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Gynecol Oncol. 2015 Jun;137(3):386-91. doi: 10.1016/j.ygyno.2015.03.042. Epub 2015 Mar 24.

A phase II evaluation of the potent, highly selective PARP inhibitor veliparib in the treatment of persistent or recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer in patients who carry a germline BRCA1 or BRCA2 mutation - An NRG Oncology/Gynecologic Oncology Group study.

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Dept. of Gynecologic Oncology & Reproductive Medicine, University of Texas, M.D. Anderson Cancer Center, Houston, TX 77030, USA. Electronic address:
NRG Statistical & Data Center, Roswell Park Cancer Institute, Buffalo, NY 14263, USA.
Medical Oncology, Memorial Sloan-Kettering Cancer Center, New York, NY 10021, USA.
University of Washington, Dept. of OB/GYN, Seattle, WA 98195, USA.
University of California Irvine Medical Center, Irvine Chao Family Comprehensive Cancer Center, Department of Obstetrics and Gynecology, Orange, CA 92868, USA.
Surgical Oncology, Fox Chase Cancer Center, Philadelphia, PA 19111, USA.
Yale University School of Medicine, Division of GYN Oncology, New Haven, CT 06520, USA.
Palo Alto Medical Foundation, San Francisco, CA 94118, USA.
Investigational Drug Branch Cancer Therapy Evaluation Program, Bethesda, MD 20892, USA.
University of Washington and the Fred Hutchinson Cancer Research Center, Puget Sound Oncology Consortium, Seattle, WA 98109, USA.



Veliparib is a potent small molecule inhibitor of PARP-1/2, which is cytotoxic in tumor cells with deficiencies in BRCA1 or BRCA2. We studied the clinical activity and toxicity of veliparib in ovarian cancer patients carrying a germline BRCA1 or BRCA2 mutation (gBRCA).


Eligibility included three or fewer prior chemotherapy regimens, measurable disease and no prior use of a PARP inhibitor. Veliparib was administered at 400mg orally BID with one cycle being 28days. The two-stage Simon design was capable of detecting a 25% response probability with 90% power while controlling alpha=10% (at a 10% assumed null response probability).


The median age of the 50 eligible patients was 57years (range 37-94) and 14, 18, and 18 patients had 1, 2, and 3 prior therapies respectively. Thirty patients (60%) were platinum-resistant. The median number of cycles administered was 6 (1-27). There was one grade 4 thrombocytopenia. Grade 3 adverse events were: fatigue (n=3), nausea (2), leukopenia (1), neutropenia (1), dehydration (1), and ALT (1). Grade 2 events >10% were: nausea (46%), fatigue (26%), vomiting (18%), and anemia (14%). The proportion responding was 26% (90% CI: 16%-38%, CR: 2, PR: 11); for platinum-resistant and platinum-sensitive patients the proportion responding was 20% and 35%, respectively. The most common reason for treatment discontinuation was progression (62%). Twenty-nine patients are alive; two with SD remain on veliparib. The median PFS is 8.18months.


The single agent efficacy and tolerability of veliparib for BRCA mutation-associated recurrent ovarian cancer warrants further investigation.


BRCA1, BRCA2 mutation; Ovarian cancer; PARP inhibitor; Phase II trial; Toxicity; Veliparib

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