Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)
Study Purpose
A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes
Recruitment Criteria
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Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms |
No |
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Study Type
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies. |
Interventional |
| Eligible Ages | 12 Years and Over |
| Gender | All |
Inclusion Criteria:
1. Patients must have a malignant solid tumor with a pathogenic inactivating TSC1 or TSC2 alteration. Genetic alterations should be identified using NGS in tumor tissue or liquid biopsy). • Patients will be enrolled after the central evaluation of NGS report confirms eligibility. 2. Patients must have solid tumors that are metastatic or locally advanced where surgical resection is not an option or likely to result in severe morbidity. 3. Patients 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 patient has no satisfactory alternative treatments. 4. Patients must have 1 or more measurable target lesions by computed tomography (CT) scan or magnetic resonance imaging (MRI) (RECIST v1.1). 5. Age: 12 years or older. 6. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1 or Karnofsky Performance Status (KPS) ≥80 or Lansky play-performance scale for pediatric patients ≥80. 7. Adequate liver function: 1. Total bilirubin ≤1.5 × upper limit of normal (ULN) (unless due to Gilbert's syndrome, then ≤3 × ULN) 2. Aspartate aminotransferase (AST) ≤2.5 × ULN (≤5 × ULN if attributable to liver metastases) 8. Adequate renal function: creatinine clearance ≥30 mL/min, Cockcroft-Gault CCr = ((140-age) × weight[kg]) / (72 × SCr[mL/min]) × 0.85, if female. 9. Adequate hematologic parameters: 1. Absolute neutrophil count (ANC) ≥1.0 × 109/L (growth factor support allowed) 2. Platelet count ≥100,000/mm3 (100 × 109/L) (transfusion and/or growth factor support allowed) 3. Hemoglobin ≥8.0 g/dL (transfusion and/or growth factor support allowed) 10. Fasting serum triglyceride must be ≤300 mg/dL; fasting serum cholesterol must be ≤350 mg/dL. 11. Minimum of 4 weeks since any major surgery, completion of radiation, or completion of prior systemic anticancer therapy, or at least 5 half-lives if the prior therapy is a single agent small-molecule therapeutic, and adequately recovered from the acute toxicities of any prior therapy, including neuropathy, to Grade ≤1. 12. Male or non-pregnant and non-breastfeeding female: 1. Females of childbearing potential must agree to use effective contraception or abstinence without interruption from 28 days prior to starting investigational product (IP) throughout 3 months after last dose of IP and have a negative serum pregnancy test (beta human chorionic gonadotropin, β-hCG) result at screening and agree to ongoing pregnancy testing during the course of the study, and after the end of study treatment. A second form of birth control is required even if she has had a tubal ligation. 2. Male patients must agree not to donate sperm and must practice abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study and throughout 3 months after last dose of IP. A second form of birth control is required even if he has undergone a successful vasectomy. 13. The patient or the patient's parent(s) or legal guardian(s) understand(s) and sign(s) the informed consent. 14. Willingness and ability to comply with scheduled visits, laboratory tests, and other study procedures.Exclusion Criteria:
1. Prior treatment with an mTOR inhibitor, including nab-sirolimus. 2. Severe (Grade ≥3) ongoing infection requiring parenteral or oral anti-infective treatment, either ongoing or completed ≤7 days prior to enrollment. 3. Patients with primary brain tumors or PEComa. 4. Patients who have any severe and/or uncontrolled medical or psychiatric conditions or other conditions that could affect their participation including: 1. Patients with meningeal carcinomatosis, leptomeningeal carcinomatosis, spinal cord compression, untreated brain metastases or symptomatic or unstable brain metastases. Note: Patients with stable brain metastases (defined as asymptomatic or no requirement for high-dose [defined as dexamethasone 10 mg daily or higher] or increasing dose of systemic corticosteroids) and without imminent need of radiation therapy are eligible. If applicable, patients must have completed brain radiation therapy and recovered adequately from any associated toxicity and/or complications prior to eligibility assessment. For patients who have received prior radiation therapy, post-treatment MRI scan should show no increase in brain lesion size/volume. 