tidsramme: Within 6 months after infusion
|
TTR is defined as the duration from cell infusion to the achievement of a hematological response |
Within 6 months after infusion
|
Samarbejdspartnere og efterforskere
Sponsor
Sponsor
Samarbejdspartnere
Samarbejdspartnere
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Studiestart
Primær færdiggørelse (Anslået)
Primær færdiggørelse
Studieafslutning (Anslået)
Studieafslutning
Datoer for studieregistrering
Først indsendt
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Først opslået
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering sendt
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
Andre undersøgelses-id-numre
- IIT2024106
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