Niraparib in the Treatment of Patients with Advanced PALB2 Mutated Tumors
- Conditions
- Solid TumorBreast TumorColon Tumor, MalignantUrologic CancerMelanomaMetastatic CancerLung TumorHead and Neck CancerPancreatic CancerEsophageal Cancer
- Interventions
- Registration Number
- NCT05169437
- Lead Sponsor
- Tempus AI
- Brief Summary
The purpose of this study is to further evaluate the efficacy and safety of niraparib in patients with locally advanced or metastatic solid tumors and a pathogenic or likely pathogenic tumor PALB2 (tPALB2) mutation.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 22
- Participants must be at least 18 years of age or older.
- Participants must have a histologically or cytologically confirmed diagnosis of locally advanced or metastatic solid tumor(s).
- Participants must have tested positive for a pathogenic or likely pathogenic tPALB2 gene mutation using a CLIA-certified laboratory as described in the Next-Generation Sequencing (NGS) Laboratory Manual.
- Participants who have stable and asymptomatic Central Nervous System (CNS) disease must be receiving a stable (for at least 7 days) or decreasing corticosteroid dose at the time of study entry.
- Participants must submit fresh or archived (collected within 24 months of enrollment) Formalin-Fixed Paraffin-Embedded (FFPE) tumor sample to the central laboratory for post-enrollment confirmation of tPALB2 status.
- Participants must have received all standard therapies appropriate for their tumor type and stage of disease or, in the opinion of the Investigator, the patient would be unlikely to tolerate or derive clinically meaningful benefit from appropriate standard of care therapy, or the participant has no satisfactory alternative treatments.
- Participants have other active concomitant malignancy that warrants systemic, biologic, or hormonal therapy.
- Participants who have ovarian or prostate cancer.
- Participants who have variants of undetermined significance (VUS), but not pathogenic variants of PALB2, at the time of screening.
- Participants who relapsed while receiving platinum based therapy in the adjuvant/curative setting.
- Participants progressing within 14-18 weeks while receiving platinum based therapy in the metastatic setting.
- Participants who have received Poly (ADP-ribose) polymerase (PARP) inhibitor(s) in prior lines of treatment.
- Participants with leptomeningeal disease, carcinomatous meningitis, symptomatic brain metastases, or radiologic signs of CNS hemorrhage.
- Participants with germline or somatic BRCA1 or BRCA2 mutations.
- Participant has systolic blood pressure (BP) over 140 mmHg or diastolic BP over 90 mmHg, despite optimal medical therapy.
- Participants have previously or are currently participating in a treatment study of an investigational agent within 3 weeks of the first dose of therapy preceding the study.
- Participants have received prior systemic cytotoxic chemotherapy, biological therapy, or hormonal therapy for cancer, or received radiation therapy within 3 weeks of the first dose therapy preceding the study.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Niraparib in Locally Advanced or Metastatic Solid Tumor Patients with PALB2 Mutations Niraparib -
- Primary Outcome Measures
Name Time Method Overall Response Rate (ORR) - Independent Central Review (ICR) Up to 4 years To evaluate overall response rate (ORR) as assessed by Independent Central Review (ICR) using RECIST v1.1
- Secondary Outcome Measures
Name Time Method Overall Survival (OS) Up to 4 years To evaluate overall survival (OS)
Progression-Free Survival (PFS) - Independent Central Review (ICR) Up to 4 years To evaluate progression-free survival (PFS) as assessed by ICR using Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1)
Clinical Benefit Rate (CBR) - Investigator and ICR Up to 4 years To evaluate Clinical Benefit Rate (CBR) as assessed by ICR and Investigator
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) Up to 4 years To evaluate safety and tolerability per the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (NCI-CTCAE v5.0)
Overall Response Rate (ORR) - Investigator Up to 4 years To evaluate ORR as assessed by Investigator using RECIST v1.1
Progression-Free Survival (PFS) - Investigator Up to 4 years To evaluate PFS as assessed by Investigator using RECIST v1.1
ORR with untreated measurable CNS lesions - Investigator Up to 4 years To evaluate intracranial ORR in participants with untreated measurable CNS lesions as assessed by Investigator using RECIST v1.1
ORR with untreated measurable CNS lesions - ICR Up to 4 years To evaluate intracranial ORR in participants with untreated measurable CNS lesions as assessed by ICR using RECIST v1.1
Duration of Response (DOR) - Independent Central Review (ICR) Up to 4 years To evaluate duration of response (DOR) as assessed by ICR using RECIST v1.1
Duration of Response (DOR) - Investigator Up to 4 years To evaluate DOR as assessed by Investigator using RECIST v1.1
Trial Locations
- Locations (79)
Yuma Regional Medical Center
🇺🇸Yuma, Arizona, United States
Highlands Oncology
🇺🇸Springdale, Arkansas, United States
Memorial Care Medical Center
🇺🇸Fountain Valley, California, United States
St Joseph Heritage Health - Fullerton
🇺🇸Fullerton, California, United States
University of California San Diego
🇺🇸La Jolla, California, United States
MemorialCare
🇺🇸Long Beach, California, United States
Cancer and Blood Specialty Clinic
🇺🇸Los Alamitos, California, United States
Cancer and Blood Specialty
🇺🇸Los Alamitos, California, United States
University of California Los Angeles
🇺🇸Los Angeles, California, United States
St Joseph Health Medical Group - Napa
🇺🇸Napa, California, United States
Scroll for more (69 remaining)Yuma Regional Medical Center🇺🇸Yuma, Arizona, United States