Phase II Trial of the PARP1 Inhibitor, Niraparib, in BAP1 and other DNA Damage Response (DDR) Pathway Deficient Neoplasms


The primary objective is to determine the response rate for subject with mutations in BAP1 and other genes involved in the DNA damage response pathway.

Inclusion Criteria:

  • Age ≥18 years
  • Histologically confirmed diagnosis of incurable cancer
  • 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
  • Measurable disease by RECIST (v 1.1) criteria
  • Adequate organ function
  • ECOG Performance Status of 0-2
  • Life expectancy ≥ 12 weeks
  • Women of childbearing potential must have a negative serum pregnancy test within 7 days prior to the first dose
  • 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)
  • Subjects must agree to not donate blood during the study or for 90 days after the last dose of study treatment.
  • Subjects receiving corticosteroids may continue as long as their dose is stable for least 4 weeks prior to initiating protocol therapy.


Cholangiocarcinoma, Renal cancer, Melanoma, mesothelioma

Principal Investigator

Thomas J George, M.D.

Contact Information


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