NCT05941637

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)

An Expanded Access Program to Axitinib is Available for Patients With Advanced Forms of Kidney Cancer (Ductal; Papillary; Chromophobic; Oncocytic), Confirmed Mutations in VHL, PBRM1 / BAP1, SETD2, VEGF), for Whom Standard Systemic Therapy Has Failed or Progressed, in Whom There Are no Satisfactory Treatment Alternatives and Who Are Not Eligible for Other Clinical Trials.

• Status: No Longer Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Lynkcell Europe · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: —

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.

Conditions

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

Interventions

Type	Name	Description
DRUG	Axitinib 5 MG	Patients will receive Axitinib (AG 013736) is a multi-target inhibitor of VEGFR1, VEGFR2, VEGFR3, PDGFRβ, and c-Kit - 5mg daily

Timeline

• First posted: 2023-07-12
• Last updated: 2024-12-31