2. Unstable angina pectoris, symptomatic congestive heart failure (New York Heart Association, NYHA class III or IV), myocardial infarction ≤6 months prior to first study treatment, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease. 3. Pre-existing severely impaired lung function. If a patient has a pre-existing pulmonary condition, eligible patients should have a spirometry and diffusing capacity for carbon monoxide (DLCO) that is >50% of the normal predicted value and/or O2 saturation that is >88% at rest on room air (Note: spirometry and pulmonary function tests [PFTs] not required to be performed unless clinically indicated). 4. Nonmalignant medical illnesses that are uncontrolled or whose control may be jeopardized by the treatment with the study therapy. 5. A history of malignancies other than the one under treatment unless the patient is disease-free for more than 5 years from diagnosis. Note, controlled non-melanoma skin cancers, carcinoma in situ of the cervix, resected incidental prostate cancer, certain low grade hematologic malignancies (eg CLL, follicular lymphoma, etc), or other adequately treated carcinoma-in-situ may be eligible, after discussion with the medical monitor. 6. Uncontrolled hypertension (systolic blood pressure ≥160 mm-Hg and/or diastolic blood pressure ≥100 mm Hg). 7. Patients with history of interstitial lung disease and/or pneumonitis, or pulmonary hypertension. 8. Individuals with known human immunodeficiency virus (HIV) infection are excluded from this study as combination antiretroviral therapy could potentially result in significant pharmacokinetic interactions. In addition, these individuals are at increased risk of serious infections due to the immunosuppressive effects of mTOR inhibition. 9. Active Hepatitis B or Hepatitis C, with detectable viral load. 5. Regarding concomitant medications with significant CYP3A4 and P-gp interactions, discontinuation of strong inhibitors (eg, ketoconazole, itraconazole, voriconazole, erythromycin, clarithromycin, telithromycin, and others), strong inducers (eg, rifampin, rifabutin), and known CYP3A4 substrates with a narrow therapeutic window (eg, fentanyl, alfentanil, astemizole, cisapride, dihydroergotamine, pimozide, quinidine, or terfenadine) is required at least 5 half lives prior to receiving the first dose of nab-sirolimus, whichever is longer.Trial Details
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Trial ID:
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries. |
NCT05103358 |
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Phase
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use. |
Phase 2 |
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Lead Sponsor
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data. |
Aadi Bioscience, Inc. |
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Principal Investigator
The person who is responsible for the scientific and technical direction of the entire clinical study. |
N/A |
| Principal Investigator Affiliation | N/A |
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Agency Class
Category of organization(s) involved as sponsor (and collaborator) supporting the trial. |
Industry |
| Overall Status | Active, not recruiting |
| Countries | Korea, Republic of, Puerto Rico, United States |
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Conditions
The disease, disorder, syndrome, illness, or injury that is being studied. |
Tumor, Tumor, Solid, Metastasis, Metastatic Cancer, Cancer, Cancer Metastatic, Tumors, Neoplasms, Neoplasm Metastasis, Solid Tumor, Advanced Solid Tumor, Advanced Cancer, Malignant Solid Tumor, Malignant Solid Neoplasm, Malignant Neoplasm, Malignant Tumor, TSC, TSC1, TSC2, Metastatic Solid Tumor, Metastatic Neoplasm |
Study TSC-007 is a prospective phase 2, open-label, multi-institutional basket trial to determine the efficacy and safety profile of nab-sirolimus administered to patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes. Patients will be treated with single agent IV nab-sirolimus until disease progression, or unacceptable toxicity, or until in the opinion of the investigator the patient is no longer benefiting from therapy, or at patient discretion.
Arms
Experimental: Arm A: Pathogenic inactivating TSC1 alterations
Patients with pathogenic inactivating TSC1 alterations.
Experimental: Arm B: Pathogenic inactivating TSC2 alterations
Patients with pathogenic inactivating TSC2 alterations.
Interventions
Drug: - nab-sirolimus
Prospective phase 2, open-label, multi-institutional basket trial to determine the efficacy and safety of nab-sirolimus administered by IV infusion to patients
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.
