- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT07611149
Study to Evaluate the Safety of UF-KURE-BCMA CAR T-Cells in Advanced Myeloma
A Phase 1, Single-Arm, Open-Label Study to Evaluate the Safety of UF-KURE-BCMA Cells in Patients With Relapsed or Refractory Multiple Myeloma
Studieoversigt
Status
Betingelser
Intervention / Behandling
Detaljeret beskrivelse
This is a Phase I, single-arm, open-label, dose-escalation study using a 3+3 design that evaluates the safety of UF (ultrafast)- KURE-BCMA for patients with relapsed/refractory multiple myeloma. Eligible patients are adults (≥18 years) with relapsed or refractory multiple myeloma after ≥3 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody. The study will accrue a total of 12 patients.
The primary objectives of this study are:
- To determine the recommended Phase 2 dose (RP2D) of UF-KURE-BCMA
- To establish the safety profile and identify dose-limiting toxicities (DLTs)
The secondary objectives:
- Manufacturing feasibility
- Overall response rate (ORR) per IMWG criteria
- Duration of response (DOR)
- Progression-free survival (PFS)
- Overall survival (OS)
The exploratory objectives include:
- CAR-T cell persistence and expansion
- Replication-competent lentivirus (RCL) testing
- Cytokine profiling
- CAR-T cell phenotype characterization
- Anti-BCMA CAR antibody development
- Spatial multi-omics analysis of the bone marrow microenvironment
The study endpoints include:
Primary Endpoint Incidence of dose-limiting toxicities occurring within 28 days of CAR-T cell infusion Secondary Endpoints
- Manufacturing success rate (≥75%)
- Treatment-emergent adverse events
- ORR, DOR, PFS, and OS per IMWG criteria
The Investigational Product is UF-KURE-BCMA, an autologous CAR-T cells manufactured using an ultra-fast process (approximately 17-20 hours), that targets specifically BCMA, a marker expressed on the surface of the myeloma cell. The study will evaluate 3 different dose levels:
- Level -1: 3 × 10⁶ cells (≥50 kg) / 2 × 10⁶ cells (<50 kg)
- Level 1 (Starting Dose): 10 × 10⁶ cells (≥50 kg) / 7 × 10⁶ cells (<50 kg)
- Level 2: 15 × 10⁶ cells (≥50 kg) / 10 × 10⁶ cells (<50 kg) Prior to the cell infusion, the patients will receive a chemotherapy regimen (lymphodepleting regimen) that includes: Cyclophosphamide 300 mg/m² IV plus fludarabine 30 mg/m² IV, administered daily for 3 days (Days -4 to -2) The study will last 28 days, which is Dose-limiting toxicity (DLT) observation period. The follow-up period is 24 months with a long-term safety follow-up of 15 years.
The study endpoints:
Primary Endpoint Incidence of dose-limiting toxicities occurring within 28 days of CAR-T cell infusion Secondary Endpoints
- Manufacturing success rate (≥75%)
- Treatment-emergent adverse events
- ORR, DOR, PFS, and OS per IMWG criteria
Statistical Methods A standard 3+3 dose-escalation design will be used. Safety and efficacy will be summarized using descriptive statistics. Time-to-event endpoints will be analyzed using the Kaplan-Meier method.
