A Study of PLX51107 in Advanced Malignancies
A Phase 1b/2a, Two-Part, Dose Escalation and Expansion Study to Assess the Safety, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of PLX51107 in Subjects With Advanced Hematological Malignancies and Solid Tumors
Study Overview
Status
Status
Conditions
Conditions
Intervention / Treatment
Intervention / Treatment
Study Type
Study Type
Enrollment (Actual)
Enrollment
Phase
Phase
- Phase 1
Contacts and Locations
Study Locations
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New York
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New York, New York, United States, 10032
- Columbia University Medical Center
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Ohio
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Columbus, Ohio, United States, 43212
- The Ohio State University Stephanie Spielman Comprehensive Breast Center
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19107
- Thomas Jefferson University
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South Carolina
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Charleston, South Carolina, United States, 29425
- MUSC/ Hollings Cancer Center
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Texas
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San Antonio, Texas, United States, 78229
- South Texas Accelerated Research Therapeutics
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Participation Criteria
Eligibility Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
Confirmed diagnosis of a relapsed or refractory malignancy in 1 of 2 treatment groups:
- Group A: Subjects with any solid tumor (including lymphomas).
- Group B: Subjects with relapsed or refractory AML, Subjects with relapsed or refractory high-risk MDS, defined as revised International Prognostic Scoring System (IPSS-R) intermediate or greater disease.
- Age ≥18 years.
- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2.
- Life expectancy ≥3 months in the judgment of the investigator.
- Adequate organ function.
- Group A subjects must have measurable or evaluable disease per the appropriate disease criteria.
- Women of child-bearing potential must have a negative serum pregnancy test at Screening and must agree to use an effective form of contraception from the time of the negative pregnancy test up to 6 months after the last dose of study drug. Effective forms of contraception include abstinence, hormonal contraceptive in conjunction with a barrier method, or a double barrier method. Women of non-child-bearing potential may be included if they are either surgically sterile or have been postmenopausal for ≥1 year.
- Fertile men must agree to use an effective method of birth control during the study and for up to 6 months after the last dose of study drug.
- All associated clinically significant toxicity from previous cancer therapy must be resolved (to ≤Grade 1 or baseline) prior to study treatment administration (Grade 2 alopecia is allowed).
- Willingness and ability to provide written informed consent prior to any study-related procedures and to comply with all study requirements.
Exclusion Criteria:
- Prior exposure to a bromodomain inhibitor, such as OTX-015 or CPI-0610.
- Allogenic or autologous transplant for hematological malignancy with infusion of stem cells within 90 days before Cycle 1 Day 1, or on active immunosuppressive therapy for graft-versus-host disease (GVHD) or GVHD prophylaxis within 2 weeks of Cycle 1 Day 1.
- Known uncontrolled fungal, bacterial, and/or viral infection ≥Grade 2.
- Uncontrolled autoimmune hemolytic anemia or thrombocytopenia.
- For Group A: Subjects with a history of brain metastases are ineligible. This includes previously treated brain metastases. For Group B (subjects with AML): Active symptomatic CNS involvement of AML. Subjects with previously treated leptomeningeal disease that has been effectively treated are eligible.
- A diagnosis of acute promyelocytic leukemia (APL) or chronic myeloid leukemia (CML) in blast crisis.
- Known or suspected allergy to the investigational agent or any agent given in association with this trial.
- Women who are pregnant or are breast feeding.
- Clinically significant cardiac disease
- Inability to take oral medication or significant nausea and vomiting, malabsorption, or significant small bowel resection that, in the opinion of the Investigator, would preclude adequate absorption.
- Subject with known human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV) infection or is known to be a carrier of hepatitis B or C.
- Strong CYP3A4 and CYP2C8 inhibitors or inducers or CYP3A4 substrate drugs with a narrow therapeutic range taken within 14 days or 5 drug half-lives before start of study drug.
- Active secondary malignancy
- Major surgery or significant traumatic injury within 14 days prior to therapy
- Receipt of anti-cancer therapy 14 days prior to Cycle 1 Day 1
- Presence of any other medical, psychological, familial, sociological, or geographic condition potentially hampering compliance with the study protocol Subjects who are participating in any other therapeutic clinical study (observational or registry trials are allowed).
- Subjects who have Burkitt's lymphoma or Burkitt-like lymphoma.
- Subjects on active anticoagulation therapy including warfarin, factor Xa inhibitors, thrombin inhibitors, or heparin.
- Subjects with documented hepatic metastases involving >50% of the hepatic parenchyma.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Number of Arms
Arms and Interventions
Participant Group / ArmParticipant Group / Arm |
Intervention / TreatmentIntervention / Treatment |
|---|---|
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Experimental: Treatment Group A
Open label, sequential PLX51107 dose escalation in approximately 30 solid tumor subjects.
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Experimental: Treatment Group B
Open label, sequential PLX51107 dose escalation in approximately 30 subjects with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS).
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What is the study measuring?
Primary Outcome Measures
Primary Outcome Measures
Outcome Measure |
Time Frame |
|---|---|
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Safety of PLX51107 as measured by adverse events and serious adverse events.
Time Frame: 1 year
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1 year
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Area under the concentration-time curve (AUC) of PLX51107.
Time Frame: 1 year
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1 year
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Maximum observed concentration (Cmax) of PLX51107.
Time Frame: 1 year
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1 year
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Time to peak concentration (Tmax) of PLX51107.
Time Frame: 1 year
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1 year
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Half life (t1/2) of PLX51107.
Time Frame: 1 year
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1 year
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Secondary Outcome Measures
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
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Overall Response Rate (ORR)
Time Frame: 1 year
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ORR is defined as the total number of patients with the best overall response according to standard criteria for the relevant malignancy divided by the total number of treated patients and expressed as a percentage.
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1 year
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Duration Of Response (DOR).
Time Frame: 1 year
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DOR is defined as the time from the initial objective response to disease progression or death, whichever occurs first.
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1 year
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Progression-Free Survival (PFS).
Time Frame: 1 year
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PFS time is defined as the time from the first dose of PLX51107 to disease progression or death, whichever occurs first.
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1 year
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Overall Survival (OS).
Time Frame: 1 year
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OS is defined as the first dose of study drug until the date of death from any cause.
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1 year
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Collaborators and Investigators
Sponsor
Sponsor
Study record dates
Study Major Dates
Study Start
Study Start
Primary Completion (Actual)
Primary Completion
Study Completion (Actual)
Study Completion
Study Registration Dates
First Submitted
First Submitted
First Submitted That Met QC Criteria
First Submitted That Met QC Criteria
First Posted (Estimate)
First Posted
Study Record Updates
Last Update Posted (Actual)
Last Update Posted
Last Update Submitted That Met QC Criteria
Last Update Submitted That Met QC Criteria
Last Verified
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Immune System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Lymphoproliferative Disorders
- Lymphatic Diseases
- Immunoproliferative Disorders
- Bone Marrow Diseases
- Hematologic Diseases
- Precancerous Conditions
- Leukemia
- Lymphoma
- Myelodysplastic Syndromes
- Leukemia, Myeloid
- Leukemia, Myeloid, Acute
- Lymphoma, Non-Hodgkin
- Preleukemia
Other Study ID Numbers
Other Study ID Numbers
- PLX122-01
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
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