A Study of PLX8394 as a Single Agent in Patients With Advanced Unresectable Solid Tumors
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ClinicalTrials.gov Identifier: NCT02428712 |
Recruitment Status :
Recruiting
First Posted : April 29, 2015
Last Update Posted : November 5, 2020
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Condition or disease | Intervention/treatment | Phase |
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Advanced Unresectable Solid Tumors BRAF-mutated Tumors | Drug: PLX8394 | Phase 1 Phase 2 |
Dose Escalation (Part 1): To evaluate safety, pharmacokinetics, pharmacodynamics of PLX8394 in adult and pediatric patients with advanced BRAF- mutated tumors, and to identify the recommended Phase 2 Dose.
Dose Extension (Part 2): To access objective tumor response to PLX8394 treatment in adult and in adolescent patients with advanced BRAF- mutated tumors, to access RECIST, and to access pharmacokinetics, pharmacodynamics, and safety.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 100 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 1/2a Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of PLX8394 in Patients With Advanced Unresectable Solid Tumors |
Study Start Date : | April 2015 |
Estimated Primary Completion Date : | February 2022 |
Estimated Study Completion Date : | June 2022 |
Arm | Intervention/treatment |
---|---|
Experimental: PLX8394
Group A: Phase 1-Dose Escalation: Adult patients. Phase 2a-RP2D Confirmation/Redefinition (Formulation 2): Adult and adolescent patients. Phase 2a-Dose Extension: Adult and adolescent patients with advanced unresectable solid tumors will be enrolled among two cohorts.
Group B: Phase 1-Dose Escalation: Pediatric patients. Enrollment to Group B is closed. Phase 2a-Dose Extension: Pediatric patients with advanced unresectable BRAF-mutated tumors, including LCH. As of Protocol Amendment 10, enrollment to Group B is closed. |
Drug: PLX8394
(Formulation 1 or Formulation 2) |
- Area under the curve (AUC) of PLX8394 (Formulation 1 and Formulation 2) [ Time Frame: First dose of PLX8394 up to 30 days after end of treatment ]
- Maximum concentration (Cmax) of PLX8394 (Formulation 1 and Formulation 2) [ Time Frame: First dose of PLX8394 up to 30 days after end of treatment ]
- Time to peak concentration (Tmax) of PLX8394 (Formulation 1 and Formulation 2) [ Time Frame: First dose of PLX8394 up to 30 days after end of treatment ]
- Half life (T1/2) of PLX8394 (Formulation 1 and Formulation 2) [ Time Frame: First dose of PLX8394 up to 30 days after end of treatment ]
- Number of participants with Treatment Emergent Adverse Events (TEAEs) as assessed by CTCAE v4.0 (Formulation 1 and Formulation 2). [ Time Frame: First dose of PLX8394 up to 30 days after end of treatment ]
- To identify the recommended Phase 2 dose (RP2D) of PLX8394 (Formulation 1) in Group A (adult patients) for further evaluation in Dose Extension. [ Time Frame: 2 years ]
- To identify the RP2D of PLX8394 (Formulation 1) in Group B (pediatric patients) for further evaluation in Dose Extension. [ Time Frame: 1 year ]
- Compare AUC of PLX8394 (Formulation 1) with PLX8394 (Formulation 2) [ Time Frame: First dose of PLX8394 up to 30 days after end of treatment ]
- Compare Cmax of PLX8394 (Formulation 1) with PLX8394 (Formulation 2) [ Time Frame: First dose of PLX8394 up to 30 days after end of treatment ]
- Compare Tmax of PLX8394 (Formulation 1) with PLX8394 (Formulation 2) [ Time Frame: First dose of PLX8394 up to 30 days after end of treatment ]
- Compare T1/2 of PLX8394 (Formulation 1) with PLX8394 (Formulation 2) [ Time Frame: First dose of PLX8394 up to 30 days after end of treatment ]
- To determine the overall response rate of PLX8394 treatment at the applicable RP2D in a) Group A, Cohort 1, b) Group A, Cohort 2, and c) Group B. [ Time Frame: 5 years ]
- To evaluate the duration of response (defined as time of initial response to progressive disease or death) at the applicable RP2D in Dose Extension. [ Time Frame: 5 years ]
- To evaluate the progression free survival (defined as time of first dose to progressive disease or death) at the applicable RP2D in Dose Extension. [ Time Frame: 5 years ]
- Clinical benefit rate (defined as stable disease, partial response and complete response) after 24 weeks on study [ Time Frame: 5 years ]

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Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria- Group A:
- Age ≥ 12 years and at least 40 kg.
