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A Study of MC001 in Patients With Advanced or Metastatic Pancreatic Ductal Adenocarcinoma (PDAC) and Other Folate Receptor Alpha (FRα)-Expressing Solid Tumors

30 de octubre de 2026 actualizado por: Medicovestor, Inc

A First-in-Human, Open-Label, Multicenter, Phase 1, Dose Escalation Study With Cohort Expansion to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Antitumor Activity of MC001, a Dimeric Antibody-Drug Conjugate (ADC), in Patients With Advanced or Metastatic Pancreatic Ductal Adenocarcinoma (PDAC) and Other Solid Tumors Expressing Folate Receptor Alpha (FRα)

The purpose of this First-in-Human, Phase 1 study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), immunogenicity, and preliminary antitumor activity of MC001 in adults with advanced or metastatic pancreatic ductal adenocarcinoma (PDAC) and other solid tumors that have relapsed or are refractory to standard therapies. MC001 is an investigational dimeric antibody-drug conjugate (ADC) that targets folate receptor alpha (FRα) and delivers a microtubule-disrupting cytotoxic payload, monomethyl auristatin E (MMAE).

The study will be conducted in two sequential parts:

Part A (Dose Escalation): Evaluates ascending intravenous (IV) dose levels of MC001 administered once every 3 weeks (Q3W) using a standard 3+3 design in patients with advanced solid tumors to identify dose-limiting toxicities (DLTs), the maximum tolerated dose (MTD), and the recommended dose for expansion (RDE).

Part B (Dose Expansion): Evaluates MC001 at the selected RDE in patients with advanced or metastatic PDAC whose tumors express FRα on at least 30% of tumor cells to further characterize safety and preliminary efficacy.

Descripción general del estudio

Tipo de estudio

Intervencionista

Inscripción (Estimado)

40

Fase

  • Fase 1

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Ubicaciones de estudio

    • New York
      • Syracuse, New York, Estados Unidos, 13210
        • SUNY Upstate Medical University
    • North Carolina
      • Huntersville, North Carolina, Estados Unidos, 28078
        • Carolina BioOncology Institute, PLLC
    • Texas
      • San Antonio, Texas, Estados Unidos, 78229
        • NEXT Oncology
    • Virginia
      • Fairfax, Virginia, Estados Unidos, 22031
        • NEXT Oncology

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

18 años y mayores (Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

Inclusion Criteria:

  1. Age 18 years or older.

  2. Histologically and radiologically confirmed advanced surgically unresectable or metastatic pancreatic ductal adenocarcinoma (PDAC) or other solid tumors.

  3. Folate Receptor alpha (FRα) expression determined by immunohistochemistry (IHC) at a central laboratory:

    • Part A (Phase 1a Dose Escalation): FRα expression status will be assessed primarily for pharmacodynamic evaluation (not required for enrollment).
    • Part B (Phase 1b Dose Expansion): FRα expression must be documented on the cell surface in at least 30% of PDAC tumor cells prior to enrollment.
  4. Failed at least one prior line of systemic chemotherapy. If daraxonrasib is accessible to the patient, the patient must have failed at least one line of systemic chemotherapy and prior treatment with daraxonrasib.

  5. Measurable disease per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

  6. Adequate organ and bone marrow function:

    • Hematologic: Absolute neutrophil count (ANC) >= 1.5 x 10^9/L, platelets >= 100 x 10^9/L, hemoglobin >= 9 g/dL.
    • Hepatic: Total bilirubin <= 1.5 x upper limit of normal (ULN); ALT and AST <= 2.5 x ULN (or <= 5 x ULN in case of liver involvement).
    • Renal: Creatinine clearance >= 60 mL/min calculated by Cockcroft-Gault formula.
    • Coagulation: Prothrombin time (PT)/International Normalized Ratio (INR) <= 1.5 x ULN.
  7. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.

  8. Estimated life expectancy >= 3 months per investigator's assessment.

  9. Female patients must not be pregnant or breastfeeding, and must be either not of childbearing potential or agree to use highly effective contraception from 1 month before study start through 6 months after completion of study treatment. Male patients with partners of childbearing potential must agree to use highly effective contraception during the study and for up to 6 months after the last dose.

  10. Left ventricular ejection fraction (LVEF) >= 50% by echocardiogram, and Fridericia-corrected QT interval (QTcF) < 470 ms for both sexes.

  11. Availability of archival or fresh tumor tissue for biomarker analysis.

Exclusion Criteria:

  1. Prior exposure to antibody-drug conjugates (ADCs) targeting FRα or conjugated to monomethyl auristatin E (MMAE).

  2. History of allergy to immunoglobulins or MMAE.

  3. Receipt of radiation therapy <= 14 days before Cycle 1 Day 1, or failure to recover to Grade <= 1 from treatment-related side effects.

  4. Symptomatic central nervous system (CNS) malignancy. (Patients with asymptomatic or treated CNS metastases may be eligible if not receiving corticosteroids or anticonvulsants and disease is stable for >= 60 days).