Status
Address
Alabama Oncology
Birmingham, Alabama, 35243
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Address
Southern Cancer Center
Mobile, Alabama, 36607
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Address
Arizona Oncology Associates
Goodyear, Arizona, 85395
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Address
Honor Health
Phoenix, Arizona, 85016
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Address
Arizona Oncology Associates
Prescott Valley, Arizona, 86301
Status
Address
Yuma Regional Medical Center
Yuma, Arizona, 85364
Status
Address
PCR Oncology
Arroyo Grande, California, 93420
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Address
Nextgen Oncology
Beverly Hills, California, 90212
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Address
City of Hope
Duarte, California, 91010
Status
Address
Providence Medical Foundation (Fullerton)
Fullerton, California, 92835
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Address
MemorialCare
Long Beach, California, 90806
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Address
USC Norris Comprehensive Cancer Center
Los Angeles, California, 90033
Status
Address
UCLA - Jonsson Comprehensive Cancer Center
Los Angeles, California, 90095
Status
Address
Providence Medical Foundation (Napa)
Napa, California, 94558
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Address
Hoag Memorial Hospital Presbyterian
Newport Beach, California, 92663
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Address
Sharp HealthCare
San Diego, California, 92123
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Address
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94115
Status
Address
Ridley-Tree Cancer Center
Santa Barbara, California, 93105
Status
Address
Sarcoma Oncology Research Center
Santa Monica, California, 90403
Status
Address
Providence Medical Foundation
Santa Rosa, California, 95403
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Address
Stanford Cancer Center
Stanford, California, 94305
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Address
The Oncology Institute of Hope & Innovation
Whittier, California, 90602
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Address
Rocky Mountain Cancer Centers
Aurora, Colorado, 80012
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Address
Rocky Mountain Cancer Centers
Boulder, Colorado, 80303
Status
Address
Rocky Mountain Cancer Centers
Colorado Springs, Colorado, 80907
Status
Address
Rocky Mountain Cancer Centers (Williams St)
Denver, Colorado, 80218
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Address
Rocky Mountain Cancer Centers
Denver, Colorado, 80220
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Address
Rocky Mountain Cancer Centers
Lakewood, Colorado, 80228
Status
Address
Rocky Mountain Cancer Centers
Littleton, Colorado, 80120
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Address
Rocky Mountain Cancer Centers
Lone Tree, Colorado, 80124
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Address
Rocky Mountain Cancer Centers
Longmont, Colorado, 80501
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Address
Rocky Mountain Cancer Centers
Pueblo, Colorado, 81008
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Address
Rocky Mountain Cancer Centers
Thornton, Colorado, 80260
Status
Address
Hartford Healthcare
Hartford, Connecticut, 06102
Status
Address
Eastern Connecticut Hematology and Oncology
Norwich, Connecticut, 06360
Status
Address
Florida Cancer Specialists - North Division
Altamonte Springs, Florida, 32701
Status
Address
Florida Cancer Specialists - South Division
Bonita Springs, Florida, 34135
Status
Address
Florida Cancer Specialists - South Division
Bradenton, Florida, 34205
Status
Address
Florida Cancer Specialists - South Division
Bradenton, Florida, 34211
Status
Address
Florida Cancer Specialists - North Division
Brandon, Florida, 33511
Status
Address
Florida Cancer Specialists - South Division
Cape Coral, Florida, 33909
Status
Address
Florida Cancer Specialists - North Division
Clearwater, Florida, 33761
Status
Address
Cancer Specialist - East
Daytona Beach, Florida, 32117
Status
Address
Holy Cross Hospital
Fort Lauderdale, Florida, 33308
Status
Address
Florida Cancer Specialists South Division
Fort Myers, Florida, 33901
Status
Address
Florida Cancer Specialists - South
Fort Myers, Florida, 33905
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Address
Florida Cancer Specialists - South
Fort Myers, Florida, 33908
Status
Address
Florida Cancer Specialists - North Division
Gainesville, Florida, 32605
Status
Address
Cancer Specialists of North Florida
Jacksonville, Florida, 32256
Status
Address
The Oncology Institute of Hope and Innovation
Lakeland, Florida, 33812
Status
Address
TOI Florida
Lakeland, Florida, 33812
Status
Address
Florida Cancer Specialists - North Division
Largo, Florida, 33770
Status
Address
Florida Cancer Specialists - North Division
Lecanto, Florida, 34461
Status
Address
Florida Cancer Specialists - South Division
Naples, Florida, 34102
Status
Address
