A Phase 1/2a, Multicenter, First-in-human, Open-label Clinical Trial Evaluating MDX2001 Monotherapy in Patients With Advanced Solid Tumors
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
Study locations (6)
Sarah Cannon Research Institute
Denver, Colorado, 80218
Sylvester Comprehensive Cancer Center - University of Miami Health System
Miami, Florida, 33136
Massachusetts General Hospital
Boston, Massachusetts, 02114
Sarah Cannon Research Institute
Nashville, Tennessee, 37203
MD Anderson Cancer Center
Houston, Texas, 77030
NEXT Oncology
San Antonio, Texas, 78229
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)