  5. Peripheral sensory neuropathy Grade >= 2.

  6. Active infection, including Human Immunodeficiency Virus (HIV), requiring systemic therapy within <= 7 days prior to Cycle 1 Day 1.

  7. Active, uncontrolled Hepatitis B virus (HBV) or Hepatitis C virus (HCV) infection:

    • HBsAg-positive patients are eligible if they have received HBV antiviral therapy for >= 4 weeks prior to first dose, have an undetectable HBV viral load at screening, and remain on antiviral therapy throughout the study.
    • HBsAg-negative, anti-HBc total positive patients are eligible if HBV viral load is undetectable.
    • Patients with a history of HCV infection are eligible if HCV viral load is undetectable at screening and curative antiviral therapy was completed >= 12 weeks prior to study treatment.
  8. History of major ventricular arrhythmias (e.g., ventricular tachycardia, ventricular fibrillation, Torsades de Pointes [TdP]).

  9. Major surgical intervention <= 28 days prior to Informed Consent Form (ICF) signature or incomplete wound healing after surgery.

  10. Vaccination with a live or live-attenuated vaccine within 30 days prior to the first dose of trial intervention.

  11. Pregnancy or lactation.

  12. Unable or unwilling to sign the ICF, or patient is incarcerated.

  13. Refractory hypokalemia, congenital long QT syndrome, or family history of long QT syndrome; implantable pacemakers or automatic implantable cardioverter defibrillators; unstable angina requiring treatment; clinically significant bradycardia (<50 beats/min), tachycardia (>100 beats/min), or clinically significant ECG abnormalities; or use of drugs with a known risk of causing Torsades de Pointes (TdP) or QT interval prolongation within 4 weeks prior to the first dose.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación Secuencial
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Part A: MC001 Dose Escalation (Phase 1a)

Patients with advanced or metastatic PDAC or other solid tumors will receive escalating doses of MC001 administered via intravenous (IV) infusion once every 21 days (Q3W) following a 3+3 design across 5 planned dose levels: Dose Level 1 (0.3 mg/kg), Dose Level 2 (0.6 mg/kg), Dose Level 3 (1.0 mg/kg), Dose Level 4 (1.6 mg/kg), and Dose Level 5 (2.2 mg/kg). Treatment continues until disease progression, unacceptable toxicity, or withdrawal.

Dimeric fully human IgG1 monoclonal antibody targeting Folate Receptor alpha (FRα) conjugated to Monomethyl Auristatin E (MMAE) via a protease-cleavable linker. Administered as an IV infusion once every 21 days (Q3W) following premedication with dexamethasone, diphenhydramine, acetaminophen, and a 5-HT3 antagonist.

Experimental: Part B: MC001 Dose Expansion in PDAC (Phase 1b)

Approximately 12 patients with advanced or metastatic PDAC with documented FRα expression in ≥30% of tumor cells will receive MC001 at the Recommended Dose for Expansion (RDE) determined in Part A, administered via IV infusion once every 21 days (Q3W) until disease progression, unacceptable toxicity, or withdrawal.

Dimeric fully human IgG1 monoclonal antibody targeting Folate Receptor alpha (FRα) conjugated to Monomethyl Auristatin E (MMAE) via a protease-cleavable linker. Administered as an IV infusion once every 21 days (Q3W) following premedication with dexamethasone, diphenhydramine, acetaminophen, and a 5-HT3 antagonist.

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Periodo de tiempo: From first dose of study drug through 30 days after the last dose (up to approximately 36 months)

Incidence and severity of AEs and SAEs, including clinically significant changes in laboratory parameters, vital signs, and ECGs, graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 6.0.

From first dose of study drug through 30 days after the last dose (up to approximately 36 months)
Number of Participants Experiencing Dose-Limiting Toxicities (DLTs) in Part A (Dose Escalation)
Periodo de tiempo: Cycle 1 (Day 1 to Day 21)

A DLT is defined as any protocol-specified treatment-related hematologic, non-hematologic, or cardiac (QTcF) toxicity graded per NCI CTCAE v6.0 occurring during the DLT evaluation period. The incidence of DLTs in Cycle 1 is used to determine the Maximum Tolerated Dose (MTD, defined as the highest dose level at which ≤1 of 6 participants experiences a DLT) and Recommended Dose for Expansion (RDE).

Cycle 1 (Day 1 to Day 21)

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Maximum Observed Plasma Concentration (Cmax) of MC001 (ADC), Total Antibody, and Unconjugated MMAE
Periodo de tiempo: Cycle 1 (Days 1, 2, 8, and 15) and Day 1 of subsequent 21-day cycles (up to approximately 36 months)

Plasma Cmax derived from intensive blood sampling in Cycle 1 and sparse sampling in subsequent cycles.

