CClinicalTrials.gg
RecruitingNCT05503797Updated Jul 27, 2026

A Study to Assess the Efficacy and Safety of FORE8394 in Participants With Cancer Harboring BRAF Alterations

A Phase 2 interventional study of Plixorafenib in Cancer Harboring BRAF Alterations, HGG and LGG, sponsored by Fore Biotherapeutics. Recruiting at 70 sites in 12 countries. Open to participants aged 8 Years and older. Per ClinicalTrials.gov, last updated 2026-07-27.

Sponsored by Fore Biotherapeutics · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
254
Allocation
Non-randomized
Ages
8 Years and older
Sex
All
01

Study summary

The objective of this Master Protocol is to evaluate the efficacy and safety of plixorafenib in participants with BRAF altered (BRAF V600E or BRAF fusions) locally advanced or metastatic solid tumors, or recurrent or progressive primary central nervous system (CNS) tumors, including rare BRAF V600E-mutated solid tumors, including anaplastic thyroid, ovarian, cholangiocarcinoma or other rare cancers.

02

Conditions studied

  • Cancer Harboring BRAF Alterations
  • HGG
  • LGG
  • Solid Tumors

Keywords

  • BRAF alterations
  • BRAF Fusions
  • BRAF V600E
  • BRAF Class 1
  • BRAF Class 2
  • High grade glioma
  • low grade glioma
  • HGG
  • LGG
  • Solid tumors
  • Biliary tract cancer
  • Ovarian cancer
  • Fallopian tube cancer
  • Primary peritoneal cancer
  • Anaplastic thyroid cancer
  • Cholangiocarcinoma
  • Bladder cancer
  • Uterine cancer
  • Hepatocellular carcinoma
  • Gastrointestinal stromal tumor (GIST)
  • Breast cancer
  • Children
  • Pediatric
  • Adolescent
  • Young adult
03

Who can participate

Ages eligible
8 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

Subprotocol A:

  1. Male and female, ≥8 years of age, and weighing ≥25 kg.
  2. Histologic diagnosis of a solid tumor or primary CNS tumor.
  3. Documentation of BRAF gene fusion in tumor and/or blood detected by an analytically validated test by DNA sequencing or RNA (transcriptome) sequencing.
  4. Have an archival tissue sample available meeting protocol requirements.
  5. Consent to provide scan(s) prior to baseline to assess change in tumor trajectory.
  6. Received all available standard therapy, is intolerant to available therapies, or the investigator has determined that treatment with standard therapy is not appropriate.
  7. All adverse events related to prior therapies (chemotherapy; radiotherapy; surgery) must have resolved to Grade 1 or baseline.

Subprotocol B:

  1. Male and female, ≥8 years of age, and weighing ≥25 kg.
  2. Histological diagnosis of a primary CNS tumor, including but not limited to the following:

    1. Adults (≥18 years) with Grade 1-4 glioma or glioneuronal tumor (including glioblastoma, anaplastic astrocytoma, high grade astrocytoma with piloid features, pilocytic astrocytoma, gliosarcoma, anaplastic pleomorphic xanthoastrocytoma, anaplastic oligodendroglioma, anaplastic oligoastrocytoma, not otherwise specified [NOS], ganglioglioma, or recurrent LGG). OR
    2. Pediatric patients (8-17 years of age) with a Grade 3 or 4 glioma or glioneuronal tumor, including those with a prior, histologically confirmed, diagnosis of a low-grade glioma or glioneuronal tumor and now have radiographic or histopathological findings consistent with WHO [2021] Grade 3 or 4 primary CNS tumor.
    3. Participants must have unresectable, locally advanced or metastatic disease that:

    i. Had prior treatment with radiotherapy and/or first-line chemotherapy or concurrent chemoradiation therapy OR

    • Note: Participants who have a WHO Grade 3 or 4 glioma for whom chemotherapy and/or radiotherapy is not considered standard of care may remain eligible for the study.

    ii. Is intolerant to available therapies OR iii. The investigator has determined that treatment with standard therapy is not appropriate.

