Phase II Trial of Niraparib in BAP1 and other DDR Deficient Neoplasms (UF-STO-ETI-001)


The purpose of this study is to investigate the use of niraparib in patients with tumors known to have mutations in BAP1 and other select DNA damage response pathway genes.

Study details

BAP1 is an ubiquitin ligase that is critical in helping to regulate the cell cycle, cellular differentiation, and cell death. This protein is also intimately involved with DNA double-strand break repair. Germline mutations in the BAP1 gene are associated with a hereditary cancer syndrome that increases the risk of uveal melanoma, mesothelioma and renal cell carcinoma.

PARP is another protein that is crucial in DNA repair and enables continued cell replication and survival. It is hypothesized that PARP inhibition with niraparib will result in significant cytoreduction in patient tumors with mutations in BAP1 and other components of the DNA damage response pathway through synthetic lethality. Synthetic lethality is the inhibition of a gene that a cell relies on to compensate for the loss of another gene, resulting in the cell's demise.


  • Age ≥18 years
  • Histologically confirmed clinical diagnosis of incurable cancer
  • Confirmed diagnosis of uveal melanoma, mesothelioma, renal cell carcinoma (clear cell subtype), or cholangiocarcinoma (Cohort A only)
  • Known DNA damage repair mutation including any one of the following: ARID1A, ATM, ATR, BACH1 (BRIP1), BAP1, BARD1, BLM, CHEK1, CHEK2, CDK2, CDK4, ERCC, FAM175A, FEN1, IDH1, IDH2, MRE11A, NBN (NBS1), PALB2, POLD1, PRKDC (DNA-PK) PTEN, RAD50, RAD51, RAD52, RAD54, RPA1, SLX4, WRN, or XRCC. Only CLIA certified next generation sequencing (NGS) assays are acceptable. Variants of unknown significance (VUS) will be allowed to enroll on study (Cohort B only)
  • Prior treatment with standard systemic therapy (must have exhausted or declined all known effective therapies)
  • Must be willing to provide blood/serum/plasma for toxicity monitoring and other research purposes
  • Must have formalin-fixed paraffin embedded (FFPE) tissue available for research purposes. Tissue must have been obtained within the last 3 years

For additional eligibility criteria please contact the study team.


  • Drug treatment
  • Physical exams


Additional eligibility criteria and study procedure details can be explained by the study team.  Please call or email:


18 to 65
65 and over




Sarcoma, Bladder cancer, Brain and nervous system, Breast Cancer, Colon Cancer

Principal Investigator

George Thomas, M.D.



Contact Information

(352) 265-0680 x 87657

Be an Informed Participant

Before deciding to participate in a research study, take time to learn about clinical research, how it's conducted and your rights as a research participant. Following are some helpful resources from independent sources. Always remember that a clinical research study is research, not treatment.

Know Who to Contact

  • Eligibility: For questions about a specific study and who is eligible to participate, call or email the contact person listed for that study.
  • Your Rights: For questions about your rights as a research participant, contact the UF Institutional Review Boards at 352-273-9600.
  • Feedback: For general questions or feedback about study listings, email the UF Clinical and Translational Science Institute at

Other Resources

  • HealthStreet: Health-focused services, classes and events, and opportunities to participate in research.
  • Join a national registry of volunteers willing to be contacted about research studies.

For Research Teams