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Badanie koszykowe fazy 2 nab-syrolimusu u pacjentów ze złośliwymi guzami litymi z patogennymi zmianami w genach TSC1 lub TSC2 (DOKŁADNOŚĆ 1)

26 października 2026 zaktualizowane przez: Aadi Bioscience, Inc.

Faza 2 wieloośrodkowa otwarta próba koszykowa Nab-syrolimusu dla dorosłych i młodzieży ze złośliwymi guzami litymi niosącymi patogenne zmiany inaktywujące w genach TSC1 lub TSC2

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

Przegląd badań

Status

Zakończony

Warunki

Interwencja / Leczenie

Szczegółowy opis

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.

Typ studiów

Interwencyjne

Zapisy (Rzeczywisty)

128

Faza

  • Faza 2

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Lokalizacje studiów

  • Korea Południowa
      • Busan, Korea Południowa, 48108
        • Inje University Haeundae Paik Hospital
      • Seoul, Korea Południowa, 03080
        • Seoul National University Hospital
      • Seoul, Korea Południowa, 05505
        • Asan Medical Center
      • Seoul, Korea Południowa, 06351
        • Samsung Medical Center
      • Seoul, Korea Południowa, 03722
        • Severance Hospital, Yonsei University Health System
      • Seoul, Korea Południowa, 08308
        • Korea University Guro Hospital
  • Portoryko
    • Puerto Rico
      • San Juan, Puerto Rico, Portoryko, 00935
        • Pan Oncology Trials, LLC
  • Stany Zjednoczone
    • Alabama
      • Birmingham, Alabama, Stany Zjednoczone, 35243
        • Alabama Oncology
      • Mobile, Alabama, Stany Zjednoczone, 36607
        • Southern Cancer Center
    • Arizona
      • Goodyear, Arizona, Stany Zjednoczone, 85395
        • Arizona Oncology Associates
      • Phoenix, Arizona, Stany Zjednoczone, 85016
        • Honor Health
      • Prescott Valley, Arizona, Stany Zjednoczone, 86301
        • Arizona Oncology Associates
      • Yuma, Arizona, Stany Zjednoczone, 85364
        • Yuma Regional Medical Center
    • California
      • Arroyo Grande, California, Stany Zjednoczone, 93420
        • PCR Oncology
      • Beverly Hills, California, Stany Zjednoczone, 90212
        • Nextgen Oncology
      • Duarte, California, Stany Zjednoczone, 91010
        • City of Hope
      • Fullerton, California, Stany Zjednoczone, 92835
        • Providence Medical Foundation (Fullerton)
      • Long Beach, California, Stany Zjednoczone, 90806
        • MemorialCare
      • Los Angeles, California, Stany Zjednoczone, 90033
        • USC Norris Comprehensive Cancer Center
      • Los Angeles, California, Stany Zjednoczone, 90095
        • UCLA - Jonsson Comprehensive Cancer Center
      • Napa, California, Stany Zjednoczone, 94558
        • Providence Medical Foundation (Napa)
      • Newport Beach, California, Stany Zjednoczone, 92663
        • Hoag Memorial Hospital Presbyterian
      • San Diego, California, Stany Zjednoczone, 92123
        • Sharp HealthCare
      • San Francisco, California, Stany Zjednoczone, 94115
        • UCSF Helen Diller Family Comprehensive Cancer Center
      • Santa Barbara, California, Stany Zjednoczone, 93105
        • Ridley-Tree Cancer Center
      • Santa Monica, California, Stany Zjednoczone, 90403
        • Sarcoma Oncology Research Center
      • Santa Rosa, California, Stany Zjednoczone, 95403
        • Providence Medical Foundation
      • Stanford, California, Stany Zjednoczone, 94305
        • Stanford Cancer Center
