Phase I combination study of the PARP inhibitor veliparib plus carboplatin and gemcitabine in patients with advanced ovarian cancer and other solid malignancies

Gynecol Oncol. 2018 Mar;148(3):507-514. doi: 10.1016/j.ygyno.2017.12.029. Epub 2018 Jan 17.

Abstract

Objective: Determine the maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of veliparib combined with carboplatin and gemcitabine in patients with advanced ovarian cancer and other nonhematologic malignancies.

Methods: In this phase I study, patients with metastatic or unresectable solid tumors and ≤2 prior chemotherapy regimens received veliparib combined with carboplatin area under the curve (AUC) 4 on day 1 and gemcitabine 800mg/m2 on days 1 and 8 of a 21-day cycle for maximum 10cycles, followed by optional veliparib maintenance therapy. Veliparib dosing commenced twice-daily (BID) continuously on day 1 of cycle 2; granulocyte colony-stimulating factor was permitted. Dose escalation used a Bayesian continual reassessment method. Safety, tolerability, and efficacy were evaluated.

Results: Seventy-five patients were enrolled (ovarian cancer, n=54; breast cancer, n=12). Thirty-six patients with ovarian cancer (67%) had known germline BRCA mutations. Most common treatment-related adverse events (TRAEs; ≥60%) were thrombocytopenia, neutropenia, nausea, and anemia. Most common grade 3/4 TRAEs (≥40%) were neutropenia and thrombocytopenia. Dose-limiting toxicities were thrombocytopenia and neutropenia. The MTD/RP2D was established at veliparib 250mg with carboplatin AUC 4 plus gemcitabine 800mg/m2. Responses were observed in 69% of patients with BRCA-deficient ovarian cancer (45% partial, 24% complete responses). Five patients remained on veliparib (80-310mg BID) for >34cycles.

Conclusions: Veliparib plus carboplatin/gemcitabine is tolerated, with a safety profile similar to carboplatin and gemcitabine alone. Combination therapy demonstrated promising preliminary antitumor activity in platinum-sensitive ovarian cancer patients with germline BRCA mutations. Trial registration ID: NCT01063816.

Keywords: BRCA1/2 mutations; Ovarian cancer; PARP inhibitor; Phase I; Veliparib.

Publication types

  • Clinical Trial, Phase I
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Anemia / chemically induced
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Area Under Curve
  • Bayes Theorem
  • Benzimidazoles / administration & dosage
  • Bile Duct Neoplasms / drug therapy
  • Breast Neoplasms / drug therapy*
  • Breast Neoplasms / genetics
  • Breast Neoplasms / pathology
  • Carboplatin / administration & dosage
  • Carcinoma, Basal Cell / drug therapy
  • Carcinoma, Hepatocellular / drug therapy
  • Carcinoma, Non-Small-Cell Lung / drug therapy
  • Carcinoma, Squamous Cell / drug therapy
  • Carcinoma, Transitional Cell / drug therapy
  • Cholangiocarcinoma / drug therapy
  • Deoxycytidine / administration & dosage
  • Deoxycytidine / analogs & derivatives
  • Female
  • Gallbladder Neoplasms / drug therapy
  • Gemcitabine
  • Genes, BRCA1
  • Genes, BRCA2
  • Germ-Line Mutation
  • Humans
  • Induction Chemotherapy
  • Kidney Neoplasms / drug therapy
  • Kidney Pelvis
  • Liver Neoplasms / drug therapy
  • Lung Neoplasms / drug therapy
  • Maintenance Chemotherapy
  • Male
  • Maximum Tolerated Dose
  • Mesothelioma / drug therapy
  • Middle Aged
  • Nausea / chemically induced
  • Neoplasm Metastasis
  • Neutropenia / chemically induced
  • Ovarian Neoplasms / drug therapy*
  • Ovarian Neoplasms / genetics
  • Ovarian Neoplasms / pathology
  • Poly(ADP-ribose) Polymerase Inhibitors / administration & dosage
  • Prostatic Neoplasms / drug therapy
  • Skin Neoplasms / drug therapy
  • Thrombocytopenia / chemically induced
  • Treatment Outcome

Substances

  • Benzimidazoles
  • Poly(ADP-ribose) Polymerase Inhibitors
  • veliparib
  • Deoxycytidine
  • Carboplatin
  • Gemcitabine

Associated data

  • ClinicalTrials.gov/NCT01063816