Undersøgelsestype
Tilmelding (Anslået)
Fase
- Fase 1
Kontakter og lokationer
Studiekontakt
- Navn: DANIEL Couriel, MD, MS, MBA
- Telefonnummer: 7343539036
- E-mail: daniel@cellserveglobal.com
Undersøgelse Kontakt Backup
- Navn: Ola Soliman, MD
- Telefonnummer: 6478650773
- E-mail: olaselkadi@gmail.com
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Beskrivelse
Inclusion Criteria:
Subjects must meet ALL of the following criteria to be eligible for study enrollment:
- Age: ≥18 years at time of signing informed consent
Diagnosis: Documented multiple myeloma meeting one of the following:
- Relapsed disease: Progression after achieving at least minimal response (MR) to prior therapy
- Refractory disease: Non-responsive or progressive disease while on therapy or within 60 days of last treatment (in subjects who achieved ≥MR on prior therapy)
Prior Therapy:
- Received ≥3 prior lines of anti-myeloma therapy
- Prior therapy must include:
At least one proteasome inhibitor At least one immunomodulatory drug (e.g., lenalidomide, pomalidomide, thalidomide) At least one anti-CD38 monoclonal antibody (e.g., daratumumab, isatuximab) o Prior anti-BCMA CAR-T therapy is permitted if subject achieved PFS ≥6 months post-infusion
Measurable Disease: At least one of the following at screening (for response assessment eligibility):
- Serum M-protein ≥0.5 g/dL by protein electrophoresis (SPEP)
- Urine M-protein ≥200 mg/24 hours by protein electrophoresis (UPEP)
- Serum free light chain (FLC) difference ≥10 mg/dL with abnormal FLC ratio Note: Subjects with non-measurable disease may enroll for safety assessment
- Performance Status: ECOG Performance Status 0-2 (see Appendix A)
Organ Function: Adequate organ function as defined by:
Hepatic:
o Total bilirubin ≤2× institutional upper limit of normal (ULN), except subjects with Gilbert's syndrome
- AST and ALT ≤2.5× institutional ULN
Renal:
o Calculated creatinine clearance ≥30 mL/min (Cockcroft-Gault formula)
Cardiac:
o Left ventricular ejection fraction (LVEF) ≥45% by echocardiogram or MUGA
Pulmonary:
o ≤Grade 1 dyspnea
o Oxygen saturation ≥92% on room air
o If PFTs performed: FEV₁ ≥50% predicted and DLCO ≥40% predicted (corrected for hemoglobin)
Prior Therapy Washout:
o ≥2 weeks since last radiation or systemic anti-myeloma therapy (standard agents)
o ≥4 weeks since last investigational therapy
o ≥6 weeks since autologous stem cell transplant
- Informed Consent: Ability to understand and willingness to provide written informed consent
- Contraception Requirements (for subjects of reproductive potential):
Female subjects:
o Women of childbearing potential must: Have negative serum pregnancy test at screening Agree to use highly effective contraception (failure rate <1% per year) from enrollment through 6 months post-CAR-T infusion
- Acceptable methods: bilateral tubal ligation, male partner sterilization, hormonal contraceptives that inhibit ovulation, hormone-releasing IUD, copper IUD
- Sexual abstinence is acceptable if consistent with subject's preferred lifestyle
Male subjects:
- Must agree to use condom plus effective contraception if partner is of childbearing potential
- Must refrain from sperm donation from enrollment through 6 months post-CAR-T infusion
Exclusion Criteria:
Subjects meeting ANY of the following criteria will be excluded:
Disease-Specific Exclusions:
- Active CNS involvement by multiple myeloma
- Plasma cell leukemia
- History of allogeneic hematopoietic stem cell transplantation
Malignancy Exclusions:
o Second active malignancy, except: Non-melanoma skin cancer Carcinoma in situ (cervix, bladder, breast) Stage 1 uterine cancer Localized prostate cancer
Cardiovascular Exclusions:
- New York Heart Association (NYHA) Class IV congestive heart failure
- Unstable angina pectoris
- Clinically significant cardiac arrhythmias
- Myocardial infarction, stroke, or TIA within 6 months of enrollment
Infectious Disease Exclusions:
- Known HIV infection or AIDS-related illness
- Active hepatitis B or C infection:
Positive HBsAg, or Positive anti-HBc or anti-HCV with detectable viral nucleic acid by PCR
- Active infection requiring systemic therapy 5. Neurological Exclusions:
- History of clinically relevant CNS pathology including:
Epilepsy or seizure disorders Paresis, aphasia Uncontrolled cerebrovascular disease Severe brain injury Dementia Parkinson's disease 6. Autoimmune Disease:
- Active autoimmune disease requiring systemic immunosuppression >15 mg/day prednisone equivalent within past 6 months
- Examples: rheumatoid arthritis, lupus
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Ikke-randomiseret
- Interventionel model: Parallel tildeling
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Aktiv komparator: Lower Dose Level
Lower Dose (Level -1): 3 × 10⁶ cells (≥50 kg)// 2 × 10⁶ cells (<50 kg)
|
The patients will receive one of 3 dose levels as outlined above.