      • Whittier, California, Stany Zjednoczone, 90602
        • The Oncology Institute of Hope & Innovation
    • Colorado
      • Aurora, Colorado, Stany Zjednoczone, 80012
        • Rocky Mountain Cancer Centers
      • Boulder, Colorado, Stany Zjednoczone, 80303
        • Rocky Mountain Cancer Centers
      • Colorado Springs, Colorado, Stany Zjednoczone, 80907
        • Rocky Mountain Cancer Centers
      • Denver, Colorado, Stany Zjednoczone, 80220
        • Rocky Mountain Cancer Centers
      • Denver, Colorado, Stany Zjednoczone, 80218
        • Rocky Mountain Cancer Centers (Williams St)
      • Lakewood, Colorado, Stany Zjednoczone, 80228
        • Rocky Mountain Cancer Centers
      • Littleton, Colorado, Stany Zjednoczone, 80120
        • Rocky Mountain Cancer Centers
      • Lone Tree, Colorado, Stany Zjednoczone, 80124
        • Rocky Mountain Cancer Centers
      • Longmont, Colorado, Stany Zjednoczone, 80501
        • Rocky Mountain Cancer Centers
      • Pueblo, Colorado, Stany Zjednoczone, 81008
        • Rocky Mountain Cancer Centers
      • Thornton, Colorado, Stany Zjednoczone, 80260
        • Rocky Mountain Cancer Centers
    • Connecticut
      • Hartford, Connecticut, Stany Zjednoczone, 06102
        • Hartford Healthcare
      • Norwich, Connecticut, Stany Zjednoczone, 06360
        • Eastern Connecticut Hematology and Oncology
    • Florida
      • Altamonte Springs, Florida, Stany Zjednoczone, 32701
        • Florida Cancer Specialists - North Division
      • Bonita Springs, Florida, Stany Zjednoczone, 34135
        • Florida Cancer Specialists - South Division
      • Bradenton, Florida, Stany Zjednoczone, 34205
        • Florida Cancer Specialists - South Division
      • Bradenton, Florida, Stany Zjednoczone, 34211
        • Florida Cancer Specialists - South Division
      • Brandon, Florida, Stany Zjednoczone, 33511
        • Florida Cancer Specialists - North Division
      • Cape Coral, Florida, Stany Zjednoczone, 33909
        • Florida Cancer Specialists - South Division
      • Clearwater, Florida, Stany Zjednoczone, 33761
        • Florida Cancer Specialists - North Division
      • Daytona Beach, Florida, Stany Zjednoczone, 32117
        • Cancer Specialist - East
      • Fort Lauderdale, Florida, Stany Zjednoczone, 33308
        • Holy Cross Hospital
      • Fort Myers, Florida, Stany Zjednoczone, 33901
        • Florida Cancer Specialists South Division
      • Fort Myers, Florida, Stany Zjednoczone, 33905
        • Florida Cancer Specialists - South
      • Fort Myers, Florida, Stany Zjednoczone, 33908
        • Florida Cancer Specialists - South
      • Gainesville, Florida, Stany Zjednoczone, 32605
        • Florida Cancer Specialists - North Division
      • Jacksonville, Florida, Stany Zjednoczone, 32256
        • Cancer Specialists of North Florida
      • Lakeland, Florida, Stany Zjednoczone, 33812
        • TOI Florida
      • Lakeland, Florida, Stany Zjednoczone, 33812
        • The Oncology Institute of Hope and Innovation
      • Largo, Florida, Stany Zjednoczone, 33770
        • Florida Cancer Specialists - North Division
      • Lecanto, Florida, Stany Zjednoczone, 34461
        • Florida Cancer Specialists - North Division
      • Naples, Florida, Stany Zjednoczone, 34102
        • Florida Cancer Specialists - South Division
      • Ocala, Florida, Stany Zjednoczone, 34474
        • Ocala Oncology
