Niraparib in the Treatment of Patients With Advanced PALB2 Mutated Tumors

NCT05169437 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: TMPS-101IND Number: 159142
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 80

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.

Conditions

Solid Tumor; Breast Tumor; Colon Tumor, Malignant; Lung Tumor; Urologic Cancer; Pancreatic Cancer; Melanoma; Metastatic Cancer; Locally Advanced Solid Tumor; Esophageal Cancer; Endometrial Cancer; Head and Neck Cancer

Keywords

PALB2; Solid Tumor; Metastatic Solid Tumor; Locally Advanced Solid Tumor; Advanced Solid Tumor; Local Solid Tumor; PALB2 Mutation; Niraparib; tPALB2; tPALB2 Mutation; Pathogenic tumor; Lung Tumor; Breast Tumor; Colon Tumor; Zejula; Pancreatic Cancer; Urologic Cancer; Melanoma; Metastatic Cancer; Head and Neck Cancer; Endometrial Cancer; Esophageal Cancer

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate (ORR) - Independent Central Review (ICR)

  To evaluate overall response rate (ORR) as assessed by Independent Central Review (ICR) using RECIST v1.1

  Up to 4 years

Secondary Outcomes (10)

• Duration of Response (DOR) - Independent Central Review (ICR)

  Up to 4 years

• Progression-Free Survival (PFS) - Independent Central Review (ICR)

  Up to 4 years

• Overall Response Rate (ORR) - Investigator

  2 years 3 months

• Duration of Response (DOR) - Investigator

  2 years 3 months

• Progression-Free Survival (PFS) - Investigator

  2 years 3 months

Study Arms (1)

Niraparib in Locally Advanced or Metastatic Solid Tumor Patients with PALB2 Mutations

EXPERIMENTAL

Drug: Niraparib

Interventions

Niraparib DRUG

Eligible participants will receive daily dosing of Niraparib.

Also known as: Zejula

Niraparib in Locally Advanced or Metastatic Solid Tumor Patients with PALB2 Mutations

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Tempus AI: lead
• GlaxoSmithKline: collaborator

Study Sites (80)

Yuma Regional Medical Center

Yuma, Arizona, 85364, United States

Location

Highlands Oncology

Springdale, Arkansas, 72762, United States

Location

Memorial Care Medical Center

Fountain Valley, California, 92708, United States

Location

St Joseph Heritage Health - Fullerton

Fullerton, California, 92835, United States

Location

University of California San Diego

La Jolla, California, 92093-0698, United States

Location

MemorialCare

Long Beach, California, 90806, United States

Location

Cancer and Blood Specialty Clinic

Los Alamitos, California, 90720, United States

Location

Cancer and Blood Specialty

Los Alamitos, California, 90720, United States

Location

University of California Los Angeles

Los Angeles, California, 90404, United States

Location

St Joseph Health Medical Group - Napa

Napa, California, 94558, United States

Location

Ventura County Hematology Oncology Specialists

Oxnard, California, 93030, United States

Location

Sharp Healthcare

San Diego, California, 92123, United States

Location

Ridley-Tree Cancer Center

Santa Barbara, California, 93105, United States

Location

St Joseph Health Medical Group - Santa Rosa

Santa Rosa, California, 95403, United States

Location

Hartford Healthcare

Hartford, Connecticut, 06102, United States

Location

Eastern Connecticut Hematology and Oncology

Norwich, Connecticut, 06360, United States

Location

Holy Cross

Fort Lauderdale, Florida, 33308, United States

Location

Cancer Specialists of North Florida

Jacksonville, Florida, 32256, United States

Location

Ocala Community Cancer Center

Ocala, Florida, 34474, United States

Location

University Cancer & Blood Center

Athens, Georgia, 30607, United States

Location

Hawaii Cancer Care

Honolulu, Hawaii, 96813, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Northwest Oncology & Hematology

