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A Study of Rucaparib in Patients With Platinum-Sensitive, Relapsed, High-Grade Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer (ARIEL2)

Phase 2
Completed
Conditions
Ovarian Cancer
Fallopian Tube Cancer
Epithelial Ovarian Cancer
Peritoneal Cancer
Interventions
Registration Number
NCT01891344
Lead Sponsor
pharmaand GmbH
Brief Summary

The purpose of this study is to determine which patients with ovarian, fallopian tube, and primary peritoneal cancer will best respond to treatment with rucaparib.

Detailed Description

Rucaparib is an orally available, small molecule inhibitor of poly-adenosine diphosphate \[ADP\] ribose polymerase (PARP) being developed for treatment of ovarian cancer associated with homologous recombination (HR) DNA repair deficiency (HRD). The safety and efficacy of rucaparib has been evaluated in several Phase 1 and Phase 2 studies. An oral formulation is the focus of current development efforts. Rucaparib is currently being investigated as monotherapy in patients with cancer associated with breast cancer susceptibility gene 1 (BRCA1) or BRCA2 mutations.

Clinical data with PARP inhibitors indicate there is an ovarian cancer patient population beyond just those with germline BRCA (gBRCA) mutations that may benefit from treatment with a PARP inhibitor. This study will define a molecular signature of HRD in ovarian cancer that correlates with response to rucaparib and enables selection of appropriate ovarian cancer patients for treatment with rucaparib. The HRD signature will be based on an association between the extent of genomic scarring (a downstream consequence of HRD) in a patient's tumor and observed clinical benefit from rucaparib treatment. Genomic scarring can be assessed by quantifying the extent of loss of heterozygosity across the tumor genome (tumor genomic LOH). One of the main advantages of detecting tumor genomic LOH is that it can identify HRD tumors regardless of the underlying mechanisms, which include both known (i.e., BRCA mutations) and unknown genetic and other mechanisms.

Once determined, this signature will be prospectively applied to ARIEL2 PART 2 and ARIEL3. This Phase 2 study (ARIEL2) will also compare archival versus recently collected tumor tissue in order to validate the use of archival tumor tissue for assessment of HRD status in ARIEL3.

This study will include 2 parts:

PART 1 (completed enrollment): Evaluation of HRD status and rucaparib efficacy in patients who received ≥1 prior platinum-based regimen and had platinum-sensitive disease

PART 2 (completed enrollment): Evaluation of HRD status and rucaparib efficacy in patients who received at least 3 prior chemotherapy regimens

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
491
Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Ovarian cancerOral rucaparibrucaparib
Primary Outcome Measures
NameTimeMethod
Progression-free Survival (PFS) According to RECIST v1.1 in Molecularly-defined HRD (Homologous Recombination Deficiency) Subgroups (Part 1 of Study)Assessments every 8 weeks from C1D1 until disease progression, death or withdrawal of consent. After 18 months on study, assessments every 16 weeks. Total follow-up was up to approximately 3 years.

The primary efficacy endpoint of PFS is calculated as 1+ the number of days from the first dose of study drug to disease progression by RECIST (Response Evaluation Criteria in Solid Tumors), as determined by the investigator or death due to any cause, whichever occurs first. Progression is defined using RECIST v1.1, as at least a 20% increase in the sum of the longest diameter of target lesions, or a measureable increase in a non-target lesion, or the appearance of new lesions.

Objective Response Rate (ORR) by RECIST v1.1 in Molecularly-defined HRD Subgroups (Part 2 of Study)Assessments every 8 weeks from C1D1 until disease progression, death or withdrawal of consent. After 18 months on study, assessments every 16 weeks. Total follow-up was up to approximately 3 years.

The confirmed response rate by RECIST v1.1 is defined as the percentage of patients with a confirmed complete response (CR) or partial response (PR) on subsequent tumor assessment at least 28 days after first response documentation. Complete response (CR) is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum of longest diameter.

Secondary Outcome Measures
NameTimeMethod
Duration of Response Per RECIST v1.1Assessments every 8 weeks from C1D1 until disease progression, death or withdrawal of consent. After 18 months on study, assessments every 16 weeks. Total follow-up was up to approximately 3 years.

Duration of response (DOR) for any confirmed RECIST CR or PR measured from the date of the first occurrence of a response until the first occurrence of progressive disease (PD) per RECIST. For patients who continued treatment post-progression, the first date of progression was used for the analysis. Any patients with an ongoing response were censored at the date of the last post-baseline scan. Complete response (CR) is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum of longest diameter.