      • Ocala, Florida, Stany Zjednoczone, 34474
        • Florida Cancer Specialists - North Division
      • Orange City, Florida, Stany Zjednoczone, 32763
        • Florida Cancer Specialists - North Division
      • Orlando, Florida, Stany Zjednoczone, 32806
        • Florida Cancer Specialists - North Division
      • Port Charlotte, Florida, Stany Zjednoczone, 33980
        • Florida Cancer Specialists - South Division
      • Sarasota, Florida, Stany Zjednoczone, 34232
        • Florida Cancer Specialists - South Division
      • Sarasota, Florida, Stany Zjednoczone, 34236
        • Florida Cancer Specialists - South Division
      • St. Petersburg, Florida, Stany Zjednoczone, 33705
        • Florida Cancer Specialists and Research Institute - North Division
      • St. Petersburg, Florida, Stany Zjednoczone, 33707
        • Florida Cancer Specialists - North
      • Stuart, Florida, Stany Zjednoczone, 34994
        • Florida Cancer Specialist - East
      • Tampa, Florida, Stany Zjednoczone, 33607
        • Florida Cancer Specialists - North Division
      • Tavares, Florida, Stany Zjednoczone, 32778
        • Florida Cancer Specialists - North Division
      • Trinity, Florida, Stany Zjednoczone, 34655
        • Florida Cancer Specialists - North Division
      • Venice, Florida, Stany Zjednoczone, 34285
        • Florida Cancer Specialists - South Division
      • Venice, Florida, Stany Zjednoczone, 34292
        • Florida Cancer Specialists - South Division
      • Vero Beach, Florida, Stany Zjednoczone, 32960
        • Florida Cancer Specialists - East
      • Wellington, Florida, Stany Zjednoczone, 33414
        • Florida Cancer Specialists - East
      • West Palm Beach, Florida, Stany Zjednoczone, 33401
        • Florida Cancer Specialists - East
    • Georgia
      • Atlanta, Georgia, Stany Zjednoczone, 30303
        • Morehouse School of Medicine
    • Hawaii
      • Honolulu, Hawaii, Stany Zjednoczone, 96813
        • Hawaii Cancer Center
    • Illinois
      • Hinsdale, Illinois, Stany Zjednoczone, 60521
        • Hope and Healing Cancer Services
      • Rolling Meadows, Illinois, Stany Zjednoczone, 60008
        • Northwest Oncology and Hematology
    • Indiana
      • Carmel, Indiana, Stany Zjednoczone, 46032
        • Urology of Indiana
      • Fort Wayne, Indiana, Stany Zjednoczone, 46804
        • Fort Wayne Medical Oncology and Hematology
      • Goshen, Indiana, Stany Zjednoczone, 46526
        • Goshen Health
    • Louisiana
      • Baton Rouge, Louisiana, Stany Zjednoczone, 70817
        • Our Lady of the Lake
      • Hammond, Louisiana, Stany Zjednoczone, 70403
        • Pontchartrain
    • Maryland
      • Bethesda, Maryland, Stany Zjednoczone, 20817
        • American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)
      • Frederick, Maryland, Stany Zjednoczone, 21702
        • Frederick Health
      • Rockville, Maryland, Stany Zjednoczone, 20850
        • Maryland Oncology Hematology
    • Massachusetts
      • Boston, Massachusetts, Stany Zjednoczone, 02215
        • Dana Farber Cancer Institute
      • Fairhaven, Massachusetts, Stany Zjednoczone, 02719
        • Southcoast Centers for Cancer Care
    • Michigan
      • Detroit, Michigan, Stany Zjednoczone, 48201
        • Barbara Ann Karmanos Cancer Institute
      • Lansing, Michigan, Stany Zjednoczone, 48912
        • Sparrow Hospital
    • Minnesota
      • Minneapolis, Minnesota, Stany Zjednoczone, 55404
