Kidney Medullary Carcinoma

P2, N=10, Terminated, M.D. Anderson Cancer Center | Trial completion date: Jul 2027 --> Nov 2025 | Active, not recruiting --> Terminated | Trial primary completion date: Jul 2027 --> Nov 2025; The study met its prespecified futility criteria during the prespecified interim futility analysis after enrolling the first cohort of 10 patients.

Selective pharmacologic inhibition of p300 suppressed this program and restored sensitivity to ICT. These findings reveal an adaptive mechanism of resistance to ICT in RMC and support targeting master myeloid regulators to enable therapeutic benefit.

P1/2, N=58, Completed, Epizyme, Inc. | Active, not recruiting --> Completed

This study represents the most extensive molecular characterization of RMC to date, identifying TROP2 and other potential therapeutic targets. Sacituzumab govitecan demonstrates potential clinical benefit, warranting further evaluation in prospective trials to confirm its efficacy and explore additional targets identified herein.

Given the exceptional rarity of RCCU-MP and its overlapping characteristics with RMC, we systematically analyze the clinical features, pathological morphology, and immunohistochemistry of RCCU-MP separately. Additionally, we explore potential molecular pathogenesis and emerging therapeutic strategies based on recent evidence.

P1/2, N=86, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Jun 2026 --> Mar 2030 | Trial primary completion date: Jun 2026 --> Mar 2030

A chemotherapy regimen of doxorubicin and cyclophosphamide, followed by weekly paclitaxel, was selected for safety in pregnancy. This case demonstrates that established chemotherapeutic regimens used in pregnant patients with other cancers can be successfully applied to manage RMC during pregnancy. Our findings underscore the importance of early, aggressive treatment and suggest that a coordinated approach can achieve favorable outcomes for both the mother and the fetus.

P1/2, N=86, Active, not recruiting, National Cancer Institute (NCI) | Suspended --> Active, not recruiting

The instituted therapy consisted of right radical nephrectomy with retroperitoneal lymphadenectomy, with nodal metastases detected. Given the rarity of unclassified RCC with a medullary phenotype, continuous documentation and analysis of individual cases not associated with sickle cell trait are crucial to understanding its behavior, prognosis, and potential therapeutic approaches, considering its aggressiveness and high metastatic potential.

We also discussed possible clinicopathological and molecular differences between SMARCB1-deficient RCC without hemoglobinopathies and RMC. The distinctions among these SMARCB1-deficient renal tumors identified in our study would significantly enhance diagnosis and develop new therapeutic strategies.

P1/2, N=86, Suspended, National Cancer Institute (NCI) | Recruiting --> Suspended

P2, N=10, Active, not recruiting, M.D. Anderson Cancer Center | Trial completion date: Jul 2025 --> Jul 2027 | Trial primary completion date: Jul 2025 --> Jul 2027