Cycle 1 (Days 1, 2, 8, and 15) and Day 1 of subsequent 21-day cycles (up to approximately 36 months)
Area Under the Plasma Concentration-Time Curve (AUC) of MC001 (ADC), Total Antibody, and Unconjugated MMAE
Periodo de tiempo: Cycle 1 (Days 1, 2, 8, and 15) and Day 1 of subsequent 21-day cycles (up to approximately 36 months)

Plasma AUC derived from pharmacokinetic blood sampling.

Cycle 1 (Days 1, 2, 8, and 15) and Day 1 of subsequent 21-day cycles (up to approximately 36 months)
Terminal Elimination Half-Life (T1/2) of MC001 (ADC), Total Antibody, and Unconjugated MMAE
Periodo de tiempo: Cycle 1 (Day 1 through Day 15)

Terminal half-life calculated from plasma concentration-time profiles during Cycle 1.

Cycle 1 (Day 1 through Day 15)
Objective Response Rate (ORR)
Periodo de tiempo: From first dose of study drug until disease progression or treatment discontinuation (assessed prior to Cycles 3 and 5, and every 3 cycles thereafter, up to approximately 36 months)

Proportion of participants in the response-evaluable analysis set achieving a confirmed Complete Response (CR) or Partial Response (PR) as determined by the investigator per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.

From first dose of study drug until disease progression or treatment discontinuation (assessed prior to Cycles 3 and 5, and every 3 cycles thereafter, up to approximately 36 months)
Duration of Response (DoR)
Periodo de tiempo: From date of first documented CR or PR until disease progression or death (up to approximately 36 months)

Time from the date of first documented confirmed objective response (CR or PR) to the date of first documented disease progression per RECIST v1.1 or death from any cause, whichever occurs first.

From date of first documented CR or PR until disease progression or death (up to approximately 36 months)
Disease Control Rate (DCR)
Periodo de tiempo: From first dose of study drug until disease progression or treatment discontinuation (up to approximately 36 months)

Proportion of participants achieving CR, PR, or Stable Disease (SD, maintained for ≥6 weeks from start of treatment) as their best overall response per RECIST v1.1.

From first dose of study drug until disease progression or treatment discontinuation (up to approximately 36 months)
Progression-Free Survival (PFS)
Periodo de tiempo: From first dose of study drug until disease progression or death (up to approximately 36 months)

Time from the date of the first dose of MC001 to the date of first documented progressive disease per RECIST v1.1 or death due to any cause, whichever occurs first.

From first dose of study drug until disease progression or death (up to approximately 36 months)
Overall Survival (OS)
Periodo de tiempo: From first dose of study drug until death from any cause (up to approximately 36 months)

Time from the date of the first dose of MC001 to the date of death due to any cause.

From first dose of study drug until death from any cause (up to approximately 36 months)
Percentage of Participants With Anti-Drug Antibodies (ADAs) to MC001
Periodo de tiempo: Screening, Cycle 1 (Days 1, 8, 15), Cycle 2 Day 1, Cycle 3 Day 1, every 2nd cycle thereafter, End of Treatment, and 30 and 90 days post-last dose (up to approximately 36 months)

Incidence and persistence of treatment-emergent anti-drug antibodies (ADAs) evaluated via a tiered analytical approach (screening, confirmatory, and titer assays).

Screening, Cycle 1 (Days 1, 8, 15), Cycle 2 Day 1, Cycle 3 Day 1, every 2nd cycle thereafter, End of Treatment, and 30 and 90 days post-last dose (up to approximately 36 months)
Concentration-QTc Relationship: Change From Baseline in Fridericia-Corrected QT Interval (ΔQTcF)
Periodo de tiempo: Cycle 1 (Day 1 pre-dose through 24 hours post-infusion, Day 8, and Day 14), Cycle 3 Day 1, and Cycle 6 Day 1

Relationship between plasma MC001 concentrations and change from baseline in QTcF (ΔQTcF) evaluated via continuous Holter ECG monitoring paired with time-matched PK sampling.

Cycle 1 (Day 1 pre-dose through 24 hours post-infusion, Day 8, and Day 14), Cycle 3 Day 1, and Cycle 6 Day 1

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Investigadores

  • Investigador principal: Seah H Lim, MD, PhD, Medicovestor, Inc

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Estimado)

1 de diciembre de 2026

Finalización primaria (Estimado)

1 de enero de 2029

Finalización del estudio (Estimado)

1 de enero de 2029

Fechas de registro del estudio

Enviado por primera vez

26 de octubre de 2026

Primero enviado que cumplió con los criterios de control de calidad

26 de octubre de 2026

Publicado por primera vez (Actual)

2 de noviembre de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

3 de noviembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

30 de octubre de 2026

Última verificación

1 de octubre de 2026

Más información

Términos relacionados con este estudio

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

NO

Descripción del plan IPD

Individual participant data will not be shared publicly due to patient privacy and confidentiality considerations in a small Phase 1 first-in-human study.

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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