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

NCT05503797 · Fore Biotherapeutics
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Official title
A Phase 2 Master Protocol to Assess the Efficacy and Safety of FORE8394, an Inhibitor of BRAF Class 1 and Class 2 Alterations, in Participants With Cancer Harboring BRAF Alterations
About this study
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.
Eligibility criteria
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: 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).
Study design
Enrollment target: 254 participants
Allocation: non_randomized
Masking: none
Age groups: child, adult, older_adult
Timeline
Starts: 2023-02-21
Estimated completion: 2028-12-28
Last updated: 2026-07-27
Interventions
Drug: Plixorafenib
Primary outcomes
  • Objective Response Rate (ORR) (Subprotocols A, B and C) (Up to approximately 4 years)
  • Pharmacokinetics (Subprotocol D) (Up to approximately 4 years)
Sponsor
Fore Biotherapeutics · industry
Contacts & investigators
ContactJessica Rine · contact · jessica.rine@fore.bio · 610-442-4517
ContactGeri Bardelli · contact · geraldine.bardelli@fore.bio · 978-835-2310
All locations (70)
Precision NextGen Oncology & Research CenterRecruiting
Beverly Hills, California, United States
UCSF Helen Diller Family Comprehensive Cancer CenterRecruiting
San Francisco, California, United States
University of California Los Angeles RheumatologyRecruiting
Westwood, Los Angeles, California, United States
Norwalk HospitalRecruiting
Norwalk, Connecticut, United States
University of Miami Hospital and ClinicsRecruiting
Miami, Florida, United States
The John Hopkins HospitalRecruiting
Baltimore, Maryland, United States
Maryland Oncology Hematology- ColumbiaRecruiting
Rockville, Maryland, United States
Tufts Medical CenterRecruiting
Boston, Massachusetts, United States
Massachusetts General HospitalRecruiting
Boston, Massachusetts, United States
Dana-Farber Cancer InstituteNot Yet Recruiting
Boston, Massachusetts, United States
St. Luke's HospitalRecruiting
Duluth, Minnesota, United States
Mosaic Life Care at Saint Joseph - Medical CenterRecruiting
Saint Joseph, Missouri, United States
Nebraska Cancer Specialists - Midwest Cancer Center - LegacyRecruiting
Omaha, Nebraska, United States
Overlook Medical CenterRecruiting
Summit, New Jersey, United States
Columbia University Irving Medical CenterRecruiting
New York, New York, United States
Memorial Sloan Kettering Cancer CenterRecruiting
New York, New York, United States
Atrium Health Wake Forest Baptist - Comprehensive Cancer CenterRecruiting
Winston-Salem, North Carolina, United States
Nationwide Children's HospitalRecruiting
Colombus, Ohio, United States
The Ohio State University Comprehensive Cancer Center (OSUCCC) - The James Cancer Hospital and Solove Research InstituteRecruiting
Columbus, Ohio, United States
Taylor Cancer Research CenterRecruiting
Maumee, Ohio, United States
Toledo Clinic Cancer CenterCompleted
Toledo, Ohio, United States
Thomas Jefferson UniversityRecruiting
Philadelphia, Pennsylvania, United States
Lifespan Cancer Institute - Rhode Island HospitalRecruiting
Providence, Rhode Island, United States
SCRI - TriStar Medical Group Children's SpecialistsRecruiting
Nashville, Tennessee, United States
SCRI Oncology PartnersRecruiting
Nashville, Tennessee, United States
Baylor Scott & White Research InstituteRecruiting
Dallas, Texas, United States
University of Texas MD Anderson Cancer CenterRecruiting
Houston, Texas, United States
Baylor Scott & White Medical CenterRecruiting
Temple, Texas, United States
University of Washington School of MedicineRecruiting
Seattle, Washington, United States
West Virginia University Health Sciences CampusRecruiting
Morgantown, West Virginia, United States
Newcastle Private HospitalRecruiting
New Lambton Heights, New South Wales, Australia
Orange Health ServiceRecruiting
Orange, New South Wales, Australia
Sydney Children's Hospital Network - RandwickRecruiting
Randwick, New South Wales, Australia
Flinders Medical CentreRecruiting
Bedford Park, South Australia, Australia
The AlfredRecruiting
Melbourne, Victoria, Australia
Sunny brook Health Sciences Centre- Bayview CampusRecruiting
Toronto, Ontario, Canada
Centre Hospitalier Universitaire Sainte-JustineRecruiting
Montreal, Quebec, Canada
Institut BergonieRecruiting
Bordeaux, Aquitaine, France
Hôpital Nord de MarseilleRecruiting
Marseille, Bouches-du-Rhône, France
Hôpital MorvanRecruiting
Brest, Finistère, France
Institut de Cancerologie de l'Ouest- AngersRecruiting
Angers, Pays de la Loire Region, France
Gustave RoussyRecruiting
Villejuif, Val-de-Marne, France
Institut Universitaire du Cancer de Toulouse OncopoleRecruiting
Toulouse, France
Hôpital Universitaire Pitié SalpêtrièreRecruiting
Paris, Île-de-France Region, France
Universitätsklinikum HeidelbergRecruiting
Heidelberg, Baden-Wurttemberg, Germany
Krankenhaus NordwestActive Not Recruiting
Frankfurt am Main, Hesse, Germany
Charité - Universitätsmedizin BerlinRecruiting
Berlin, Germany
Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori" - IRSTRecruiting
Meldola, Forli-Cesena, Italy
Istituto Nazionale Tumori IRCCS Fondazione G. PascaleRecruiting
Naples, Naples, Italy
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San RaffaeleRecruiting
Milan, Italy
Istituto Europeo di OncologiaRecruiting
Milan, Italy
Erasmus Medisch CentrumRecruiting
Rotterdam, South Holland, Netherlands
Haukeland UniveritetssjukehusRecruiting
Bergen, Hordaland, Norway
Oslo Universitetssykehus-RadiumhospitaletRecruiting
Oslo, Oslo, Norway
Catholic University of Korea Saint Vincent's HospitalRecruiting
Suwon, Gyeonggi-do, South Korea
Seoul National University HospitalRecruiting
Suwon, Gyeonggido, South Korea
Dong-A University HospitalRecruiting
Pusan, Gyeongsangnam-do, South Korea
Chonnam National University Hwasun HospitalRecruiting
Hwasun, Jeollanam-do, South Korea
Seoul National University HospitalRecruiting
Seoul, Seoul Teugbyeoisi, South Korea
Severance HospitalRecruiting
Seoul, Seoul Teugbyeolsi, South Korea
Hospital Clinico Universitarlo de SantiagoRecruiting
Santiago de Compostela, A Coruña, Spain
Hospital Universitari Vall d'HebronRecruiting
Barcelona, Barcelona, Spain
Hospital Infantil Universitario Niño JesúsRecruiting
Madrid, Madrid, Spain
Hospital Universitario 12 de OctubreRecruiting
Madrid, Madrid, Spain
Hospital Universitario Virgen del RocíoRecruiting
Seville, Sevilla, Spain
Hospital Clinico Universitarlo de ValenciaRecruiting
Valencia, Valencia, Spain
Skånes UniversitetssjukhusRecruiting
Lund, Skåne County, Sweden
Karolinska UniversitetssjukhusetRecruiting
Solna, Stockholm County, Sweden
The Christie NHS Foundation TrustRecruiting
Manchester, England, United Kingdom
Sarah Cannon Research InstituteRecruiting
London, United Kingdom