|
|
Aktiv komparator: Starting Dose Level
Starting Dose Level (Level 1) : 10 × 10⁶ cells (≥50 kg) / 7 × 10⁶ cells (<50 kg)
|
The patients will receive one of 3 dose levels as outlined above.
|
|
Aktiv komparator: Higher Dose Level
Higher Dose Level (Level 2): 15 × 10⁶ cells (≥50 kg) / 10 × 10⁶ cells (<50 kg)
|
The patients will receive one of 3 dose levels as outlined above.
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Incidence of dose-limiting toxicities (DLT occurring within 28 days of CAR-T cell infusion)
Tidsramme: Within 28 days of CAR-T cell infusion
|
DLT
|
Within 28 days of CAR-T cell infusion
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Manufacturing success rate
Tidsramme: 28 days post infusion
|
Manufacturing success rate is defined as manufacturing process leading to an adequate product per protocol standards.
We expect this outcome to occur in ≥75% of the products manufactured.
|
28 days post infusion
|
|
Treatment-emergent adverse events
Tidsramme: At 24 months
|
Treatment adverse events related to the administration of CART-cells
|
At 24 months
|
|
ORR per IMWG criteria
Tidsramme: At 24 months
|
Overall response rate
|
At 24 months
|
|
DOR
Tidsramme: At day 24 months
|
Duration of Response
|
At day 24 months
|
|
PFS
Tidsramme: At 24 months
|
Progression-Free Survival
|
At 24 months
|
|
OS
Tidsramme: At 24 months
|
Overall Survival
|
At 24 months
|
Samarbejdspartnere og efterforskere
Sponsor
Publikationer og nyttige links
Generelle publikationer
- Munshi NC, Anderson LD Jr, Shah N, Madduri D, Berdeja J, Lonial S, Raje N, Lin Y, Siegel D, Oriol A, Moreau P, Yakoub-Agha I, Delforge M, Cavo M, Einsele H, Goldschmidt H, Weisel K, Rambaldi A, Reece D, Petrocca F, Massaro M, Connarn JN, Kaiser S, Patel P, Huang L, Campbell TB, Hege K, San-Miguel J. Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma. N Engl J Med. 2021 Feb 25;384(8):705-716. doi: 10.1056/NEJMoa2024850.
- Lee DW, Santomasso BD, Locke FL, Ghobadi A, Turtle CJ, Brudno JN, Maus MV, Park JH, Mead E, Pavletic S, Go WY, Eldjerou L, Gardner RA, Frey N, Curran KJ, Peggs K, Pasquini M, DiPersio JF, van den Brink MRM, Komanduri KV, Grupp SA, Neelapu SS. ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells. Biol Blood Marrow Transplant. 2019 Apr;25(4):625-638. doi: 10.1016/j.bbmt.2018.12.758. Epub 2018 Dec 25.
- Kumar S, Paiva B, Anderson KC, Durie B, Landgren O, Moreau P, Munshi N, Lonial S, Blade J, Mateos MV, Dimopoulos M, Kastritis E, Boccadoro M, Orlowski R, Goldschmidt H, Spencer A, Hou J, Chng WJ, Usmani SZ, Zamagni E, Shimizu K, Jagannath S, Johnsen HE, Terpos E, Reiman A, Kyle RA, Sonneveld P, Richardson PG, McCarthy P, Ludwig H, Chen W, Cavo M, Harousseau JL, Lentzsch S, Hillengass J, Palumbo A, Orfao A, Rajkumar SV, Miguel JS, Avet-Loiseau H. International Myeloma Working Group consensus criteria for response and minimal residual disease assessment in multiple myeloma. Lancet Oncol. 2016 Aug;17(8):e328-e346. doi: 10.1016/S1470-2045(16)30206-6.