  3. Documented BRAF V600E mutation in tumor and/or liquid biopsy detected by an analytically validated test at CLIA or CLIA-equivalent laboratory approved by sponsor or sponsor-designated central test.
  4. An archival tissue sample available meeting protocol requirements, or fresh biopsy is required if the archival sample is not available for retrospective confirmation test.
  5. Consent to provide scan(s) prior to baseline to assess change in tumor trajectory.
  6. Measurable disease based upon specified response criteria, as determined by the radiographic BICR.
  7. All adverse events related to prior therapies (eg, chemotherapy, radiotherapy, surgery) must have resolved to Grade 1 or baseline.
  8. Participants who are receiving corticosteroid treatment must be on a stable or decreasing dose of ≤8 mg/day of dexamethasone or equivalent corticosteroid treatment for 7 days prior to first dose of study treatments.

Subprotocol C:

  1. Male and female, ≥8 years of age, and weighing ≥25 kg.
  2. Histologic diagnosis of a rare BRAF V600E-mutated solid tumor that is unresectable, locally advanced or metastatic.
  3. Measurable disease on CT, MRI, or physical exam
  4. Documented BRAF V600E mutation in tumor and/or liquid biopsy detected by an analytically validated test.
  5. Have an archival tissue sample available meeting protocol requirements.
  6. Consent to provide scan(s) prior to baseline to assess change in tumor trajectory
  7. Received all available standard therapy, is intolerant to available therapies, or the investigator has determined that treatment with standard therapy is not appropriate.

Subprotocol D:

  1. Male and female, ≥8 years of age, and weighing ≥25 kg.
  2. Histologic diagnosis of a solid tumor harboring a BRAF V600E mutation and not eligible for other subprotocols.
  3. Measurable disease on CT, MRI, or physical exam.
  4. Evidence of BRAF V600E mutation in tumor and/or blood detected by genomic tests.
  5. Consent to provide a tumor biopsy.
  6. Willingness to comply with the ECG substudy procedures.
  7. All adverse events related to prior therapies (chemotherapy; radiotherapy; surgery) must have resolved to Grade 1 or baseline.

Exclusion criteria

Exclusion Criteria:

Subprotocol A:

  1. Prior treatment with RAF/BRAF inhibitors active for Class 2 BRAF alterations for advanced unresectable or metastatic disease.
  2. Prior treatment with a MEK inhibitor.
  3. Tyrosine kinase inhibitor(s) and/or targeted therapies are allowed (other than BRAF/MAPK pathway inhibitors per Exclusion Criteria 3 and 4) and will be restricted to no more than the number of lines of therapy that are consistent with standard treatment guidelines.
  4. Malignancy with co-occurring activating RAS mutation(s) at any time.
  5. Uncontrolled intercurrent illness that would limit compliance with study requirements.
  6. HIV infection with exceptions; discuss with treating physician.
  7. Have impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral plixorafenib or cobicistat (such as ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, and small bowel resection).
  8. Grade ≥2 changes in AST, ALT, GGT, or bilirubin attributed to prior immune checkpoint inhibitor treatment are exclusionary, even if resolved.

Subprotocol B:

  1. Prior treatment with BRAF, ERK, and/or MEK inhibitor(s).
  2. Known or suspected neurofibromatosis-1 (NF-1) and/or RAS related gene alterations.
  3. Uncontrolled intercurrent illness that would limit compliance with study requirements.
  4. Active infection requiring systemic therapy.
  5. HIV infection with exceptions; discuss with treating physician.
  6. Have impairment of GI function or GI disease that may significantly alter the absorption of oral plixorafenib (such as ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection).
  7. Grade ≥ 2 changes in AST, ALT, gamma-glutamyl transaminase (GGT), or bilirubin attributed to prior immune checkpoint inhibitor treatment are exclusionary, even if resolved.

Subprotocol C:

  1. Diagnosis of colorectal adenocarcinoma or pancreatic ductal adenocarcinoma (neuroendocrine or acinar tumors are eligible).
  2. Diagnosis of BRAF V600E-mutated cutaneous melanoma, papillary thyroid cancer, or NSCLC.
  3. Participant has CNS metastases.
  4. Prior treatment with BRAF, ERK, and/or MEK inhibitor(s), unless otherwise specified for specific tumor types (i.e. low grade serous or borderline ovarian cancer).
  5. Known or suspected neurofibromatosis-1 (NF-1) and/or RAS related gene alterations.
  6. Participants with prostate, breast, or gynecologic cancers with known activating mutations that lead to constitutive hormone receptor activation (AR-V7, ESR1).
  7. Uncontrolled intercurrent illness that would limit compliance with study requirements.
  8. Active infection requiring systemic therapy.
  9. HIV infection with exceptions; discuss with treating physician.