Progression-free Survival (PFS) According to RECIST v1.1 in Molecularly-defined HRD Subgroups (Part 2 of Study)Assessments every 8 weeks from C1D1 until disease progression, death or withdrawal of consent. After 18 months on study, assessments every 16 weeks. Total follow-up was up to approximately 3 years.

Progression-Free Survival (PFS) is calculated as 1+ the number of days from the first dose of study drug to disease progression by RECIST, as determined by the investigator or death due to any cause, whichever occurs first. Progression is defined using RECIST v1.1, as at least a 20% increase in the sum of the longest diameter of target lesions, or a measureable increase in a non-target lesion, or the appearance of new lesions.

Objective Response Rate (ORR) by RECIST v1.1 (Part 1 of Study)Assessments every 8 weeks from C1D1 until disease progression, death or withdrawal of consent. After 18 months on study, assessments every 16 weeks. Total follow-up was up to approximately 3 years.

The confirmed response rate by RECIST v1.1 is defined as the percentage of patients with a confirmed CR or PR on subsequent tumor assessment at least 28 days after first response documentation. Complete response (CR) is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum of longest diameter.

Overall Survival (Part 2 of Study)All patients in Part 2 were followed for survival, subsequent therapy, and secondary malignancy every 12 weeks until death, loss to follow-up, withdrawal of consent from study or study closure, whichever happened first, up to 7 years.

Overall survival (OS) is defined as the number of days from the date of first dose of study drug to the date of death (due to any cause). Patients without a known date of death will be censored on the date the patient was last known to be alive.

Objective Response Rate (ORR) by RECIST v1.1 and GCIG CA-125 CriteriaAssessments every 8 weeks from C1D1 until disease progression, death or withdrawal of consent. After 18 months on study, assessments every 16 weeks. Total follow-up was up to approximately 3 years.

The endpoint of ORR defined as the percentage of patients with a best response of CR or PR using RECIST v 1.1 or a response per Gynecologic Cancer InterGroup cancer antigen 125 (GCIG CA-125) criteria. Complete response (CR) is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial response (PR) is at least a 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum of longest diameter. A response to CA-125 has occurred if there is at least a 50% decrease from baseline: 1. in a sample collected after initiation of study treatment AND 2. that is confirmed in a subsequent sample collected ≥21 days after the prior sample. The absolute value of this confirmatory sample must be ≤110% of the prior sample. The date when the first sample with a 50% decrease from baseline is observed is the date of the CA-125 response.

Steady State Trough (Cmin) Level Rucaparib ConcentrationsCycle 1 Day 15 to Cycle 4 Day 1, or approximately 10 weeks

Per protocol, the secondary PK endpoint, trough (Cmin) concentrations of rucaparib were summarized with descriptive statistics overall and by cycle in all patients with at least one PK sample collected. Blood samples for trough level PK analysis of rucaparib were drawn at the following timepoints only: on Day 15 of Cycle 1 and on Day 1 of Cycles 2, 3, and 4. Data for other timepoints is not available.

Trial Locations

Locations (75)

Saint Jude Heritage Medical Center

🇺🇸

Fullerton, California, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

Prince of Wales Hospital

🇦🇺

Sydney, New South Wales, Australia

Johns Hopkins Kimmel Cancer Center

🇺🇸

Baltimore, Maryland, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Dana-Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

The University of Texas MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

University of Washington - Seattle Cancer Care Alliance

🇺🇸

Seattle, Washington, United States

UC San Diego

🇺🇸

San Diego, California, United States

University of Miami Hospital & Clinics Sylvester Comprehensive Cancer Center

🇺🇸

Miami, Florida, United States

The Ohio State University Wexner Medical Center

🇺🇸

Columbus, Ohio, United States

Royal Melbourne Hospital

🇦🇺

Parkville, Victoria, Australia

Tom Baker Cancer Centre

🇨🇦

Calgary, Alberta, Canada

University of Cincinnati Physicians Company

🇺🇸

Cincinnati, Ohio, United States

Crown Princess Mary Cancer Centre (Westmead Hospital)

🇦🇺

Westmead, Wentworthville, Australia

Royal North Shore Hospital

🇦🇺

Saint Leonards, New South Wales, Australia

UPMC Cancer Center

🇺🇸

Pittsburgh, Pennsylvania, United States

Horizon BioAdvance

🇺🇸

Lafayette, Indiana, United States

Flinders Cancer Clinic - Flinders Medical Centre (FMC)

🇦🇺

Bedford Park, South Australia, Australia

Vancouver Cancer Centre, British Columbia Cancer Agency (BCCA)