Rolling Meadows, Illinois, 60008, United States

Location

Fort Wayne Medical Oncology and Hematology

Fort Wayne, Indiana, 46845, United States

Location

Goshen Health

Goshen, Indiana, 46526, United States

Location

Community Health Network

Indianapolis, Indiana, 46250, United States

Location

Beacon Health System

South Bend, Indiana, 46601, United States

Location

Pontchartrain Cancer Center

Hammond, Louisiana, 70403, United States

Location

The Center for Cancer and Blood Disorders - Maryland

Bethesda, Maryland, 20817, United States

Location

Frederick Health

Frederick, Maryland, 21702, United States

Location

Maryland Oncology Hematology

Rockville, Maryland, 20850, United States

Location

Southcoast Health

Fairhaven, Massachusetts, 02719, United States

Location

Sparrow Health

Lansing, Michigan, 48912, United States

Location

Central Care Cancer Center

Bolivar, Missouri, 65613, United States

Location

Mosaic Life Care

Saint Joseph, Missouri, 64507, United States

Location

Oncology Hematology Associates

Springfield, Missouri, 65807, United States

Location

Nebraska Cancer Specialists

Omaha, Nebraska, 68130, United States

Location

OptumCare Cancer Care

Las Vegas, Nevada, 89102, United States

Location

New Jersey Cancer Care and Blood Disorders

Belleville, New Jersey, 07109, United States

Location

Englewood Health

Englewood, New Jersey, 07631, United States

Location

Summit Medical Group

Florham Park, New Jersey, 07932, United States

Location

Novant Health Inc. - Charlotte

Charlotte, North Carolina, 28204, United States

Location

Southeastern Medical Oncology Center

Goldsboro, North Carolina, 27534, United States

Location

Novant Health Inc. - Winston-Salem

Winston-Salem, North Carolina, 27103, United States

Location

Aultman Medical Group

Canton, Ohio, 44708, United States

Location

TriHealth

Cincinnati, Ohio, 45220, United States

Location

University Hospitals Seidman

Cleveland, Ohio, 44106, United States

Location

Cleveland Clinic - Taussig Cancer Center

Cleveland, Ohio, 44195, United States

Location

OhioHealth

Columbus, Ohio, 43214, United States

Location

The Toledo Clinic

Toledo, Ohio, 43623, United States

Location

Oklahoma Cancer Specialists

Tulsa, Oklahoma, 74146, United States

Location

Oregon Oncology Specialists

Salem, Oregon, 97301, United States

Location

Gettysburg Cancer Center

Gettysburg, Pennsylvania, 17325, United States

Location

Bon Secours - St. Francis Cancer Center

Greenville, South Carolina, 29607, United States

Location

Avera Cancer Institute

Sioux Falls, South Dakota, 57105, United States

Location

Sanford Health

Sioux Falls, South Dakota, 57117, United States

Location

Baptist Cancer Center

Memphis, Tennessee, 38120, United States

Location

Vanderbilt University

Nashville, Tennessee, 37232, United States

Location

Texas Oncology - Austin Midtown

Austin, Texas, 78705, United States

Location

Texas Oncology - Austin Central Pharmacy

Austin, Texas, 78731, United States

Location

Texas Oncology - South Austin

Austin, Texas, 78745, United States

Location

Mary Crowley Cancer Research

Dallas, Texas, 75230, United States

Location

Texas Oncology - Baylor Charles A. Sammons Cancer Center

Dallas, Texas, 75246, United States

Location

The University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Oncology Consultants

Houston, Texas, 77030, United States

Location

Texas Oncology - Longview Cancer Center

Longview, Texas, 75601, United States

Location

Texas Oncology - Palestine Cancer Center

Palestine, Texas, 75801, United States

Location

Texas Oncology - Paris Cancer Center

Paris, Texas, 75460, United States

Location

Lumi Research

Sugar Land, Texas, 77479, United States

Location

Texas Oncology - Tyler Pharmacy

Tyler, Texas, 75702, United States

Location

Community Cancer Trials of Utah

Ogden, Utah, 84405, United States

Location

Utah Cancer Specialists

Salt Lake City, Utah, 84106, United States

Location

Inova Schar Institute

Fairfax, Virginia, 22031, United States

Location

Hematology Oncology Associates of Fredericksburg

Fredericksburg, Virginia, 22408, United States

Location

Virginia Cancer Institute

Richmond, Virginia, 23229, United States

Location

Virginia Cancer Institute

Richmond, Virginia, 23230, United States

Location

PeaceHealth

Bellingham, Washington, 98225, United States

Location

Northwest Medical Specialties

Tacoma, Washington, 98405, United States

Location

ThedaCare

Appleton, Wisconsin, 54911, United States

Location

SSM Health

Madison, Wisconsin, 53717, United States

Location

MeSH Terms

Conditions

Breast Neoplasms; Colonic Neoplasms; Lung Neoplasms; Urologic Neoplasms; Pancreatic Neoplasms; Melanoma; Neoplasm Metastasis; Esophageal Neoplasms; Endometrial Neoplasms; Head and Neck Neoplasms

Interventions

niraparib

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Breast Diseases; Skin Diseases; Skin and Connective Tissue Diseases; Colorectal Neoplasms; Intestinal Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Colonic Diseases; Intestinal Diseases; Respiratory Tract Neoplasms; Thoracic Neoplasms; Lung Diseases; Respiratory Tract Diseases; Urogenital Neoplasms; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Urologic Diseases; Endocrine Gland Neoplasms; Pancreatic Diseases; Endocrine System Diseases; Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Nerve Tissue; Nevi and Melanomas; Skin Neoplasms; Neoplastic Processes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Esophageal Diseases; Uterine Neoplasms; Genital Neoplasms, Female; Uterine Diseases; Genital Diseases, Female; Genital Diseases

Results Point of Contact

• Title: Virginia Rhodes, MD
• Organization: Tempus AI, Inc

support@tempus.com

(800) 976-5448

Study Officials

• Virginia Rhodes

  Tempus AI, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 13, 2021
• First Posted: December 27, 2021
• Study Start: March 15, 2022
• Primary Completion: August 6, 2024
• Study Completion: August 27, 2024
• Last Updated: October 29, 2025
• Results First Posted: October 29, 2025

Record last verified: 2025-10

Locations

US (80)