- Phase 1-Dose Escalation (no longer enrolling as of Protocol Amendment 10): Patients with histologically confirmed advanced solid tumors who are refractory to, relapsed after, or intolerant to standard therapy or for whom no standard therapy exists.
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Phase 2a-RP2D Confirmation/Redefinition (Formulation 2) and Phase 2a-Dose Extension: Patients with a history of histologically confirmed solid tumors with a BRAF mutation. Following RP2D confirmation and redefinition, extension subjects must meet criteria for Cohort 1 and Cohort 2 as specified below:
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Phase 2a-Dose Extension-Cohort 1
- Patients with solid tumors (as of Amendment 10, only subjects with glioma tumors) driven by a BRAF-V600 mutation
- Patients with no prior exposure to BRAF-directed therapy and for whom no standard therapy exists.
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Phase 2a-Dose Extension-Cohort 2
- Patients with solid tumors driven by BRAF non-V600 mutation.
- Patients with prior exposure to BRAF-directed therapy will be allowed, pending confirmation of mutations in either a re-biopsy or ctDNA, if the mutational profile identifies a potential for response based on PLX8394 mechanism of action and after investigator discussion.
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- Measurable disease by RECIST 1.1.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-1.
- Adequate hematologic, hepatic, and renal function.
- Women of child-bearing potential must have a negative pregnancy test and must agree to use an effective form of contraception from the time of the negative pregnancy test up to 3 months after the last dose of study drug. 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 3 months after the last dose of study drug.
- Completion of previous anti-cancer therapy at least 2 weeks before study drug initiation.
Exclusion Criteria- Group A:
- Phase 1 and Phase 2a RP2D confirmation-Dose Escalation: Investigational drug use within 28 days (or 5 half-lives, whichever is shorter) of the first dose of PLX8394.
- Major surgical procedure, open biopsy (excluding skin cancer resection), or significant traumatic injury within 14 days of initiating study drug or anticipation of the need for major surgery during the study.
- Uncontrolled intercurrent illness.
- Patients with colorectal cancer or pancreatic cancer
- Active secondary malignancy unless the malignancy is not expected to interfere with the evaluation of safety and is approved by the Medical Monitor. Patients with a completely treated prior malignancy and no evidence of disease for ≥ 2 years are eligible.
- Refractory nausea and vomiting, malabsorption, external biliary shunt, or significant bowel resection that would preclude adequate absorption.
- Clinically significant cardiac disease.
- Known infection with HIV, HBV, or HCV or a known carrier of HBV or HCV.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02428712
Contact: Ghassan Ahmed | Ghassan.Ahmed@precisionformedicine.com |
United States, Arizona | |
HonorHealth | Recruiting |
Scottsdale, Arizona, United States, 85258 | |
United States, California | |
University of California, San Francisco | Withdrawn |
San Francisco, California, United States, 94143 | |
Stanford Hospitals and Clinics | Completed |
Stanford, California, United States, 94305 | |
United States, Florida | |
University of Miami | Recruiting |
Miami, Florida, United States, 33136 | |
United States, Massachusetts | |
Massachusetts General Hospital | Active, not recruiting |
Boston, Massachusetts, United States, 02114 | |
Beth Israel Deaconess Medical Center | Withdrawn |
Boston, Massachusetts, United States, 02215 | |
United States, Michigan | |
Karmanos Cancer Institute | Completed |
Detroit, Michigan, United States, 48201 | |
United States, New York | |
Memorial Sloan Kettering Cancer Center | Recruiting |
New York, New York, United States, 10065 | |
United States, Pennsylvania | |
Thomas Jefferson University Medical Oncology Clinica | Withdrawn |
Philadelphia, Pennsylvania, United States, 19107 | |
United States, Tennessee | |
Vanderbilt University | Withdrawn |
Nashville, Tennessee, United States, 37232 | |
United States, Texas | |
MD Anderson Cancer Center | Recruiting |
Houston, Texas, United States, 77030 | |
Texas Children's Hospital (Baylor College of Medicine) | Recruiting |
Houston, Texas, United States, 77030 | |
United States, Utah | |
Huntsman Cancer Institute | Completed |
Salt Lake City, Utah, United States, 84112 |
Responsible Party: | NovellusDx |
ClinicalTrials.gov Identifier: | NCT02428712 |
Other Study ID Numbers: |
PLX120-03 |
First Posted: | April 29, 2015 Key Record Dates |
Last Update Posted: | November 5, 2020 |
Last Verified: | October 2020 |
Neoplasms |