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RecruitingInterventionalPhase 1/Phase 2

A Phase 1/2a, Multicenter, First-in-human, Open-label Clinical Trial Evaluating MDX2001 Monotherapy in Patients With Advanced Solid Tumors

NCT ID: NCT06239194Sponsor: ModeX Therapeutics, An OPKO Health CompanyLast updated: 2026-05-06

Summary

This study is designed to characterize the safety, tolerability, and anti-tumor activity of MDX2001 in patients with advanced solid tumors.

Detailed description

This study consists of Phase 1a dose escalation, Phase 1b dose expansion in a single indication, and Phase 2a expansion in a single indication. Primary Objectives * All Phases: Evaluate the safety and tolerability of MDX2001 in patients with advanced solid tumor malignancies * Phase 1 only: Identify a recommended Phase 2 dose (RP2D) for further development of MDX2001 * For Phase 1b and Phase 2: Assess the anti-tumor efficacy of MDX2001 in patients with selected advanced solid tumor malignancies Secondary Objectives: * Further characterize the anti-tumor activity of MDX2001 based on additional assessments of clinical benefit * Characterize the pharmacokinetics of MDX2001 * Characterize the immunogenicity of MDX2001 * Characterize relationship of baseline target protein expression in tumor tissue and clinical benefit The expected duration of study intervention for patients may vary, based on progression date. The median expected duration of study per patient is estimated to be 10 months (up to 1 month for screening, a median of 6 months for treatment, and a median of 3 months for long term follow-up).

Arms & interventions

  • DrugMDX2001

    MDX2001 intravenous infusion

Outcome measures

Primary

  • All Phases: Adverse events (AEs)

    Incidence and severity of AEs and serious AEs (SAEs) graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 including changes in clinical laboratory parameters

    Time frame: Baseline until end of study, up to approximately 9 months

  • Phase 1b and Phase 2a: Objective response rate of MDX2001

    Objective response rate is defined as the proportion of patients who achieve a complete response (CR) or partial response (PR) per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

    Time frame: From date of enrollment until the end of treatment, up to approximately 6 months

  • Phase 1: Recommended Phase 2 dose (RP2D)

    Recommended Phase 2 dose is determined following the evaluation of MDX2001 safety including the incidences of dose limiting toxicities (DLTs), MDX2001 anti-tumor activity, and MDX2001 pharmacokinetics

    Time frame: Baseline until end of study, up to approximately 9 months

Secondary

  • Phase 1a: Objective response rate of MDX2001

    Time frame: From date of enrollment until the end of treatment, up to approximately 6 months

  • All Phases: Duration of response (DOR)

    Time frame: From date of enrollment until the end of treatment, up to approximately 6 months

  • All Phases: Time to response (TTR)

    Time frame: From date of enrollment until the first documentation of response (CR or PR), approximately 4 months

  • All Phases: Disease control rate (DCR)

    Time frame: From date of enrollment until the end of treatment, up to approximately 6 months

  • All Phases: Progression free survival (PFS)

    Time frame: From date of enrollment until the end of treatment, up to approximately 6 months

  • All Phases: Pharmacokinetic Parameter Cmax of MDX2001

    Time frame: From date of enrollment until completion of the 6th cycle of treatment, up to approximately 6 months

  • All Phases: Pharmacokinetic parameter area under the curve (AUC(0-T)) of MDX2001

    Time frame: From date of enrollment until the completion of the 3rd cycle of treatment, up to approximately 3 months

  • All Phases: Evaluation of MDX2001 immunogenicity

    Time frame: Baseline until end of study, up to approximately 9 months

  • All Phases: Correlation between tumor antigen expression and anti-tumor activity of MDX2001

    Time frame: Baseline until the end of treatment, up to approximately 6 months

Eligibility criteria

Sex: AllAge: 18 Years and olderHealthy volunteers: No
Inclusion Criteria: * Patients must be ≥ 18 years of age * Histologically or cytologically confirmed diagnosis of metastatic solid tumors * Eastern Cooperative Oncology Group (ECOG) performance status 0-1 * All patients should have at least 1 measurable disease per RECIST v1.1. An irradiated lesion can be considered measurable only if progression has been demonstrated on the irradiated lesion. * All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. * Adequate hematologic, hepatic and renal function * Capable of giving signed informed consent Exclusion Criteria: * Any clinically significant cardiac disease * Unresolved toxicities from previous anticancer therapy * Prior solid organ or hematologic transplant * Known untreated, active, or uncontrolled brain metastases * Known positivity with human immunodeficiency virus (HIV), known active hepatitis B or C, or uncontrolled chronic or ongoing infectiion requiring intravenous treatment. * Receipt of a live-virus vaccination within 28 days of planned treatment start * Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions. * Participation in a concurrent clinical study in the treatment period. * Known hypersensitivity to MDX2001 or any of its ingredients * Supplemental oxygen use for activities of daily living The above information is not intended to contain all considerations relevant to the potential participation in a clinical trial.

Study locations (6)

Sarah Cannon Research Institute

Denver, Colorado, 80218

Recruiting
ModeX Therapeutics · Contact
Gerald Falchook, MD · Principal Investigator

Sylvester Comprehensive Cancer Center - University of Miami Health System

Miami, Florida, 33136

Recruiting
ModeX Therapeutics · Contact
Jaime Merchan, MD · Principal Investigator

Massachusetts General Hospital

Boston, Massachusetts, 02114

Recruiting
ModeX Therapeutics · Contact
Rebecca Heist, MD, MPH · Principal Investigator

Sarah Cannon Research Institute

Nashville, Tennessee, 37203

Recruiting
ModeX Therapeutics · Contact
Melissa Johnson, MD · Principal Investigator

MD Anderson Cancer Center

Houston, Texas, 77030

Recruiting
ModeX Therapeutics · Contact
Ecaterina Dumbrava, MD · Principal Investigator

NEXT Oncology

San Antonio, Texas, 78229

Recruiting
ModeX Therapeutics · Contact
David Sommerhalder, MD · Principal Investigator

References

  • Dumbrava E, Minchom A, Henry J, Johnson M, Sommerhalder D, Merchan J, Heist R, Cabanas EG, Cotreau M, Goenaga AL, Burzyn D, Makris L, Culm K, Abbadessa G. MDX-2001-101 study protocol: a phase I/IIa, multicenter, first-in-human, open-label clinical trial evaluating MDX2001 monotherapy in patients with advanced solid tumors. Future Oncol. 2026 Feb;22(3):305-312. doi: 10.1080/14796694.2025.2610468. Epub 2026 Jan 6.(PubMed)