Florida Cancer Specialists - North Division
Ocala, Florida, 34474
Status
Address
Ocala Oncology
Ocala, Florida, 34474
Status
Address
Florida Cancer Specialists - North Division
Orange City, Florida, 32763
Status
Address
Florida Cancer Specialists - North Division
Orlando, Florida, 32806
Status
Address
Florida Cancer Specialists - South Division
Port Charlotte, Florida, 33980
Status
Address
Florida Cancer Specialists and Research Institute - North Division
Saint Petersburg, Florida, 33705
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Address
Florida Cancer Specialists - North
Saint Petersburg, Florida, 33707
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Address
Florida Cancer Specialists - South Division
Sarasota, Florida, 34232
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Address
Florida Cancer Specialists - South Division
Sarasota, Florida, 34236
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Address
Florida Cancer Specialist - East
Stuart, Florida, 34994
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Address
Florida Cancer Specialists - North Division
Tampa, Florida, 33607
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Address
Florida Cancer Specialists - North Division
Tavares, Florida, 32778
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Address
Florida Cancer Specialists - North Division
Trinity, Florida, 34655
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Address
Florida Cancer Specialists - South Division
Venice, Florida, 34285
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Address
Florida Cancer Specialists - South Division
Venice, Florida, 34292
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Address
Florida Cancer Specialists - East
Vero Beach, Florida, 32960
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Address
Florida Cancer Specialists - East
Wellington, Florida, 33414
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Address
Florida Cancer Specialists - East
West Palm Beach, Florida, 33401
Status
Address
Morehouse School of Medicine
Atlanta, Georgia, 30303
Status
Address
Hawaii Cancer Center
Honolulu, Hawaii, 96813
Status
Address
Hope and Healing Cancer Services
Hinsdale, Illinois, 60521
Status
Address
Northwest Oncology and Hematology
Rolling Meadows, Illinois, 60008
Status
Address
Urology of Indiana
Carmel, Indiana, 46032
Status
Address
Fort Wayne Medical Oncology and Hematology
Fort Wayne, Indiana, 46804
Status
Address
Goshen Health
Goshen, Indiana, 46526
Status
Address
Our Lady of the Lake
Baton Rouge, Louisiana, 70817
Status
Address
Pontchartrain
Hammond, Louisiana, 70403
Status
Address
American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)
Bethesda, Maryland, 20817
Status
Address
Frederick Health
Frederick, Maryland, 21702
Status
Address
Maryland Oncology Hematology
Rockville, Maryland, 20850
Status
Address
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Status
Address
Southcoast Centers for Cancer Care
Fairhaven, Massachusetts, 02719
Status
Address
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, 48201
Status
Address
Sparrow Hospital
Lansing, Michigan, 48912
Status
Address
Minnesota Oncology Hematology
Minneapolis, Minnesota, 55404
Status
Address
Central Care Cancer Center
Bolivar, Missouri, 65613
Status
Address
Lake Regional
Osage Beach, Missouri, 65065
Status
Address
Mosaic Life Care
Saint Joseph, Missouri, 64507
Status
Address
Washington University School of Medicine
Saint Louis, Missouri, 63110
Status
Address
Oncology Hematology Associates
Springfield, Missouri, 65807
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Address
Nebraska Cancer Specialists
Grand Island, Nebraska, 68803
Status
Address
Nebraska Methodist Hospital
Omaha, Nebraska, 68114
Status
Address
OptumCare Cancer Care-Parent
Las Vegas, Nevada, 89102
Status
Address
Comprehensive Cancer Centers of Nevada
Las Vegas, Nevada, 89169
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Address
New Jersey Cancer Care and Blood Disorders
Belleville, New Jersey, 07109
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Address
Englewood Hospital and Medical Center
Englewood, New Jersey, 07631
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Address
Summit Medical Group - NJ
Florham Park, New Jersey, 07932
Status
Address
Atlantic Health System - Morristown Medical Center
Morristown, New Jersey, 07960
Status
Address
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263
Status
Address
HOACNY
East Syracuse, New York, 13057
Status
Address
Cayuga Medical Center
Ithaca, New York, 14850
Status
Address
David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Status
Address
Southeastern Medical Oncology
Goldsboro, North Carolina, 27534
Status
Address
Sanford Health-Fargo
Fargo, North Dakota, 58102
Status
Address
Aultman Medical Group
Canton, Ohio, 44708
Status
Address
TriHealth
Cincinnati, Ohio, 45220
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Address
University of Cincinnati (UC) - Cancer Institute
Cincinnati, Ohio, 45627
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Address
Cleveland Clinic
Cleveland, Ohio, 44195