- Hankey BF, Ries LA, Edwards BK. The surveillance, epidemiology, and end results program: a national resource. Cancer Epidemiol Biomarkers Prev. 1999 Dec;8(12):1117-21. No abstract available.
- San-Miguel J, Dhakal B, Yong K, Spencer A, Anguille S, Mateos MV, Fernandez de Larrea C, Martinez-Lopez J, Moreau P, Touzeau C, Leleu X, Avivi I, Cavo M, Ishida T, Kim SJ, Roeloffzen W, van de Donk NWCJ, Dytfeld D, Sidana S, Costa LJ, Oriol A, Popat R, Khan AM, Cohen YC, Ho PJ, Griffin J, Lendvai N, Lonardi C, Slaughter A, Schecter JM, Jackson CC, Connors K, Li K, Zudaire E, Chen D, Gilbert J, Yeh TM, Nagle S, Florendo E, Pacaud L, Patel N, Harrison SJ, Einsele H. Cilta-cel or Standard Care in Lenalidomide-Refractory Multiple Myeloma. N Engl J Med. 2023 Jul 27;389(4):335-347. doi: 10.1056/NEJMoa2303379. Epub 2023 Jun 5.
- Shah N, Chari A, Scott E, Mezzi K, Usmani SZ. B-cell maturation antigen (BCMA) in multiple myeloma: rationale for targeting and current therapeutic approaches. Leukemia. 2020 Apr;34(4):985-1005. doi: 10.1038/s41375-020-0734-z. Epub 2020 Feb 13.
- Ghassemi S, Durgin JS, Nunez-Cruz S, Patel J, Leferovich J, Pinzone M, Shen F, Cummins KD, Plesa G, Cantu VA, Reddy S, Bushman FD, Gill SI, O'Doherty U, O'Connor RS, Milone MC. Rapid manufacturing of non-activated potent CAR T cells. Nat Biomed Eng. 2022 Feb;6(2):118-128. doi: 10.1038/s41551-021-00842-6. Epub 2022 Feb 21.
- Rodriguez-Otero P, Ailawadhi S, Arnulf B, Patel K, Cavo M, Nooka AK, Manier S, Callander N, Costa LJ, Vij R, Bahlis NJ, Moreau P, Solomon SR, Delforge M, Berdeja J, Truppel-Hartmann A, Yang Z, Favre-Kontula L, Wu F, Piasecki J, Cook M, Giralt S. Ide-cel or Standard Regimens in Relapsed and Refractory Multiple Myeloma. N Engl J Med. 2023 Mar 16;388(11):1002-1014. doi: 10.1056/NEJMoa2213614. Epub 2023 Feb 10.
Datoer for undersøgelser
Studer store datoer
Studiestart (Anslået)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
- Karsygdomme
- Hjerte-kar-sygdomme
- Neoplasmer
- Sygdomme i immunsystemet
- Neoplasmer efter histologisk type
- Hæmatologiske sygdomme
- Lymfoproliferative lidelser
- Immunproliferative lidelser
- Neoplasmer, Plasmacelle
- Hæmostatiske lidelser
- Paraproteinæmier
- Blodproteinforstyrrelser
- Hæmoragiske lidelser
- Hemiske og lymfatiske sygdomme
- Myelomatose
Andre undersøgelses-id-numre
- HEM02
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
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Kliniske forsøg med Administration of CAR T-cells at 3 different dose levels
-
Mayo ClinicNational Cancer Institute (NCI)RekrutteringRefraktær Myelom | Tilbagevendende myelomatoseForenede Stater