Subprotocol D:

  1. Known or suspected neurofibromatosis-1 (NF-1) and/or RAS related gene alterations or other co-occurring driver mutations.
  2. Participant has a non-CNS solid tumor with CNS metastases.
  3. Uncontrolled intercurrent illness that would limit compliance with study requirements.
  4. Active infection requiring systemic therapy.
  5. HIV infection with exceptions; discuss with treating physician.
  6. Use or anticipate the need for medications with known risk for QT-prolonging potential and Torsades de Pointes.
  7. History of acute or chronic cardiovascular disease or surgery, hypertension, with systolic blood pressure >160mm HG, history of QTc abnormalities, or clinical significantly ECG abnormalities (for participants in the ECG substudy).
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
254 participants (estimated)

Study arms

  • Experimental
    Subprotocol A

    Participants with unresectable, locally advanced or metastatic solid tumors or primary CNS tumors harboring BRAF fusions will receive plixorafenib which will be increased as tolerated, continuously in 3-week cycles until disease progression, unacceptable toxicity, or other reason for withdrawal.

    Drug: Plixorafenib

  • Experimental
    Subprotocol B

    Participants with recurrent primary CNS tumors harboring BRAF V600E mutations will receive plixorafenib, continuously in 3-week cycles until disease progression, unacceptable toxicity, or other reason for withdrawal.

    Drug: Plixorafenib

  • Experimental
    Subprotocol C

    Participants with advanced, rare, non-CNS solid tumors harboring BRAF V600E mutations will receive plixorafenib, continuously in 3-week cycles until disease progression, unacceptable toxicity, or other reason for withdrawal.

    Drug: Plixorafenib

  • Experimental
    Subprotocol D

    Participants with BRAF V600E-mutated advanced solid tumors will receive plixorafenib until disease progression, unacceptable toxicity, or other reason for withdrawal.

    Drug: Plixorafenib

Interventions

  • DrugPlixorafenib

    Oral tablets

    Also known as: FORE8394, PLX8394, PLX-8394

05

What researchers measure

Primary outcomes

  1. Objective Response Rate (ORR) (Subprotocols A, B and C)

    ORR will be determined by standard tumor response criteria by blinded independent central review (BICR).

    Time frame: Up to approximately 4 years

  2. Pharmacokinetics (Subprotocol D)

    Systemic exposure of plixorafenib measured by Cmax and AUC

    Time frame: Up to approximately 4 years

Secondary outcomes

  1. Duration of Response (DOR) by BICR (Subprotocols A, B and C)

    DOR will be determined by standard tumor response criteria per BICR (subprotocols A-C)

    Time frame: Up to approximately 4 years

  2. ORR per Investigator Assessment

    ORR will be determined by standard tumor response criteria by Investigator Assessment.

    Time frame: Up to approximately 4 years

  3. DOR per Investigator Assessment

    DOR will be determined by standard tumor response criteria.

    Time frame: Up to approximately 4 years

  4. Percentage of Participants with DOR at 6 months, 12 months, and 18 months

    Time frame: 6 months, 12 months and 18 months

  5. Time to Response by BICR (Subprotocols A, B and C)

    Time frame: Up to approximately 4 years

  6. Progression Free Survival (PFS) by BICR (Subprotocols A, B and C)

    Time frame: Up to approximately 4 years

  7. PFS per Investigator's Assessment

    Time frame: Up to approximately 4 years

  8. Overall Survival

    Time frame: Up to approximately 4 years

  9. Percentage of Participants with PFS at 6 months, 12 months and 24 months

    BICR (Subprotocols A, B and C) and by Investigator Assessment (Subprotocols A, B, C and D)