🇨🇦

Vancouver, British Columbia, Canada

Cross Cancer Centre

🇨🇦

Edmonton, Alberta, Canada

The Christie NHS Foundation Trust

🇬🇧

Manchester, United Kingdom

Jewish General Hospital

🇨🇦

Montreal, Quebec, Canada

Institut Bergonie

🇫🇷

Bordeaux, Aquitaine, France

Princess Margaret Cancer Centre

🇨🇦

Toronto, Ontario, Canada

Royal Marsden NHS Foundation Trust

🇬🇧

London, United Kingdom

Institut Claudius Regaud

🇫🇷

Toulouse, Midi-Pyrenees, France

St James University Hospital

🇬🇧

Leeds, West Yorkshire, United Kingdom

Hospital Clinico Universitario de Valencia

🇪🇸

Valencia, Spain

Royal Marsden Sutton Hospital

🇬🇧

Sutton, Surrey, United Kingdom

Addenbrooke's Hospital

🇬🇧

Cambridge, United Kingdom

Centre Hospitalier de L'Universite de Montreal

🇨🇦

Montreal, Quebec, Canada

Hospital Vall d'Hebron

🇪🇸

Barcelona, Spain

Hôpital Européen Georges-Pompidou

🇫🇷

Paris, Ile-de-France, France

Charles Gairdner Hospital

🇦🇺

Nedlands, Western Australia, Australia

Memorial Sloan-Kettering Cancer Center

🇺🇸

New York, New York, United States

Rocky Mountain Cancer Centers

🇺🇸

Lakewood, Colorado, United States

Mercy Hospital for Women

🇦🇺

Heidelberg, Victoria, Australia

British Columbia Cancer Agency

🇨🇦

Kelowna, British Columbia, Canada

BC Cancer Agency - Fraser Valley Centre

🇨🇦

Surrey, British Columbia, Canada

Beatson West of Scotland Cancer Centre

🇬🇧

Glasgow, Scotland, United Kingdom

Sir Bobby Robson Cancer Trials Research Centre, Northern Centre for Cancer Care

🇬🇧

Newcastle upon Tyne, United Kingdom

Institut de cancerologie Gustave Roussy

🇫🇷

Villejuif, Ile-de-France, France

Centre Leon Berard

🇫🇷

Lyon, Rhone-Alpes, France

Providence Alaska Medical Center

🇺🇸

Anchorage, Alaska, United States

University of Arizona Cancer Center

🇺🇸

Tucson, Arizona, United States

Coastal Integrative Cancer Care

🇺🇸

San Luis Obispo, California, United States

Central Coast Medical Oncology

🇺🇸

Santa Maria, California, United States

Mayo Clinic Jacksonville

🇺🇸

Jacksonville, Florida, United States

Stanford University

🇺🇸

Stanford, California, United States

Altus Research

🇺🇸

Lake Worth, Florida, United States

New York University Langone Medical Center

🇺🇸

New York, New York, United States

Women's Cancer Care Associates

🇺🇸

Albany, New York, United States

Hope - A Woman's Cancer Institute

🇺🇸

Asheville, North Carolina, United States

Fox Chase Cancer Center

🇺🇸

Philadelphia, Pennsylvania, United States

London Regional Cancer Centre

🇨🇦

London, Ontario, Canada

Ottawa Hospital Cancer Centre

🇨🇦

Ottawa, Ontario, Canada

Hopital Tenon

🇫🇷

Paris, Ile-de-France, France

CHU de Québec - Université Laval

🇨🇦

Québec, Canada

Centre Catherine de Sienne

🇫🇷

Nantes, Pays De La Loire, France

Instituto Valencia de Oncologia

🇪🇸

Valencia, Spain

Centre Hospitalier Lyon Sud

🇫🇷

Pierre-Benite, Rhone-Alpes, France

Imperial College Healthcare NHS Trust - Hammersmith Hospital

🇬🇧

London, United Kingdom

University College London

🇬🇧

London, United Kingdom

University of California Los Angeles

🇺🇸

Los Angeles, California, United States

Royal Brisbane & Women's Hospital

🇦🇺

Herston, Queensland, Australia

Comprehensive Cancer Centers of Nevada

🇺🇸

Henderson, Nevada, United States

University of California, San Francisco

🇺🇸

San Francisco, California, United States

Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

California Pacific Medical Center

🇺🇸

San Francisco, California, United States

Florida Hospital Cancer Institute

🇺🇸

Orlando, Florida, United States

UF Health Cancer Center

🇺🇸

Orlando, Florida, United States

Norton Cancer Institute

🇺🇸

Louisville, Kentucky, United States

University of Oklahoma

🇺🇸

Oklahoma City, Oklahoma, United States

Washington University School of Medicine

🇺🇸

Saint Louis, Missouri, United States

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