RecruitingPhase 2

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

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
8 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests how well plixorafenib (FORE8394) works and how safe it is for people with cancer that has certain BRAF gene changes. It includes cancers that have grown nearby or spread, and tumors that started in the brain or spinal cord and have returned or gotten worse. There are four study groups, each with different rules.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • All groups: Be at least 8 years old and weigh at least 25 kg (about 55 pounds).
  • Group A: Have a solid tumor or a tumor that started in the brain or spinal cord, with a confirmed BRAF fusion (joined genes).
  • Group B: Have a brain or spinal cord tumor with a confirmed BRAF V600E gene change. Adults may have grade 1–4 glioma or glioneuronal tumors; children ages 8–17 must have grade 3–4 tumors. The tumor must be unable to be removed by surgery, have grown nearby, or have spread.
  • Group C: Have a rare solid tumor with a confirmed BRAF V600E change that cannot be removed by surgery, has grown nearby, or has spread. Group D: Have a solid tumor with this gene change and not qualify for the other groups.
  • Groups A and C: Have tried all available standard treatments, been unable to tolerate them, or have a doctor decide they are not suitable. Group B has prior radiation or chemotherapy rules, with exceptions when treatment is unsuitable or not tolerated.
  • Groups A, B, and C: Provide earlier scans and a stored tumor sample. Group B allows a new sample if none is stored. Group D: Agree to a tumor biopsy and ECG heart-recording study procedures.
  • Groups B, C, and D: Have cancer that can be measured on scans or by the required exam.
  • Groups A, B, and D: Side effects from past treatment must be mild or back to their starting level. Group B: Steroid doses must be steady or falling for 7 days before treatment, at no more than 8 mg of dexamethasone daily or an equal dose of another steroid.

Who can't join

  • All groups: An uncontrolled illness that would make study requirements hard to follow, or HIV infection unless an exception applies. Groups B, C, and D also exclude active infections needing treatment throughout the body.
  • Group A: Past use of certain RAF/BRAF drugs or any MEK inhibitor. Groups B and C: Past BRAF, ERK, or MEK inhibitors—targeted cancer drugs—with some tumor-specific exceptions in Group C.
  • Group A: Certain active RAS gene changes in the cancer. Groups B, C, and D: Known or suspected NF-1 (a condition linked to tumors) or RAS-related gene changes. Group D also excludes other gene changes that drive cancer growth.
  • Groups A and B: Gut problems that could keep the study medicine from being absorbed, or past moderate or worse liver blood-test changes caused by certain immune treatments, even if they went away.
  • Group C: Certain colon, rectal, or pancreatic cancers; skin melanoma with BRAF V600E; papillary thyroid cancer; or non-small cell lung cancer. Pancreatic neuroendocrine and acinar tumors may qualify.
  • Group C: Cancer that has spread to the brain or spinal cord, or certain hormone-related gene changes in prostate, breast, or gynecologic cancers. Group D: A solid tumor that started elsewhere and spread to the brain or spinal cord.
  • Group D: Taking or expecting to need medicines known to cause certain dangerous heart rhythm changes.
  • Group D: Listed heart or blood vessel diseases or surgery, or blood pressure with a top number above 160. For the ECG study, certain past or current heart-recording problems also exclude participation.

Simplified for readability. The official criteria below are what the study team uses.

Official 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 sites (30)

CA

CT

FL

MA

MD

MN

MO

NC

NE

NJ

NY

OH

PA

RI

TN

TX

WA

WV