    Time frame: 6 months, 12 months and 24 months

  10. Disease Control Rate (DCR)

    Time frame: Up to approximately 4 years

  11. Number of Participants who Experience Treatment-emergent Adverse Events (TEAEs)

    Time frame: Up to approximately 4 years

  12. Plasma Concentrations of Plixorafenib

    Time frame: Up to approximately 4 years

  13. Plasma Concentrations of Plixorafenib Metabolites

    Time frame: Up to approximately 4 years

06

Study locations

67 of 70 sites recruiting
  • Precision NextGen Oncology & Research Center
    Beverly Hills, California 90210, United States
    Recruiting
  • UCSF Helen Diller Family Comprehensive Cancer Center
    San Francisco, California 94143, United States
    Recruiting
  • University of California Los Angeles Rheumatology
    Westwood, Los Angeles, California 90095-6984, United States
    Recruiting
  • Norwalk Hospital
    Norwalk, Connecticut 06856, United States
    Recruiting
  • University of Miami Hospital and Clinics
    Miami, Florida 33136, United States
    Recruiting
  • The John Hopkins Hospital
    Baltimore, Maryland 21287, United States
    Recruiting
  • Maryland Oncology Hematology- Columbia
    Rockville, Maryland 20850, United States
    Recruiting
  • Tufts Medical Center
    Boston, Massachusetts 02111, United States
    Recruiting
  • Massachusetts General Hospital
    Boston, Massachusetts 02214, United States
    Recruiting
  • Dana-Farber Cancer Institute
    Boston, Massachusetts 02215, United States
    Not yet recruiting
  • St. Luke's Hospital
    Duluth, Minnesota 55805, United States
    Recruiting
  • Mosaic Life Care at Saint Joseph - Medical Center
    Saint Joseph, Missouri 64506, United States
    Recruiting
  • Nebraska Cancer Specialists - Midwest Cancer Center - Legacy
    Omaha, Nebraska 68130, United States
    Recruiting
  • Overlook Medical Center
    Summit, New Jersey 07901, United States
    Recruiting
  • Columbia University Irving Medical Center
    New York, New York 10032, United States
    Recruiting
  • Memorial Sloan Kettering Cancer Center
    New York, New York 10065, United States
    Recruiting
  • Atrium Health Wake Forest Baptist - Comprehensive Cancer Center
    Winston-Salem, North Carolina 27157, United States
    Recruiting
  • Nationwide Children's Hospital
    Colombus, Ohio 43205, United States
    Recruiting
  • The Ohio State University Comprehensive Cancer Center (OSUCCC) - The James Cancer Hospital and Solove Research Institute
    Columbus, Ohio 43221, United States
    Recruiting
  • Taylor Cancer Research Center
    Maumee, Ohio 43537, United States
    Recruiting
  • Toledo Clinic Cancer Center
    Toledo, Ohio 43623, United States
    Completed
  • Thomas Jefferson University
    Philadelphia, Pennsylvania 19107, United States
    Recruiting
  • Lifespan Cancer Institute - Rhode Island Hospital
    Providence, Rhode Island 02903, United States
    Recruiting
  • SCRI - TriStar Medical Group Children's Specialists
    Nashville, Tennessee 37203, United States
    Recruiting
  • SCRI Oncology Partners
    Nashville, Tennessee 37203, United States
    Recruiting
  • Baylor Scott & White Research Institute
    Dallas, Texas 75246, United States
    Recruiting
  • University of Texas MD Anderson Cancer Center
    Houston, Texas 77030, United States
    Recruiting
  • Baylor Scott & White Medical Center
    Temple, Texas 43205, United States
    Recruiting
  • University of Washington School of Medicine
    Seattle, Washington 98109, United States
    Recruiting
  • West Virginia University Health Sciences Campus
    Morgantown, West Virginia 26506, United States
    Recruiting
  • Newcastle Private Hospital
    New Lambton Heights, New South Wales 2305, Australia
    Recruiting
  • Orange Health Service
    Orange, New South Wales 2800, Australia
    Recruiting
  • Sydney Children's Hospital Network - Randwick
    Randwick, New South Wales 2031, Australia
    Recruiting
  • Flinders Medical Centre
    Bedford Park, South Australia 5042, Australia
    Recruiting
  • The Alfred
    Melbourne, Victoria 3004, Australia
    Recruiting
  • Sunny brook Health Sciences Centre- Bayview Campus
