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
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.
Subprotocol A:
Subprotocol B:
Histological diagnosis of a primary CNS tumor, including but not limited to the following:
i. Had prior treatment with radiotherapy and/or first-line chemotherapy or concurrent chemoradiation therapy OR
ii. Is intolerant to available therapies OR iii. The investigator has determined that treatment with standard therapy is not appropriate.
Subprotocol C:
Subprotocol D:
Exclusion Criteria:
Subprotocol A:
Subprotocol B:
Subprotocol C:
Subprotocol D:
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
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
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
Participants with BRAF V600E-mutated advanced solid tumors will receive plixorafenib until disease progression, unacceptable toxicity, or other reason for withdrawal.
Drug: Plixorafenib
Oral tablets
Also known as: FORE8394, PLX8394, PLX-8394
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
Pharmacokinetics (Subprotocol D)
Systemic exposure of plixorafenib measured by Cmax and AUC
Time frame: Up to approximately 4 years
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
ORR per Investigator Assessment
ORR will be determined by standard tumor response criteria by Investigator Assessment.
Time frame: Up to approximately 4 years
DOR per Investigator Assessment
DOR will be determined by standard tumor response criteria.
Time frame: Up to approximately 4 years
Percentage of Participants with DOR at 6 months, 12 months, and 18 months
Time frame: 6 months, 12 months and 18 months
Time to Response by BICR (Subprotocols A, B and C)
Time frame: Up to approximately 4 years
Progression Free Survival (PFS) by BICR (Subprotocols A, B and C)
Time frame: Up to approximately 4 years
PFS per Investigator's Assessment
Time frame: Up to approximately 4 years
Overall Survival
Time frame: Up to approximately 4 years
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
Disease Control Rate (DCR)
Time frame: Up to approximately 4 years
Number of Participants who Experience Treatment-emergent Adverse Events (TEAEs)
Time frame: Up to approximately 4 years
Plasma Concentrations of Plixorafenib
Time frame: Up to approximately 4 years
Plasma Concentrations of Plixorafenib Metabolites
Time frame: Up to approximately 4 years
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
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