Clinical Trials

Pfizer is sponsoring multiple Phase 1 clinical trials evaluating the central nervous system-penetrant BRAF inhibitor Claturafenib (PF-07799933) as monotherapy or in combined targeted regimens. These studies assess safety, tolerability, pharmacokinetics, and therapeutic efficacy across BRAF-mutant malignancies, including melanoma, glioma, non-small cell lung cancer, thyroid cancer, colorectal cancer, and advanced solid tumors. A completed clinical trial evaluated drug bioavailability in healthy participants, while active, non-recruiting trials focus on dose escalation, expansion, and master protocols in refractory neoplastic conditions.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05538130 ACTIVE_NOT_RECRUITING
Melanoma; Glioma; Thyroid Cancer; Non-Small Cell Lung Cancer; Malignant Neoplasms; Brain Neoplasms; Advanced or Metastatic Solid Tumors; HGG; LGG; Low Grade Glioma; High Grade Glioma; Differentiated Thyroid Cancer; NSCLC (Non-small Cell Lung Cancer)
Pfizer
 2022-11-30 PHASE1
NCT05355701 ACTIVE_NOT_RECRUITING
Melanoma; Non-Small-Cell Lung Cancer; Thyroid Cancer; Glioma; Advanced Colorectal Cancer (Part 1)
Pfizer
 2022-07-05 PHASE1
NCT07563894 COMPLETED
Healthy
Pfizer
 2026-04-10 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-09-01)

Check the Claturafenib (PF-07799933) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Claturafenib selectively binds to and inhibits mutant forms of BRAF kinase, preventing downstream phosphorylation of ERK within the MAPK signaling cascade and inhibiting tumor cell proliferation. This brain-penetrant target inhibition halts oncogenic driver activity, offering clinical therapeutic utility in treating BRAF-mutated malignancies such as melanoma, glioma, thyroid cancer, and non-small cell lung cancer.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.