        • Minnesota Oncology Hematology
    • Missouri
      • Bolivar, Missouri, Stany Zjednoczone, 65613
        • Central Care Cancer Center
      • Osage Beach, Missouri, Stany Zjednoczone, 65065
        • Lake Regional
      • Saint Joseph, Missouri, Stany Zjednoczone, 64507
        • Mosaic Life Care
      • Springfield, Missouri, Stany Zjednoczone, 65807
        • Oncology Hematology Associates
      • St Louis, Missouri, Stany Zjednoczone, 63110
        • Washington University School of Medicine
    • Nebraska
      • Grand Island, Nebraska, Stany Zjednoczone, 68803
        • Nebraska Cancer Specialists
      • Omaha, Nebraska, Stany Zjednoczone, 68114
        • Nebraska Methodist Hospital
    • Nevada
      • Las Vegas, Nevada, Stany Zjednoczone, 89169
        • Comprehensive Cancer Centers of Nevada
      • Las Vegas, Nevada, Stany Zjednoczone, 89102
        • OptumCare Cancer Care-Parent
    • New Jersey
      • Belleville, New Jersey, Stany Zjednoczone, 07109
        • New Jersey Cancer Care and Blood Disorders
      • Englewood, New Jersey, Stany Zjednoczone, 07631
        • Englewood Hospital and Medical Center
      • Florham Park, New Jersey, Stany Zjednoczone, 07932
        • Summit Medical Group - NJ
      • Morristown, New Jersey, Stany Zjednoczone, 07960
        • Atlantic Health System - Morristown Medical Center
    • New York
      • Buffalo, New York, Stany Zjednoczone, 14263
        • Roswell Park Comprehensive Cancer Center
      • East Syracuse, New York, Stany Zjednoczone, 13057
        • HOACNY
      • Ithaca, New York, Stany Zjednoczone, 14850
        • Cayuga Medical Center
      • New York, New York, Stany Zjednoczone, 10065
        • David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center
    • North Carolina
      • Goldsboro, North Carolina, Stany Zjednoczone, 27534
        • Southeastern Medical Oncology
    • North Dakota
      • Fargo, North Dakota, Stany Zjednoczone, 58102
        • Sanford Health-Fargo
    • Ohio
      • Canton, Ohio, Stany Zjednoczone, 44708
        • Aultman Medical Group
      • Cincinnati, Ohio, Stany Zjednoczone, 45220
        • TriHealth
      • Cincinnati, Ohio, Stany Zjednoczone, 45627
        • University of Cincinnati (UC) - Cancer Institute
      • Cleveland, Ohio, Stany Zjednoczone, 44195
        • Cleveland Clinic
      • Toledo, Ohio, Stany Zjednoczone, 43623
        • The Toledo Clinic
    • Oklahoma
      • Oklahoma City, Oklahoma, Stany Zjednoczone, 73104
        • Oklahoma State University (OSU) - Stephenson Cancer Center
      • Tulsa, Oklahoma, Stany Zjednoczone, 74146
        • Oklahoma Cancer Specialist
    • Pennsylvania
      • Gettysburg, Pennsylvania, Stany Zjednoczone, 17325
        • Gettysburg-PCSRI
      • Langhorne, Pennsylvania, Stany Zjednoczone, 19047
        • Alliance Cancer Specialists
      • Philadelphia, Pennsylvania, Stany Zjednoczone, 19107
        • Thomas Jefferson University
      • York, Pennsylvania, Stany Zjednoczone, 17403
        • Cancer Care Associates of York - Parent
    • South Carolina
      • Greenville, South Carolina, Stany Zjednoczone, 29605
        • Prisma Health Cancer Institute
    • South Dakota
      • Sioux Falls, South Dakota, Stany Zjednoczone, 57105
        • Avera Cancer Institute
      • Sioux Falls, South Dakota, Stany Zjednoczone, 57117
        • Sanford Health
    • Tennessee
      • Dickson, Tennessee, Stany Zjednoczone, 37055
