An Expanded Access Program to Axitinib is Available for Patients With Advanced Forms of Kidney Cancer (Ductal; Papillary; Chromophobic; Oncocytic) With Mutations in VHL, PBRM1 / BAP1, SETD2, VEGF)

• Conditions: Clear Cell Kidney Cancer; Kidney Cancer; Kidney Cancer With PBRM1/BAP1/VHL/SETD2 Mutations

• Registration Number: NCT05941637
• Lead Sponsor: Lynkcell Europe

Brief Summary

Kidney cancer belongs to a heterogeneous group of tumors and is the most common oncourological disease; up to 80% of cases are clear cell carcinoma.

Detailed Description

The study of rare hereditary forms of clear cell kidney cancer (CRP) made it possible to identify the VHL gene, germline mutations which lead to the development of the Hippel-Lindau syndrome, and somatic mutations are characteristic of sporadic CRP. The second most frequent mutation independent of VHLmut is the PBRM1 gene involved in chromatin remodeling. PBRM1 mutations are positively correlated with SETD2 mutations and negatively correlated with BAP1 mutations. Depending on the status of PBRM1/BAP1 mutations, tumors are characterized by different pathomorphological features and prognoses. The main stages of the clonal evolution of SRP, which is already at the early stages and is characterized by pronounced intratumoral genetic heterogeneity, have been determined. However, as PRP progresses, subclones acquire different secondary mutations that contribute to the activation of the same mTOR and VEGF signaling pathways, as well as disrupting the mechanisms of chromatin remodeling and the functioning of TP53. The present program will determine the efficacy of standard doses of axitinib in patients with differentiated mutations in kidney cancer following partial exome sequencing.

Study Timeline

• Study First Submitted: 2023-07-03
• Study First Submitted QC: 2023-07-03
• Study First Posted: 2023-07-12
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-26
• Last Update Posted: 2024-12-31

Recruitment & Eligibility

• Status: NO_LONGER_AVAILABLE
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• Histologically documented metastatic renal cell cancer or cell kidney cancer
• Evidence of measurable disease.
• Adequate renal function (serum creatinine level)
• ECOG Status 0-1
• Patient must provide signed informed consent
• Male or female, age >/= 18 years

Exclusion Criteria

• Current use or anticipated need for treatment with drugs that are known potent CYP3A4 inhibitors
• Current use or anticipated need for treatment with drugs that are known as potent CYP3A4 or CYP1A2.
• Active gastrointestinal bleeding.
• Severe allergic reactions
• Unwillingness or inability to comply with mandated pretreatment biopsy or therapeutic regimen

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified