NCT04603365

Pamiparib and Temozolomide for the Treatment of Hereditary Leiomyomatosis and Renal Cell Cancer

A Phase 2 Study of Pamiparib (BGB-290) Plus Temozolomide for Hereditary Leiomyomatosis and Renal Cell Cancer (HLRCC)

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Jonsson Comprehensive Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial investigates how well pamiparib and temozolomide work in treating patients with hereditary leiomyomatosis and renal cell (kidney) cancer. Poly adenosine diphosphate-ribose polymerase (PARPs) are proteins that help repair DNA mutations. PARP inhibitors, such as pamiparib, can keep PARP from working, so tumor cells can't repair themselves, and they may stop growing. Chemotherapy drugs, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving pamiparib and temozolomide may help treat patients with hereditary leiomyomatosis and renal cell cancer.

Detailed description

PRIMARY OBJECTIVE: I. Evaluate response rate of pamiparib plus low-dose temozolomide. SECONDARY OBJECTIVES: I. To evaluate the progression-free survival with pamiparib plus low-dose temozolomide. II. To evaluate the safety (adverse events \[AE's\]) with pamiparib plus low-dose temozolomide. TRANSLATIONAL OBJECTIVES: I. To determine the association between plasma and tumor 2-hydroxyglutarate (2HG), fumarate, and succinate levels and response to treatment. II. To analyze the association of the genomic mutational signature (by whole genome sequencing) of the tumor to treatment. OUTLINE: Patients receive orally (PO) twice daily (BID) on days 1-28 and temozolomide PO once daily (QD) on days 1-7. Cycles repeat every 28 days for up to 36 months in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up every 4 months.

Conditions

• Clear Cell Papillary Renal Neoplasm
• Collecting Duct Carcinoma
• Hereditary Leiomyomatosis and Renal Cell Carcinoma
• Hereditary Papillary Renal Cell Carcinoma
• Metastatic Renal Cell Carcinoma
• Metastatic Unclassified Renal Cell Carcinoma
• Papillary Renal Cell Carcinoma
• Stage III Renal Cell Cancer American Joint Committee on Cancer (AJCC) v8
• Stage IV Renal Cell Cancer AJCC v8
• Tubulocystic Renal Cell Carcinoma
• Unresectable Renal Cell Carcinoma

Interventions

Type	Name	Description
DRUG	Pamiparib	Given PO
DRUG	Temozolomide	Given PO

Timeline

• Start date: 2021-10-18
• Primary completion: 2023-05-07
• Completion: 2023-05-07
• First posted: 2020-10-26
• Last updated: 2023-05-17

Regulatory

• FDA-regulated drug study