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NCT01078662

Open Label Study to Assess Efficacy and Safety of Olaparib in Confirmed Genetic BRCA1 or BRCA2 Mutation Pats

Completed Phase 2 Results posted Last updated 13 August 2025
What this trial tests

Phase 2 trial testing olaparib in Ovarian in 298 participants. Completed in 12 August 2024.

Timeline
21 February 2010
Primary endpoint
31 July 2012
12 August 2024

Quick facts

Lead sponsorAstraZeneca
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationnon randomized
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment298
Start date21 February 2010
Primary completion31 July 2012
Estimated completion12 August 2024
Sites14 locations across Sweden, Israel, Germany, Australia, United States, Spain

Drugs / interventions tested

Conditions studied

Sponsor

AstraZeneca — full company profile →

Who can join

Adults 18 to 130, any sex, with Ovarian or Breast. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Tumour Response Rate Primary · Tumour assessments carried out at baseline ie 28 days before first study drug dose and then every 8 weeks up to 6 months after starting study treatment, then every 12 weeks until objective disease progression, assessed maximum up to 29 months

Tumour response rate is the proportion of patients who experienced complete or partial response at least once during the assessment period, according to the definitions of Response Evaluation Criteria In Solid Tumours (RECIST version 1.1).

GroupValue95% CI
Breast Cancer12.95.74 – 23.85
Ovarian Cancer31.124.64 – 38.13
Pancreatic Cancer21.77.46 – 43.7
Prostate Cancer5015.7 – 84.3
Other Cancers8.30.21 – 38.48
All Patients26.221.27 – 31.55
Objective Response Rate Secondary · Tumour assessments carried out at baseline ie 28 days before first study drug dose and then every 8 weeks up to 6 months after starting study treatment, then every 12 weeks until objective disease progression, assessed maximum up to 29 months

Objective response rate is the proportion of patients with at least one measurable lesion at baseline, who experienced complete or partial response at least once during the assessment period, according to the definitions of Response Evaluation Criteria In Solid Tumours (RECIST version 1.1).

GroupValue95% CI
Breast Cancer13.86.15 – 25.38
Ovarian Cancer35.928.66 – 43.7
Pancreatic Cancer21.77.46 – 43.7
Prostate Cancer57.118.41 – 90.1
Other Cancers9.10.23 – 41.28
All Patients29.323.92 – 35.19
Progression Free Survival Secondary · Tumour assessments are carried out at baseline ie 28 days before first study drug dose and then every 8 weeks up to 6 months after starting study treatment, then every 12 weeks until objective disease progression, assessed maximum up to 29 months

Progression free survival is defined as the duration from first dose till objective progression or death. In absence of progression or death, the time is calculated from first dose till last evaluable scanning visit.

GroupValue95% CI
Breast Cancer3.681.76 – 7.52
Ovarian Cancer7.033.65 – 11.24
Pancreatic Cancer4.551.81 – 8.21
Prostate Cancer7.152.63 – 17.45
Overall Survival Secondary · Survival follow-up from first dose till death of the patient or till end of study in absence of death, assessed maximum up to 29 months

Overall survival is defined as the duration from first dose till death. In absence of death, the time is calculated from first dose till the date subject last known to be alive.

GroupValue95% CI
Breast Cancer11.015.68 – 24.18
Ovarian Cancer16.629.43 – NA
Pancreatic Cancer9.813.84 – 16.62
Prostate Cancer18.386.24 – 25.46
Overall Survival Rate at 12 Months Secondary · Survival follow-up from first dose till death of the patient or till end of study in absence of death, assessed maximum up to 29 months

Overall survival rate at 12 months is defined as the proportion of patients who are alive 12 months after date of first dose

GroupValue95% CI
Breast Cancer44.75.68 – 24.18
Ovarian Cancer64.49.43 – NA
Pancreatic Cancer40.93.84 – 16.62
Prostate Cancer506.24 – 25.46
Duration of Response Secondary · From onset of first occurrence of complete or partial response till documented progression or death by any cause in the absence of progression, assessed maximum up to 29 months

Duration of response is calculated from the date of first documented response (complete or partial) until date of documented progression (as defined by RECIST 1.1) or death (by any cause) in the absence of disease progression.