        • Sarah Cannon and HCA Research Institute
      • Franklin, Tennessee, Stany Zjednoczone, 37067
        • Sarah Cannon and HCA Research Institute
      • Gallatin, Tennessee, Stany Zjednoczone, 37066
        • Sarah Cannon and HCA Research Institute
      • Germantown, Tennessee, Stany Zjednoczone, 38138
        • West Cancer Center
      • Henderson, Tennessee, Stany Zjednoczone, 37075
        • Sarah Cannon and HCA Research Institute
      • Hermitage, Tennessee, Stany Zjednoczone, 37076
        • Sarah Cannon and HCA Research Institute
      • Lebanon, Tennessee, Stany Zjednoczone, 37090
        • Sarah Cannon and HCA Research Institute
      • Memphis, Tennessee, Stany Zjednoczone, 38120
        • Baptist Cancer Center
      • Murfreesboro, Tennessee, Stany Zjednoczone, 37129
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Stany Zjednoczone, 37203
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Stany Zjednoczone, 37205
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Stany Zjednoczone, 37207
        • Sarah Cannon and HCA Research Institute
      • Nashville, Tennessee, Stany Zjednoczone, 37211
        • Sarah Cannon and HCA Research Institute
      • Shelbyville, Tennessee, Stany Zjednoczone, 37160
        • Sarah Cannon and HCA Research Institute
      • Smyrna, Tennessee, Stany Zjednoczone, 37167
        • Sarah Cannon and HCA Research Institute
    • Texas
      • Dallas, Texas, Stany Zjednoczone, 75230
        • Mary Crowley Cancer Research
      • Dallas, Texas, Stany Zjednoczone, 75246
        • Texas Oncology - DFW
      • El Paso, Texas, Stany Zjednoczone, 79915
        • Texas Oncology
      • Houston, Texas, Stany Zjednoczone, 77030
        • MD Anderson Cancer Center
      • Houston, Texas, Stany Zjednoczone, 77030
        • Oncology Consultants
      • Irving, Texas, Stany Zjednoczone, 75063
        • Arizona Oncology Associates
      • Kingwood, Texas, Stany Zjednoczone, 77339
        • Lumi Research
      • McAllen, Texas, Stany Zjednoczone, 78503
        • Texas Oncology Central-South
      • San Antonio, Texas, Stany Zjednoczone, 78229
        • South Texas Accelerated Research Therapeutics (START)
      • Tyler, Texas, Stany Zjednoczone, 75702
        • Texas Oncology
    • Utah
      • Ogden, Utah, Stany Zjednoczone, 84405
        • Community Cancer Trials of Utah
      • Salt Lake City, Utah, Stany Zjednoczone, 84106
        • Utah Cancer Specialists
    • Virginia
      • Fairfax, Virginia, Stany Zjednoczone, 22031
        • Virginia Cancer Specialists
      • Fredericksburg, Virginia, Stany Zjednoczone, 22408
        • Hematology Oncology Associates of Fredericksburg
      • Richmond, Virginia, Stany Zjednoczone, 23235
        • Virginia Urology
    • Washington
      • Seattle, Washington, Stany Zjednoczone, 98109
        • University of Washington Cancer Consortium
      • Spokane, Washington, Stany Zjednoczone, 99216
        • Cancer Care Northwest
      • Spokane, Washington, Stany Zjednoczone, 99202
        • Spokane Urology
      • Tacoma, Washington, Stany Zjednoczone, 98405
        • Northwest Medical Specialties
    • Wisconsin
      • Appleton, Wisconsin, Stany Zjednoczone, 54911
        • ThedaCare
      • La Crosse, Wisconsin, Stany Zjednoczone, 54601
        • Gunderson Health System
      • Madison, Wisconsin, Stany Zjednoczone, 53705
        • University of Wisconsin - Carbone Cancer Center

