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A Study to Evaluate Rucaparib in Participants With Solid Tumors and With Deleterious Mutations in HRR Genes

Phase 2
Terminated
Conditions
Solid Tumor
Interventions
Registration Number
NCT04171700
Lead Sponsor
pharmaand GmbH
Brief Summary

A Phase 2, open-label, single-arm trial to evaluate the response of rucaparib in participants with various solid tumors and with deleterious mutations in Homologous Recombination Repair (HRR) genes.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
83
Inclusion Criteria
  • Unresectable, locally advanced or metastatic solid tumor and relapsed/progressive disease
  • Measurable disease per RECIST v1.1 or modified RECIST v1.1 and PCWG3 (for prostate cancer)
  • Have a deleterious mutation (germline or somatic) in BRCA1, BRCA2, PALB2, RAD51C, RAD51D, BARD1, BRIP1, FANCA, NBN, RAD51 or RAD51B. Note: Breast cancer patients that are HER2 negative and have germline BRCA1 or BRCA2 mutations AND patients with epithelial ovarian cancer, fallopian tube cancer, primary peritoneal cancer or metastatic castration-resistant prostate cancer with BRCA1 or BRCA2 mutations are ineligible for this trial.
  • At least one prior line of therapy extending overall survival or standard of care therapy for advanced disease. Note: Some tumor types have specific inclusion/exclusion criteria for previous treatments.
  • ECOG 0 or 1
  • Tumor tissue available for genomic analysis, or must be willing to have a biopsy if no archival tumor tissue available
  • Adequate organ function
  • Life expectancy of 4 months

Key

Exclusion Criteria
  • Active central nervous system brain metastases, leptomeningeal disease or primary tumor of CNS origin
  • Active second malignancy (Exceptions: Successfully treated malignancy with no active disease for 1 year, surgically cured and/or low-risk tumors, or patients receiving ongoing anticancer hormonal therapy for a previously treated cancer)
  • Pre-existing gastrointestinal disorders/conditions interfering with ingestion/absorption of rucaparib
  • Prior treatment with a PARP inhibitor
  • More than 3 prior lines of chemotherapy in the locally advanced/metastatic setting
  • History of myelodysplastic syndrome or acute myeloid leukemia

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
RucaparibRucaparibEligible participants will be enrolled in either Cohort A or Cohort B. Cohort A: Up to 200 participants with deleterious mutations in BRCA1, BRCA2, PALB2, RAD51C or RAD51D. Cohort B (Exploratory): Up to 20 participants with deleterious mutations in BARD1, BRIP1, FANCA, NBN, RAD51 or RAD51B.
Primary Outcome Measures
NameTimeMethod
Best Overall Response Rate by InvestigatorFrom first dose of study drug until disease progression (up to approximately 2 years)

Best overall response rate as assessed by the investigator by RECIST v1.1 (or by RECIST v1.1 and PCWG3 in participants with advanced prostate cancer).

Secondary Outcome Measures
NameTimeMethod
Disease Control RateFrom first dose of study drug until disease progression (up to approximately 2 years)

Measure of clinical benefit, defined as the percentage of complete response (CR), partial response (PR), and stable disease (SD) beyond 16 weeks.

Overall Response Rate by Independent Radiology ReviewFrom first dose of study drug until disease progression (up to approximately 2 years)

Best overall response rate by independent radiology review by RECIST v1.1 (or by RECIST v1.1 and PCWG3 in participants with advanced prostate cancer).

Progression-free SurvivalFrom first dose of study drug until disease progression (up to approximately 2 years)

Measure of clinical benefit, defined as the duration from study enrollment to objective tumor progression. Progression was defined using RECIST v1.1, as a 20% increase in the sum of diameters of target lesions (and an absolute increase of at least 5 mm), or unequivocal progression of existing non-target lesions, or the appearance of new lesions. For mCRPC disease, the PCWG3 confirmed bone disease progression criteria (2+2) were also incorporated.

Number of Participants Experiencing Treatment-emergent Adverse EventsFrom first dose of study drug until disease progression (up to approximately 2 years)
Duration of ResponseFrom first dose of study drug until disease progression (up to approximately 2 years)

Measure of clinical benefit, defined as the time from initial tumor response to documented tumor progression.

Overall SurvivalFrom first dose of study drug until disease progression (up to approximately 2 years)

Measure of clinical benefit, defined as the duration from study enrollment to death.

Steady State Minimum Concentration [Cmin]From first dose of study drug until disease progression (up to approximately 2 years)

Rucaparib pharmacokinetics

Trial Locations

Locations (18)

University of Iowa Hospital and Clinics

🇺🇸

Iowa City, Iowa, United States

Columbia University Irving Medical Center

🇺🇸

New York, New York, United States

New York Cancer and Blood Specialists

🇺🇸

Port Jefferson Station, New York, United States

New York Cancer And Blood Specialists

🇺🇸

Bronx, New York, United States

UCLA Medicine Hematology and Oncology

🇺🇸

Los Angeles, California, United States

Robert H. Lurie Comprehensive Cancer Center of Northwestern University

🇺🇸

Chicago, Illinois, United States

Beth Israel Deaconess Medical Cancer Surgical Pavilion

🇺🇸

Boston, Massachusetts, United States

Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

UPMC Hillman Cancer Center

🇺🇸

Pittsburgh, Pennsylvania, United States

Seattle Cancer Care Alliance/University of Washington

🇺🇸

Seattle, Washington, United States

UCSF Helen Diller Family Comprehensive Cancer Center

🇺🇸

San Francisco, California, United States

Tennessee Oncology

🇺🇸

Nashville, Tennessee, United States

Stephenson Cancer Center - The University of Oklahoma

🇺🇸

Oklahoma City, Oklahoma, United States

SCRI/Tennessee Oncology - Chattanooga

🇺🇸

Chattanooga, Tennessee, United States

Florida Cancer Specialists

🇺🇸

Saint Petersburg, Florida, United States

H. Lee Moffitt Cancer Center & Research Institute

🇺🇸

Tampa, Florida, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

Ohio State University

🇺🇸

Columbus, Ohio, United States

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