GroupValue95% CI
Breast Cancer204149.5 – 405
Ovarian Cancer225143 – 410
Pancreatic Cancer134131 – 141
Prostate Cancer326.5164 – 476
Other Cancers165165 – 165
All Patients208134 – 410
Disease Control Rate at Week 16 Secondary · Tumour assessments carried out at baseline ie 28 days before first study drug dose and then at week 8 and week 16

Disease control rate is the proportion of patients with best response of complete or partial response or stable disease according to definitions of Response Evaluation Criteria In Solid Tumours (RECIST version 1.1) till week 16.

GroupValue95% CI
Breast Cancer37.125.16 – 50.31
Ovarian Cancer5850.73 – 65.08
Pancreatic Cancer47.826.82 – 69.41
Prostate Cancer62.524.49 – 91.48
Other Cancers33.39.92 – 65.11
All Patients5246.18 – 57.81

Adverse events — posted to ClinicalTrials.gov

Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

OLAPARIB
Serious: 90/298 (30%)
Deaths:

Serious adverse events (78 terms)

ReactionSystemOLAPARIB
ANAEMIABlood and lymphatic system disorders
ABDOMINAL PAINGastrointestinal disorders
INTESTINAL OBSTRUCTIONGastrointestinal disorders
SMALL INTESTINAL OBSTRUCTIONGastrointestinal disorders
VOMITINGGastrointestinal disorders
PNEUMONIAInfections and infestations
DYSPNOEARespiratory, thoracic and mediastinal disorders
PLEURAL EFFUSIONRespiratory, thoracic and mediastinal disorders
PULMONARY EMBOLISMRespiratory, thoracic and mediastinal disorders
THROMBOCYTOPENIABlood and lymphatic system disorders
NEUTROPENIABlood and lymphatic system disorders
DYSPHAGIAGastrointestinal disorders
PYREXIAGeneral disorders
GASTROENTERITISInfections and infestations
URINARY TRACT INFECTIONInfections and infestations
FEBRILE NEUTROPENIABlood and lymphatic system disorders
PERICARDIAL EFFUSIONCardiac disorders
GASTROINTESTINAL OBSTRUCTIONGastrointestinal disorders
HAEMOGLOBIN DECREASEDInvestigations
MUSCULAR WEAKNESSMusculoskeletal and connective tissue disorders
CEREBROVASCULAR ACCIDENTNervous system disorders
SYNCOPENervous system disorders
ENTERITISGastrointestinal disorders
INTESTINAL MASSGastrointestinal disorders
LARGE INTESTINAL OBSTRUCTIONGastrointestinal disorders
Other adverse events (41 terms — click to expand)