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

12 lat i starsze (Dziecko, Dorosły, Starszy dorosły)

Akceptuje zdrowych ochotników

Nie

Płeć kwalifikująca się do nauki

Wszystko

Opis

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.

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Nielosowe
  • Model interwencyjny: Zadanie dla jednej grupy
  • Maskowanie: Brak (otwarta etykieta)

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Eksperymentalny: Arm A: Pathogenic inactivating TSC1 alterations

Patients with pathogenic inactivating TSC1 alterations.
Lek: 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

Inne nazwy:
  • ABI-009
Eksperymentalny: Arm B: Pathogenic inactivating TSC2 alterations

Patients with pathogenic inactivating TSC2 alterations.
Lek: 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

Inne nazwy:
  • ABI-009

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Overall Response Rate (ORR)
Ramy czasowe: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression , up to 30 months.

Based on CT or MRI scans from the time of study treatment initiation until disease progression, overall response rate (ORR) is defined as the proportion of patients with best overall response (BOR) of confirmed partial response (PR) or complete response (CR) in accordance with Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 as determined by independent Radiographic Review (IRR).

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression , up to 30 months.

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
Duration of Response (DOR)
Ramy czasowe: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.

DOR is defined as the number of months from the start of CR or PR (whichever response is recorded first) and subsequently confirmed to the first date of documented progressive disease (PD) or death using CT or MRI scans in accordance with RECIST v1.1.

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.

PD is defined as ≥20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.
Disease Control Rate
Ramy czasowe: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression, up to 30 months.

Disease control rate (DCR) is defined as BOR of confirmed CR or PR (either of any duration) or SD ≥12 weeks following study treatment initiation.

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression, up to 30 months.
Time to Response
Ramy czasowe: The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.

Time to response (TTR) is defined as the time from first dose of study drug to initial measurement of CR or PR, where CR or PR is subsequently confirmed.

CR is defined as disappearance of all target lesions. PR is defined as ≥30% decrease in the sum of diameters of target lesions from baseline, with no new lesions and no progression of non-target lesions.
The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression.
Progression-free Survival
Ramy czasowe: From first dose of study treatment to the date of disease progression or death

Number of months from study treatment initiation to the date of disease progression (PD) or death due to any cause PD is defined as ≥20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
From first dose of study treatment to the date of disease progression or death
Overall Survival
Ramy czasowe: From first dose of study treatment to the date of death

Number of months from study treatment initiation to the date of death due to any cause
From first dose of study treatment to the date of death
Patient-Reported Outcome
Ramy czasowe: D1 of every cycle up to Cycle 24

Changes from baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QOQ-C30) v3.0 scores EORTC QOQ-C30 includes functional scales (physical, role, cognitive, emotional, social), global health status, symptom scales (fatigue, pain, nausea/vomiting), and other (dyspnoea, appetite loss, insomnia, constipation/diarrhea, financial difficulties). Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); two used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score = better quality of life.
D1 of every cycle up to Cycle 24
Incidence and Severity of Treatment-emergent and Treatment-related Adverse Events (AEs)
Ramy czasowe: Monitored continuously from signing of Informed Consent until last dose of IP (estimated up to 30 months) plus safety follow-up of 28 days after the last dose.

Incidence and severity of treatment-emergent and treatment-related AEs as assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v5.0
Monitored continuously from signing of Informed Consent until last dose of IP (estimated up to 30 months) plus safety follow-up of 28 days after the last dose.

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Aadi Bioscience, Inc.

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Rzeczywisty)

10 marca 2022

Zakończenie podstawowe (Rzeczywisty)

23 grudnia 2024

Ukończenie studiów (Rzeczywisty)

23 grudnia 2024

Daty rejestracji na studia

Pierwszy przesłany

22 października 2021

Pierwszy przesłany, który spełnia kryteria kontroli jakości

22 października 2021

Pierwszy wysłany (Rzeczywisty)

2 listopada 2021

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

28 października 2026

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

26 października 2026

Ostatnia weryfikacja

1 września 2026

Więcej informacji

Terminy związane z tym badaniem

Słowa kluczowe

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • TSC-007

Plan dla danych uczestnika indywidualnego (IPD)

Planujesz udostępniać dane poszczególnych uczestników (IPD)?

NIE

Informacje o lekach i urządzeniach, dokumenty badawcze

Bada produkt leczniczy regulowany przez amerykańską FDA

Tak

Bada produkt urządzenia regulowany przez amerykańską FDA

Nie

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

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