Evidence map›Paper›PMID 39626160›Full record

Trial reportJCO precision oncology2024

Phase II Trial of the PARP Inhibitor, Niraparib, in BAP1 and Other DNA Damage Response Pathway-Deficient Neoplasms.

Thomas J George, Ji-Hyun Lee, David L DeRemer, Peter J Hosein, Steven Staal, Merry Jennifer Markham, Dennie Jones, Karen C Daily, Jonathan A Chatzkel, Brian H Ramnaraign and 10 more

Erratum issuedAbstract readClinical Trial, Phase IIMulticenter Study
In one paragraph

Trial report in JCO precision oncology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 23 papers.

0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

23 citing papers in PubMed.

  1. Trial
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  9. Novel Insights into the Enigmatic Genetics of Male Breast Cancer in China.Pathophysiology : the official journal of the International Society for Pathophysiology · 2026
    Article
  10. Review
  11. Review
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

20 authors.

Thomas J GeorgeDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.ORCID 0000-0002-6249-9180
Ji-Hyun LeeUniversity of Florida Health Cancer Center, Gainesville, FL.
David L DeRemerUniversity of Florida Health Cancer Center, Gainesville, FL.
Peter J HoseinSylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami, FL.ORCID 0000-0001-8537-6397
Steven StaalDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.
Merry Jennifer MarkhamDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.ORCID 0000-0003-3567-3494
Dennie JonesDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.
Karen C DailyDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.ORCID 0000-0002-3218-9739
Jonathan A ChatzkelDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.
Brian H RamnaraignDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.ORCID 0000-0003-4051-4551
Julia L CloseDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.ORCID 0000-0002-7649-0320
Nkiruka EzenwajiakuSylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami, FL.
Martina C MurphyDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.
Carmen J AllegraDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.
Sherise RogersDivision of Hematology Oncology, Department of Medicine, University of Florida, Gainesville, FL.
Zhongyue ZhangUniversity of Florida Health Cancer Center, Gainesville, FL.
Derek LiUniversity of Florida Health Cancer Center, Gainesville, FL.
Gayathri SrinivasanMays Cancer Center, University of Texas at San Antonio, San Antonio, TX.
Montaser ShaheenMays Cancer Center, University of Texas at San Antonio, San Antonio, TX.
Robert HromasMays Cancer Center, University of Texas at San Antonio, San Antonio, TX.ORCID 0000-0002-1916-6276

Funding

TISSUE CULTURE---COREP30CA054174 · NCI · UNIVERSITY OF TEXAS HLTH SCIENCE CENTER · PI Robin Jean Leach · 1991 to 2026
$59.1M
University of Florida Health Cancer Center Support GrantP30CA247796 · NCI · UNIVERSITY OF FLORIDA · PI JI-HYUN LEE · 2023 to 2026
$10.9M
EEPD1 Repair of Stressed Replication ForksR01CA205224 · NCI · UNIVERSITY OF TEXAS HLTH SCIENCE CENTER · PI Robert A Hromas · 2016 to 2026
$3.3M
NCI NIH HHS P30 CA054174NCI NIH HHS P30 CA247796NCI NIH HHS R01 CA205224
6 · The paper itself

Abstract

purposeBRCA1-associated protein 1 (BAP1) is a critical cell cycle and DNA damage response (DDR) regulator with mutations (mBAP1) causing a functional protein loss. PARP inhibitors (PARPis) demonstrate synthetic lethality in mBAP1 preclinical models, independent of underlying BRCA status. This study aimed to explore the clinical activity of niraparib in patients with advanced tumors likely to harbor mBAP1.

methodsThis was a phase II multicenter trial in which refractory solid tumor patients were assigned to cohort A (histology-specific tumors likely to harbor mBAP1) or cohort B (histology-agnostic tumors with other known non-BRCA-confirmed DDR mutations). All patients received niraparib 300 mg orally once daily on a 28-day cycle. The primary end point was objective response rate, and secondary end points included progression-free survival (PFS) and overall survival.

resultsFrom August 2018 through December 2021, 37 patients were enrolled with 31 evaluable for response (cohort A, n = 18; cohort B, n = 13). In cohort A, the best response was one partial response (PR; 6%), eight stable disease (SD; 44%), and nine progressive disease (PD; 50%). This cohort stopped at the first stage following the prespecified Simon's design. mBAP1 was confirmed in 7/9 patients (78%) with PR or SD but in only 3/9 (33%) in those with PD. The median PFS in patients with mBAP1 (n = 10) was 6.7 months (95% CI, 1.0 to 9.2) versus 1.8 months (95% CI, 0.9 to 4.5) for wild-type (n = 8;

conclusionNiraparib failed to meet the prespecified efficacy end point for response. However, clinical benefit was suggested in a proportion of patients who had a confirmed mBAP1, supporting further investigation.

Indexed as

DNA DamageIndazolesNeoplasmsPiperidinesPoly(ADP-ribose) Polymerase InhibitorsTumor Suppressor ProteinsAdultAgedAged, 80 and overFemaleHumansMaleMiddle AgedIndazolesniraparibPiperidinesPoly(ADP-ribose) Polymerase InhibitorsTumor Suppressor Proteins

Identifiers

PMID39626160
PMCPMC11616782

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.