ReactionSystemOLAPARIB
NAUSEAGastrointestinal disorders
FATIGUEGeneral disorders
VOMITINGGastrointestinal disorders
ANAEMIABlood and lymphatic system disorders
DIARRHOEAGastrointestinal disorders
ABDOMINAL PAINGastrointestinal disorders
DECREASED APPETITEMetabolism and nutrition disorders
DYSPEPSIAGastrointestinal disorders
HEADACHENervous system disorders
DYSGEUSIANervous system disorders
CONSTIPATIONGastrointestinal disorders
COUGHRespiratory, thoracic and mediastinal disorders
OEDEMA PERIPHERALGeneral disorders
PYREXIAGeneral disorders
BACK PAINMusculoskeletal and connective tissue disorders
DYSPNOEARespiratory, thoracic and mediastinal disorders
DIZZINESSNervous system disorders
ABDOMINAL DISTENSIONGastrointestinal disorders
URINARY TRACT INFECTIONInfections and infestations
THROMBOCYTOPENIABlood and lymphatic system disorders
ARTHRALGIAMusculoskeletal and connective tissue disorders
MUSCLE SPASMSMusculoskeletal and connective tissue disorders
WEIGHT DECREASEDInvestigations
ASTHENIAGeneral disorders
ABDOMINAL PAIN UPPERGastrointestinal disorders
PAIN IN EXTREMITYMusculoskeletal and connective tissue disorders
LEUKOPENIABlood and lymphatic system disorders
FLATULENCEGastrointestinal disorders
NASOPHARYNGITISInfections and infestations
UPPER RESPIRATORY TRACT INFECTIONInfections and infestations
INSOMNIAPsychiatric disorders
BLOOD CREATININE INCREASEDInvestigations
DRY MOUTHGastrointestinal disorders
DEPRESSIONPsychiatric disorders
HOT FLUSHVascular disorders
MUSCULOSKELETAL PAINMusculoskeletal and connective tissue disorders
ANXIETYPsychiatric disorders
OROPHARYNGEAL PAINRespiratory, thoracic and mediastinal disorders
MUSCULOSKELETAL CHEST PAINMusculoskeletal and connective tissue disorders
DYSPNOEA EXERTIONALRespiratory, thoracic and mediastinal disorders

Most-reported serious reactions: ANAEMIA, ABDOMINAL PAIN, INTESTINAL OBSTRUCTION, SMALL INTESTINAL OBSTRUCTION, VOMITING, PNEUMONIA, DYSPNOEA, PLEURAL EFFUSION.

Data from ClinicalTrials.gov NCT01078662 adverse events section.

Sponsor's own description

To assess the efficacy of oral olaparib in patients with advanced cancer who have a confirmed genetic BRCA1 and/or BRCA2 mutation, by assessment of tumour response

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Olaparib monotherapy in patients with advanced cancer and a germline BRCA1/2 mutation.
    Kaufman B, Shapira-Frommer R, Schmutzler RK, Audeh MW, et al · · 2015 · cited 1293× · PMID 25366685 · DOI 10.1200/jco.2014.56.2728
  2. The molecular biology of pancreatic adenocarcinoma: translational challenges and clinical perspectives.
    Wang S, Zheng Y, Yang F, Zhu L, et al · · 2021 · cited 252× · PMID 34219130 · DOI 10.1038/s41392-021-00659-4
  3. Triple-negative breast cancer: treatment challenges and solutions.
    Collignon J, Lousberg L, Schroeder H, Jerusalem G. · · 2016 · cited 246× · PMID 27284266 · DOI 10.2147/bctt.s69488
  4. Targeting DNA damage response in cancer therapy.
    Hosoya N, Miyagawa K. · · 2014 · cited 232× · PMID 24484288 · DOI 10.1111/cas.12366
  5. Efficacy and safety of olaparib monotherapy in germline BRCA1/2 mutation carriers with advanced ovarian cancer and three or more lines of prior therapy.
    Domchek SM, Aghajanian C, Shapira-Frommer R, Schmutzler RK, et al · · 2016 · cited 231× · PMID 26723501 · DOI 10.1016/j.ygyno.2015.12.020
  6. PARP Inhibitors in the Management of Ovarian Cancer: ASCO Guideline.
    Tew WP, Lacchetti C, Ellis A, Maxian K, et al · · 2020 · cited 217× · PMID 32790492 · DOI 10.1200/jco.20.01924
  7. PARP inhibitors in pancreatic cancer: molecular mechanisms and clinical applications.
    Zhu H, Wei M, Xu J, Hua J, et al · · 2020 · cited 203× · PMID 32122376 · DOI 10.1186/s12943-020-01167-9
  8. PARP inhibitors in the management of breast cancer: current data and future prospects.
    Livraghi L, Garber JE. · · 2015 · cited 188× · PMID 26268938 · DOI 10.1186/s12916-015-0425-1

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