Status
Address
The Toledo Clinic
Toledo, Ohio, 43623
Status
Address
Oklahoma State University (OSU) - Stephenson Cancer Center
Oklahoma City, Oklahoma, 73104
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Address
Oklahoma Cancer Specialist
Tulsa, Oklahoma, 74146
Status
Address
Gettysburg-PCSRI
Gettysburg, Pennsylvania, 17325
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Address
Alliance Cancer Specialists
Langhorne, Pennsylvania, 19047
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Address
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
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Address
Cancer Care Associates of York - Parent
York, Pennsylvania, 17403
Status
Address
Prisma Health Cancer Institute
Greenville, South Carolina, 29605
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Address
Avera Cancer Institute
Sioux Falls, South Dakota, 57105
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Address
Sanford Health
Sioux Falls, South Dakota, 57117
Status
Address
Sarah Cannon and HCA Research Institute
Dickson, Tennessee, 37055
Status
Address
Sarah Cannon and HCA Research Institute
Franklin, Tennessee, 37067
Status
Address
Sarah Cannon and HCA Research Institute
Gallatin, Tennessee, 37066
Status
Address
West Cancer Center
Germantown, Tennessee, 38138
Status
Address
Sarah Cannon and HCA Research Institute
Henderson, Tennessee, 37075
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Address
Sarah Cannon and HCA Research Institute
Hermitage, Tennessee, 37076
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Address
Sarah Cannon and HCA Research Institute
Lebanon, Tennessee, 37090
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Address
Baptist Cancer Center
Memphis, Tennessee, 38120
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Address
Sarah Cannon and HCA Research Institute
Murfreesboro, Tennessee, 37129
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Address
Sarah Cannon and HCA Research Institute
Nashville, Tennessee, 37203
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Address
Sarah Cannon and HCA Research Institute
Nashville, Tennessee, 37205
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Address
Sarah Cannon and HCA Research Institute
Nashville, Tennessee, 37207
Status
Address
Sarah Cannon and HCA Research Institute
Nashville, Tennessee, 37211
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Address
Sarah Cannon and HCA Research Institute
Shelbyville, Tennessee, 37160
Status
Address
Sarah Cannon and HCA Research Institute
Smyrna, Tennessee, 37167
Status
Address
Mary Crowley Cancer Research
Dallas, Texas, 75230
Status
Address
Texas Oncology - DFW
Dallas, Texas, 75246
Status
Address
Texas Oncology
El Paso, Texas, 79915
Status
Address
MD Anderson Cancer Center
Houston, Texas, 77030
Status
Address
Oncology Consultants
Houston, Texas, 77030
Status
Address
Arizona Oncology Associates
Irving, Texas, 75063
Status
Address
Lumi Research
Kingwood, Texas, 77339
Status
Address
Texas Oncology Central-South
McAllen, Texas, 78503
Status
Address
South Texas Accelerated Research Therapeutics (START)
San Antonio, Texas, 78229
Status
Address
Texas Oncology
Tyler, Texas, 75702
Status
Address
Community Cancer Trials of Utah
Ogden, Utah, 84405
Status
Address
Utah Cancer Specialists
Salt Lake City, Utah, 84106
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Address
Virginia Cancer Specialists
Fairfax, Virginia, 22031
Status
Address
Hematology Oncology Associates of Fredericksburg
Fredericksburg, Virginia, 22408
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Address
Virginia Urology
Richmond, Virginia, 23235
Status
Address
University of Washington Cancer Consortium
Seattle, Washington, 98109
Status
Address
Spokane Urology
Spokane, Washington, 99202
Status
Address
Cancer Care Northwest
Spokane, Washington, 99216
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Address
Northwest Medical Specialties
Tacoma, Washington, 98405
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Address
ThedaCare
Appleton, Wisconsin, 54911
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Address
Gunderson Health System
La Crosse, Wisconsin, 54601
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Address
University of Wisconsin - Carbone Cancer Center
Madison, Wisconsin, 53705
International Sites
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Address
Inje University Haeundae Paik Hospital
Busan, , 48108
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Seoul National University Hospital
Seoul, , 03080
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Address
Severance Hospital, Yonsei University Health System
Seoul, , 03722
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Asan Medical Center
Seoul, , 05505
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Samsung Medical Center
Seoul, , 06351
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Address
Korea University Guro Hospital
Seoul, , 08308
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Address
Pan Oncology Trials, LLC
San Juan, , 00935
