Biliary Tract CancerPhase 1/2NCT06239194
Dose Escalation and Dose Expansion Study of MDX2001 in Patients With Advanced Solid Tumors
A Phase 1/2 study testing MDX2001 in people with Biliary Tract Cancer and Breast Cancer, run by ModeX Therapeutics, An OPKO Health Company at 6 sites in United States.
Trial summary
This study is designed to characterize the safety, tolerability, and anti-tumor activity of MDX2001 in patients with advanced solid tumors. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Biliary Tract Cancer, Breast Cancer, Cervical Cancer
- What is being tested?
- MDX2001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 330 participants
- Where
- 6 sitesUnited States
- Study dates
- 12 Jun 2024to Feb 2029
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
ModeX Therapeutics, +1 857-233-9936, info@modextx.com
Sarah Waliany, MD, MPH
- Recruiting
Sarah Cannon Research Institute
Denver, Colorado, 80218
ModeX Therapeutics, +1 857-233-9936, info@modextx.com
Gerald Falchook, MD
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
ModeX Therapeutics, +1 857-233-9936, info@modextx.com
Ecaterina Dumbrava, MD
- Recruiting
Sylvester Comprehensive Cancer Center - University of Miami Health System
Miami, Florida, 33136
ModeX Therapeutics, +1 857-233-9936, info@modextx.com
Jaime Merchan, MD
- Recruiting
Sarah Cannon Research Institute
Nashville, Tennessee, 37203
ModeX Therapeutics, +1 857-233-9936, info@modextx.com
Melissa Johnson, MD
- Recruiting
NEXT Oncology
San Antonio, Texas, 78229
ModeX Therapeutics, +1 857-233-9936, info@modextx.com
David Sommerhalder, MD
About this study
Official title: A Phase 1/2a, Multicenter, First-in-human, Open-label Clinical Trial Evaluating MDX2001 Monotherapy in Patients With Advanced Solid Tumors
This study is designed to characterize the safety, tolerability, and anti-tumor activity of MDX2001 in patients with advanced solid tumors.
Read the 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).
- Conditions
- Biliary Tract Cancer, Breast Cancer, Cervical Cancer, Colon Cancer, Endometrial Cancer, Esophageal Cancer
- Being tested
- MDX2001
- Medical subject headings
- Biliary Tract Neoplasms, Breast Neoplasms, Uterine Cervical Neoplasms, Colonic Neoplasms, Endometrial Neoplasms, Esophageal Neoplasms, Stomach Neoplasms, Head and Neck Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MDX2001Drug
MDX2001 intravenous infusion or subcutaneous (SC) injection.
| Study group | Role | What they receive |
|---|---|---|
| Phase 1a - MDX2001 Dose Escalation | Experimental | Patients with metastatic solid tumors will receive MDX2001 as intravenous (IV) infusion or subcutaneous (SC) injection. Receives: Drug: MDX2001 |
| Phase 1b - Indication Optimization | Experimental | Patients with a single tumor indication receive MDX2001 as intravenous (IV) infusion or subcutaneous (SC) injection. Receives: Drug: MDX2001 |
| Phase 1b - Dose Optimization | Experimental | Patients with a single tumor indication will receive MDX2001 as intravenous (IV) infusion or subcutaneous (SC) injection. Receives: Drug: MDX2001 |
| Phase 2a - Cohort Expansion | Experimental | Patients with a single tumor indication will receive MDX2001 as intravenous (IV) infusion or subcutaneous (SC) injection at the recommended Phase 2 dose. Receives: Drug: MDX2001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: 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
Measured: Baseline until end of study, up to approximately 9 months
Secondary outcomes (9)
Phase 1a: 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 RECIST v1.1.
Measured: From date of enrollment until the end of treatment, up to approximately 6 months
All Phases: Duration of response (DOR)
Duration of response is defined as the time from first documentation of response (complete response [CR] or partial response [PR]) to documentation of objective disease progression or death due to any cause, whichever occurs first
Measured: From date of enrollment until the end of treatment, up to approximately 6 months
All Phases: Time to response (TTR)
Time to response is defined as the time from first dose to first documentation of response (CR or PR)
Measured: From date of enrollment until the first documentation of response (CR or PR), approximately 4 months
All Phases: Disease control rate (DCR)
Disease control rate is defined as the proportion of evaluable patients with a best overall response (BOR) of stable disease, CR or PR
Measured: From date of enrollment until the end of treatment, up to approximately 6 months
All Phases: Progression free survival (PFS)
Progression-free survival is defined as the time from the first dose to the date of disease progression or death (any cause), whichever occurs first
Measured: From date of enrollment until the end of treatment, up to approximately 6 months
All Phases: Pharmacokinetic Parameter Cmax of MDX2001
Maximum observed plasma concentration
Measured: 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
Area under the plasma concentration versus time curve
Measured: From date of enrollment until the completion of the 3rd cycle of treatment, up to approximately 3 months
All Phases: Evaluation of MDX2001 immunogenicity
The presence and persistence of anti-MDX2001 antibodies
Measured: Baseline until end of study, up to approximately 9 months
All Phases: Correlation between tumor antigen expression and anti-tumor activity of MDX2001
Relationship between H score cell surface target protein expression in tumor tissue at baseline and objective responses with MDX2001
Measured: Baseline until the end of treatment, up to approximately 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2024
- Study start12 Jun 2024
- Primary completionAug 2028 (estimated)
- Study completionFeb 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Biliary Tract CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 330 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ModeX Therapeutics, An OPKO Health Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06239194
- Sponsor's protocol ID
- MDX-2001-101
- Status last verified
- Oct 2026
- First submitted
- 18 Jan 2024
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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 41495607