    Toronto, Ontario M4N 3M5, Canada
    Recruiting
  • Centre Hospitalier Universitaire Sainte-Justine
    Montreal, Quebec H3T 1C5, Canada
    Recruiting
  • Institut Bergonie
    Bordeaux, Aquitaine 33000, France
    Recruiting
  • Hôpital Nord de Marseille
    Marseille, Bouches-du-Rhône 13005, France
    Recruiting
  • Hôpital Morvan
    Brest, Finistère 29200, France
    Recruiting
  • Institut de Cancerologie de l'Ouest- Angers
    Angers, Pays de la Loire Region 49055, France
    Recruiting
  • Gustave Roussy
    Villejuif, Val-de-Marne 94805, France
    Recruiting
  • Institut Universitaire du Cancer de Toulouse Oncopole
    Toulouse, 31059, France
    Recruiting
  • Hôpital Universitaire Pitié Salpêtrière
    Paris, Île-de-France Region 75013, France
    Recruiting
  • Universitätsklinikum Heidelberg
    Heidelberg, Baden-Wurttemberg 69120, Germany
    Recruiting
  • Krankenhaus Nordwest
    Frankfurt am Main, Hesse 60488, Germany
    Active, not recruiting
  • Charité - Universitätsmedizin Berlin
    Berlin, 13353, Germany
    Recruiting
  • Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori" - IRST
    Meldola, Forli-Cesena 47014, Italy
    Recruiting
  • Istituto Nazionale Tumori IRCCS Fondazione G. Pascale
    Naples, Naples 80131, Italy
    Recruiting
  • Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele
    Milan, 20132, Italy
    Recruiting
  • Istituto Europeo di Oncologia
    Milan, 20141, Italy
    Recruiting
  • Erasmus Medisch Centrum
    Rotterdam, South Holland 3015 GD, Netherlands
    Recruiting
  • Haukeland Univeritetssjukehus
    Bergen, Hordaland 5021, Norway
    Recruiting
  • Oslo Universitetssykehus-Radiumhospitalet
    Oslo, Oslo 0379, Norway
    Recruiting
  • Catholic University of Korea Saint Vincent's Hospital
    Suwon, Gyeonggi-do 16247, South Korea
    Recruiting
  • Seoul National University Hospital
    Suwon, Gyeonggido 443-721, South Korea
    Recruiting
  • Dong-A University Hospital
    Pusan, Gyeongsangnam-do 602-812, South Korea
    Recruiting
  • Chonnam National University Hwasun Hospital
    Hwasun, Jeollanam-do 58128, South Korea
    Recruiting
  • Seoul National University Hospital
    Seoul, Seoul Teugbyeoisi 03080, South Korea
    Recruiting
  • Severance Hospital
    Seoul, Seoul Teugbyeolsi 03722, South Korea
    Recruiting
  • Hospital Clinico Universitarlo de Santiago
    Santiago de Compostela, A Coruña 15706, Spain
    Recruiting
  • Hospital Universitari Vall d'Hebron
    Barcelona, Barcelona 08035, Spain
    Recruiting
  • Hospital Infantil Universitario Niño Jesús
    Madrid, Madrid 28009, Spain
    Recruiting
  • Hospital Universitario 12 de Octubre
    Madrid, Madrid 28041, Spain
    Recruiting
  • Hospital Universitario Virgen del Rocío
    Seville, Sevilla 41013, Spain
    Recruiting
  • Hospital Clinico Universitarlo de Valencia
    Valencia, Valencia 46010, Spain
    Recruiting
  • Skånes Universitetssjukhus
    Lund, Skåne County 221 85, Sweden
    Recruiting
  • Karolinska Universitetssjukhuset
    Solna, Stockholm County 171 64, Sweden
    Recruiting
  • The Christie NHS Foundation Trust
    Manchester, England M20 4BX, United Kingdom
    Recruiting
  • Sarah Cannon Research Institute
    London, W1G 6AD, United Kingdom
    Recruiting
07

References and documents

Individual participant data

Plan to share: Yes — Fore is committed to sharing with qualified external researchers access to deidentified patient-level data and related study documents (eg. study protocol) from eligible studies following publication of the study results.

Supporting information: Study protocol

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT05503797
Lead sponsor
Fore Biotherapeutics
Responsible party
Sponsor
First posted
Aug 17, 2022
Start date
Feb 21, 2023
Primary completion
Dec 31, 2027 (estimated)
Completion
Dec 28, 2028 (estimated)
Last update
Jul 27, 2026

Study contacts

Jessica Rine
Contact
jessica.rine@fore.bio
610-442-4517
Geri Bardelli
Contact
geraldine.bardelli@fore.bio
978-835-2310

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
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